Research Studies and Clinical Trials Search
Results
A tool used to collect information about Juvenile Idiopathic Arthritis
PR-COIN is a registry that focuses on improving the quality of the care patients of all ages with juvenile idiopathic arthritis (JIA) receive. Data is collected from the participant's rheumatology appointments and entered into a registry database. With the data collected...
PR-COIN is a registry that focuses on improving the quality of the care patients of all ages with juvenile idiopathic arthritis (JIA) receive. Data is collected from the participant's rheumatology appointments and entered into a registry database. With the data collected rheumatologists around the country can work on improving the standard of care they give to their patients. This study is designed to improve disease control, improve quality of life for patients, support families in managing their child's condition, and many other quality improvement objectives.
A tool used to collect information about juvenile rheumatic diseases
The CARRA Registry is a database that monitors patients for 10 years and is limited to patients with rheumatic conditions under the age of 18 years old depending on their diagnosis. The information collected from participants is gathered from their rheumatology appointments every...
The CARRA Registry is a database that monitors patients for 10 years and is limited to patients with rheumatic conditions under the age of 18 years old depending on their diagnosis. The information collected from participants is gathered from their rheumatology appointments every 6 months, with some short questionnaires for patients and families to answer about their overall health. With the data collected rheumatologists around the country can evaluate effectiveness of medication and monitor patient health.
Acute Leukemia or Myelodysplastic Syndrome: A study comparing matched unrelated donor versus haploidentical related myeloablative hematopoietic cell transplantation
This study is for children and young adults: -Ages 6 months to 21 years old -With a diagnosis of acute leukemia or myelodysplastic syndrome (MDS)...
This study is for children and young adults: -Ages 6 months to 21 years old -With a diagnosis of acute leukemia or myelodysplastic syndrome (MDS) This study is being done to learn more about hematopoietic cell transplantation (HCT) that use mismatched related donor (haploidentical or haplo) versus a matched unrelated donor (MUD). The research team will use bone marrow or stem cells from a mismatched related family donor or from a MUD and then compare the transplant results.
Acute Liver Failure (ALF): A clinical trial of study drug (eATG or high dose methylprednisolone) compared to placebo (saline)
To participate in this study the participant must between 1 year and up to 18 years of age. This study is being done to learn if one medicine is better, the same, or worse for treating children with acute liver failure compared to another medicine and a placebo (a fake medicine).
To participate in this study the participant must between 1 year and up to 18 years of age. This study is being done to learn if one medicine is better, the same, or worse for treating children with acute liver failure compared to another medicine and a placebo (a fake medicine).
Acute Lymphoblastic Leukemia (ALL): A clinical study of carfilzomib and induction chemotherapy in relapsed/refractory ALL
This study will have 2 parts: induction and consolidation. The induction part of the study is being done to learn more about carfilzomib in infants, children, and young adults with relapsed or refractory ALL. It will evaluate the side effects (good and/or bad) and the response...
This study will have 2 parts: induction and consolidation. The induction part of the study is being done to learn more about carfilzomib in infants, children, and young adults with relapsed or refractory ALL. It will evaluate the side effects (good and/or bad) and the response rate of carfilzomib in combination with chemotherapy. The treatment response to this new combination will be compared to responses observed on a similar group of patients that received available chemotherapy as standard treatment. The consolidation part of the study is being done to learn more about carfilzomib in infants, children, and young adults with relapsed or refractory ALL. It will evaluate the side effects (good and/or bad) of carfilzomib in combination with chemotherapy that is based on a regimen that has been commonly used in both United States and Europe. It will also measure how effective the combination of carfilzomib with the modified chemotherapy is at treating infants, children, and young adults with relapsed or refractory ALL. The study will measure whether carfilzomib in combination with the consolidation chemotherapy can deepen the response of the leukemia to the induction chemotherapy and the ability to receive further therapy after this treatment is completed. Study participants must be age 21 years or younger at the time of initial ALL diagnosis and age > 1 year at the time of study treatment initiation.
Acute Lymphoblastic Leukemia (ALL): A clinical study of ponatinib with chemotherapy in patients with philadelphia chromosome-postive ALL
This study is being done to learn more about the investigational drug, ponatinib, for the treatment of acute lymphoblastic leukemia in pediatric patients.
This study is being done to learn more about the investigational drug, ponatinib, for the treatment of acute lymphoblastic leukemia in pediatric patients.
Acute Lymphoblastic Leukemia or Lymphoma (ALL): A study on premedicating pediatric patients receiving PEG-asparaginase with antihistamines
Patients who are ages 1 to 26 may be eligible for this study. The purpose of this study is to see if taking an antihistamine (Benadryl® and either Pepcid® or Tagamet®) before PEG-asparaginase reduces allergic reactions. We also want to see if it reduces the need to switch to...
Patients who are ages 1 to 26 may be eligible for this study. The purpose of this study is to see if taking an antihistamine (Benadryl® and either Pepcid® or Tagamet®) before PEG-asparaginase reduces allergic reactions. We also want to see if it reduces the need to switch to Erwinia asparaginase or another PEG-asparaginase substitute. In this study, we will document any side effects that occur within 30 days of treatment with PEG-asparaginase. We will also look at the level of asparaginase in patients' blood after each dose. We may also look at patients' genetic information to see how the body reacts to treatment with PEG-asparaginase.
Acute Lynphoblastic Leukemia (ALL): A clinical study of ruxolitinib and chemotherapy in de novo high-risk CRLF2-rearranged or JAK pathway-mutant ALL
HR B-ALL is a type of cancer of the blood cells that occurs in the bone marrow. This research study will find out the best dose of ruxolitinib to use with standard chemotherapy and if the study drug, ruxolitinib, in combination with standard HR B-ALL treatment is safe and...
HR B-ALL is a type of cancer of the blood cells that occurs in the bone marrow. This research study will find out the best dose of ruxolitinib to use with standard chemotherapy and if the study drug, ruxolitinib, in combination with standard HR B-ALL treatment is safe and effective in children, adolescents, and young adults with HR B-ALL. This study is for participants 1–21 years old.
Acute Myelogenous Leukemia (AML): A clinical trial of standard therapy compared to chemotherapy with CPX-351 investigational drug
To join this study, patients must be younger than 22 years old and have a diagnosis of de novo Acute Myelogenous Leukemia (AML). Treatment for AML involves high doses of a common class of chemotherapy drugs called anthracyclines. These drugs are effective at treating AML, but...
To join this study, patients must be younger than 22 years old and have a diagnosis of de novo Acute Myelogenous Leukemia (AML). Treatment for AML involves high doses of a common class of chemotherapy drugs called anthracyclines. These drugs are effective at treating AML, but high doses are known to cause long-term side effects, especially to the heart. This study will compare standard chemotherapy using daunorubicin, cytarabine and gemtuzumab ozogamicin (GO) to chemotherapy using an experimental drug called CPX-351. Researchers will also study the effects, good and/or bad, of adding gilteritinib to AML therapy for patients with high amounts of FLT3/ITD or other FLT3 mutations. Last, researchers will study changes in heart function during and after treatment for AML.
Acute Myeloid Leukemia (AML): A clinical study of enasidenib to treat relapsed/refractory AML with IDH-2 mutations
The overall goal of this study is to find out what effects, good and/or bad, enasidenib has on children and adolescents with AML with IDH2 mutations. This study is for patients who are 2 to 18 years old.
The overall goal of this study is to find out what effects, good and/or bad, enasidenib has on children and adolescents with AML with IDH2 mutations. This study is for patients who are 2 to 18 years old.
Alagille Syndrome (ALGS): A study on the long-term safety and effectiveness of Livmarli
This study is being done for children: • With a Alagille Syndrome (AlGS) diagnosis • Currently taken Livmarli. The purpose of this research study is to evaluate the long-term safety and outcomes of patients with Alagille Syndrome...
This study is being done for children: • With a Alagille Syndrome (AlGS) diagnosis • Currently taken Livmarli. The purpose of this research study is to evaluate the long-term safety and outcomes of patients with Alagille Syndrome (ALGS) who are prescribed Livmarli (maralixibat).
Amblyopia (Lazy Eye): A study on glasses and eye patches in children 3-12 for treatment
Age 3-12 years to evaluate if treating amblyopia (lazy eye) with glasses and eye patching at the same time improves vision as well as treating amblyopia (lazy eye) first with glasses and then with patching, if needed. This study will also use a small wearable temperature sensor...
Age 3-12 years to evaluate if treating amblyopia (lazy eye) with glasses and eye patching at the same time improves vision as well as treating amblyopia (lazy eye) first with glasses and then with patching, if needed. This study will also use a small wearable temperature sensor to record actual patch wear time with prescribed patching treatment.
Angelman Syndrome: Clinical Needs Survey
This study is for children/adults : • Diagnosed with Angelman Syndrome • Who are seen or will be seen at the Angelman Syndrome Clinic at Children's Mercy • Previously enrolled in the Linking Angelman and Dup15q Data for Expanded Research (LADDER) database or who would like...
This study is for children/adults : • Diagnosed with Angelman Syndrome • Who are seen or will be seen at the Angelman Syndrome Clinic at Children's Mercy • Previously enrolled in the Linking Angelman and Dup15q Data for Expanded Research (LADDER) database or who would like to join The goal of the study is to better understand Angelman and Dup15q syndromes. The Clinical Needs Survey will be used as the intake form for the clinic but will be stored in the LADDER database. The Angelman Syndrome Clinic is partnering with LADDER that will allow information about individuals seen at the clinic to be housed in a centralized database. The information held in LADDER is private and no information that could identify a patient or their family is shared with anyone outside the LADDER team. The goal is that information from these surveys will help researchers to create therapies and advance clinical trials more quickly and easily.
Appetite and Exercise: A study to examine the connection between physical activity and appetite control in teenagers
This study is for: - Teenagers (14-17 years old) -Healthy individuals who participate in less than 60 minutes of physical activity per day...
This study is for: - Teenagers (14-17 years old) -Healthy individuals who participate in less than 60 minutes of physical activity per day The purpose of this research study is to see if there is an association between physical activity and appetite control in teenagers.
Asthma: A study to see how histamine response can guide treatment
Who can be in this study: • Children ages 6-17 years • Diagnosed with asthma that has been uncontrolled • African-American or Caucasian...
Who can be in this study: • Children ages 6-17 years • Diagnosed with asthma that has been uncontrolled • African-American or Caucasian The purpose of this research study is to find out if a Laser Doppler (HILD) type test that looks at histamine response can help determine if certain groups of children with asthma respond differently to antihistamines (drugs that block histamine) such as levocetirizine (study drug) than others. We also want to see if certain gene patterns in children with asthma may be related to how a child responds to levocetirizine.
B-Cell Acute Lymphoblastic Leukemia (B-ALL): A study eliminating Total Body Irradiation (TBI) for NGS-MRD Negative Children, Adolescents, and Young Adults
There are two groups involved in this study, Observational and Treatment. Patients of any age diagnosed with B-Cell acute lymphoblastic leukemia (B-ALL), T-cell acute lymphoblastic leukemia (T-ALL), or Mixed phenotype acute leukemia (MPAL) may join the Observational group. B-ALL...
