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 Myeloid Leukemia (AML): A clinical study of enasidenib to treat relapsed/refractory AML with IDH-2 mutations
This study is for: • Patients who are between the age of 2 and 18 years old • Have been diagnosed with Acute Myeloid Leukemia (AML) that has relapsed (has come back) or is refractory (has not responded to earlier treatment)...
This study is for: • Patients who are between the age of 2 and 18 years old • Have been diagnosed with Acute Myeloid Leukemia (AML) that has relapsed (has come back) or is refractory (has not responded to earlier treatment) • The AML also must have a specific genetic change, also called a mutation, in a protein called IDH2 The overall goal of this study is to find out what effects, good and/or bad, the drug enasidenib has on children and adolescents with AML with IDH2 mutations.
Acute Myeloid Leukemia (AML): A study of chemotherapy with or without venetoclax in children with relapsed AML without FLT3/ITD mutation
This study is for: • Patients who have enrolled in APAL2020SC • Who are greater than 28 days old to less than 22 years old • Who have been diagnosed with acute myeloid leukemia without FLT3/ITD mutation that has relapsed (has come back)...
This study is for: • Patients who have enrolled in APAL2020SC • Who are greater than 28 days old to less than 22 years old • Who have been diagnosed with acute myeloid leukemia without FLT3/ITD mutation that has relapsed (has come back) This is a randomized trial (like the flip of a coin) to find what effects, good and/or bad, chemotherapy with venetoclax verus without venetoclax has on children and young adults with AML.
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
This study is for: • Those ages 3-12 years old • Who have amblyopia (lazy eye) in one eye • And who have normal vision in their good eye...
This study is for: • Those ages 3-12 years old • Who have amblyopia (lazy eye) in one eye • And who have normal vision in their good eye This study will 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.
Amblyopia (Lazy Eye): A study watching dichoptic movies/shows wearing the Luminopia headset and eye patches.
This study is for children: • Those ages 4-7 years old • Who have amblyopia (lazy eye) in one eye • And who have normal vision in their good eye...
This study is for children: • Those ages 4-7 years old • Who have amblyopia (lazy eye) in one eye • And who have normal vision in their good eye This study is designed to find out if watching shows or movies wearing the virtual reality headset 1 hour a day for 6 days a week, will improve vision as good as patching 2 hours a day for 7 days.
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.
Anxiety and Depression: A study of how the body breaks down medicine
This study involves: - Children and teens age 6-18 - Taking the prescribed medicine every day for 2 weeks without missing any doses - A one-time 24-hour visit in the Pediatric Clinical Research Unit...
This study involves: - Children and teens age 6-18 - Taking the prescribed medicine every day for 2 weeks without missing any doses - A one-time 24-hour visit in the Pediatric Clinical Research Unit Researchers at Children's Mercy are doing this study to learn more about how the body breaks down medicine for depression and/or anxiety. Children and teens who are prescribed fluoxetine (Prozac®), sertraline (Zoloft®), or escitalopram (Lexapro®) are eligible to participate
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-ALL: Eliminating Total Body Irradiation (TBI) for NGS-MRD Negative Children, Adolescents, and Young Adults
This study is being done children and adults. • Between 1-30 years old for one of the groups • Diagnosed with B-cell acute lymphoblastic leukemia (B-ALL) There are two groups in this study. For the treatment group, patients age 1 through 30 are eligible. In this group...
This study is being done children and adults. • Between 1-30 years old for one of the groups • Diagnosed with B-cell acute lymphoblastic leukemia (B-ALL) There are two groups in this study. For the treatment group, patients age 1 through 30 are eligible. In this group, researchers would like to know if not giving a child total body irradiation (also called TBI) therapy before a child's transplant is as good as giving a child radiation before transplant and a child's growth and development will not be affected by radiation. For the observation group, all patients are eligible. The purpose of this part of the study is to use the NGS-MRD test to see if the patient would qualify for the Non-TBI Group, and to gather more research data.
B-Cell Acute Lymphoblastic Leukemia (B-ALL): A clinical trial to study inotuzumab ozogamicin (InO) in combination with chemotherapy in children and young adults with relapsed or refractory B-ALL
This study is for: • Patients who are between the ages of 1 year and 22 years old • Who have been diagnosed with B-lymphoblastic leukemia (B-ALL) that is refractory (has not responded to earlier treatment) or is in second or greater relapse (has come back)...
