Neuro Oncology

No one investigator is going to find solutions for pediatric brain and spinal cord tumors alone. That’s why our team is deeply rooted in global initiatives around the world to provide solutions and advance discovery. If there is a collaborative effort to find new discoveries for pediatric brain and spinal cord tumors, our team is a part of it.

DIPG (Diffuse Intrinsic Pontine Glioma)

A Phase 1 Open Label, Multi Center Study to Evaluate the Safety and Tolerability of BXQ-350 in Children with Newly Diagnosed Diffuse Intrinsic Pontine Glioma (DIPG) and Diffuse Midline Glioma (DMG)

Summary: This study will evaluate the safety of BXQ-350 and determine the maximum tolerated dose (MTD) in children with newly diagnosed DIPG or DMG. All patients will receive BXQ-350 by intravenous (IV) infusion and radiation therapy. The study is divided into two parts: Part 1 will enroll patients at increasing dose levels of BXQ-350 in order to determine the MTD. Part 2 will enroll patients requiring a biopsy in order to assess BXQ-350 concentrations in the biopsied tumor. ClinicalTrials.gov Identifier: NCT04771897 Primary investigator: Dr. Maryam Fouladi

A Phase 1b Study of PTC596 in Children with Newly Diagnosed Diffuse Intrinsic Pontine Glioma and High Grade Glioma

Summary: In this research study the investigators want to learn more about the safety of the study drug, PTC596 has when taken during radiation. The investigators also want to learn about the effects, if any, these drugs have on children and young adults with brain tumors. ClinicalTrials.gov Identifier: NCT03605550 Primary investigator: Dr. Maryam Fouladi

PNOC 007, H3.3K27M Specific Peptide Vaccine Combined with poly-ICLC for the Treatment of newly diagnosed HLA-A2+ H3.3K27M Positive Diffuse Intrinsic Pontine Glioma (DIPG) as well as other newly diagnosed HLA-A2+ H3.3K27M Positive Gliomas

Summary: Eligible patients will undergo focal radiation therapy after initial diagnosis as part of their standard of care per institutional guidelines. Patients must begin radiation therapy within 4 weeks of diagnosis by imaging, pathology, or surgery, whichever is later. Clinical Trials NCT Number: NCT02960230 Primary investigator: Dr. Margot Lazow

A Safety and Pharmacokinetic Study of Single Agent REGN2810 in Pediatric Patients with Relapsed or Refractory Solid or CNS Tumors and a Safety and Efficacy Trial of REGN2810 in Combination with Radiotherapy in Pediatric Patients with ndDIPG, ndHGG and rHGG

Summary: To confirm the safety and anticipated recommended phase 2 dose (RP2D) of REGN2810 (cemiplimab) for children with recurrent or refractory solid or Central Nervous System (CNS) tumors Clinical Trials NCT Number: NCT03690869 Primary investigator: Dr. Margot Lazow

A Target Validation Study of Fimepinostat in Children and Young Adults with Newly Diagnosed Diffuse Intrinsic Pontine Glioma (DIPG), Recurrent Medulloblastoma, or Recurrent HGG

Summary: This trial studies how well fimepinostat works in treating patients with newly diagnosed diffuse intrinsic pontine glioma, or medulloblastoma, or high-grade glioma that have come back. Fimepinostat may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Clinical Trials NCT Number: NCT03893487 Primary investigator: Dr. Margot Lazow

Medulloblastoma

ACNS1422 A Phase 2 Study of Reduced Therapy for Newly Diagnosed Average-RiskWNT-Driven Medulloblastoma Patients

Summary: This phase II trial studies how well reduced doses of radiation therapy to the brain and spine (craniospinal) and chemotherapy work in treating patients with newly diagnosed type of brain tumor called WNT)/Wingless (WNT)-driven medulloblastoma Clinical Trials NCT Number: NCT02724579 Primary investigator: Dr. Mark Ranalli