There are two groups involved in this study, Observational and Treatment. Patients of any age diagnosed with B-Cell acute lymphoblastic leukemia (B-ALL), T-cell acute lymphoblastic leukemia (T-ALL), or Mixed phenotype acute leukemia (MPAL) may join the Observational group. B-ALL patients 1 to 31 years of age requiring hematopoietic cell transplantation and NGS-MRD negative may join the Treatment group. In this study, researchers would like to see if not giving patients total body irradiation (also called TBI) therapy before transplant is as good as giving patients radiation before transplant.
B-cell acute lymphoblastic leukemia (B-ALL): A study on cell therapy medication to treat cancer cells and prevent relapse
This study is for individuals between 1 year of age and 25 years of age who have been diagnosed with B-cell acute lymphoblastic leukemia (B-ALL). It involves children or young adults who have recently started receiving cancer treatment for the first time and have a small number...
This study is for individuals between 1 year of age and 25 years of age who have been diagnosed with B-cell acute lymphoblastic leukemia (B-ALL). It involves children or young adults who have recently started receiving cancer treatment for the first time and have a small number of cancer cells still detected in their bodies after the first part of treatment. When these small number of cancer cells are present after the induction and consolidation treatment phases (initial months of treatment) for B-ALL, the patients are considered to be in a high risk group. This means their cancer is harder to treat and may be more likely to return after treatment. The goal of this study is to see if a specific type of cell therapy medication may treat the remaining small amount of cancer cells and help prevent relapse in children and young adults who are part of this high-risk group.
Bedside MRI: A study to compare portable low-field MRI imaging to traditional MRI.
This study is for children and young adults: -Radiology outpatients 0 days-22 years of age -Scheduled for an MRI of the brain...
This study is for children and young adults: -Radiology outpatients 0 days-22 years of age -Scheduled for an MRI of the brain -Adele Hall campus Different medical imaging techniques, such as MRI, CT and US, are used in Radiology to image different parts of the body. The purpose of this research study is to capture, compare and assess the quality and performance of Hyperfine MRI, or low-field strength MRI, imaging. Researchers will compare Hyperfine MRI images to clinical exams. Any patient that consents will receive a bedside MRI exam.
Bone Cancer: A study comparing two surgery methods to treat cancer in the lung
This study is for: • Patients less than 50 years old • Who have osteosarcoma (OST), a type of bone cancer, that has spread into the lungs...
This study is for: • Patients less than 50 years old • Who have osteosarcoma (OST), a type of bone cancer, that has spread into the lungs The overall goal of this study is to compare the effects, good and/or bad, of two different surgery methods to find out which is better. The two types of surgeries are called open thoracic surgery and thoracoscopy. Study patients will be randomly assigned to one of the two types of surgery.
Brain or Spinal Cord Disease/Injury: Blood or saliva samples collected and stored for future research
A research repository collects and stores information and samples so that researchers can learn more about a disease or condition. This study is for a repository that will collect saliva or blood samples from many individuals with functional impairments in order to learn more...
A research repository collects and stores information and samples so that researchers can learn more about a disease or condition. This study is for a repository that will collect saliva or blood samples from many individuals with functional impairments in order to learn more about the way medicines interact with the body and the way that your genes affect this.
Brain Tumors: A study to see if memantine protects the brain during radiation therapy treatment in children
This study is for: • Patients ages 4 to 18 • Who have been diagnosed with a brain tumor...
This study is for: • Patients ages 4 to 18 • Who have been diagnosed with a brain tumor The goal of this study is to see if the drug memantine makes a difference in changes to attention, memory, or other thought processes (called cognitive function changes) in children and adolescents treated with radiation therapy for a brain tumor. In this study participants will get 1 of 2 treatments: memantine (active drug) or placebo (no active drug). The treatment (memantine or placebo) participants receive is decided by a process called randomization, which means that the treatment is assigned based on chance like flipping a coin. Participants will also complete questionnaires and short computer testing sessions.
Bronchopulmonary Dysplasia (BPD): A study of Sildenafil in premature babies to evaluate its safety and ability to prevent pulmonary hypertension
This study is for infants: • Born prematurely • Less than 1 year of age • Have a form of lung disease called bronchopulmonary dysplasia (BPD), that may develop an additional problem called...
This study is for infants: • Born prematurely • Less than 1 year of age • Have a form of lung disease called bronchopulmonary dysplasia (BPD), that may develop an additional problem called pulmonary hypertension.This study will learn more about the safety of a drug called sildenafil which is an approved drug available by prescription for use in adult patients with pulmonary hypertension, but is not approved for use in patients less than 1 year of age. Researchers want to learn more about sildenafil side effects and how well it works to prevent long-term problems of BPD, and the best amount (dose) to give in premature babies with BPD. Pulmonary hypertension may worsen the lung disease, and are currently no medicines available to prevent it. Researchers want to find new ways to prevent long-term problems such as pulmonary hypertension in babies with BPD because infants with those conditions are at a higher risk of dying.
Cancer - Solid or CNS Tumors: A clinical study of sirolimus and metronomic chemotherapy for recurrent and/or refractory tumors
This is a study of a drug called sirolimus. Sirolimus has not been approved for use by the FDA for this use and is considered an investigational drug. "Investigational" means that it is still experimental because it has not been tested for use for the treatment of solid or CNS...
This is a study of a drug called sirolimus. Sirolimus has not been approved for use by the FDA for this use and is considered an investigational drug. "Investigational" means that it is still experimental because it has not been tested for use for the treatment of solid or CNS tumors. In this study, sirolimus is given in combination with other chemotherapy drugs. The drugs etoposide, cyclophosphamide and celecoxib are used in combination in children with your type of cancer when it returns or stops responding to treatment. When this combination of etoposide, cyclophosphamide and celecoxib is given by mouth, at lower doses than given through the vein, it is much better tolerated by patients. It also helps stop tumor growth by preventing blood from getting to the tumor. Blood supply is needed for tumors to grow. In this study, sirolimus is added to these three drugs. Study doctors want to learn if patients can tolerate this. They also want to learn whether it improves the effect that this therapy has on tumor growth.
Cancer - Solid Tumors: A clinical study of palbociclib with irinotecan and temozolomide and/or topotecan and cyclophasphamide in recurrent or refractory tumors
The purpose of this study is to compare the effects of the study drug called palbociclib (IBRANCE®) when it is given in combination with two other study drugs (temozolomide and irinotecan) with the effects of the combination of temozolomide and irinotecan alone to find out which...
The purpose of this study is to compare the effects of the study drug called palbociclib (IBRANCE®) when it is given in combination with two other study drugs (temozolomide and irinotecan) with the effects of the combination of temozolomide and irinotecan alone to find out which combination is better for treating Ewing sarcoma. The palbociclib, in combination with IRN and TMZ, in this research is investigational because it is not currently approved for use in children. Temozolomide (TEMODAR®) is approved in US for a treatment of certain brain tumors in adults. Irinotecan (CAMPTOSAR®) is approved in US for a treatment of adult colon cancer. Researchers will compare the results of taking IRN with TMZ to the results of taking palbociclib with IRN and TMZ to see if there are any differences
Cancer or Cancer Related Illness: Blood and/or tissue samples collected and stored for future research
Patients with a suspected or confirmed oncology or bone marrow failure diagnosis are eligible to participate. If enrolled, blood and/or tissue samples will be collected, deidentified, and stored for use in future research projects. De-identified clinical data will also be...
Patients with a suspected or confirmed oncology or bone marrow failure diagnosis are eligible to participate. If enrolled, blood and/or tissue samples will be collected, deidentified, and stored for use in future research projects. De-identified clinical data will also be collected for future research.
Cancer: A study screening for genetic changes (mutations) that may respond to specific drug therapies for tumors
The main purpose of this study is to learn how well tumors that have specific genetic changes (mutations) respond to drugs that "target" those changes. This combination of a tumor with a mutation and a drug that aims at that mutation is called a "match."
The main purpose of this study is to learn how well tumors that have specific genetic changes (mutations) respond to drugs that "target" those changes. This combination of a tumor with a mutation and a drug that aims at that mutation is called a "match."
Cancer: An informational registry and tissue/blood storage bank for future research
This study is available for those who are 25 years old or younger. The goal of this study is to find better ways to diagnose, treat and prevent cancers in children, adolescents and young adults. We will collect some information about each participant's cancer and treatment. We...
This study is available for those who are 25 years old or younger. The goal of this study is to find better ways to diagnose, treat and prevent cancers in children, adolescents and young adults. We will collect some information about each participant's cancer and treatment. We will also collect extra tumor, blood, bone marrow, or other tissue from procedures done by doctors to help make a diagnosis and learn more about different cancers now and in the future.
CHAMP GI: A remote tube weaning study using CHAMP® App
This study is for children who are: • Ages 1 month to 5 years 11 months • Approved for tube weaning by Children's Mercy Interdisciplinary Feeding Clinic providers The goal of this study is to evaluate the use of data collection while at home and using the CHAMP® App during a...
This study is for children who are: • Ages 1 month to 5 years 11 months • Approved for tube weaning by Children's Mercy Interdisciplinary Feeding Clinic providers The goal of this study is to evaluate the use of data collection while at home and using the CHAMP® App during a feeding tube weaning period. Tube weaning can be a stressful time, and researchers are interested in finding ways to make the experience better. Researchers are trying to learn if the CHAMP App would be a helpful tool for parents and providers to support monitoring children that are tube feeding because of feeding difficulties.
Chemotherapy-induced vomiting: A study of Olanzapine in children receiving chemotherapy for Hematopoietic Stem Cell Transplant conditioning
Patients 2.5 to 18 years of age and are about to receive chemotherapy before a blood or bone marrow transplant may join this study. Chemotherapy drugs are used to prepare children for blood or bone marrow transplant. A few of the side effects of these drugs are nausea and...
Patients 2.5 to 18 years of age and are about to receive chemotherapy before a blood or bone marrow transplant may join this study. Chemotherapy drugs are used to prepare children for blood or bone marrow transplant. A few of the side effects of these drugs are nausea and vomiting. The purpose of this research study is to find out whether adding the drug olanzapine to standard antiemetics (drugs that help prevent vomiting) will be helpful in controlling chemotherapy-induced vomiting (CINV). To find this out, some of the participants in this study will receive olanzapine and others will receive a placebo (a substance that looks like olanzapine but does not have any active or medicinal ingredients). This will allow us to compare treatment of CINV with and without olanzapine.
Chronic Anterior Uveitis: A study testing levels of Humira in the blood for uveitis treatment
This study is for children: •Under 18 years of age • Must have chronic anterior uveitis • Must be starting Adalimumab (brand name Humira) for active uveitis...
This study is for children: •Under 18 years of age • Must have chronic anterior uveitis • Must be starting Adalimumab (brand name Humira) for active uveitis • No previous Adalimumab treatment can have occurred The purpose of this study is to understand whether blood levels of adalimumab (brand name: Humira) can be used to predict how well the uveitis will respond to adalimumab. Patients who participate will have blood samples and questionnaires collected at 2, 4, and 6 months after starting Adalimumab. Both a rheumatology and ophthalmology exam must be completed during those study visits.
Chronic Kidney Disease (CKD) and Iron Deficiency Anemia: A study on ferumoxytol treatment for CKD and iron deficiency
Iron deficiency anemia (IDA) is common in adults and children who have chronic kidney disease (CKD). Iron therapy is the usual treatment for IDA. Iron therapy has been shown to restore iron levels and help the body normalize levels of hemoglobin (the part of your blood that...