This study is for: • Patients who are between the ages of 1 year and 22 years old • Who have been diagnosed with B-lymphoblastic leukemia (B-ALL) that is refractory (has not responded to earlier treatment) or is in second or greater relapse (has come back) • The ALL also must have a specific protein present called CD22 The overall goal of this study is to find out what effect, good and/or bad, the study drug inotuzumab ozogamicin (InO) in combination with chemotherapy has on children and young adults with relapsed or refractory B-ALL. The treatment used in this study involves cancer fighting medicine called chemotherapy plus the medicine InO. The treatment on this study takes about 2 months, divided into 2 phases of therapy.
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 Non-Hodgkin Lymphoma (B-NHL): A study of glofitumab with or without chemotherapy in children with relapsed or refractory mature B-NHL
This study is for: • Part 1: Patients who are greater than 6 months old to less than 18 years old • Part 2: Patients who are greater than 6 months old to less than 31 years old • Who have been diagnosed with mature B-Cell non-Hodgkin lymphoma that has relapsed (has...
This study is for: • Part 1: Patients who are greater than 6 months old to less than 18 years old • Part 2: Patients who are greater than 6 months old to less than 31 years old • Who have been diagnosed with mature B-Cell non-Hodgkin lymphoma that has relapsed (has come back) or is refractory (has not responded to earlier treatment) The goal of this study are to find what effects, good and/or bad, the drug glofitumab with or without chemotherapy has on children and young adults with relapsed or refractory mature B-NHL. Part 1 is looking learn more about the pharmacology (for example, the best dose) of the medication. Part 2 increases the number of participants based on what is learned from Part 1.
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.
Bedside Ultrasound: A study to determine if Ultrasound can be used to detect the development of Hepatic Sinusoidal Obstruction Syndrome.
This study is being done for children and adults: • Ages 1 month through 22 years. • Undergoing a Bone Marrow Transplant...
This study is being done for children and adults: • Ages 1 month through 22 years. • Undergoing a Bone Marrow Transplant Patients having a Bone Marrow Transplant are at great risk for developing Hepatic Sinusoidal Obstructive Syndrome (SOS). The purpose of this research study is to determine if researchers can detect the development of SOS using Ultrasound before clinical symptoms present.
Blood Glucose: A study on healthy infants and their development
This study is for: • Infants who are 1-3 months old • Participants much be able to wear a continuous glucose monitor and an active watch for four days, both on various parts of their leg...
This study is for: • Infants who are 1-3 months old • Participants much be able to wear a continuous glucose monitor and an active watch for four days, both on various parts of their leg • Be able to be a part of this study for 10 months, which is the full length of the study Participants and their family must be willing to come to the Baby Health Behavior Lab at Children's Mercy for a total of 6 visits. The continuous glucose monitor will track changes in blood glucose levels. The watch will track sleep and wakefulness activity. Participants will also be asked to complete a record of their infant's diet for 4 days. The purpose of this research study is to learn how the different ways babies are fed affects their development.
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 Response to Medicines: A study to learn how the brain of a person with ADHD or depression responds to medicine
This study is being done for: -Children and adults ages 7 to 25 years old -Children and adults with ADHD previously enrolled in the ATX PBPK-PD-Clinical Outcomes Study at Children's Mercy -Children and adults with depression who previously participated in the GOLDILOKs PRISM...
This study is being done for: -Children and adults ages 7 to 25 years old -Children and adults with ADHD previously enrolled in the ATX PBPK-PD-Clinical Outcomes Study at Children's Mercy -Children and adults with depression who previously participated in the GOLDILOKs PRISM Study at Children's Mercy The goal of the study is to learn more about how exactly brain cells respond to ADHD and/or depression medicines and learn if researchers can predict whether a child or adult's ADHD or depression symptoms will improve with these certain medicines.
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 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 with ALK or ROS1 mutations: A study of ensartinib as treatment for tumors
This study is for: • Patients who have enrolled in APEC1621SC • Who are between the ages of 12 months old and 21 years old • Have been diagnosed with cancer that is relapsed (has come back) or is refractory (has not responded to earlier...
This study is for: • Patients who have enrolled in APEC1621SC • Who are between the ages of 12 months old and 21 years old • Have been diagnosed with cancer that is relapsed (has come back) or is refractory (has not responded to earlier treatment) • The cancer also must have a specific genetic change, also called a mutation, in a gene called ALK or ROS1 • Are able to swallow pills The medicine, ensartinib, has been shown to block the growth of cancer cells with ALK or ROS1 in laboratory experiments. Early studies with ensartinib have been conducted in adults. The study drug is taken by mouth for up to 24 months. The goal of this study is to find what effects ensartinib has on a child's tumor, any side effects it may cause, and to learn more about the pharmacology of the medication.