HEAD START 4 PROTOCOL: Newly Diagnosed Children (Less Than 10 Years Old) With Medulloblastoma And Other Central Nervous System Embryonal Tumors. Clinical And Molecular Risk-Tailored Intensive And Compressed Induction Chemotherapy Followed By Consolidation With Randomization To Either Single-Cycle Or To Three Tandem Cycles Of Marrow-Ablative Chemotherapy With Autologous Hematopoietic Progenitor Cell Rescue

Summary: This is a prospective randomized clinical trial, to determine whether dose-intensive tandem Consolidation, in a randomized comparison with single cycle Consolidation, provides an event-free survival and overall survival. Clinical Trials NCT Number: NCT02875314 Primary investigator: Dr. Randall Olshefski

PNOC 005: A Phase 1 Study of Modified Measles Virus (MV-NIS) for the Treatment of Children and Young Adults with Recurrent Medulloblastoma or Recurrent ATRT

Summary: This study will look to determine the safety and recommended phase 2 dose of the modified measles virus (MV-NIS) in children and young adults with recurrent medulloblastoma or atypical teratoid rhabdoid tumor (ATRT). ClinicalTrials.gov Identifier: NCT02962167 Primary Investigator: Dr. Margot Lazow

Central Nervous System (CNS) 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

APEC1621D NCI-COG Pediatric Match (Molecular Analysis For Therapy Choice)- Phase 2 Subprotocol Of LY3023414 In Patients With Solid Tumors

Summary: To determine the objective response rate in pediatric patients treated with LY3023414 with advanced solid tumors, non-Hodgkin lymphomas or CNS tumors that harbor TSC loss of function mutations. ClinicalTrials.gov Identifier: NCT03213678 Primary Investigator: Dr. Mark Ranalli

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

Summary: This phase II Pediatric MATCH trial studies how well ensartinib works in treating patients with solid tumors, non-Hodgkin lymphoma, or histiocytic disorders with ALK or ROS1 genomic alterations that have come back or do not respond to treatment (refractory) and have spread to other places in the body. ClinicalTrials.gov Identifier: NCT03213652 Primary Investigator: Dr. Mark Ranalli

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

Summary: To determine the objective response rate in pediatric patients treated with olaparib with advanced solid tumors (including CNS tumors), non-Hodgkin lymphomas or histiocytic disorders that harbor activating genetic alterations in the deleterious genetic alterations in the DNA damage repair pathway. ClinicalTrials.gov Identifier: NCT03233204 Primary Investigator: Dr. Mark Ranalli

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

Summary: This phase II Pediatric MATCH trial studies how well palbociclib works in treating patients with Rb positive solid tumors, non-Hodgkin lymphoma, or histiocytic disorders with activating alterations in cell cycle genes that have spread to other places in the body and have come back or do not respond to treatment. ClinicalTrials.gov Identifier: NCT03526250 Primary Investigator: Dr. Mark Ranalli

A Phase 1/2, Open-Label, Dose-Escalation And Expansion Study Of Entrectinib (RXDX-101) In Pediatrics And Young Adults With No Curative First-Line Treatment Option Or Recurrent/Refractory Solid Tumors And Primary CNS Tumors

Summary: This is an open-label, Phase 1/2 multicenter dose escalation study in pediatric patients with relapsed or refractory extracranial solid tumors (Phase 1), with additional expansion cohorts (Phase 2) in patients with primary brain tumors harboring NTRK1/2/3 or ROS1 gene fusions, and extracranial solid tumors harboring NTRK1/2/3 or ROS1 gene fusions. ClinicalTrials.gov Identifier: NCT02650401 Primary Investigator: Dr. Ralph Salloum

Gliomas

A Phase 3 Randomized Study of Selumetinib (IND # 77782) versus Carboplatin/Vincristine in Newly Diagnosed or Previously Untreated Neurofibromatosis Type 1 (NF1) Associated Low-Grade Glioma (LGG)