Iron deficiency anemia (IDA) is common in adults and children who have chronic kidney disease (CKD). Iron therapy is the usual treatment for IDA. Iron therapy has been shown to restore iron levels and help the body normalize levels of hemoglobin (the part of your blood that carries oxygen). The purpose of this research study is to evaluate the experimental drug ferumoxytol when compared to iron sucrose in children with CKD and iron deficiency anemia or who are at risk of developing IDA. Another purpose of the study is to see what happens to ferumoxytol in children's bodies and how the body clears the drug from blood. This is called pharmacokinetics (PK). PK blood samples will be obtained from some ferumoxytol-treated subjects who agree to have these samples taken. The study doctor may ask subjects to participate in this part of the study or inform them that PK samples are not needed.
Chronic Kidney Disease (CKD): A study on ferric citrate to maintain or improve iron and phosphate levels
The purpose of this placebo-controlled study is to determine how well ferric citrate will lower the level of FGF23, maintain or correct anemia and iron levels in the blood and maintain normal levels of serum phosphate in children with CKD stages 3 and 4 that is, between 15% and...
The purpose of this placebo-controlled study is to determine how well ferric citrate will lower the level of FGF23, maintain or correct anemia and iron levels in the blood and maintain normal levels of serum phosphate in children with CKD stages 3 and 4 that is, between 15% and 60% of normal function. Ferric citrate is an oral medication that is approved by the U.S. Food and Drug Administration (FDA) for use in adults with kidney disease to control serum phosphate levels, but it has not been approved for use in children. The use of ferric citrate is considered investigational, which means it has not been approved for the way it is being used in this study.
Chronic Migraine: A study on Erenumab treatment in children and adolescents
This study is for children and adolescents: • Currently enrolling males ages 6 -17 • Currently enrolling females ages 6-11 • Have over 15 headaches in a month (with at least 8 being classified as a migraine)...
This study is for children and adolescents: • Currently enrolling males ages 6 -17 • Currently enrolling females ages 6-11 • Have over 15 headaches in a month (with at least 8 being classified as a migraine) The purpose of the OASIS PEDIATRIC (CM) study is to evaluate the safety and effectiveness of a study medicine called erenumab to reduce the frequency of chronic migraine headaches. In this study, erenumab is being compared to a placebo, which allows researchers to evaluate the effects of the study medicine. Eligible subjects are agree to have two injections under the skin once a month, and be willing to follow all study instructions. Study participants will have approximately 8 monthly visits to the clinic, and if interested in the optional extension phase, there will be an additional 10 visits. The visits take about 2-3 hours.
Chronic Myeloid Leukemia (CML): A study on stopping TKI medication in children
To be eligible in this study, patients must be younger than 18 years of age and have been diagnosed with Chronic Myeloid Leukemia (CML). In this study, researchers would like to determine if children, adolescents, and young adults can stop taking their TKI medication and remain...
To be eligible in this study, patients must be younger than 18 years of age and have been diagnosed with Chronic Myeloid Leukemia (CML). In this study, researchers would like to determine if children, adolescents, and young adults can stop taking their TKI medication and remain in molecular remission. Researchers would also like to see if children, adolescents, and young adults who restart TKI medication will re-achieve molecular remission. Those who join this study will be asked to stop taking their TKI medication. If at any point during the study, the CML comes back (loss of molecular remission), participants will restart TKI medication.
Colectomy for Familial Adenomatous Polyposis (FAP): A study of tissue samples to understand bacteria in the gut
This study is currently enrolling patients who: • Are ages 7 to 20 years • Have been diagnosed with Familial Adenomatous Polyposis • Have been referred to colectomy within 12 months of surveillance colonoscopy...
This study is currently enrolling patients who: • Are ages 7 to 20 years • Have been diagnosed with Familial Adenomatous Polyposis • Have been referred to colectomy within 12 months of surveillance colonoscopy Familial Adenomatous Polyposis (FAP) is an inherited syndrome where polyps develop in the gut and will become cancerous. The purpose of this research study is to learn more about the relationship between bacteria in the gut and polyps. Being in this study involves the participant providing a stool sample and a blood sample. The parent/guardian will also give the study team permission to take tissue samples from their child's colon once it has been removed.
Colonoscopy with or without Polyposis: Tissue samples collected for research on cancer prevention and treatment
This study is currently enrolling patients who: • Are ages 1 month to 21 years • Have been diagnosed with polyposis, either clinically or through genetic testing with adenomas found on colonoscopy...
This study is currently enrolling patients who: • Are ages 1 month to 21 years • Have been diagnosed with polyposis, either clinically or through genetic testing with adenomas found on colonoscopy The main purpose of this study is to use normal tissues (not polyps) and non-normal tissues (polyps) to test candidate drugs or compounds against and ensure there is not significant toxicity to these normal cells. A study participant will have biopsies of tissue collected from different locations in their intestinal tract. The tissues will be stored in a way that allows cell to grow and multiply. The cell lines that potentially grow will be used to test the toxicity of the drugs or compounds that are being tested to see if they possibly help tissue affected by polyposis.
Congenital Diaphragmatic Hernia (CDH): A study in infants to evaluate the effectiveness of milrinone vs. placebo in improving heart and lung function and outcomes
This research study is being done to find out whether a drug called milrinone, when given to infants with congenital diaphragmatic hernia (CDH), will help the heart work better by supplying oxygen to the lungs and tissues. The main purpose of this initial pilot study is to...
This research study is being done to find out whether a drug called milrinone, when given to infants with congenital diaphragmatic hernia (CDH), will help the heart work better by supplying oxygen to the lungs and tissues. The main purpose of this initial pilot study is to understand better how milrinone works to help the heart and lungs give oxygen to the tissues and other organs in infants with CDH. We are hoping this pilot study will support and look at the possibility and safety of conducting a much larger study. The following things are part of this study: the study drug (milrinone), or placebo (sugar solution), head ultrasounds, maternal and baby medical chart review, and follow up questionnaires.
Cystic Fibrosis: A clinical trial of combination study drug therapy (VX-121/TEZ/D-IVA)
This is a clinical study to learn more about the safety and tolerability of VX-121/tezacaftor/deutivacaftor (VX-121/TEZ/D-IVA) in patients with Cystic Fibrosis. This study is open to patients who are 6 -11 years old. A clinical trial is a way to study new drugs and treatments...
This is a clinical study to learn more about the safety and tolerability of VX-121/tezacaftor/deutivacaftor (VX-121/TEZ/D-IVA) in patients with Cystic Fibrosis. This study is open to patients who are 6 -11 years old. A clinical trial is a way to study new drugs and treatments to see if they are safe. VX-121/TEZ/D-IVA is the combination study treatment that is being used for this study and is an investigational drug. "Investigational" means the drug is not approved by the United States Food and Drug Administration (FDA), the European Medicines Agency (EMA) in the European Union or health authorities in other countries and is still being tested for safety and effectiveness.
Depression: A study of fluoxetine (Prozac®) and escitalopram (Lexapro®) in teens and adolescents
This study is for teens: • Ages (12-18) • With depression diagnosis The goal of this study is to understand why the antidepressant medicines fluoxetine (Prozac®) and escitalopram...
This study is for teens: • Ages (12-18) • With depression diagnosis The goal of this study is to understand why the antidepressant medicines fluoxetine (Prozac®) and escitalopram (Lexapro®) work for some teens with depression and not for others. Every teen and their depression are unique, and there is no one-size fits all treatment. What researchers learn from this study could help the care team find the right treatment sooner, which could lead teens on a quicker path to recovery.
Desmoplastic Small Round Cell Tumor (relapsed, recurrent or refractory): A clinical study of ramucirumab in pediatric patients and young adults
This study is being done to learn more about the efficacy of the investigational drug ramucirumab in combination with low-dose cyclophosphamide and vinorelbine for the treatment of pediatric and young adult patients with Desmoplastic Small Round Cell Tumor (DSRCT).
This study is being done to learn more about the efficacy of the investigational drug ramucirumab in combination with low-dose cyclophosphamide and vinorelbine for the treatment of pediatric and young adult patients with Desmoplastic Small Round Cell Tumor (DSRCT).
Dystonia: A study using MRI images to potentially predict dystonia in children who experience low oxygen levels and brain injury at birth
There are two groups for this research study: Group 1 • Infants less than 30 days old...
There are two groups for this research study: Group 1 • Infants less than 30 days old • Have a diagnosis of Hypoxic-Ischemic Encephalopathy • Are receiving cooling therapy (hypothermia) • Already having a brain scan (MRI) as part of normal medical care Group 2 • Children who are 18 months - 7 years • Have a diagnosis of Dystonia • Already having a brain scan (MRI) as part of normal medical care The purpose of this research study is to gain information that will be used to help predict which children with low oxygen levels and brain injury at birth may go on to develop dystonia. This could allow for earlier treatments for at-risk children that could help improve their long-term development. This research study will add specialized Magnetic Resonance Imaging (MRI) images to the clinical MRI that babies are already receiving to get more information on their brain networks.
Eating Disorders: A study to understand brain response to Naltrexone in teens and young adults
Children's Mercy (Research Institute/Division of Adolescent Medicine and Clinical Pharmacology) is doing a research study to understand brain differences in the reward system of teens and young adults with eating disorders. The study involves a one-time dose of a medication...
Children's Mercy (Research Institute/Division of Adolescent Medicine and Clinical Pharmacology) is doing a research study to understand brain differences in the reward system of teens and young adults with eating disorders. The study involves a one-time dose of a medication called naltrexone. Naltrexone is used in many conditions in adults and children from autism to eating disorders. It is most known for preventing drug abuse in adults. In patients with eating disorders, it is used to reduce binge eating and purging (such as vomiting). The study team previous work showed that naltrexone helps some patients with eating disorders, but not all. The study team wants to understand why some patients respond and others don't.
Episodic migraine: A study on erenumab treatment in children and adolescent
This study is for children/teens: • 12-17 years old • Who have up to 14 headaches in a month (with at least 4 being classified as a migraine)...
This study is for children/teens: • 12-17 years old • Who have up to 14 headaches in a month (with at least 4 being classified as a migraine) The purpose of the OASIS PEDIATRIC (EM) study is to evaluate the safety and effectiveness of a study medicine called erenumab to reduce the frequency of episodic migraine headaches. In this study, erenumab is being compared to a placebo, which allows researchers to evaluate the effects of the study medicine. Participants have to be willing to have two injections under the skin once a month, and follow all study instructions. Study participants will have approximately 8 monthly visits to the clinic, and if interested in the optional extension phase, there will be an additional 10 visits. The visits take about 2-3 hours.
Functional Impairments: A study collecting data for researchers to find better treatments for children and adults who take specific medications.
This study is being done on child/adults: • 1 - 26 Years old • Patients that have schedule procedures that will involve the collection of CSF and blood...
This study is being done on child/adults: • 1 - 26 Years old • Patients that have schedule procedures that will involve the collection of CSF and blood Centrally Acting Molecules Study is a database that will collect information about how certain medications cross the blood-brain barrier in patients who have functional, spinal and or brain impairments. This research study is done to see how much medication crosses a barrier to get into fluid surrounding the brain and spinal cord. This research study involves collecting blood, left over cerebrospinal fluid(CSF) samples and data from standard care services that are a part of the care that the patient will already receive, including a lumbar puncture or surgery.
Functional Impairments: DNA samples collected and stored for future research.
This study is being done for children and adults: 1. One month to age 26 2. Have an impairment limiting their function. The primary purpose of this study is to provide a bank of specimens that will be used for future research involving the...
