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Experimental Therapeutics in Pediatric Cancer

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Children's Mercy patient at the Cancer Center

The Experimental Therapeutics in Pediatric Cancer program makes clinical trials available to your child when traditional cancer therapies have not been successful. 

We are the only pediatric hospital in the region that provides access to cancer clinical trials, which means your child can benefit from the latest innovations in research while staying close to home. This is important because family connection and familiarity with surroundings has been shown to improve the success of cancer treatment.

What is experimental therapeutics?


Our trials test treatment methods that have not yet been used for your child’s diagnosis. Many medications involved in clinical trials have been proven effective and safe for other cancers or for use in adult patients, but not specifically for pediatric cancer. Experimental Therapeutics is the science of taking those treatments and trialing them with different cancers to test their effectiveness.

What are the phases of a cancer research trial?

 

  • Phase 1: A test of a drug to determine its safety and proper dosage for pediatric patients. At this phase, we do not know yet if a drug is effective.

  • Phase 2: This phase comes after a drug’s safety has been proven and tests the drug’s effectiveness.

  • Phase 3: This phase ensures a drug meets our standard of care. Phase 3 is designed to verify if a drug can improve on how we currently treat a diagnosis. 

Financial assistance to help fund your trial participation


If your child qualifies for a clinical trial in our program, we provide guidance in finding financial resources to help cover the cost of the trial. In addition to working with Missouri and Kansas Medicaid programs, we also assist with access to Medicaid programs in other states. Many parts of the clinical trial’s process, such as labs, X-rays, and other essentials tests, are covered by your health insurance. 

The research team


The Experimental Therapeutics Research Team works to provide the best in cancer research expertise while providing the emotional support your family needs. Our team includes four doctors, one nurse practitioner, four research coordinators, and one social worker. 

Investigational Drug Service (IDS) Pharmacy


The Children’s Mercy IDS Pharmacy is a full service pharmacy providing clinical knowledge and operational support for all studies involving investigational drugs at Children’s Mercy. As part of the Experimental Therapeutics Team, the IDS Pharmacy staff provides protocol and sponsor directed security, accountability, education, and dispensing of all Investigational as well as ancillary medications.

Cancer trials currently available


This list changes frequently and does not include most of our Children’s Oncology Group trials.

For more information about current available trials please email us.