Cancer: A study screening for genetic changes (mutations) that may respond to specific drug therapies for tumors
This study is for: • Patients who are between the ages of 12 months and 21 years old • Who have been diagnosed with solid tumors that have relapsed (have come back) or are refractory (have not responded to earlier treatment)...
This study is for: • Patients who are between the ages of 12 months and 21 years old • Who have been diagnosed with solid tumors that have relapsed (have come back) or are refractory (have not responded to earlier treatment) The goal of this study is to find better ways to diagnose and treat relapsed or refractory tumors in children, adolescents, and young adults. The study team will collect some information about each participant's cancer and treatment. The study team will also collect extra tumor, blood, and/or other tissue from procedures done by doctors to help make a diagnosis and learn more about different cancers now and in the future. If the study team finds out that the leukemia has a specific genetic change (mutation) that may respond to a drug that "targets" those changes, they may present a treatment study using that specific drug to the participant. 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.
Capture IBD: A tool used to collect information about Irritable Bowel Disease
This study is for children and young adults: • 4-21 years old • Who have or may have Irritable Bowel Disease (e.g., Ulcerative Colitis, Crohn's)...
This study is for children and young adults: • 4-21 years old • Who have or may have Irritable Bowel Disease (e.g., Ulcerative Colitis, Crohn's) This study will develop a research resource for future studies of IBD, including Crohn's disease and ulcerative colitis, and other inflammatory diseases and diseases of the intestines. This resource will be available to scientists to answer many questions about treatments, therapies, and surgeries for patients with IBD.
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
This study is being done for children: • Between the ages of 13-17 years • Diagnosed with Chronic Kidney Disease; CKD 3-4...
This study is being done for children: • Between the ages of 13-17 years • Diagnosed with Chronic Kidney Disease; CKD 3-4 The purpose of this study is to determine how well ferric citrate will lower the level of FGF23 (the hormone that regulates phosphate levels in the blood) , 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.
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.
Craniopharyngioma: A Study Identifying Clinically Relevant Biological Targets for Medical Therapy
This study is being done for children and adults: • 1 to 21 years old. • Diagnosed with craniopharyngioma...
This study is being done for children and adults: • 1 to 21 years old. • Diagnosed with craniopharyngioma. This study wants to learn what might be the best treatment for children with a tumor called craniopharyngioma. The main goal of this study is to collect extra tissue at the time of surgery and medical information after surgery. Researchers will also collect and test a small amount of blood.
Cystic Fibrosis-Related Diabetes: A Study Comparing the Safety of the Bionic Pancreas Device Relative to Usual Care.
This study is being done to learn how well the iLet Bionic Pancreas system works in patients with Cystic Fibrosis-Related Diabetes (CFRD). - 14 years old or older - Have Cystic Fibrosis Related Diabetes...
This study is being done to learn how well the iLet Bionic Pancreas system works in patients with Cystic Fibrosis-Related Diabetes (CFRD). - 14 years old or older - Have Cystic Fibrosis Related Diabetes - Be on treatment of insulin for at least 3 months
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.
Diffuse Intrinsic Pontine Glioma (DIPG) and High-Grade Glioma (HGG): A study of Selinexor with Radiation Therapy
This study is for children and young adults: -Ages 12 months to 21 years old -With a new diagnosis of high-grade glioma (HGG) or diffuse intrinsic pontine glioma (DIPG)...
This study is for children and young adults: -Ages 12 months to 21 years old -With a new diagnosis of high-grade glioma (HGG) or diffuse intrinsic pontine glioma (DIPG). This study is being done to find the highest and safest dose of selinexor with radiation therapy.
DOMINICA: A study of severe pediatric eosinophilic asthma and time to first exacerbation using Benralizumab
This study is for children who are/have: • Ages 6 to 17 • Eosinophilic asthma • Severe or hard to treat asthma...
This study is for children who are/have: • Ages 6 to 17 • Eosinophilic asthma • Severe or hard to treat asthma • Currently using a high dose maintenance inhaler for at least 6 months • Have gone to urgent care, emergency room or have been hospitalized 3 times in the past year The goal of this study is to see how Benralizumab affects children ages 6 to 17 with eosinophilic asthma that have a history of frequent asthma attacks that are hard to treat with inhaled medication. Benralizumab is approved for children as young as 6 years of age for hard to treat asthma. Researchers want to see how Benralizumab helps the daily life of children with eosinophilic asthma.
Dystonia and Cerebral Palsy: A study of a medicine called Trihexyphenidyl
This study is being done for children: • Ages between 5-17 years • Diagnosed with Dystonia and Cerebral Palsy • Parents/Guardians may participate in this research study...
