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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
PEPN22P1: A Pharmacokinetic Study of Vincristine in Infants Dosed According to BSA-Banded Infant Dosing Tables and Older Children Dosed by Traditional BSA Methods
A correlative study of vincristine pharmacokinetics to evaluate a new infant dosing method and determine whether it results in uniform drug exposure (AUC) across the age groups.
  • Age: ≤ 12 years
  • Newly diagnosed and relapsed cancer diagnosis that is being treated with vincristine at the 1.5 mg/m2 dose level
Children's Oncology Group AeRang Kim, M.D. Any
PEPN2121: A Phase 1/2 Study of Tiragolumab and Atezolizumab in Patients with Relapsed or Refractory SMARCB1 or SMARCA4 Deficient Tumors
A Phase 1/2 study to evaluate the safety of tiragolumab as monotherapy in pediatric patients (<18 years) with SMARCB1 or SMARCA4 deficient tumors (Part A), to evaluate antitumor activity of the combination of tiragolumab and atezolizumab in patients with SMARCB1 or SMARCA4 deficient tumors (Part B), and to evaluate the safety and adverse event profile of this combination therapy in subjects with SMARCB1 or SMARCA4 deficient tumors, with a particular focus in pediatric patients < 12 years of age.
  • Patients must have SMARCB1 (INI1) or SMARCA4 deficient tumors
  • Part A age: ≥ 12 years and < 18 years
  • Part B age: ≥ 18 years
Children's Oncology Group AeRang Kim, M.D.Renal medullary carcinoma, malignant rhabdoid tumor (extra-CNS), atypical teratoid rhabdoid tumor (CNS), poorly differentiated chordoma, epithelioid sarcoma, other SMARCB1 or SMARCA4 deficient tumors
ONC-028 Intermediate-size Expanded Access to ONC201 for Patients with H3 K27M-mutant and/or Midline Gliomas
ONC201 will be administered once weekly in an outpatient setting. The small molecule has demonstrated efficacy in a range of preclinical models of advanced cancers.
  • Patient with evidence of leptomeningeal disease or CSF dissemination of disease are not eligible
OncoceuticsLindsay Kilburn, M.D.DIPG, high grade glioma, diffuse midline gliomas
PNOC-022 A Combination Therapy Trial using an Adaptive Platform Design for Children and Young Adults with Diffuse Midline Gliomas (DMGs) including Diffuse Intrinsic Pontine Gliomas (DIPGs) at Initial Diagnosis, Post-Radiation Therapy and at Time of Progression
This trial will randomize participants at study entry who are at different stages of disease.
  • Three cohorts open for participants with newly diagnosed DMG prior to radiation, DMG who have completed radiation and participants with DMG at progression
Pacific Neuro-Oncology ConsortiumLindsay Kilburn, M.D.Diffuse midline gliomas & DIPGs at initial diagnosis, or post radiation at the time of progression

PNOC-026/Day101: A Multicenter Study to 
Evaluate the Safety and Efficacy of the Oral Pan-RAF Inhibitor DAY 101

DAY101 is an oral, selective, small-molecule, Type II pan-RAF kinase inhibitor that is being developed for the treatment of recurrent or progressive pediatric low-grade gliomas.
  • Patients must have received at least one line of prior systemic therapy and have documented evidence of radiographic progression
Pacific Neuro-Oncology ConsortiumLindsay Kilburn, M.D.Recurrent low grade glioma, Solid tumors
SDT201: A dose-escalation study examining the safety preliminary efficacy of ascending drug and energy dose combinations for sonodynamic therapy using SONALA-001 in combination with Exablate 4000 Type-2 MR-Guided Focused Ultrasound
The aim is to introduce a new therapeutic modality to a patient population that currently has no effective standard-of-care option; therefore, we will test the combination therapy of SONALA-001 and MRgFUS delivered with the Exablate 4000 Type-2 device.
  • Prior treatment consisting of a minimum of 54 Gy standard focal irradiation administered over 42-49 days
  • Minimum of 5 years of age
SonalasenseLindsay Kilburn, M.D.Newly diagnosed DIPG
BT005:  A Feasibility Safety Study Using the Exablate 4000 System in the Management of Benign Centrally-Located Intracranial Tumors Which Require Clinical Intervention
A feasibility study is to develop data to evaluate the safety and feasibility of Exablate 4000 treatment of benign intracranial tumors.
  • Young adult and pediatric patients with MR-proven centrally located intracranial tumors which have demonstrated slow (benign) growth, and for whom the only other treatment option is conventional surgery
  • Young adult and pediatric patients with MR-proven centrally located intracranial tumors which have demonstrated slow (benign) growth, and for whom the only other treatment option is conventional surgery
InsightecRobert Keating, M.D.Benign centrally located tumors
PNOC-021: A Phase I Trial Evaluating the Combination of Trametinib and Everolimus in Pediatric and Young Adult Patients with Recurrent Low Grade Gliomas
This will be the first study to determine the optimal dosing schedule and probable side effects for a combination therapy of trametinib and everolimus in pediatric LGG.
  • Participants must have histologically confirmed diagnosis of a LGG (WHO grade I-II) that is recurrent or progressive after prior treatment
  • Participants who have had surgery alone are not eligible
Pacific Neuro-Oncology ConsortiumLindsay Kilburn, M.D.Low grade glioma

