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Brain Tumor Clinical Trials

For questions about new and recurrent brain tumor clinical trials, please email the research nurse coordinator, Julia Mascia, or call 202-476-2907.

Title Description Key Eligibility Criteria Sponsor Investigator Disease Type
ADVL1711: A Phase 1/2 Study of Lenvatinib in Combination With Everolimus in Recurrent and Refractory Pediatric Solid Tumors, Including CNS Tumors
NCT03245151
A Phase 2 study to evaluate the efficacy and safety of Lenvatinib in combination with ifosfamide and etoposide in children, adolescents, and young adults with relapsed or refractory osteosarcoma.Phase I
  • Recurrent/refractory solid tumors or brain tumors
  • Ages ≥2 years old to <18 years old
Phase 2
  • Recurrent/refractory Ewing sarcoma/pPNET, rhabdomyosarcoma or high grade glioma
  • Ages ≥2 years old to ≤21 years old
Eisai, Inc.AeRang Kim, M.D., Ph.D.Ewing sarcoma, pPNET, rhabdomyosarcoma, high grade glioma, solid tumors, brain tumors, diffuse intrinsic pontine glioma
ADVL1823: Larotrectinib (LOXO-101, NSC# 788607, IND# 141824) for Previously Untreated TRK Fusion Pediatric Solid Tumors and TRK Fusion Relapsed Pediatric Acute Leukemias
NCT03834961
A Phase 2 study of Larotrectinib, a highly selective oral small molecule inhibitor of the TRK family of tyrosine kinases which are encoded by the NTRK genes in select pediatric cancers.
  • Cohort A: histologic diagnosis of infantile fibrosarcoma with an NTRK1, NTRK2, or NTRK3 fusion
  • Cohort B: histologic diagnosis of any solid tumor other than infantile fibrosarcoma, including CNS tumors but excluding high grade gliomas, with an NTRK1, NTRK2, or NTRK3 fusion
  • Cohort C: histologic diagnosis of relapsed/refractory acute leukemia with an NTRK1, NTRK2, or NTRK3 fusion
  • Ages ≤ 30 years of age
Children's Oncology GroupAeRang Kim, M.D., Ph.D.TRK fusion solid tumors, including CNS and TRK Fusion acute leukemias
ADVL1414: A Phase 1 Study of Selinexor (KPT-330, IND #125052), a Selective XPO1 Inhibitor, in Recurrent and Refractory Pediatric Solid Tumors, including CNS Tumors
NCT02323880
This phase 1 trial of selinexor in relapsed or refractory pediatric solid tumors or high-grade gliomas.
  • Part A: Recurrent/refractory solid tumors, including lymphoma and CNS tumors
  • Part B: Recurrent/refractory high grade glioma (WHO Grade III/IV) including disseminated tumors (excluding diffuse intrinsic pontine glioma), not requiring surgical resection
  • Part C: Patients with recurrent/refractory high grade glioma (WHO Grade III/IV) and requiring surgical resection (excluding diffuse intrinsic pontine glioma and disseminated tumors)
  • Ages ≥ 12 months old to ≤ 21 years old
Children's Oncology GroupJeffrey Dome, M.D., Ph.D.High grade glioma, all solid tumors, all CNS, lymphoma
ADVL1514: A Phase 1 Study of ABI-009 (nab-rapamycin) in Pediatric Patients with Recurrent or Refractory Solid Tumors, including CNS Tumors as a Single Agent and in Combination with Temozolomide and Irinotecan
NCT02975882
This phase 1 of the nanoparticle albumin-bound rapamycin when given together with temozolomide and irinotecan in treating pediatric patients with refractory or relapsed solid tumors
  • Recurrent/refractory solid tumors, including CNS tumors
  • Ages ≥ 12 months old to ≤ 21 years old
Children's Oncology GroupAeRang Kim, M.D., Ph.D.All CNS, all solid tumors
ADVL1615: A Phase 1 Study of Pevonedistat (MLN4924, IND# 136078), a NEDD8 Activating Enzyme (NAE) Inhibitor, in Combination with Temozolomide and Irinotecan in Pediatric Patients with Recurrent or Refractory Solid Tumors
NCT03323034
This is a phase 1 dose escalation of pevonedistat as a single agent and in combination with irinotecan and temozolomide for pediatric patients with refractory or recurrent solid tumors, including CNS tumors and lymphoma.