This study is being done for children: • Ages between 5-17 years • Diagnosed with Dystonia and Cerebral Palsy • Parents/Guardians may participate in this research study This research study is being done to learn how a child's body breaks down and responds to the medicine Trihexyphenidyl, the sides effects of this medicine and how well it works.
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.
Early onset Scoliosis (EOS): The social factors that determine the quality of life for young children.
This study is being done for children: • Early onset Scoliosis (EOS) Diagnosis The aim of this study is to understand the social factors (such as where a family lives) that may affect how well a...
This study is being done for children: • Early onset Scoliosis (EOS) Diagnosis The aim of this study is to understand the social factors (such as where a family lives) that may affect how well a child does now and later in life. Researchers believe that these social factors can affect the child's outcome, such as how well they do with spine bracing, casting, or surgery.
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.
Fontan Circulation: A study on liver function
This study is being done for children and adults: • Ages 8 and older • Diagnosed with a congenital Heart Defect treated with Fontan Operation...
This study is being done for children and adults: • Ages 8 and older • Diagnosed with a congenital Heart Defect treated with Fontan Operation The purpose of this research study is to see how two different medications are processed by the liver and cleared from the body in patients with Fontan circulation. The liver is essential in moving and breaking down drugs. Fontan circulation may affect the ability of the liver to function.
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.
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.
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.
Hemophilia: A study investigating inflammation and iron homeostasis
The primary objective of this study is to determine if there is an association of factor 8 or 9 activity with inflammatory markers, hepcidin, and iron stores in children and young adults with hemophilia A and B. Inclusion Criteria...
The primary objective of this study is to determine if there is an association of factor 8 or 9 activity with inflammatory markers, hepcidin, and iron stores in children and young adults with hemophilia A and B. Inclusion Criteria: • Less than 22 years of age • Able to comprehend English and/or Spanish • Participants must be diagnosed with Hemophilia A (Factor 8 deficiency) or Hemophilia B (Factor 9 deficiency) Being in this study will involve one study visit which will last approximately 20-30 minutes. The study visit can happen at the same time as the child regular clinic visit. The biggest risks from being in this study are bleeding and discomfort related to the blood draw. There may be direct benefit to being in this study: any iron deficiency discovered from study labs can be treated by the primary hematologist. By being in this study, children may help researchers find better treatments for patients with hemophilia in the future.
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
This study is for: • Patients who are between the ages of 0 and 21 years old • Who have been diagnosed with molecular high risk medulloblastoma, very high risk medulloblastoma, or medulloblastoma that has relapsed (has come back) or is refractory (has not responded to earlier...
This study is for: • Patients who are between the ages of 0 and 21 years old • Who have been diagnosed with molecular high risk medulloblastoma, very high risk medulloblastoma, or medulloblastoma that has relapsed (has come back) or is refractory (has not responded to earlier treatment) The goal of this research study is to find the effects, good and/or bad, the investigational drug eflornithine HCl (DFMO) has on high risk and very high risk 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-grade Glioma (HGG) with BRAFV600-mutation: A clinical trial of dabrafenib and trametinib after radiation therapy
This study is for: • People who are ages 3 through 21 • Who are newly diagnosed with high-grade glioma...
This study is for: • People who are ages 3 through 21 • Who are newly diagnosed with high-grade glioma (HGG) with BRAFV600-mutation High-grade gliomas (HGGs) are hard to treat successfully. Two drugs that will be used in this study are dabrafenib and trametinib. This study will look at how well the combination of dabrafenib and trametinib works when given to children and young adults with HGGs after they receive radiation therapy. The use of this combination to treat pediatric brain tumors is experimental.
High-Risk Neuroblastoma (HRNBL): A Study of the Addition of Dinutuximab to Therapy
This study is being done for children and adults: • 30 years old or younger. • Diagnosed with High-Risk Neuroblastoma (HRNBL)...
This study is being done for children and adults: • 30 years old or younger. • Diagnosed with High-Risk Neuroblastoma (HRNBL) The overall goal of this study is to compare the outcomes of dinutuximab given early with Induction therapy to the effects of standard Induction therapy with the addition of dinutuximab later in therapy in people with HRNBL to find out which is better.
Hodgkin Lymphoma: A study to compare standard therapy to the use of two drugs, Brentuximab vedotin and Nivolumab
This study is being done for children and adults: • 5 to 60 years old. • Diagnosed with Hodgkin lymphoma (HL)...
This study is being done for children and adults: • 5 to 60 years old. • Diagnosed with Hodgkin lymphoma (HL) The overall goal of this study is to compare the effects, good and/or bad, of brentuximab vedotin and nivolumab against standard therapy for people with HL to find out which is better.
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.