Treatment Area

Study Name

Acute Lymphoblastic Leukemia AALL1521: A Phase 2 Study of the JAK1/JAK2 Inhibitor Ruxolitinib With Chemotherapy in Children With De Novo High-Risk CRLF2-Rearranged and/or JAK Pathway–Mutant Acute Lymphoblastic Leukemia
High Grade Glioma ACNS1721: A Phase 2 Study of Veliparib and Local Irradiation, Followed by Maintenance Veliparib and Temozolomide, in Patients with Newly Diagnosed High-Grade Glioma without H3 K27M or BRAFV600E Mutations
Relapsed/Refractory Juvenile Myelomonocytic Leukemia ADVL1521: Phase 2 Study of the MEK inhibitor Trametinib in Children with Relapsed or Refractory Juvenile Myelomonocytic Leukemia
Refractory Solid Tumors  ADVL1622: A Phase 2 Trial of XL184 (Cabozantinib) an Oral Small-Molecule Inhibitor of Multiple Kinases in Children and Young Adults with Refractory Sarcomas, Wilms Tumor, and Other Rare Tumors
Relapsed/Refractory Rhabdomyosarcoma, Nonrhabdomyosarcoma Soft Tissue Sarcoma and Ewing Sarcoma ADVL1722: A Phase 2, Multicenter, Open-Label Study to Assess Safety and Preliminary Activity of Eribulin Mesylate in Pediatric Subjects with Relapsed/Refractory Rhabdomyosarcoma, Nonrhabdomyosarcoma Soft Tissue Sarcoma and Ewing Sarcoma
Relapsed/Refractory Solid Tumors ADVL1921: Phase 1 Study to Evaluate the Safety and Pharmacokinetics of Palbociclib in Combination with Irinotecan and Temozolomide in Pediatric Patients with Recurrent or Refractory Solid Tumors
Relapsed/Refractory Classic Hodgkin Lymphoma AHOD1721: A Phase 2 Trial of Nivolumab + Brentuximab Vedotin for Children, Adolescents, and Young Adults with Relapsed/Refractory CD30 + Classic Hodgkin Lymphoma After Failure of First-Line Therapy, Followed by Brentuximab + Bendamustine for Participants with a Suboptimal Response.
Relapsed/Refractory or Progressive Neuroblastoma ANBL1821: Phase 2 Randomized Study of Irinotecan/Temozolomide/Dinutuximab with or without Eflornithine (DFMO) (IND# 141913) in Children with Relapsed, Refractory or Progressive Neuroblastoma
Recurrent/Refractory Solid Tumors APEC1621SC: NCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice) Screening Protocol
Recurrent/Refractory Solid Tumors APEC1621A: NCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice) Phase 2 Subprotocol of LOXO-101 (Larotrectinib) in Patients with Tumors Harboring Actionable NTRK Fusions
Recurrent/Refractory Solid Tumors APEC1621B: NCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice) Phase 2 Subprotocol of JNJ-42756493 (Erdafitinib) in Patients with Tumors Harboring FGFR1/2/3/4 Alterations.
Recurrent/Refractory Solid Tumors APEC1621C: NCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice) Phase 2 Subprotocol of Tazemetostat for Patients with Tumors Harboring Alterations in EZH2 or Members of the SWI/SNF Complex
Recurrent/Refractory Solid Tumors APEC1621D: NCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice) Phase 2 Subprotocol of LY3023414 in Solid Tumors
Recurrent/Refractory Solid Tumors APEC1621E: NCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice) Phase 2 Subprotocol of Selumetinib in Patients with Tumors Harboring Activating MAPK Pathway Mutations
Recurrent/Refractory Solid Tumors APEC1621F: NCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice) Phase 2 Subprotocol of Ensartinib in Patients with Tumors Harboring ALK or ROS1 Genomic Alterations
Recurrent/Refractory Solid Tumors APEC1621G: NCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice) Phase 2 Subprotocol of Vemurafenib in Patients with Tumors Harboring Actionable BRAF V600 Mutations
Recurrent/Refractory Solid Tumors APEC1621H: NCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice) Phase 2 Subprotocol of Olaparib in Patients with Tumors Harboring Defects in DNA Damage Repair Genes
Relapsed/Refractory Solid Tumors APEC1621I: NCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice) Phase 2 Subprotocol of Palbociclib in Patients with Tumors Harboring Activating Alterations in Cell Cycle Genes
Recurrent/Refractory Solid Tumors APEC1621J: NCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice) Phase 2 Subprotocol of BVD-523FB in Patients with Tumors Harboring Activating MAPK Pathway Mutations
BRAF V600 Mutation Positive Relapsed/Refractory High Grade Glioma CDRB436G2201: Phase 2 Open-Label Global Study to Evaluate the Effect of Dabrafenib in Combination with Trametinib in Children and Adolescent Patients with BRAF V600 Mutation Positive Relapsed or Refractory High Grade Glioma (HGG)
Newly Diagnosed High-Risk Neuroblastoma NMTRC012: A Study Using Molecular Guided Therapy with Induction Chemotherapy Followed by a Randomized Controlled Trial of Standard Immunotherapy with or without DFMO Followed by DFMO Maintenance for Subjects with Newly Diagnosed High-Risk Neuroblastoma
Neuroblastoma in Remission NMTRC014: Neuroblastoma Maintenance Therapy Trial Using Difluoromethylornithine (DFMO)
Relapsed/Refractory Solid Tumors or CNS tumors AflacST1502: A Phase 2 Study of Sirolimus in Combination with Metronomic Chemotherapy in Children with Recurrent and/or Refractory Solid and CNS Tumors
Relapsed T Cell Lymphoblastic Leukemia/Lymphoma AflacLL1602: A Phase 1 Trial Using Everolimus in Combination with Nelarabine, Cyclophosphamide and Etoposide in Relapsed T Cell Lymphoblastic Leukemia/Lymphoma

 

Children's Research Institute

The Children’s Research Institute at Children's Mercy is creating an integrated research environment where no boundaries exist between science and medicine. In our quest to find answers to pediatric medicine’s most challenging questions, we are collaborating with physicians, scientists, academic colleagues, philanthropic partners and others within our community, and around the world.