A Phase 1/2 Trial of CBL0137 (NSC# 825802, IND# 155843) in Patients with Relapsed or Refractory Solid Tumors including CNS Tumors and Lymphoma

This phase I/II trial evaluates the best dose, side effects and possible benefit of CBL0137 in treating patients with relapsed, or refractory solid tumors, including central nervous system (CNS) tumors or lymphoma.
  • Parts A and B1: ≥ 12 months and ≤ 21 years old
  • Part B2 (relapsed/refractory osteosarcoma): ≥ 12 months and ≤ 30 years old
Children's Oncology GroupAeRang Kim, M.D.Solid tumors, lymphoma
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
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
This is a Phase 1 study of palbociclib in combination with chemotherapy (irinotecan and temozolomide 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-045: Pembrolizumab in Treating Younger Patients With Recurrent, Progressive, or Refractory High-Grade Gliomas, Diffuse Intrinsic Pontine Gliomas, Hypermutated Brain Tumors, Ependymoma or Medulloblastoma
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-053: A Pediatric Brain Tumor Consortium Phase I/ II and Surgical Study of CX-4945 in Patients with Recurrent SHH Medulloblastoma
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
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-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
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
REMIND: Phase I Research on Multi-antigen T Cell Infusion Against Neuro-oncologic Disease
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
CONNECT1702: A Phase 1b Study of PTC596 in Children With Newly Diagnosed Diffuse Intrinsic Pontine Glioma and High Grade Glioma
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
VAL-10-001: Pilot Pharmacokinetic Study of VAL-413 (Orotecan®) in Patients with Recurrent Pediatric Solid Tumors


An open-label Phase I clinical trial designed to establish the recommended Phase II dose and to characterize the pharmacokinetic profile of VAL-413 when combined with temozolomide
  • Recurrent Pediatric Solid Tumors
  • ≥ 1 year of age or ≤ 30 years of age
Valent Technologies LLCAeRang Kim, M.D.Solid tumors, including CNS tumors 
BT-016A: Safety and Feasibility Study to Evaluate Blood Brain Barrier Disruption Using Exablate MR Guided Focused Ultrasound in Combination with Doxorubicin in Treating Pediatric Patients with Diffuse Intrinsic Pontine Gliomas


A feasibility of Blood Brain Barrier disruption using the Exablate 4000 Type 2 System in patients with DIPG undergoing Doxorubicin chemotherapyNewly diagnosed DIPG with a Tumor size volume approximately between 10 cc and 30 cc, At least 4-week and not greater than 12 weeks from completion of radiation therapyInsightecRoger Packer, MDNewly diagnosed diffuse intrinsic pontine glioma, high grade glioma
Firefly-2: A Phase 3, Randomized, International Multicenter Trial Of DAY101 Monotherapy Versus Standard Of Care Chemotherapy In Patients With Pediatric Low-Grade Glioma Harboring An Activating RAF Alteration Requiring First-Line Systemic Therapy


Comparing the objective response rates of DAY101 monotherapy versus standard of care (SoC) chemotherapy in patients with pediatric low grade glioma harboring an activating RAF alteration
Low grade gliomas who has not been treated prior, Tumor must harbor an RAF alteration
Day101 Pharmaceuticals
Lindsay Kilburn, MD
Low grade glioma
PBTC-060A Pilot Study of Safety, Tolerability, and Immunological Effects of SurVaxM in  Pediatric Patients with Progressive or Relapsed Medulloblastoma, High Grade Glioma,  Ependymoma and Newly Diagnosed Diffuse Intrinsic Pontine Glioma


The primary objective is to assess the toxicity profile of SurVaxM Vaccines in emulsion with Montanide plus sargramostim in children with relapsed or progressive medulloblastoma and high-grade glioma (HGG), ependymoma and non-recurrent diffuse intrinsic pontine glioma (DIPG) post-radiation therapy (RT)Demonstration  of survivin expression as assessed after screening consent/assent of at least 1% on tumor tissue by immunohistochemistry, patients must be progressive or relapsed and Patients must have received their last dose of known myelosuppressive anticancer therapy at least 21 days prior to enrollmentPediatric Brain Tumor Consortium
Eugene Hwang, MD
Progressive or Relapsed Medulloblastoma, High Grade Glioma, 
Ependymoma and Newly Diagnosed Diffuse Intrinsic Pontine Glioma
ACTION: Adoptive Cellular Therapy Following Dose-Intensified Temozolomide in Newly diagnosed Pediatric High-grade Gliomas (Phase I)


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 Florida
Eugene Hwang, MD High Grade Glioma