Infant Leukemia: A study of revumenib (SNDX-5613) in combination with chemotherapy in infants with relapsed or refractory KMT2A-rearranged infant leukemia
This study is for: • Patients ages 1 month to less than 6 years old • Who have been diagnosed with KMT2A-rearranged infant leukemia that has relapsed (has come back) or is refractory (has not responded to earlier treatment)...
This study is for: • Patients ages 1 month to less than 6 years old • Who have been diagnosed with KMT2A-rearranged infant leukemia that has relapsed (has come back) or is refractory (has not responded to earlier treatment) The medicine, revumenib (SNDX-5613), has been shown to specifically kill these cancer cells with KMT2A rearrangement in laboratory experiments. Early studies with revumenib have been conducted in adults and pediatrics. The goals of this study are to find what effects, good and/or bad, the drug revumenib in combination with chemotherapy has on infants with relapsed or refractory leukemia and to learn more about the pharmacology (for example, the best dose) of the medication.
Inflammatory Bowel Disease: Transitioning to Adult Care Survey
This study is for: • Those who are diagnosed with Inflammatory Bowel Disease (IBD) • Are between ages 15-22 • Both patients with IBD and their caregivers...
This study is for: • Those who are diagnosed with Inflammatory Bowel Disease (IBD) • Are between ages 15-22 • Both patients with IBD and their caregivers 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 longitudinal tears portion of this study is for: • Juvenile Idiopathic Arthritis without Uveitis (JIA-no-U): Diagnosed with JIA (RF-negative, oligoarticular, psoriatic, or undifferentiated) within the last four years and are less than 18 years of age at the time of...
The longitudinal tears portion of this study is for: • Juvenile Idiopathic Arthritis without Uveitis (JIA-no-U): Diagnosed with JIA (RF-negative, oligoarticular, psoriatic, or undifferentiated) within the last four years and are less than 18 years of age at the time of enrollment • or Juvenile Idiopathic Arthritis associated Uveitis (JIA-U): diagnosed with JIA (RF-negative, oligoarticular, psoriatic, or undifferentiated) and uveitis and are less than 18 years of age at the time of enrollment The one time DNA collection for genetics portion of this study is for: • JIA-no-U patients who must be 1) diagnosed with JIA ≥ 4 years and 2) have at least one ophthalmology exam performed after having JIA ≥ 4 years. • or JIA-U patients must be 1) diagnosed with JIA, 2) diagnosed with uveitis, and 3) have at least one ophthalmology exam performed confirming uveitis diagnosis. 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 researchers 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.
Langerhans Cell Histiocytosis (LCH): Treatment study for Children and Adolescents
This study is being done for children and adolescents: • age 17 or younger. • Diagnosed with Langerhans Cell Histiocytosis (LCH)...
This study is being done for children and adolescents: • age 17 or younger. • Diagnosed with Langerhans Cell Histiocytosis (LCH) The purpose of this research study is to find out if extending the treatment even further (from 12 months to 24 months) and adding another drug called 6-mercaptopurine will result in fewer LCH relapses (when the disease comes back) and fewer long-term disease-related problems. Study patients will receive study treatment for about 12 or 24 months. After treatment, study patients will then be followed for at least 5 years.
Leukemia & Lymphoma: A study of tagraxofusp with or without chemotherapy in pediatric patients with relapsed or refractory CD123 expressing hematologic malignancies
This study is for: • Patients who are between the ages of 1 and 21 years old • Who have been diagnosed with a hematologic maligancy (including, but not limited to acute lymphoblastic leukemia, acute myeloid leukemia, myelodysplastic syndrome, mixed phenotype acute leukemia...
This study is for: • Patients who are between the ages of 1 and 21 years old • Who have been diagnosed with a hematologic maligancy (including, but not limited to acute lymphoblastic leukemia, acute myeloid leukemia, myelodysplastic syndrome, mixed phenotype acute leukemia, acute undifferentiated leukemia, blastic plasmacytoid dendritic cell neoplasm, Hodgkin lymphoma, and non-Hodgkin lymphoma) that has relapsed (has come back) or is refractory (has not responded to earlier treatment). • The cancer also must have a specific protein present called CD123 The goal of this study is to improve survival rates in children and young adults with relapsed hematological malignancies, determine the recommended phase 2 dose (RP2D) of tagraxofusp given alone and in combination with chemotherapy, as well as to find the effects, both good and/or bad, tagraxofusp has in pediatric patients.
Leukemia or Lymphoma: A study to see if levocarnitine protects the liver from chemotherapy
This study is being done for children and adults: • Ages 15-40. • Diagnosed with B-ALL, T-ALL, Lymphoblastic Lymphoma (LLy), or Mixed-Phenotype Acute Leukemia/Lymphoma (MPAL)...