This study is being done for children and adults: 1. One month to age 26 2. Have an impairment limiting their function. The primary purpose of this study is to provide a bank of specimens that will be used for future research involving the response of medicines used to treat patients with disabilities commonly seen in the rehabilitation clinic (i.e. spasticity, tone management, cognitive impairment, etc.). Additionally, this will serve as a database of patient interested in participating in genetic studies in the future. This study is for a repository that will collect saliva or blood samples from many individuals with functional impairments to learn more about the way medicines interact with the body and the way genes affect this.
Gastroenterology Repository: A tool used to collect information for future and current research
This study is enrolling patients age 6 months to 23 years of age who have been seen by a Children's Mercy gastroenterologist or other providers and are undergoing any standard of care procedure or visit at which time samples could be collected for the repository.
This study is enrolling patients age 6 months to 23 years of age who have been seen by a Children's Mercy gastroenterologist or other providers and are undergoing any standard of care procedure or visit at which time samples could be collected for the repository.
Gastrointestinal symptoms: A study of Sucraid® treatment in babies to teens
This study is for children: -6 months to 17 years old -Who may have a diagnosis of CSID -Who have diarrhea, abdominal pain, gas/bloating, and/or nausea...
This study is for children: -6 months to 17 years old -Who may have a diagnosis of CSID -Who have diarrhea, abdominal pain, gas/bloating, and/or nausea. The goal of this study is to look at the effects of a medicine called Sucraid on gastrointestinal (GI) symptoms in children with different enzyme levels in their GI tract.
Gastrojejunal (G-J Tube): A study on megestrol to transition children to oral feeding
This study is being done for children: • 9 months to 9 years old • Who are dependent on g-tube for at least 80% of daily caloric intake • Have the motor and emotional skills necessary to eat by mouth...
This study is being done for children: • 9 months to 9 years old • Who are dependent on g-tube for at least 80% of daily caloric intake • Have the motor and emotional skills necessary to eat by mouth The goal of this study is to transition children from g-tube to all oral feeding. Study lasts 6 months and includes bi-weekly, remote coaching sessions, 4 in-person visits and a 50% chance of receiving megestrol, a medication used to stimulate appetite.
Germ Cell Tumors (GCT): A study comparing accelerated versus standard chemotherapy
Patients who are ages 11 to 45 years with germ cell tumors (GCT) may be eligible to participate in this study. In this study, researchers want to determine whether accelerated BEP (standard chemotherapy drugs Bleomycin, Etoposide, and cisPlatin) will be effective and well...
Patients who are ages 11 to 45 years with germ cell tumors (GCT) may be eligible to participate in this study. In this study, researchers want to determine whether accelerated BEP (standard chemotherapy drugs Bleomycin, Etoposide, and cisPlatin) will be effective and well-tolerated for patients with advanced GCTs. Researchers don't know yet if accelerated treatment is helpful in advanced GCTs and are hoping this study will answer that question.
Germ Cell Tumors (GCT): Monitoring patients with low risk GCT and using the drug carboplatin instead of cisplatin for patients with standard risk GCT.
Eligible patients must be diagnosed with having a germ cell tumor (GCT). Patients who are diagnosed with Stage I GCT may be eligible no matter the age. Those ages 0-25 who are diagnosed with Stage II-IV may be eligible depending on the location of the GCT. Researchers want to see...
Eligible patients must be diagnosed with having a germ cell tumor (GCT). Patients who are diagnosed with Stage I GCT may be eligible no matter the age. Those ages 0-25 who are diagnosed with Stage II-IV may be eligible depending on the location of the GCT. Researchers want to see if monitoring patients with low risk germ cell tumors after their tumor is removed is as effective as following surgery with chemotherapy and/or more surgery. Researchers also want to compare the effects of carboplatin with cisplatin on patients with standard risk germ cell tumors to find out which is better.
GI pathologies detection: A tool used to collect information for future developments.
This study is for patients: • Having a capsule endoscopy The study purpose is to collect PillCamTM SB3 videos/cases to support future development and validation of improved algorithms and technics for pathologies detection in the GI.
This study is for patients: • Having a capsule endoscopy The study purpose is to collect PillCamTM SB3 videos/cases to support future development and validation of improved algorithms and technics for pathologies detection in the GI.
Growth Hormone Deficienct (GHD): A study of an investigational test compared to a standard test to diagnose GHD
A study, for those ages 2 to <18 years old, to determine how effective a macimorelin stim test is compared to standard arginine and clonidine stim tests to diagnose kids as Growth Hormone Deficient (GHD).
A study, for those ages 2 to <18 years old, to determine how effective a macimorelin stim test is compared to standard arginine and clonidine stim tests to diagnose kids as Growth Hormone Deficient (GHD).
Hemodialysis Registry: A tool used to collect health information on children receiving hemodialysis for future research
The purpose of this research study is to continuously monitor outcomes in children around the world that are on hemodialysis. This registry should provide much needed information to doctors, nurses, dieticians, psychologists, social workers, and health care administrators, and...
The purpose of this research study is to continuously monitor outcomes in children around the world that are on hemodialysis. This registry should provide much needed information to doctors, nurses, dieticians, psychologists, social workers, and health care administrators, and help improve the wellbeing of children currently on HD. Being in this study involves having data collected from the child's medical record by a study team member and entered into an international database (registry). The data to be entered will include the child's date of birth, a unique study number, dates of service, and relevant medical information.
Hepatitis B Infection: A clinical study of tenofovir alafenamide in children and adolescents with long term hepatitis B infection
The purpose of this study is to see if Tenofovir Alafenamide is safe, tolerable, and effective in treating children and adolescents (2 to less than 18 years of age) with chronic (long term) hepatitis B virus infection.
The purpose of this study is to see if Tenofovir Alafenamide is safe, tolerable, and effective in treating children and adolescents (2 to less than 18 years of age) with chronic (long term) hepatitis B virus infection.
High Risk Medulloblastoma: A clinical study of elfornithine/DFMO as maintenance therapy after standard treatment
The purpose of this research study is to see how well the investigational drug, eflornithine HCl (DFMO) works for Medulloblastoma. An investigational drug is one that has not been approved by the U.S. Food and Drug Administration (FDA). This study will look at the ability of this...
The purpose of this research study is to see how well the investigational drug, eflornithine HCl (DFMO) works for Medulloblastoma. An investigational drug is one that has not been approved by the U.S. Food and Drug Administration (FDA). This study will look at the ability of this study drug to either keep the tumors in remission or, if for active tumors, for the tumors to respond to the treatment. It will also look at the safety and tolerability of DFMO.
High-Risk Neuroblastoma (NBL): A study of immunotherapy with or without DFMO after molecular therapy and induction chemotherapy in newly diagnosed patients
In this study we are testing the effectiveness of adding an experimental agent called DFMO to the standard immunotherapy for high-risk neuroblastoma. This portion of the study will be a randomized trial (like flipping a coin). We will use a computer system to randomly decide...
In this study we are testing the effectiveness of adding an experimental agent called DFMO to the standard immunotherapy for high-risk neuroblastoma. This portion of the study will be a randomized trial (like flipping a coin). We will use a computer system to randomly decide which group you will be placed in. Your physicians will not be able to help decide which group you will be placed in. Half of subjects, Group A, will receive standard immunotherapy with Dinutuximab, GM-CSF, and Isotretinoin. The other half of subjects, Group B, will receive standard immunotherapy with Dinutuximab, GM-CSF, Isotretinoin in combination with the study drug DFMO. We cannot guarantee which group you will be placed in. At the end of the 6 cycles of immunotherapy, all patients (both Arm A and Arm B) will receive DFMO twice a day for 730 days as maintenance therapy.
Hodgkin Lymphoma: A clinical study of ipilimubab, nivolumab and brentuximab vedotin in relapse/refractory hodgkin lymphoma
This study is being done to answer the following question: What effects, good and/or bad, do two different combinations of multiple drugs: brentuximab vedotin, with either one or two immune activating drugs: ipilimumab, and nivolumab, have on relapsed or refractory Hodgkin...
This study is being done to answer the following question: What effects, good and/or bad, do two different combinations of multiple drugs: brentuximab vedotin, with either one or two immune activating drugs: ipilimumab, and nivolumab, have on relapsed or refractory Hodgkin lymphoma? We are doing this study because we want to find out which combination approach is better or worse for this type of relapsed or refractory Hodgkin's Lymphoma.
Hyperfine MRI: A study of a portable MRI machine
This study is for patients: • Between the ages of 0-22 years • Can have a portable MRI exams at specific time points. The purpose of this research study is to capture, compare and assess the quality and performance of Hyperfine MRI, or...
This study is for patients: • Between the ages of 0-22 years • Can have a portable MRI exams at specific time points. The purpose of this research study is to capture, compare and assess the quality and performance of Hyperfine MRI, or low-field strength MRI, imaging. Additionally, for patients that have other clinically available imaging techniques, researchers will compare Hyperfine MRI images to clinical exams such as traditional MRI, CT and US.
Hypophosphatasia (HPP) Registry: A tool used to collect information about HPP
This study aims to enroll patients from the ages of 1 month to 99 years old with a clinical diagnosis of Hypophosphatasia (HPP). Hypophosphatasia is a rare, serious, and possibly fatal genetic disorder. The purpose of this research study is to collect information on patients...
This study aims to enroll patients from the ages of 1 month to 99 years old with a clinical diagnosis of Hypophosphatasia (HPP). Hypophosphatasia is a rare, serious, and possibly fatal genetic disorder. The purpose of this research study is to collect information on patients who have HPP. HPP is a rare disease so there is not a lot of information known about the condition or how it changes as you get older. By collecting information from HPP patients we hope to be able to help other patients in the future.
Hypophosphatasia (HPP): A study of how the body reacts to asfotase alfa treatment over time
This study collects observational Standard of Care (SOC) data in patients currently receiving asfotase alfa for Hypophosphatasia or about to start treatment with asfotase alfa as determined by their doctor. Some blood samples deemed appropriate by the participant's doctor will be...
This study collects observational Standard of Care (SOC) data in patients currently receiving asfotase alfa for Hypophosphatasia or about to start treatment with asfotase alfa as determined by their doctor. Some blood samples deemed appropriate by the participant's doctor will be drawn at their regular clinic visits alongside any blood draws the participant may already have. This additional blood will be used for research purposes in the context of this observational study. The purpose of this research study is to do the following: - Collect information on patients who have HPP - Examine the body's immune response to the medication the participant takes for their disease - Examine how the participant's body reacts to their medication for HPP over time.
Improve treatment: Understanding the amount of fluoxetine, escitalopram, pimozide, pantoprazole, and lansoprazole that reaches the brain in teens and young adults
This study is being done for teens and young adults: • Ages 12 to 21 • Currently taking or recently stopped taking fluoxetine, escitalopram, pimozide, pantoprazole, or lansoprazole • Able to have a special brain scan (MRI), a blood draw, and answer brief questionnaires...
This study is being done for teens and young adults: • Ages 12 to 21 • Currently taking or recently stopped taking fluoxetine, escitalopram, pimozide, pantoprazole, or lansoprazole • Able to have a special brain scan (MRI), a blood draw, and answer brief questionnaires The goal of this study is to measure the amount of certain medications in the brain.
Inflammatory Bowel Disease (IBD): A registry to improve care for children and young adults with Crohn's or colitis
All patients with a clinical diagnosis of Inflammatory Bowel Disease, regardless of age, gender, race or ethnicity will be recruited. This is a multi-site registry study through Improve Care Now. Our purpose is to transform the health, care and costs for all children and...