  • Recurrent/refractory solid tumors, including CNS tumors and lymphoma, for which no standard therapy is available are eligible
  • Part A2: Ages ≥ 6 months to < 12 months
Children's Oncology GroupAeRang Kim, M.D., Ph.D.All CNS, all solid tumors, lymphoma
ADVL1921: Phase 1 study to evaluate the safety and pharmacokinetics of Palbociclib (Ibrance®) in combination with Irinotecan and Temozolomide and in combination with Topotecan and Cyclophosphamide in pediatric patients with recurrent or refractory solid tumors
NCT03709680
This is a Phase 1 study of palbociclib in combination with chemotherapy (irinotecan and temozolmide or topotecan and cyclophosphamide) in pediatric patients with recurrent/refractory solid tumors
  • Relapsed/refractory solid tumors, including CNS tumors
  • Ages ≥ 2 to < 21 years
PfizerAeRang Kim, M.D., Ph.D.All solid tumors, CNS
PBTC-029: A Phase 1 and Phase II and Re-Treatment Study of AZD6244 for Recurrent or Refractory Pediatric Low Grade Glioma
NCT01089101
Use of oral Selumetinib a MEK inhibitor for patients with recurrent, refractory low grade gliomas.
  • Recurrent/refractory low grade glioma
  • Stratum 2: NF-1 without BRAF abberation, BRAF V600e mutation and/or fusion
  • Stratum 5: Non-NF-1 LGG with BRAF abberarion
  • Stratum 6: Non NF-1 LGG who have inadequate tissue quality, assay failure
  • Ages ≥ 3 to ≤ 21 years
Pediatric Brain Tumor ConsortiumLindsay Kilburn, M.D.Low grade glioma
PBTC-045: Pembrolizumab in Treating Younger Patients With Recurrent, Progressive, or Refractory High-Grade Gliomas, Diffuse Intrinsic Pontine Gliomas, Hypermutated Brain Tumors, Ependymoma or Medulloblastoma
NCT02359565
Uses intravenous Pembrolizumab, a humanized immunoglobulin, which binds to PD-1 receptor for patients with progressive or refractory brain tumors.
  • Recurrent, progressive, or refractory non-brainstem high grade glioma, hypermutant high grade glioma, ependymoma and medulloblastoma
  • Multi-focal parenchymal disease is ineligible
  • Ages ≥ 1 to ≤ 18 years
Pediatric Brain Tumor ConsortiumEugene Hwang, M.D.High-grade gliomas, diffuse intrinsic pontine gliomas, hypermutated brain tumors, ependymoma or medulloblastoma
PBTC-047: Phase 1 trial of Panobinostat in Children with Diffuse Intrinsic Pontine Glioma
NCT02717455
Use of oral Panobinostat a pan-HDAC inhibitor for patients with DIPGs.
  • Non-progressed diffuse intrinsic pontine glioma
  • Only received radition therapy as prior treatment
  • Ages ≥ 2 to ≤ 22 years
Pediatric Brain Tumor ConsortiumLindsay Kilburn, M.D.Diffuse intrinsic pontine glioma
PBTC-053: A Pediatric Brain Tumor Consortium Phase I/ II and Surgical Study of CX-4945 in Patients with Recurrent SHH Medulloblastoma
NCT03904862
Use of oral CX-4945 which is a tetracyclic synthetically derived small molecule carboxylate acid salt that exhibits. potent and highly selective inhibition of Casein Kinase 2 (CK2).
  • Recurrent or progressive medulloblastoma with the SHH subtype only
  • Tissue is required to send to sponsor for confirmatory results of subgroup type
  • Ages ≥ 3 and ≤ 18 years
Pediatric Brain Tumor ConsortiumLindsay Kilburn, M.D.Recurrent SHH medulloblastoma, for skeletally mature and skeletally immature patients
PBTC-055: Phase I/II trial of Dabrafenib, Trametinib, and Hydroxychloroquine (HCQ) for BRAF V600E-mutant or Trametinib and HCQ for BRAF fusion/duplication positive or NF1-associated recurrent or progressive gliomas in children and young adults
NCT04201457
Use of combination of Trametinib, Dabrafenib and Hydroxychloroquine or combination of Trameinib and Hydroxychloroquine for patients with gliomas with BRAF aberration and or V600E mutant.