This study is being done for children and adults: • Ages 15-40. • Diagnosed with B-ALL, T-ALL, Lymphoblastic Lymphoma (LLy), or Mixed-Phenotype Acute Leukemia/Lymphoma (MPAL). The study involves taking the drug levocarnitine during standard of care chemotherapy. The overall goal of this study is to determine whether taking levocarnitine will reduce the rate of severe liver damage caused by asparaginase chemotherapy during the first month of treatment.
Leukemia: An expanded access program of revumenib (SNDX-5613) in patients with relapsed or refractory leukemia with genetic alterations associated with HOXA overexpression
This expanded access program is for: • Patients who are older than 6 months old • Who have been diagnosed with acute leukemia with a gene alternation that has been associated with HOXA overexpression that has relapsed (has come back) or is refractory (has not responded to...
This expanded access program is for: • Patients who are older than 6 months old • Who have been diagnosed with acute leukemia with a gene alternation that has been associated with HOXA overexpression that has relapsed (has come back) or is refractory (has not responded to earlier treatment). • Who are not otherwise eligible to participate in other Syndax-sponsored clinical studies and have no approved treatment options. This expanded access program will provide revumenib in a controlled clinical setting.
Leukemia: An informational registry & study screening for genetic changes (mutations) that may respond to specific drug therapies for relapsed or refractory leukemia
This study is for: • Patients less than 22 years old • Who have a known or suspected diagnosis of relapsed (has come back) or refractory (has not responded to earlier treatment) leukemia...
This study is for: • Patients less than 22 years old • Who have a known or suspected diagnosis of relapsed (has come back) or refractory (has not responded to earlier treatment) leukemia The goal of this study is to find better ways to diagnose and treat relapsed or refractory leukemia in children, adolescents and young adults. The Study Team will collect some information about each participant's cancer and treatment. The Study Team will also collect extra blood and/or bone marrow from procedures done by doctors to help make a diagnosis and learn more about different cancers now and in the future. If the Study Team finds out that the leukemia has a specific genetic change (mutation) that may respond to a drug that "targets" those changes, they may present a treatment study using that specific drug to the participant.
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.
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.
MUAC Z-score tape: Monitoring malnutrition for pediatric patients.
This study is being done for children and parents: • Children who are being seen in the GI Interdisciplinary Feeding and Swallowing Program. • Parents will complete a one-time survey to determine if they can accurately obtain infant/child MUAC Z-score measurements using CMH...
This study is being done for children and parents: • Children who are being seen in the GI Interdisciplinary Feeding and Swallowing Program. • Parents will complete a one-time survey to determine if they can accurately obtain infant/child MUAC Z-score measurements using CMH MUAC Z-score tapes provided to them. This study begins with teaching parents how to measure at visit one, then they demonstrate a measurement at visit two on their own and get reteaching if needed. Once that is completed, the survey is sent to the parent to see how they felt about the education and feasibility of actually measuring on their child.
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
This study is for: • Patients who are between the ages of 0 and 31 years old • Who have been diagnosed with neuroblastoma that has relapsed (has come back) or is refractory (has not responded to earlier treatment)...
This study is for: • Patients who are between the ages of 0 and 31 years old • Who have been diagnosed with neuroblastoma that has relapsed (has come back) or is refractory (has not responded to earlier treatment) The goal of this study is to find what effects, good and/or bad, the investigational drug eflornithine HCl (difluoromethylornithine DFMO) along with etoposide has on 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
This study is for: • Patients who are between the ages of 0 and 21 years old • Who have been diagnosed with high-risk neuroblastoma (HRNB) that is now in remission and no longer seen as active disease on scans and in bone marrow samples...
This study is for: • Patients who are between the ages of 0 and 21 years old • Who have been diagnosed with high-risk neuroblastoma (HRNB) that is now in remission and no longer seen as active disease on scans and in bone marrow samples. • Who have completed prior treatment for HRNB The goal this study is to find what effects, good and/or bad, the investigational drug eflornithine HCl (DFMO) has on 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.
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.
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.
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.
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
Polycystic Ovary Syndrome (PCOS) : A study of collect samples to improve diagnosis, evaluation and treatment.
This study is for children and adults: • With PCOS diagnosis • Have been evaluated at Children's Mercy for PCOS or a related condition The purpose of this study is to improve the diagnosis, evaluation, and treatment of PCOS and other related conditions...
This study is for children and adults: • With PCOS diagnosis • Have been evaluated at Children's Mercy for PCOS or a related condition The purpose of this study is to improve the diagnosis, evaluation, and treatment of PCOS and other related conditions. In addition, information collected will be used to further our understanding of how PCOS & other conditions affect the body, other related health concerns, and the effectiveness of current treatments for these conditions.