All patients with a clinical diagnosis of Inflammatory Bowel Disease, regardless of age, gender, race or ethnicity will be recruited. This is a multi-site registry study through Improve Care Now. Our purpose is to transform the health, care and costs for all children and adolescents with Crohn's disease and ulcerative colitis (Inflammatory Bowel Disease or IBD) by building a sustainable collaborative chronic care network.
Inflammatory Bowel Disease: Transitioning to Adult Care Survey
The purpose of this research study is to understand the things that make it difficult for a young person living with IBD to transition from a pediatric GI doctor to an adult GI doctor. By being in this study, it may help researchers learn how to better work with patients moving...
The purpose of this research study is to understand the things that make it difficult for a young person living with IBD to transition from a pediatric GI doctor to an adult GI doctor. By being in this study, it may help researchers learn how to better work with patients moving to adult health care.
Intermittent Exotropia (IXT): A study on full time eye patching
This study is being done for children: • Between 3 and 8 years old • Have intermittent exotropia (eyes that turn out some of the time) • Have good vision...
This study is being done for children: • Between 3 and 8 years old • Have intermittent exotropia (eyes that turn out some of the time) • Have good vision • Be wearing glasses for at least 2 weeks if needed The goal of this study is see if wearing a patch during 3 months all the time while awake, will help children with Intermittent Exotropia (IXT) to improve eye misalignment (keep their eyes straight); better than not wearing a patch.
Juvenile Idiopathic Arthritis (JIA): A study of tears and blood markers for JIA and uveitis
The main reason for this research study is to help researchers to learn more about children with juvenile idiopathic arthritis (JIA) and/or uveitis (inflammation of the eye), and to help us identify who is at the greatest risk for uveitis development. Researchers also hope to...
The main reason for this research study is to help researchers to learn more about children with juvenile idiopathic arthritis (JIA) and/or uveitis (inflammation of the eye), and to help us identify who is at the greatest risk for uveitis development. Researchers also hope to discover whether certain markers in a child's tears and blood can better help to determine one's risk for developing uveitis. Investigators hope that early detection of uveitis, regular monitoring for eye disease and complications, and appropriate, timely therapy can improve the quality of life and long-term outcomes of children with arthritis and/or uveitis.
Juvenile Spondyloarthritis: A study to Compare the Likelihood of Disease Flare Associated with Continuing, Tapering, or Stopping Medication in Children
• 8-21 years of age • patients must have been diagnosed with Juvenile Spondyloarthritis • patients must be currently taking a TNFi medication (Adalimumab, Certolizumab, Etanercept, Golumumab, Infliximab) • patients must not have active disease for a minimum of 6 months...
• 8-21 years of age • patients must have been diagnosed with Juvenile Spondyloarthritis • patients must be currently taking a TNFi medication (Adalimumab, Certolizumab, Etanercept, Golumumab, Infliximab) • patients must not have active disease for a minimum of 6 months Patients who participate will be randomly assigned to either continue their current medication schedule, taper down their current medication schedule, or completely stop their medication. The patient will follow their routine care schedule approximately every 3 months, and monitored for any flare of disease.
Kidney Disease (FSGS, Alport syndrome, IgA nephropathy): A clinical trial of sparsentan treatment for kidney diseases
Travere Therapeutics, Inc. (the Sponsor) is running a research study to see if a study drug, not yet approved for use by the United States Food and Drug Administration (FDA), named sparsentan, will help in the treatment of kidney diseases and if it is safe to use in people.
Travere Therapeutics, Inc. (the Sponsor) is running a research study to see if a study drug, not yet approved for use by the United States Food and Drug Administration (FDA), named sparsentan, will help in the treatment of kidney diseases and if it is safe to use in people.
Kidney Failure Registry: A tool used to collect health information on children less than 21 years old with kidney failure for future research
The purpose of this research study is to gather data on all children less than 21 years of age with kidney failure who receive treatment in the United States, Canada, and Mexico in order to evaluate patient characteristics and outcomes of treatment used in pediatric patients in...
The purpose of this research study is to gather data on all children less than 21 years of age with kidney failure who receive treatment in the United States, Canada, and Mexico in order to evaluate patient characteristics and outcomes of treatment used in pediatric patients in North America. Being in this study involves the collection of data from the person's medical record by a qualified member of the nephrology team. Children who are entered into this study will not have procedures or treatments which are different from those routinely performed as part of their ongoing medical care. The child's date of birth, a unique study number, dates of service, and relevant medical information will be collected on special computerized forms. These forms will be transmitted by computer to EMMES (a data collection company used by NAPRTCS). The data transmission process is secured for patient confidentiality.
Leukemia (B-cell acute lymphoblastic leukemia (B-ALL), Mixed Phenotype Acute Leukemia (MPAL), and B-Lly: A clinical trial adding study treatment to standard treatment
Those who are ages 1 to 25 and diagnosed with High Risk B-ALL, MPAL, or B-LLy are eligible to join this study. We would like to collect information about participants' leukemia and the effects of the first two phases of treatment, called Induction and Consolidation. For those...
Those who are ages 1 to 25 and diagnosed with High Risk B-ALL, MPAL, or B-LLy are eligible to join this study. We would like to collect information about participants' leukemia and the effects of the first two phases of treatment, called Induction and Consolidation. For those diagnosed with High Risk B-ALL, we would like to see if adding inotuzumab ozogamicin to standard chemotherapy maintains or improves outcomes. For those diagnosed with MPAL, we would like to determine the outcomes to treatment using High Risk B-ALL therapy. For those diagnosed with B-LLy, we would like to determine the outcomes to treatment using High Risk B-ALL therapy.
Leukemia: A clinical study of SNDX-5613 and chemotherapy in relapsed/refractory leukemia with a KMT2A/MLL Gene arrangement or nucleophosmin 1 mutation (mNPM1)
This study is being done to: find the best dose of the study drug, SNDX-5613, in combination with chemotherapy, for patients with leukemia; find out what side effects happen when people take the study drug; look at whether this drug may slow the progression of cancer; learn...
This study is being done to: find the best dose of the study drug, SNDX-5613, in combination with chemotherapy, for patients with leukemia; find out what side effects happen when people take the study drug; look at whether this drug may slow the progression of cancer; learn whether there is something in the blood that can be measured to see how the therapy will work and/or how well future patients may respond to the therapy; and investigate how the study drug is processed in the body (this is called pharmacokinetics).
Low-Grade Glioma: A Study of the Drugs Selumetinib vs. Carboplatin/Vincristine
Study participants must be ages 2 through 21 and newly diagnosed or Previously Untreated Low-Grade Glioma (LGG). In this study, researchers want to if selumetinib works just as well as the standard treatment using carboplatin and vincristine (called CV) for patients with LGG...
Study participants must be ages 2 through 21 and newly diagnosed or Previously Untreated Low-Grade Glioma (LGG). In this study, researchers want to if selumetinib works just as well as the standard treatment using carboplatin and vincristine (called CV) for patients with LGG. Selumetinib is a drug that works by blocking a protein (a basic building block of the human body) that lets cancer cells grow without stopping. Early studies have shown that selumetinib was successful in treating patients with LGG that came back after a first attempt at treatment. However, the use of selumetinib in patients with previously-untreated LGG is considered experimental because selumetinib has not yet been FDA approved for this type of cancer.
Lymphoblastic Leukemia/Lymphoma (LL): A clinical study of everolimus with nelarabine, cyclosphosphamide, and etoposide for relapsed T-cell leukemia and lymphoma
The purpose of this research study is to learn if a drug called everolimus is safe when used together with standard chemotherapy in treating patients who have relapsed or refractory disease.
The purpose of this research study is to learn if a drug called everolimus is safe when used together with standard chemotherapy in treating patients who have relapsed or refractory disease.
Magnetic Resonance Imaging (MRI): Low-field Bedside MRI in Pediatric and Neonatal ECMO patients
This study is for: -Patients aged 0-17 currently undergoing Extracorporeal Membrane Oxygenation (ECMO) and admitted to the NICU, PICU, or CICU at Children's Mercy...
This study is for: -Patients aged 0-17 currently undergoing Extracorporeal Membrane Oxygenation (ECMO) and admitted to the NICU, PICU, or CICU at Children's Mercy The purpose of this study is: -To determine the feasibility and rates of serious adverse events of performing a portable MRI on subject's on ECMO. -To power calculation for subsequent efficacy study on sensitivity and specificity for detection of acute brain injury utilizing hyperfine MRI versus conventional MRI, CT and/or US.
Mold Infections: A study to lower risk and improve diagnosis
The purpose of this research study is to explore a lower risk and more accurate way to diagnose invasive mold infections. The study team wants to see if an existing test called a biomarker would work and also see if some newer blood tests (which test for DNA of molds or RNA...
The purpose of this research study is to explore a lower risk and more accurate way to diagnose invasive mold infections. The study team wants to see if an existing test called a biomarker would work and also see if some newer blood tests (which test for DNA of molds or RNA produced by the patient in response to a mold presence) would work. Another purpose of this study is to compare the different ways that care teams manage patients who might have a mold infection in their lungs so as to better understand how these different care decisions might impact patients. To be eligible for this study, patient must have at least one condition with a known higher risk of mold infection, had lung images showing a nodule in the last 96 hours, be older than 120 days and younger than 22 years, and be able to comply with the study requirements and blood sampling.
Myelomonocytic Leukemia: A clinical study of trametinib in relapsed or refractory juvenile myelomonocytic leukemia
Recent developments in laboratories around the world have led to discoveries about the changes in the genes of patients with Juvenile Myelomonocytic Leukemia (JMML). The identification of these changes (also called mutations) may be helpful in the treatment of JMML. Trametinib is...
Recent developments in laboratories around the world have led to discoveries about the changes in the genes of patients with Juvenile Myelomonocytic Leukemia (JMML). The identification of these changes (also called mutations) may be helpful in the treatment of JMML. Trametinib is a drug that works to target some of these mutations commonly found in JMML cells. This study looks at how well trametinib works when given to children and young adults whose JMML comes back (recurs) or does not respond to therapy (is refractory). This study is for patients who are older than 1 month old but less than 22 years old.
Neonatal Diseases: A study on genetic risk factors
More and more often, doctors and researchers are understanding that there may be a genetic reason why some infants have a greater risk of becoming sick from certain diseases. Some of these diseases are directly or indirectly caused by germs, including bacteria, viruses or fungi...
More and more often, doctors and researchers are understanding that there may be a genetic reason why some infants have a greater risk of becoming sick from certain diseases. Some of these diseases are directly or indirectly caused by germs, including bacteria, viruses or fungi. If we can identify the genetic links that make some infants more likely to become sick from these germs, we may be able to target treatment to these higher-risk babies in the future, which could prevent severe illness and even death. The study team is looking for genetic differences that might make certain babies more likely to become sick from Necrotizing Enterocolitis (NEC), Bronchopulmonary Dysplasia (BPD) and specific blood infections (Group B Strep and Herpes Simplex) during the birth to 12-month-old period.
Neonatal Enterovirus and Human Parechovirus Viral Sepsis: A study on observing the infection in neonates.
This study is being done for children: • ≤ 30 days old • Suspected or proven neonatal viral sepsis • Only patients currently admitted at Children's Mercy may participate in this study...