  • Stratum 1: Low grade gliomas or high grade gliomas with BRAF V600e/D/K mutation
  • Stratum 2: Low grade gliomas with BRAF duplication or fusion or with NF-1, or High grade glioma with BRAF duplication/fusion with NF-1
  • Ages ≥ 1 to ≤ 30 years
Pediatric Brain Tumor ConsortiumEugene Hwang, M.D.Low grade glioma with BRAF aberration, high grade glioma with BRAF aberration, low grade glioma with neurofibromatosis type 1
PNOC-012: A Randomized, Blinded Study of Fluorescence Detection of Pediatric Primary Central Nervous System Tumors in Subjects Receiving Tozuleristide and Imaged With the Canvas System
NCT03579602
The purpose of this study is to evaluate how well Tozuleristide imaged with Canvas work at helping to distinguish between tumor and normal tissue during surgery in pediatric primary central nervous system tumors.
  • Undergoing a known total surgical resection
  • Prior therapy is eligible as long as recovered from toxic effects
  • Ages ≥ 1 month to ≤ 30 years
Pacific Neuro-Oncology ConsortiumLindsay Kilburn, M.D.Any brain tumor that requires a complete surgical resection
PNOC-013: A Safety and Pharmacokinetic Study of Single Agent REGN2810 in Pediatric Patients With Relapsed or Refractory Solid or Central Nervous System (CNS) Tumors and a Safety and Efficacy Trial of REGN2810 in Combination With Radiotherapy in Pediatric Patients With Newly Diagnosed Diffuse Intrinsic Pontine Glioma, Newly Diagnosed High-Grade Glioma, or Recurrent High-Grade Glioma
NCT03690869
Uses the investigational agent REGN2810, a human monoclonal antibody targeting the PD-1 receptor shown to be present in many High Grade Gliomas.
  • Newly diagnosed diffuse intrinsic pontine glioma, high grade glioma, recurrent high grade glioma and solid tumors
  • Primary diagnosis of high grade glioma of the spinal cord or disseminated disease not eligible
  • Ages 3 to 25 years
Pacific Neuro-Oncology ConsortiumLindsay Kilburn, M.D.Diffuse intrinsic pontine glioma, high grade glioma, recurrent high grade glioma, solid tumors
PNOC-007: H3.3K27M Specific Peptide Vaccine Combined With Poly-ICLC With and Without PD-1 Inhibition Using Nivolumab for the Treatment of Newly Diagnosed HLA-A2 + H3.3K27M Positive Diffuse Intrinsic Pontine Glioma (DIPG) and Newly Diagnosed HLA-A2 + H3.3K27M Positive Gliomas
NCT02960230
Uses biological agent K27M peptide vaccines along with PD-1 inhibitor infusion of Nivolumab.
  • Newly diagnosed diffuse intrinsic pontine glioma or midline glioma who have a positive H3.327M mutation
  • Must not have received any prior chemotherapy or treatments other than radiation
  • Time from diagnosis to start of radiation therapy must be no longer that 4 weeks
  • Must be on a stable or decreasing dose of corticosteroids
  • Ages 3 to 21 years
Pacific Neuro-Oncology ConsortiumLindsay Kilburn, M.D.Diffuse intrinsic pontine glioma, diffuse midline glioma, H3 K27M mutant
PNOC-008: 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 Intrinsic Pontine Glioma)
NCT03739372
Uses a new treatment approach based on each patient's tumor gene expression, whole-exome sequencing (WES), targeted panel profile (UCSF 500 gene panel), and RNA-Seq.