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.
Recurrent or Progressive Low-Grade Glioma: A Study to Compare Treatment with the Drug Selumetinib Alone vs. Selumetinib and Vinblastine
Study participants must be ages 2 through 25 and are suspected of having progressive or recurrent low-grade glioma (LGG). This study looks at how well selumetinib and vinblastine work when given together to children and young adults with recurrent or progressive LGGs. Vinblastine...
Study participants must be ages 2 through 25 and are suspected of having progressive or recurrent low-grade glioma (LGG). This study looks at how well selumetinib and vinblastine work when given together to children and young adults with recurrent or progressive LGGs. Vinblastine is often given to children with progressive or recurrent LGGs. 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 with selumetinib have had some success in treating patients with LGG that came back after a first attempt at treatment. However, the use of selumetinib in children and young adults with recurrent or progressive LGG is considered experimental because selumetinib has not yet been FDA approved for this type of cancer.
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 combined immunodeficiency (SCID): A study to see how different amounts of a drug called busulfan help improve the immune system of infants after a stem cell transplant
This study is being done for infants: •Diagnosed with severe combined immunodeficiency (SCID). Researchers want to find out if lower doses of a chemotherapy drug called busulfan before stem cell transplant can help...
This study is being done for infants: •Diagnosed with severe combined immunodeficiency (SCID). Researchers want to find out if lower doses of a chemotherapy drug called busulfan before stem cell transplant can help patients with Severe Combined Immune Deficiency (SCID).
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.
Sickle Cell Disease and Other Hemoglobinopathies: A Non-Myeloablative Conditioning and Transplantation of Partially HLA-Mismatched and HLA-Matched Bone Marrow
This study is for children and adults: • 1-70 years old • Undergoing transplant...
This study is for children and adults: • 1-70 years old • Undergoing transplant. This research study is done to collect data on a type of bone marrow stem cell transplant for patients with sickle cell anemia or severe hemoglobinopathy such as thalassemia. Being in this study includes a review of patient medical history and completing questionnaires.
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.
Slipped Capital Femoral Epiphysis (SCFE): A study to identify social risk factors
This study is for: • Patients who have obesity and are diagnosed with Slipped Capital Femoral Epiphysis (SCFE) • Children 9-16 years old...
This study is for: • Patients who have obesity and are diagnosed with Slipped Capital Femoral Epiphysis (SCFE) • Children 9-16 years old. • A parent or legal guardian of the study participant will be asked to complete a one-time 9-question survey via email as well as a one-time interview over the phone or via Zoom call The purpose of this study is to help understand underlying health disparities and improve other aspects of a child's health that are related to SCFE. Researchers wants to know if a SCFE diagnosis can be a "wake up call to action" for a family and health care community by identifying and addressing ways for the child to regain their health.
Solid and CNS Tumors: A study of tiragolumab and atezolizumab in patients with relapsed or refractory SMARCB1 or SMARCA4 deficient tumors
This study is for: • Patients who are either older than or equal to 1 year old • Who have been diagnosed a tumor that is SMARCB1 (INI1) or SMARCA4 deficient that has relapsed (has come back) or is refractory (has not responded to earlier treatment)...
This study is for: • Patients who are either older than or equal to 1 year old • Who have been diagnosed a tumor that is SMARCB1 (INI1) or SMARCA4 deficient that has relapsed (has come back) or is refractory (has not responded to earlier treatment) • The specific types of tumors for this study include: - Renal medullary carcinoma - Malignant rhabdoid tumor (extra-CNS) - Atypical teratoid rhabdoid tumor (CNS) - Poorly differentiated chordoma - Epithelioid sarcoma - Other SMARCB1 or SMARCA4 deficient tumors The goal of this study is to find what effects, good and/or bad, the drugs tiragolumab and atezolizumab have on patients with relapsed or refractory solid or CNS tumors. Note: Children's Mercy is not participating in Part A of this study.
Solid Tumors: A study of CBL0137 for relapsed or refractory solid tumors
This study is for: • Part B1: - Patients who are between the ages of 1 and 21 years old - Who have been diagnosed with progressive (growing) or recurrent (has come back) diffuse intrinsic pontine...
This study is for: • Part B1: - Patients who are between the ages of 1 and 21 years old - Who have been diagnosed with progressive (growing) or recurrent (has come back) diffuse intrinsic pontine glioma (DIPG) or other diffuse midline gliomas that has a mutation in the H3 K27M gene • Part B2: - Patients who are between the ages of 1 and 30 years old - Who have been diagnosed with osteoscaroma that has relapsed (has come back) or is refractory (has not responded to earlier treatment) The goal of this study is to find what effects, good and/or bad, the drug CBL0137 has on children and young adults with relapsed or refractory solid tumors. Note: Children's Mercy is not participating in Part A of 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.