This study is being done for children: • ≤ 30 days old • Suspected or proven neonatal viral sepsis • Only patients currently admitted at Children's Mercy may participate in this study. The main reason for this research study is to get a better understanding of what causes neonatal viral sepsis and to assess the impact of the infection on the babies' health. Viruses called enterovirus (EV) or human parechovirus (HPeV) are very common in the population and can cause neonatal viral sepsis. By gaining a better understanding of the condition, researchers hope this information can be used to guide diagnosis and treatment of babies with neonatal viral sepsis in the future. Researchers are asking your baby to be in this research study because your baby was brought to the hospital with symptoms of neonatal viral sepsis.
Nephrotic Syndrome Registry: A tool used to collect health information and laboratory samples to learn more about kidney diseases
Childhood onset nephrotic syndrome is a condition which affects the kidneys, causing them to leak protein from the blood into the urine. Nephrotic syndrome is a disease that can improve (remission) and worsen (relapse) at different times throughout childhood. NEPTUNE is a large...
Childhood onset nephrotic syndrome is a condition which affects the kidneys, causing them to leak protein from the blood into the urine. Nephrotic syndrome is a disease that can improve (remission) and worsen (relapse) at different times throughout childhood. NEPTUNE is a large North American study consisting of over 500 individuals and more than 5 years of research. By collecting health information and laboratory samples, our goal is to learn more about these kidney diseases and find better ways to prevent and treat people with nephrotic syndrome. New knowledge will be shared with researchers and the public.
Neuroblastoma: A clinical trial of elfornithine/DFMO and etoposide for relapsed/refractory neuroblastoma
The purpose of this research study is to evaluate the investigational drug eflornithine HCl (DFMO) along with etoposide for Neuroblastoma. An investigational drug is one that has not been approved by the U.S. Food and Drug Administration (FDA). Currently there are no standard of...
The purpose of this research study is to evaluate the investigational drug eflornithine HCl (DFMO) along with etoposide for Neuroblastoma. An investigational drug is one that has not been approved by the U.S. Food and Drug Administration (FDA). Currently there are no standard of care treatments for patients with neuroblastoma in remission or with relapsed/refractory neuroblastoma. This study will look at the ability of this study drug to either keep the tumors in remission or, if for active tumors, for the tumors to respond to the treatment. The study will also look at the safety and tolerability of DFMO.
Neuroblastoma: A study of DFMO as maintenance therapy
The purpose of this research study is to evaluate the investigational drug eflornithine HCl (DFMO) for neuroblastoma that is in remission (cured according to medical tests). An investigational drug is one that has not been approved by the U.S. Food and Drug Administration (FDA)...
The purpose of this research study is to evaluate the investigational drug eflornithine HCl (DFMO) for neuroblastoma that is in remission (cured according to medical tests). An investigational drug is one that has not been approved by the U.S. Food and Drug Administration (FDA). This study will look at the ability of this study drug to keep tumors in remission and will also look at the safety and tolerability of DFMO.
Neurofibromatosis Type 1 (NF1) Associated Low-Grade Glioma (LGG) in Children: A study of selumetinib versus standard treatment
Study participants must be ages 2 through 21, and have been diagnosed with Neurofibromatosis type 1 (NF1) and a cancer called low-grade glioma (LGG). In this study, researchers would like to see if a drug called Selumetinib works just as well as the standard treatment for NF1...
Study participants must be ages 2 through 21, and have been diagnosed with Neurofibromatosis type 1 (NF1) and a cancer called low-grade glioma (LGG). In this study, researchers would like to see if a drug called Selumetinib works just as well as the standard treatment for NF1-associated LGG. Researchers would also like to see if selumetinib is better than the standard treatment in improving vision. Selumetinib is a drug that works by blocking proteins (a basic building block of the human body) needed for cell growth and killing cancer cells. The use of selumetinib in the treatment of previously untreated NF1-associated LGG is considered experimental because selumetinib has not yet been FDA approved for this type of cancer.
NGGCT Brain Cancer (Non-Germinomatous Central Nervous System Germ Cell Tumor): A clinical trial of radiation therapy compared to standard treatment
This clinical study is available for those ages 3 to 30 who have been diagnosed with a type of brain cancer called NGGCT and your tumor is "localized". The term localized means that the tumor has not spread to other parts of your brain or body. One goal for this study is to see...
This clinical study is available for those ages 3 to 30 who have been diagnosed with a type of brain cancer called NGGCT and your tumor is "localized". The term localized means that the tumor has not spread to other parts of your brain or body. One goal for this study is to see if treatment with RT to the spine and a portion of the brain works just as well as the standard treatment for people with localized NGGCT whose disease responds well to induction chemotherapy, or who have no signs of disease following surgery. The other goal is to see if we can lower the number of relapses in the spine for patients who receive RT to the spine and a portion of the brain, as compared to patients on previous studies who received RT to a portion of the brain only. Treatment is divided into 2 phases of therapy. In the first phase, participants will receive induction chemotherapy. In the second phase, participants will receive RT or additional chemotherapy plus RT. Some people on this study will also have surgery before the second phase of treatment to remove any tumor that remains after the first phase of treatment.
NICU Babies: Abdominal Ultrasound vs. X-Ray to Diagnose Necrotizing Enterocolitis (NEC)
This study is being done for: • NICU babies • Infants with concern for Necrotizing Enterocolitis (NEC) The purpose of this observational research study is to determine the clinical usefulness of bowel ultrasound (BUS) for...
This study is being done for: • NICU babies • Infants with concern for Necrotizing Enterocolitis (NEC) The purpose of this observational research study is to determine the clinical usefulness of bowel ultrasound (BUS) for the evaluation of necrotizing enterocolitis (NEC) in diverse NICU settings.
Non-infectious Uveitis: A study trying to predict the response of Methotrexate on non-infectious uveitis using eye testing
This study is for those who are: • less than 18 years of age • patients must have been diagnosed with non-infectious uveitis • patients must be starting Methotrexate for treatment of non-infectious uveitis...
This study is for those who are: • less than 18 years of age • patients must have been diagnosed with non-infectious uveitis • patients must be starting Methotrexate for treatment of non-infectious uveitis The main reason for this research study is to improve the way of selecting treatment for Non-infectious uveitis (NIU) patients who started to have NIU as a child. The study team will look at several different items to improve this process including demographics, eye exams, physical exams, imaging results, blood test results, and other potential risk factors. Pediatric NIU leads to vision-threatening complications or permanent vision loss in 50% of affected children. Methotrexate is the usual first chosen medication that works by suppressing the immune system.
NTRK gene tumors: A study of LOXO-101 (larotrectinib) treatment for tumors
This study is for children This study is for children, adolescents, and young adults who: • Are 12 months- 21 years old • Have solid tumors that are not responding to therapy, or have come back, and also have NTRK gene fusion proteins • Are able to swallow pills...
This study is for children This study is for children, adolescents, and young adults who: • Are 12 months- 21 years old • Have solid tumors that are not responding to therapy, or have come back, and also have NTRK gene fusion proteins • Are able to swallow pills The medicine that is used in the study, LOXO-101 (larotrectinib), has been approved by the FDA for children with NTRK gene tumor changes because it has shown to block the growth of cancer cells. It is taken by mouth for up to 26 months. The goals of the study are to find out what effects the drug has on the child's tumor, evaluate the drug's side effects, and to learn more about how the child's body handles the drug.
Parenting Teens Videos & Toolkit: Caregiver Survey
This study aims to get feedback from parents and caregivers about a series of videos they watch about parenting teenagers. Those who can participate are: • A parent or caregiver who is willing to: 1. Watch a series of 3-5 minute videos (in total lasting ~40 minutes) and...
This study aims to get feedback from parents and caregivers about a series of videos they watch about parenting teenagers. Those who can participate are: • A parent or caregiver who is willing to: 1. Watch a series of 3-5 minute videos (in total lasting ~40 minutes) and 2. Complete online surveys. Researchers are providing education about steps parents can take to improve the health and safety of their children as they become teens. The aim is to help parents learn what steps can be taken before a crisis develops with their teenager. The "Prepped and Ready" video presentations provides a brief overview on the following topics, with an emphasis on suicide prevention: -Eating disorder prevention -Home safety (including storage of firearms, medications, etc.) -Self-care -Substance use (including vaping) -Suicide prevention To learn if this education is helpful, researchers hope to learn about parents experience of attending the talk, and what parents learn. Researchers also want to understand what steps parents may take after the talk and what barriers they encounter.
Peanut Allergy: A study on a skin patch called ViaskinTM Peanut.
This study is being done for children: • Between 4-7 years old • With diagnosis of Peanut Allergy • Can use the skin patch for 1 year (12 months)...
This study is being done for children: • Between 4-7 years old • With diagnosis of Peanut Allergy • Can use the skin patch for 1 year (12 months) The main purpose of this study is to learn how well ViaskinTM Peanut works and how safe it is compared with a placebo patch over a 12-month study treatment period in children aged 4 to 7 years with peanut allergy. A placebo patch is an identical patch with inactive material that looks like the ViaskinTM Peanut patch, but does not have any active study drug. Researchers use a placebo to see if the ViaskinTM Peanut patch works better or is safer than taking nothing.
Pediatric Heart Disease: A remote monitoring study exploring parental & healthcare team experience using a wearable device
This study is for children who fit the following criteria as well as their parents/guardians: • Who are less than 2 years of age • Who have congenital heart disease...
This study is for children who fit the following criteria as well as their parents/guardians: • Who are less than 2 years of age • Who have congenital heart disease • Who are monitored at home with CHAMP App(R) The purpose of this research study is to find out what parents think about using the Pediarity System, which includes the Gabi Wi-Fi Monitor and Gabi Band. The band goes around the child's upper arm. This device does not alert for values that are out of range. For this study, the healthcare team will only see the values from the Pediarity system after the device has been turned back in.
Pediatric Joint Hypermobility and Ehlers Danlos Syndrome: Patient quality of life and symptoms survey
This study is for children: • 5-22 years of age • Patient must have Pediatric Joint Hypermobility or Ehlers Danlos Syndrome • Patient must be seen in the EDS clinic at Children's Mercy or the Rheumatology clinic with Dr. Jones...
This study is for children: • 5-22 years of age • Patient must have Pediatric Joint Hypermobility or Ehlers Danlos Syndrome • Patient must be seen in the EDS clinic at Children's Mercy or the Rheumatology clinic with Dr. Jones This study is being done to understand what other conditions are commonly seen in patients who have Pediatric Joint Hypermobility or Ehlers Danlos Syndrome. Surveys are sent to families asking about patient pain levels, quality of life, fatigue, and gastrointestinal symptoms. The study team will also review the patient's chart to see what other diagnoses are affecting the patient alongside joint hypermobility and Ehlers Danlos.
Perinatal Research Biorepository (PRB) Registry: A tool used to collect biological samples during pregnancy for future research
The goal of the Biorepository is to collect, store, and dispense de-identified biological samples with health information to only approved researchers within the hospital network. Any families who are over the age of 18, English-speaking, and have a planned delivery at Children's...
The goal of the Biorepository is to collect, store, and dispense de-identified biological samples with health information to only approved researchers within the hospital network. Any families who are over the age of 18, English-speaking, and have a planned delivery at Children's Mercy and collaborating institutions (University of Kansas Medical Center and AdventHealth Shawnee Mission) can donate remaining biological samples from scheduled clinical procedures at any time of pregnancy.