Summary: This phase III trial studies if selumetinib works just as well as the standard treatment with carboplatin/vincristine (CV) for subjects with NF1-associated low grade glioma (LGG), and to see if selumetinib is better than CV in improving vision in subjects with LGG of the optic pathway (vision nerves). ClinicalTrials.gov Identifier: NCT03871257 Primary Investigator: Dr. Mark Ranalli

A Phase 3 Randomized Non-Inferiority Study of Carboplatin and Vincristine versus Selumetinib (NSC# 748727, IND# 77782) in Newly Diagnosed or Previously Untreated Low-Grade Glioma (LGG) not associated with BRAFV600E Mutations or Systemic Neurofibromatosis Type 1 (NF1)

Summary: This phase 3 trial compares the effect of selumetinib versus the standard of care treatment with carboplatin and vincristine (CV) in treating patients with newly diagnosed or previously untreated low-grade glioma (LGG) that does not have a genetic abnormality called BRAFV600E mutation and is not associated with systemic neurofibromatosis type 1. ClinicalTrials.gov Identifier: NCT04166409 Primary Investigator: Dr. Mark Ranalli

A Phase 3 Study of Selumetinib (NSC# 748727, IND# 77782) or Selumetinib in Combination with Vinblastine for non-NF1, non-TSC Patients with Recurrent or Progressive Low-Grade Gliomas (LGGs) Lacking BRAFV600E or IDH1 Mutations

Summary: The purpose of this study is to see if adding vinblastine to treatment with the drug selumetinib is more effective than selumetinib alone for children and young adults with low-grade glioma (a common type of brain cancer) that has come back after prior treatment or does not respond to therapy. ClinicalTrials.gov Identifier: NCT04576117 Primary Investigator: Dr. Mark Ranalli

A Pilot and Surgical Study of Larotrectinib from Treatment of Children with Newly Diagnosed High-Grade Glioma with NTRK Fusion

Summary: This is a pilot study that will evaluate disease status in children that have been newly diagnosed high-grade glioma with TRK fusion. The evaluation will occur after 2 cycles of the medication (Larotrectinib) have been given. ClinicalTrials.gov Identifier: NCT04655404 Primary Investigator: Dr. Maryam Fouladi

A Phase III Study Comparing Two Carboplatin Containing Regimens for Children and Young Adults with Previously Untreated Low Grade Glioma

Summary: This study is trying to learn and understand if the chemotherapy drug called carboplatin works as well as the standard therapy. ClinicalTrials.gov Identifier: NCT02455245 Primary Investigator: Dr. Margot Lazow

A Pilot Trial Testing the Clinical Benefit of Using Molecular Profiling to Determine an Individualized Treatment Plan in Children and Young Adults with High-Grade Glioma (Excluding Diffuse Instrinsic Pontine Glioma)

Summary: The study will use a new treatment approach based on each patient’s tumor gene expression, whole-exome sequencing, targeted panel profile (UCSF 500 gene panel), and RNA-Seq. The current study will test the efficacy of such an approach in children with High-grade gliomas HGG. ClinicalTrials.gov Identifier: NCT03739372 Primary Investigator: Dr. Margot Lazow

A Target Validation/Phase 1 Study of BGB-290 in Combination with Temozolomide in Adolescent and Young Adult IDH1/2 Newly Diagnosed and Recurrent Mutant Gliomas

Summary: This phase I trial studies the side effects and best dose of BGB-290 and temozolomide in treating adolescents and young adults with IDH1/2-mutant grade I-IV glioma that is newly diagnosed or has come back. ClinicalTrials.gov Identifier: NCT03749187 Primary Investigator: Dr. Margot Lazow

Multiple Diseases

Larotrectinib (LOXO-101, NSC# 788607, IND# 141824) for Previously Untreated TRK Fusion Pediatric Solid Tumors and TRK Fusion Relapsed Pediatric Acute Leukemias