The Remote CaRe CHD Trial: An in-home exercise study for teens with congenital heart disease
This study is for: • Adolescents, aged 12-19 years old • Diagnosed with either: Fontan physiology, Tetralogy of Fallot, or D-Transposition of the great arteries...
This study is for: • Adolescents, aged 12-19 years old • Diagnosed with either: Fontan physiology, Tetralogy of Fallot, or D-Transposition of the great arteries Most patients and families with congenital heart disease (CHD) have little or no access to a supervised cardiac rehabilitation program specifically designed for teens with CHD. The Remote CaRe CHD Trial is studying the effects of a 12-week supervised exercise program delivered IN YOUR HOME using live video conferencing software - allowing everyone to have access to exercise training specifically for teens with CHD. Researchers aim is to see if the in-home, Remote CaRe exercise program can increase fitness, physical function, and quality of life in teens with CHD. To learn more, you can watch the Remote CaRe video by copying/pasting this link into your browser: https://youtu.be/WO4JEpXOZDM?si=psbhbtUre5nW9SDf You can call or text us at 816-764-5605 or you can email us at remotecare@cmh.edu
Turner Syndrome (TS), The Brain, and Hormone Replacement Therapy (HRT): A study evaluating brain structure and function in adolescents and young adults with Turner syndrome who use hormone replacement therapy as compared to those without Turner syndrome
This study is for teen girls and young adult women: • Ages 14-21 years • With and without Turner syndrome...
This study is for teen girls and young adult women: • Ages 14-21 years • With and without Turner syndrome The goal of this study is to understand how hormones from the ovaries affect brain development. Every person is unique and the ovaries of some teens and young adults do not produce enough hormones. What researchers learn from this study could help the care team find better treatments for teens and young adults whose ovaries do not make enough hormones.
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 Happy Bob App
This study is being done for teens: • Ages 13-17 years old • Diagnosed with Type 1 Diabetes • Own/Use an iPhone to view Dexcom readings...
This study is being done for teens: • Ages 13-17 years old • Diagnosed with Type 1 Diabetes • Own/Use an iPhone to view Dexcom readings The goal of the study is to see what impact the Happy Bob app has on diabetes management, mood, feelings of peer support, and more. The app connects to Dexcom and sends users time in range notifications in the personality of their choosing - Happy Bob, Snarky Bob, Pumpkin Bob, Snowman Bob, and more. Users can also earn awards in the app and join the Children's Mercy Teens group page to encourage one another with posts and respond to fun icebreaker questions, among other features.
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 or Type 2 Diabetes: A Study to Continuous Monitoring and Control of Hyploglycemia
This study is being done for young adults: • Be at least 18 years or older. • Have been diagnosed with Type 1 or Type 2 diabetes mellitus with at least one (1) week of being on insulin at time of study entry...
This study is being done for young adults: • Be at least 18 years or older. • Have been diagnosed with Type 1 or Type 2 diabetes mellitus with at least one (1) week of being on insulin at time of study entry. • Use a smartphone and/or have access to a computer The purpose of the study is to confirm the safety of using the Continuous Glucose Monitoring CGM System for making diabetes treatment decisions instead of fingerstick testing.
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.
Weight control and Asthma: The process of steroid medications
This study is being done for children and teens: • 5-17 years • Asthma diagnosis • Steroid use during hospitalization...
This study is being done for children and teens: • 5-17 years • Asthma diagnosis • Steroid use during hospitalization The purpose of this study is to find out if children with obesity process steroid medication differently than children who do not have obesity when having an asthma attack. this study will help us figure out how to give each person prescribed the medicine the "just right" dose for them—not too high and not too low.
Wilms Tumors: A study of Combination Chemotherapy
This study is being done for children and adults: • 30 years old or younger. • Diagnosed with Diffuse Anaplastic Wilms Tumor (DAWT) or Relapsed Favorable Histology Wilms Tumor (FHWT)...
This study is being done for children and adults: • 30 years old or younger. • Diagnosed with Diffuse Anaplastic Wilms Tumor (DAWT) or Relapsed Favorable Histology Wilms Tumor (FHWT) In this study, we want to find out if we can improve treatment for patients with newly diagnosed DAWT and those with high-risk or very high-risk relapsed FHWT. Patients on study will be given a combination of chemotherapy drugs plus radiation therapy.
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.