Peritoneal Dialysis Registry: A tool used to collect health information on children receivin peritoneal dialysis for future research
The purpose of this research study is to continuously monitor outcomes in children around the world that are on peritoneal dialysis. This registry should provide much needed information to doctors, nurses, dieticians, psychologists, social workers, and health care administrators...
The purpose of this research study is to continuously monitor outcomes in children around the world that are on peritoneal dialysis. This registry should provide much needed information to doctors, nurses, dieticians, psychologists, social workers, and health care administrators, and help improve the wellbeing of children currently on PD. Being in this study involves having data collected from the child's medical record by a study team member and entered into an international database (registry). The data to be entered will include the child's date of birth, a unique study number, dates of service, and relevant medical information.
Ph+ ALL: A study of adding Imatinib to two different chemotherapy treatments in Children and Young Adults with Philadelphia Chromosome-Positive Acute Lymphoblastic Leukemia
This study is available for those who are 1 through 21 and have been diagnosed with a form of Acute Lymphoblastic Leukemia (ALL) called Philadelphia chromosome positive (Ph+) ALL or ABL-class fusion positive ALL. The overall goal of this study is to determine if using a less...
This study is available for those who are 1 through 21 and have been diagnosed with a form of Acute Lymphoblastic Leukemia (ALL) called Philadelphia chromosome positive (Ph+) ALL or ABL-class fusion positive ALL. The overall goal of this study is to determine if using a less toxic chemotherapy in combination with a drug called imatinib, can effectively treat standard risk Ph+ ALL and standard risk ABL-class fusion positive ALL.
Polycystic Kidney Disease (PKD): Blood, urine, and information collected and stored for research on better understanding PKD
This study is for ages 4-18. EPOC is an observational cohort of young patients with early ADPKD. A repository or biological samples and kidney imaging from these patients will be made available to the PKD research community to discover new prognostic biomarkers. Children...
This study is for ages 4-18. EPOC is an observational cohort of young patients with early ADPKD. A repository or biological samples and kidney imaging from these patients will be made available to the PKD research community to discover new prognostic biomarkers. Children diagnosed with PKD and undiagnosed siblings of PKD participants may be enrolled
Prader-Willi Syndrome: A study of a nasal spray called Carbetocin and its potential to slow down hunger
This study is for people: • Who are ages 5-21 • With Willi syndrome (PWS) diagnosis...
This study is for people: • Who are ages 5-21 • With Willi syndrome (PWS) diagnosis The purpose of this research study is to investigate the efficacy and safety of carbetocin nasal spray for a symptom of PWS called hyperphagia, or increased appetite. Carbetocin is related to a hormone called oxytocin, which is involved in regulating hunger, bodyweight, and energy use. Low levels of oxytocin in the brain have been found in PWS. Carbetocin may help reduce the hyperphagia caused by PWS.
Prediabetes/Diabetes, Polycystic Ovary Syndrome (PCOS), Rapid Weight Gain, and/or Insulin Resistance: A study of metformin in youth ages 7 to 21 years old
Researchers at Children's Mercy want to learn more about Metformin. Metformin is a pill dosed every day to treat things like insulin resistance and diabetes. The purpose of this study is to learn how Metformin is processed in the body and to discover how to best dose Metformin in...
Researchers at Children's Mercy want to learn more about Metformin. Metformin is a pill dosed every day to treat things like insulin resistance and diabetes. The purpose of this study is to learn how Metformin is processed in the body and to discover how to best dose Metformin in children. This study is for children currently taking Metformin between the ages 7 to 21 years old.
Primary Mediastinal B-cell lymphoma (PMBCL): A study combining nivolumab and chemo-immunotherapy as a treatment option
This study is for children: • 2 years old and older • Received a diagnosis of primary mediastinal B-cell lymphoma (PMBCL)...
This study is for children: • 2 years old and older • Received a diagnosis of primary mediastinal B-cell lymphoma (PMBCL) The goal of this study is to compare the effects, good and/or bad, of nivolumab with chemo-immunotherapy versus chemo-immunotherapy alone. In this study children will get 1 of 2 treatment plans.
Rare/Unknown Genetic Diseases: A study of genetics in children to understand unknown or rare conditions and find treatments
Thousands of genetic diseases affecting children remain undiscovered and untreatable. Children's Mercy is in the unique position of having the technology, scientists, physicians, as well as patient volume and diversity to address this challenge...
Thousands of genetic diseases affecting children remain undiscovered and untreatable. Children's Mercy is in the unique position of having the technology, scientists, physicians, as well as patient volume and diversity to address this challenge. Studying the genetics of thousands of children will help us make rapid progress against these unknown diseases and lead the way for future children and families in need. Genomic Answers for Kids is a rich resource for researchers studying genetic conditions, leading to answers and new treatments for children. Our progress on Genomic Answers for Kids relies on clinicians to identify possible participants in the program and for patients to volunteer to take part. Although all of today's participants may not directly benefit, they will help future children and families in need. With each participant's help, we will find genomic answers for kids. We are seeking children who may have a genetic condition or a genetic component to their symptoms, as well as their families.
Relapsed and Refractory Solid Tumors: A clinical study of nabpaclitaxel and gemcitabine treatment
This is a study of a drug called nab-paclitaxel used together with gemcitabine. Nab-paclitaxel has not been approved for use by the FDA for this use and is considered an investigational drug. "Investigational" means that it is still not fully tested because it has not been...
This is a study of a drug called nab-paclitaxel used together with gemcitabine. Nab-paclitaxel has not been approved for use by the FDA for this use and is considered an investigational drug. "Investigational" means that it is still not fully tested because it has not been approved for this use in children. We are using nab-paclitaxel because it seems to work against cancer in test tubes and animals, and in some patients. Nab-paclitaxel is FDA-approved to treat adults with a variety of cancers including pancreatic, breast, lung ovarian, and melanoma. It is approved to be given in combination with gemcitabine for adults with pancreatic cancer. Nab-paclitaxel has been tested in children but not in combination with gemcitabine. Because it is not yet approved for children there is a lot that we do not know about it yet.
Remote CaRe: An in-home cardiac rehabilitation study for adolescents with congenital heart disease
This study is for: • Adolescents, aged 12-19 years old • Diagnosed with one of three congenital heart diseases: Fontan physiology, Tetralogy of Fallot, or D-Transposition of the great arteries"...
This study is for: • Adolescents, aged 12-19 years old • Diagnosed with one of three congenital heart diseases: Fontan physiology, Tetralogy of Fallot, or D-Transposition of the great arteries" This study aims to see if 12-weeks of in-home cardiac rehab (exercise training) using live video conferencing software can increase physical fitness in adolescents with congenital heart disease. To learn more about this study please watch this video: https://www.youtube.com/watch?v=2oOt5lTL1ow You can call or text us at 816-764-5605
Respiratory syncytial virus (RSV): An study treatment for RSV
This study is being done for children: • Ages 28 days to 36 months • Can be in the study for around 29 days • Hospitalized or non-hospitalized...
This study is being done for children: • Ages 28 days to 36 months • Can be in the study for around 29 days • Hospitalized or non-hospitalized The purpose of this research study is to obtain information on how effective and how safe the Study Drug is in treating children with RSV infection and to determine the dose of Study Drug that works best. This will be done by checking how the children are feeling, and any side effects or changes to the children's laboratory tests.
Severe respiratory illness: Exposure to an antibiotic called Bactrim
This study is for children: 1. 3-18 years old 2. Who had greater than or equal to 6 days of exposure to treatment doses of Bactrim 3. Who developed severe respiratory failure leading to an ICU admission with need for a ventilator and/or ECMO...
This study is for children: 1. 3-18 years old 2. Who had greater than or equal to 6 days of exposure to treatment doses of Bactrim 3. Who developed severe respiratory failure leading to an ICU admission with need for a ventilator and/or ECMO The goal of this study is to further define the lung failure process the follows that can rarely follow Bactrim exposure. There are several well understood adverse reactions to Bactrim but severe lung failure is only recently recognized and reported. Researchers need to evaluate more patients to understand why this rare reaction happens to some children and not others.
Single Ventricle Condition: A Clinical Trial of a Medication to Increase Exercise Endurance for Adolescents who had a Fontan Procedure
This study is for children: • Age 12-18 years old • Born with a single ventricle and had a Fontan procedure • Currently on blood thinner (i.e. taking medications such as aspirin, heparin, coumadin/warfarin, etc.)...
This study is for children: • Age 12-18 years old • Born with a single ventricle and had a Fontan procedure • Currently on blood thinner (i.e. taking medications such as aspirin, heparin, coumadin/warfarin, etc.) The purpose of this study is to find out if the investigational study drug, udenafil, is effective and safe in adolescent participants who have single ventricle heart disease (SVHD) and have undergone a Fontan procedure. Udenafil works to help dilate (open) the blood vessels, which allows for better function of the heart and lungs. The study aims to (1) find out how well udenafil works to improve a patient's ability to exercise and perform everyday activities (2) potentially prevent liver fibrosis, a common complication of SVHD. Patients who participate in the study will be randomly assigned, like flipping of a coin, to receive either udenafil or placebo (does not contain active medication) and have an equal chance (50%) of receiving either udenafil or placebo. Participants will take one tablet by mouth, twice a day, for 26 weeks and be in the study for approximately 7 months.
Single Ventricle Heart Condition: How Skeletal Muscle Health Contributes Toward Overall Health
This study is for children: • 12-18 years old • Born with a single ventricle heart and had the Fontan Procedure • Be able to wear an accelerometer for a week. (Accelerometer is a small device worn on the hip to measure physical...
This study is for children: • 12-18 years old • Born with a single ventricle heart and had the Fontan Procedure • Be able to wear an accelerometer for a week. (Accelerometer is a small device worn on the hip to measure physical activity) OR • 12-18 years old • Born with a normal heart (comparison group) • Be able to wear an accelerometer for a week. (Accelerometer is a small device worn on the hip to measure physical activity) The goal of the research study is to better understand the importance of skeletal muscle health (especially leg muscles) and how it contributes to the overall health of an adolescent living with a single ventricle heart. Researchers know that healthy muscles play a larger role in helping blood return to the lungs in these adolescents, but they do not understand the details of this relationship. In addition, adolescents with normal hearts will be enrolled as a comparison group.
Solid tumors with PI3K/MTOR pathway mutations: A study of the drug LY3023414 as treatment for tumors
This study is for children, adolescents, and young adults who: • Are 12 months- 21 years old • Have solid tumors that are not responding to therapy, or have come back, and also have mutations of the PI3K/MTOR pathway...
This study is for children, adolescents, and young adults who: • Are 12 months- 21 years old • Have solid tumors that are not responding to therapy, or have come back, and also have mutations of the PI3K/MTOR pathway • Are able to swallow pills The medicine that is used in the study, LY3023414, blocks the growth of cancer cells with this pathway in test tubes and in animals. It has been given to adults but this will be the first time it is being used in children and adolescents. LY3023414 is taken by mouth twice a day for 28 days for up to 6 months. The goals of this study are to find out what effects the drug has on the child's tumor, evaluate the drug's side effects, and to learn more about how the child's body handles the drug.
Solid tumors with RET alterations: A study of LOXO-292 (selpercatinib) as treatment for tumors
This study is for children, adolescents, and young adults who: • Are 12 months – 21 years old • Have solid tumors that are not responding to therapy, or have come back, and also have RET alterations...