  • Newly diagnosed high grade glioma
  • Patients with disseminated disease are not eligible
  • Ages ≤ 21 years
Pacific Neuro-Oncology ConsortiumLindsay Kilburn, M.D.High grade glioma
Dabrafenib: 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 Intrinsic Pontine Glioma)
NCT02684058
Patients will be stratified to either the combination of Trametinib & Dabrafenib vs Carboplatin and Vincristine.
  • High grade glioma with BRAF V600e who have relapsed, progressed or failed frontline therapy
  • Low grade glioma with BRAF V600e with progressive disease following surgery
  • Ages ≥ 12 months to ≤ 18 years
Novartis PharmaceuticalsLindsay Kilburn, M.D.Diffuse astrocytoma, anaplastic astrocytoma, astrocytoma, oligodendroglioma, anaplastic oligodendroglioma, glioblastoma, ganglioglioma
ACTION: Adoptive Cellular Therapy Following Dose-Intensified Temozolomide in Newly diagnosed Pediatric High-grade Gliomas (Phase I).
NCT03334305
Uses cellular therapy with Dendritic cells and T-cells to activate the immune system against the tumor cells.
  • Newly diagnosed high grade glioma
  • Must not have had any prior treatments
  • Ages 3 to 21 years
University of FloridaEugene Hwang, M.D.High grade glioma
EH102: A Phase 1 Study of the EZH2 Inhibitor Tazemetostat in Pediatric Subjects with Relapsed or Refractory INI1-Negative Tumors or Synovial Sarcoma
NCT02601937
This is a phase 1 study of Tazemetostat for patients with INI1 negative tumors of CNS or rhabdoid solid tumors.
  • Relapsed/refractory disease of malignant rhabdoid tumors, including CNS
  • Ages > 6 months to < 18 years
EpizymeLindsay Kilburn, M.D.Rhabdoid tumors, INI1 negative tumors includes CNS tumors
OZM-077: Phase I/Ib Trial of Combined 5'Azacitidine and Carboplatin for Recurrent/Refractory Pediatric Brain and Solid Tumors
NCT03206021
Phase 1/1b study of the combination of azacitidine and carboplatin for CNS Ependymomas and childhood solid tumors.
  • Recurrent/refractory brain or solid tumor including recurrent/refractory ependymomas
  • Must not have received any investigational treatment within 30 days prior
  • Need tissue from diagnosis or resection
  • Ages 1 to < 18 years
The Hospital for Sick ChildrenEugene Hwang, M.D.CNS ependymoma, solid tumors
REMIND: Phase I Research on Multi-antigen T Cell Infusion Against Neuro-oncologic Disease
NCT03652545
This Phase I dose-escalation trial is designed to determine the safety and feasibility of rapidly generated tumor multi-antigen associated specific cytotoxic T lymphocytes (TAA-T) in patients with newly diagnosed diffuse intrinsic pontine gliomas DIPGs (Group A) or recurrent, progressive, or refractory non-brainstem CNS malignancies (Group B).
  • High risk CNS tumors
  • Ages 6 months to 80 years
Catherine Bollard, M.D. and Children's National HospitalEugene Hwang, M.D.Diffuse pontine gliomas, high grade brain tumor
PBTC-048: Feasibility trial of Optune device for Children with recurrent or progressive Supratentorial High grade Gliomas & Ependymomas
NCT03033992
Uses a device that works with tumor treatment fields by means of transducer arrays placed on shaved scalp.
  • Supratentorial high grade gliomas, ependymomas that are recurrent, progressive or refractory
  • Ages ≥ 5 to ≤ 21 years
Pediatric Brain Tumor ConsortiumLindsay Kilburn, M.D.Brain tumors located above the supratentorium
CONNECT1702: A Phase 1b Study of PTC596 in Children With Newly Diagnosed Diffuse Intrinsic Pontine Glioma and High Grade Glioma
NCT03605550
Uses investigational agent PTC-596 in combination with radiotherapy for patients with High grade brain tumors.
  • Newly diagnosed diffuse intrinsic pontine glioma, high grade glioma H3K27 mutant diffuse midline glioma, anaplastic astrocytoma, glioblastoma
  • Ages ≥ 12 months to ≤ 21 years
Nationwide Children's HospitalEugene Hwang, M.D.High grade glioma, diffuse intrinsic pontine glioma