Summary: This phase II trial studies the side effects and how well larotrectinib works in treating patients with previously untreated TRK fusion solid tumors and TRK fusion acute leukemia that has come back. ClinicalTrials.gov Identifier: NCT03834961 Primary Investigator: Dr. Mark Ranalli

ADVL1921: PHASE 1 STUDY TO EVALUATE THE SAFETY AND PHARMACOKINETICS OF PALBOCICLIB (IBRANCE®) IN COMBINATION WITH IRINOTECAN AND TEMOZOLOMIDE IN PEDIATRIC PATIENTS WITH RECURRENT OR REFRACTORY SOLID TUMORS

Summary: This study will evaluate palbociclib in combination with chemotherapy (temozolomide with irinotecan and/or topotecan with cyclophosphamide) in children, adolescents and young adults with recurrent or refractory solid tumors. ClinicalTrials.gov Identifier: NCT03709680 Primary Investigator: Dr. Mark Ranalli

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

Summary: Determine the objective response rate in pediatric patients treated with erdafitinib with advanced solid tumors (including CNS tumors), non-Hodgkin lymphomas or histiocytic disorders that harbor genetic alterations in the FGFR1/2/3/4 pathway. ClinicalTrials.gov Identifier: NCT03210714 Primary Investigator: Dr. Mark Ranalli

APEC1621G NCI-COG PEDIATRIC MATCH (MOLECULAR ANALYSIS FOR THERAPY CHOICE)- PHASE 2 SUBPROTOCOL OF VEMURAFENIB IN PATIENTS WITH TUMORS HARBORING BRAF V600 MUTATIONS

Summary: This phase II Pediatric MATCH trial studies how well vemurafenib works in treating patients with solid tumors, non-Hodgkin lymphoma, or histiocytic disorders with BRAF V600 mutations that have spread to other places in the body (advanced) and have come back (recurrent) or do not respond to treatment (refractory). ClinicalTrials.gov Identifier: NCT03220035 Primary Investigator: Dr. Mark Ranalli

PHASE 2 SUBPROTOCOL OF AG-120 (IVOSIDENIB) IN PATIENTS WITH TUMORS HARBORING IDH1 MUTATIONS

Summary: This phase II Pediatric MATCH trial studies how well ivosidenib works in treating patients with solid tumors that have spread to other places in the body (advanced), lymphoma, or histiocytic disorders that have IDH1 genetic alterations (mutations). ClinicalTrials.gov Identifier: NCT04195555 Primary Investigator: Dr. Mark Ranalli

PHASE 2 SUBPROTOCOL OF TIPIFARNIB IN PATIENTS WITH TUMORS HARBORING HRAS GENOMIC ALTERATIONS

Summary: This phase II pediatric MATCH trial studies how well tipifarnib works in treating patients with solid tumors that have recurred or spread to other places in the body (advanced), lymphoma, or histiocytic disorders, that have a genetic alteration in the gene HRAS. ClinicalTrials.gov Identifier: NCT04284774 Primary Investigator: Dr. Mark Ranalli

APEC1621SC NCI-COG PEDIATRIC MATCH (MOLECULAR ANALYSIS FOR THERAPY CHOICE) SCREENING PROTOCOL

Summary: The main purpose of this study is to learn how well tumors that have specific genetic changes (mutations) respond to drugs that target those changes. ClinicalTrials.gov Identifier: NCT03155620 Primary Investigator: Dr. Mark Ranalli

A Phase 2 Study of Erdafitinib in Subjects with Advanced Solid Tumors and Selected FGFR Gene Alterations

Summary: The purpose of this study is to evaluate the efficacy of erdafitinib in terms of overall response rate (ORR) in adult and pediatric participants with advanced solid tumors with fibroblast growth factor receptor (FGFR) alterations (mutations or gene fusions). ClinicalTrials.gov Identifier: NCT04083976 Primary Investigator: Dr. Maryam Fouladi