This study is for children, adolescents, and young adults who: • Are 12 months – 21 years old • Have solid tumors that are not responding to therapy, or have come back, and also have RET alterations The overall goal of this study is to test any good and bad effects of the study drug selpercatinib (LOXO-292) on a participant's tumor. Selpercatinib has been shown to block the growth of cancer cells that have RET alterations and has had some success in adult cancers. Selpercatinib is taken by mouth twice daily for 28 days for up to 26 months. It is not necessary to be able to swallow pills to participate in this study.
Statin: A study to understand how liver fat affects liver cell processes
This study is for children: • 8-21 years • LDL cholesterol >130mg/dl (>95% percentile) • Fasting overnight (~8 hrs.)...
This study is for children: • 8-21 years • LDL cholesterol >130mg/dl (>95% percentile) • Fasting overnight (~8 hrs.) The purpose of this research study is to determine the role that liver fat has on movement of drug into the liver and how liver fat affects the response to a drug used to lower cholesterol (statin). A goal of the study is to determine if increased liver fat leads to less movement of a drug into the liver and less response to statins, potentially placing a patient with higher liver fat at risk for side effects and treatment failure.
TLC Study: Implementing COVID-19 tests in School Communities
Those who can participate are: • Children with or without symptoms • Parents/Legal guardian must be able to provide child's information to enroll • Individuals who "walk up" for testing at the community-based site also will be able to enroll...
Those who can participate are: • Children with or without symptoms • Parents/Legal guardian must be able to provide child's information to enroll • Individuals who "walk up" for testing at the community-based site also will be able to enroll The purpose of the School TLC study is to help prevent the spread of COVID-19 in schools and promote safe in-person learning. All KCPS students, staff, and community members can get tested for COVID-19 You can learn more at http://childrensmercy.org/TLC.
Tuberous Sclerosis Complex (TSC): A study of a drug called Ganaxolone (GNX) to reduce seizures.
This study is for children and adults: • Ages 1 and above • Who have been diagnosed with Tuberous Sclerosis Complex (TSC)-related Epilepsy. • Are able to be in the study for 16-20 weeks...
This study is for children and adults: • Ages 1 and above • Who have been diagnosed with Tuberous Sclerosis Complex (TSC)-related Epilepsy. • Are able to be in the study for 16-20 weeks. The purpose of this research study is to find out if the study drug Ganaxolone (GNX) is safe and reduces the number of seizures you have when added to standard epilepsy treatment and drug(s).
Tumors with HRAS gene mutations: A study of tipifarnib treatment for tumors
This study is for children, adolescents, and young adults who: • Are 12 months – 21 years old • Have tumors that are not responding to therapy, or have come back, and also have the HRAS gene mutation...
This study is for children, adolescents, and young adults who: • Are 12 months – 21 years old • Have tumors that are not responding to therapy, or have come back, and also have the HRAS gene mutation The medicine tipifarnib has been shown to block the growth of cancer cells with HRAS in laboratory experiments and has reduced tumor size in some adults. It is taken by mouth for up to 24 months. The goal of this study is to find out what effects tipifarnib has on the child's tumor and any side effects it may cause. Participants do not need to be able to swallow pills for this study.
Tumors with IDH pathway mutations: A study of AG-120 (iosidenib) treatment for tumors
This study is for children, adolescents, and young adults who: • Are 12 months – 21 years old • Have tumors that are not responding to therapy, or have come back, and also have mutations of the IDS pathway • Are able to swallow pills...
This study is for children, adolescents, and young adults who: • Are 12 months – 21 years old • Have tumors that are not responding to therapy, or have come back, and also have mutations of the IDS pathway • Are able to swallow pills AG-120 (ivosidenib) has been shown to block the growth of cancer cells that have specific genetic changes in the IDH pathway and is used in other cancers that have this pathway. The treatment is given by mouth daily for 28 days up to 26 months. The goals of the study are to find out what effects the drug has on the child's tumor, evaluate the drug's side effects, and to learn more about how the child's body handles the drug and how the drug affects the child's body.
Type 1 Diabetes Stage 2: A study to possibly prevent or delay progression to Stage 3 T1D
This study is for children: • Able to have low dose Antithymocyte Globulin (ATG) • Able to have infusion therapy...
This study is for children: • Able to have low dose Antithymocyte Globulin (ATG) • Able to have infusion therapy The goal of this study is to learn if ATG can help prevent or delay progression to clinical diagnosis of type 1 diabetes in people that is having multiple autoantibodies with abnormal glucose status (called stage 2 diabetes) and at least one of the high-risk marker(s) who are at greater risk of progression to clinical diabetes.
Type 1 Diabetes: A study of Garmin Vivosmart devices to measure change in physical activity
This study is for youths: -8-21 years old -With a diagnosis of Type 1 Diabetes -Who are patients in the Children's Mercy Kansas City...
This study is for youths: -8-21 years old -With a diagnosis of Type 1 Diabetes -Who are patients in the Children's Mercy Kansas City The goal of this study is to see if wearing the Garmin physical activity tracker increases activity and control over Type 1 diabetes (T1D). Another purpose is to see if wearing the Garmin tracker changes how people feel about managing their diabetes. The study will look at activity levels, blood sugar, hospital admissions, time in range, A1c, and reported activity levels. The study also will ask about feelings about T1D and managing T1D in surveys for both patients and parents of child patients.
Type 1 Diabetes: A study of the oral medication DFMO in newly diagnosed type 1 diabetic children and adults for treatment.
This study is being done for children/adults: -Newly diagnosed (within past 100 days) type 1 diabetes -Ages 6-40 years...
This study is being done for children/adults: -Newly diagnosed (within past 100 days) type 1 diabetes -Ages 6-40 years DFMO is a medication taken by mouth which may reduce the stress of cells that make insulin and preserve body's own insulin production.
Type 1 Diabetes: A study to help preserve remaining insulin secretion
This study is being done for children/adults: • With type 1 Diabetes Diagnosis • Able to wear a Continuous Glucose Monitoring...
This study is being done for children/adults: • With type 1 Diabetes Diagnosis • Able to wear a Continuous Glucose Monitoring This study is to see if giving the medications rituximab-pvvr (Ruxience®) followed by abatacept can help preserve remaining insulin secretion if given within three months of being diagnosed with type 1 diabetes (T1D).This study will help researchers to learn more about how to help people with diabetes Type 1.
Type 1 Diabetes: Reduce feelings of diabetes distress
This study is for children: • Ages 8-12.99 • Who have Type 1 Diabetes • Are on intensive insulin treatment...
This study is for children: • Ages 8-12.99 • Who have Type 1 Diabetes • Are on intensive insulin treatment • English is their primary language The purpose of this research study is to test whether a treatment program works to help families of school-age children with T1D reduce any feelings of diabetes distress and improve glucose levels. Families participate via a computer, tablet, or smartphone. Families will also upload continuous glucose monitoring (CGM) data.
Type I Diabetes: A study of biomarkers for Type I Diabetes
A study to learn more about how Type 1 Diabetes works. This study will try and develop new biomarkers in participants of all ages to help identify what causes Type 1 Diabetes. In order to participate in this study, a potential participant must be enrolled in the Trial Net TN01...
A study to learn more about how Type 1 Diabetes works. This study will try and develop new biomarkers in participants of all ages to help identify what causes Type 1 Diabetes. In order to participate in this study, a potential participant must be enrolled in the Trial Net TN01 Monitoring study.
Type I Diabetes: A study of children's ability to wear a device called Kiddo
This study is for children: • Between 3-9 years old • Has a diagnosis of type 1 diabetes • Uses insulin...
This study is for children: • Between 3-9 years old • Has a diagnosis of type 1 diabetes • Uses insulin • Wears a continuous glucose monitor The goal of this study is to test if a device called Kiddo, that is worn on the wrist, will be comfortable and easy for children to wear. Researchers will see how the measurements (Heart rate, temperature, oxygen level, sweat levels, and activity levels) will relate to blood sugar control, and also see how parent interaction with the KIDDO phone app is associated with diabetes outcomes.
Type I Diabetes: A study on the effect of individualized eating plans for teens
This study is for teens: -12-17 years old -T1D Diagnosis for 1+ years...
This study is for teens: -12-17 years old -T1D Diagnosis for 1+ years -Last HbA1c test 7.2+ The purpose of this research study is to see if a personalized eating plan that spaces out carbohydrates evenly throughout the day is something teens can follow and is helpful for their glycemic management. The research will study how closely the eating plan is followed. Average blood glucose at the beginning of the study will also be compared with average blood glucose at the end of the study to see if blood glucose control is related to following an eating plan. The total duration of project participation is 6 months.
Type I Diabetes: A study on two interventions to improve blood sugar control
This study is for teenagers: • Diagnosed with type 1 diabetes • Between 12-17 years old • Currently using an insulin pump for insulin delivery...
This study is for teenagers: • Diagnosed with type 1 diabetes • Between 12-17 years old • Currently using an insulin pump for insulin delivery The overall purpose of this study is to help adolescents with Type 1 Diabetes (T1D) follow their treatment plans. T1D is a serious disease that happens because the body cannot control blood glucose (sugar) levels. It is very common for adolescents to forget to give an insulin bolus for meals. When insulin doses are missed, there is a greater chance for poor blood sugar control. It is very common for adolescents to forget to give an insulin bolus for meals. When insulin doses are missed, there is a greater chance for poor blood sugar control. When adolescents follow their diabetes plan closely, they have better blood sugar control and overall health.
Type I Diabetes: A study to better understand those at risk for developing Type I Diabetes
The overall objective of this study is to perform baseline and repeat assessments over time of the metabolic and immunologic status of individuals at risk for type 1 diabetes (T1D) to: (a) characterize their risk for developing T1D and identify subjects eligible for prevention...
The overall objective of this study is to perform baseline and repeat assessments over time of the metabolic and immunologic status of individuals at risk for type 1 diabetes (T1D) to: (a) characterize their risk for developing T1D and identify subjects eligible for prevention trials, (b) describe the pathogenic evolution of T1D, and (c) increase the understanding of the pathogenic factors involved in the development of T1D.
Type I Diabetes: A study to observe the preservation of C-peptides in the pancreas of children newly diagnosed with Type I Diabetes
This study is being done for children and adults: • Age 12-35 • Within 100 days of being newly diagnosed with Type I Diabetes (T1D) • At least 1 autoantibody positive...
This study is being done for children and adults: • Age 12-35 • Within 100 days of being newly diagnosed with Type I Diabetes (T1D) • At least 1 autoantibody positive • Mixed Meal Tolerance Tests with most visits Researchers are performing this study to observe the preservation of C-peptides in the pancreas of children newly diagnosed with T1D. The goal of this study is to learn if either abrocitinib or ritlecitinib could help delay or stop further destruction of remaining beta cells in people recently diagnosed with Type I Diabetes (T1D). Researchers will follow people in the study by performing blood tests and will collect information about their diabetes to learn more about how these drugs work.
Youth with Insulin Treated Diabetes: A study to collect feedback on a diabetes phone app
This study is for: - Participants include Parent(s)/Guardians of children age 1-18 who have insulin treated diabetes or youth age 13-22 who have insulin treated diabetes The goal of this study is to test if a diabetes phone app has an impact on engagement and understanding of...
This study is for: - Participants include Parent(s)/Guardians of children age 1-18 who have insulin treated diabetes or youth age 13-22 who have insulin treated diabetes The goal of this study is to test if a diabetes phone app has an impact on engagement and understanding of diabetes care.