Brain Tumor Institute

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.

ONC-028 Intermediate-size Expanded Access to ONC201 for Patients with H3 K27M-mutant and/or Midline Gliomas

ClinicalTrials.gov Identifier: NCT04617002

Description

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.

Key Eligibility Criteria

Patient with evidence of leptomeningeal disease or CSF dissemination of disease are not eligible;

Sponsor

Oncoceutics

Investigator

Lindsay Kilburn, M.D.

Disease Type

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

ClinicalTrials.gov Identifier: NCT05009992

Description

This trial will randomize participants at study entry who are at different stages of disease.

Key Eligibility Criteria

Three cohorts open for participants with newly diagnosed DMG prior to radiation, DMG who have completed radiation and participants with DMG at progression

Sponsor

Pacific NeuroOncology Consortium

Investigator

Lindsay Kilburn, M.D.

Disease Type

Diffuse midline gliomas & DIPGs at initial diagnosis, or post radiation at the time of progression

PNOC-019 A Randomized, Double-Blinded, Pilot Trial of Neoadjuvant Checkpoint Inhibition followed by Combination Adjuvant Checkpoint Inhibition in Children and Young Adults with Recurrent or Progressive High Grade Glioma (HGG)

ClinicalTrials.gov Identifier: NCT04323046

Description

A phase 1 trial to evaluate the immunological local and systemic changes following neoadjuvant checkpoint inhibition.

Key Eligibility Criteria

Participants with recurrent or progressive HGG who are candidates for surgical tumor debulking
Surgery is required

Sponsor

Pacific NeuroOncology Consortium

Investigator

Lindsay Kilburn, M.D.

Disease Type

Recurrent, progressive high grade glioma

ACTION: Adoptive Cellular Therapy Following Dose-Intensified Temozolomide in Newly diagnosed Pediatric High-grade Gliomas (Phase I)

ClinicalTrials.gov Identifier: NCT03334305

Description

Uses cellular therapy with dendritic cells and T-cells to activate the immune system against the tumor cells.

Key Eligibility Criteria

Newly diagnosed high grade glioma
Must not have had any prior treatments
Ages 3 to 21 years

Sponsor

University of Florida

Investigator

Eugene Hwang, M.D.

Disease Type

High grade glioma

PNOC-016 A Target Validation Study of Fimepinostat in Children and Young Adults with Newly Diagnosed Diffuse Intrinsic Pontine Glioma (DIPG), Recurrent Medulloblastoma, or Recurrent High-Grade Glioma (HGG)

ClinicalTrials.gov Identifier: NCT03893487

Description

This study will include children and young adults (ages 3-39) with recurrent medulloblastoma who will undergo tissue collection.

Key Eligibility Criteria

Patients must undergo tumor tissue collection as part of their standard of care
Patients in the medulloblastoma and HGG strata will be allowed to have undergone prior therapy including surgery, chemotherapy and radiation therapy

Sponsor

Pacific NeuroOncology Consortium

Investigator

Lindsay Kilburn, M.D.

Disease Type

Recurrent medulloblastoma

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

ClinicalTrials.gov Identifier: NCT04775485

Description

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.

Key Eligibility Criteria

Patients must have received at least one line of prior systemic therapy and have documented evidence of radiographic progression

Sponsor

Pacific NeuroOncology Consortium

Investigator

Lindsay Kilburn, M.D.

Disease Type

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

ClinicalTrials.gov Identifier: NCT05123534

Description

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.

Key Eligibility Criteria

Prior treatment consisting of a minimum of 54 Gy standard focal irradiation administered over 42-49 days
Minimum of 5 years of age

Sponsor

Sonalasense

Investigator

Lindsay Kilburn, M.D.

Disease Type

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

ClinicalTrials.gov Identifier: NCT03028246

Description

A feasibility study is to develop data to evaluate the safety and feasibility of Exablate 4000 treatment of benign intracranial tumors.

Key Eligibility Criteria

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

Sponsor

Insightec

Investigator

Robert Keating, M.D.

Disease Type

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

ClinicalTrials.gov Identifier: NCT04485559

Description

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.

Key Eligibility Criteria

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

Sponsor

Pacific NeuroOncology Consortium

Investigator

Lindsay Kilburn, M.D.

Disease Type

Low grade glioma

PBTC-051: This is a multicenter phase 1 trial of APX005M for children with recurrent or refractory primary malignant CNS tumor or newly diagnosed DIPG.

ClinicalTrials.gov Identifier: NCT03389802

Description

The primary objectives of the study are to evaluate the safety of APX005M administered intravenously every 3 weeks to children with central nervous system tumors.

Key Eligibility Criteria

Patients must have not received any prior therapy for treatment of their current CNS malignancy other than radiation therapy

Sponsor

Pediatric Brain Tumor Consortium

Investigator

Eugene Hwang, M.D.

Disease Type

Newly diagnosed DIPG post RT

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

ClinicalTrials.gov Identifier: NCT04870944

Description

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.

Key Eligibility Criteria

Parts A and B1: ≥ 12 months and ≤ 21 years old
Part B2 (relapsed/refractory osteosarcoma): ≥ 12 months and ≤ 30 years old

Sponsor

Children's Oncology Group

Investigator

AeRang Kim, M.D.

Disease Type

Solid tumors, lymphoma

ADVL1823: Larotrectinib (LOXO-101, NSC# 788607, IND# 141824) for Previously Untreated TRK Fusion Pediatric Solid Tumors and TRK Fusion Relapsed Pediatric Acute Leukemias

ClinicalTrials.gov Identifier: NCT03834961

Description

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.

Key Eligibility Criteria

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

Sponsor

Children's Oncology Group

Investigator

AeRang Kim, M.D., Ph.D.

Disease Type

TRK fusion solid tumors, including CNS and TRK Fusion acute leukemias

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

ClinicalTrials.gov Identifier: NCT03323034

Description

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.

Key Eligibility Criteria

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

Sponsor

Children's Oncology Group

Investigator

AeRang Kim, M.D., Ph.D.

Disease Type

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

ClinicalTrials.gov Identifier: NCT03709680

Description

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

Key Eligibility Criteria

Relapsed/refractory solid tumors, including CNS tumors
Ages ≥ 2 to < 21 years

Sponsor

Pfizer

Investigator

AeRang Kim, M.D., Ph.D.

Disease Type

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

ClinicalTrials.gov Identifier: NCT02359565

Description

Uses intravenous Pembrolizumab, a humanized immunoglobulin, which binds to PD-1 receptor for patients with progressive or refractory brain tumors.

Key Eligibility Criteria

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

Sponsor

Pediatric Brain Tumor Consortium

Investigator

Eugene Hwang, M.D.

Disease Type

High-grade gliomas, diffuse intrinsic pontine gliomas, hypermutated brain tumors, ependymoma or medulloblastoma

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

ClinicalTrials.gov Identifier: NCT04201457

Description

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.

Key Eligibility Criteria

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

Sponsor

Pediatric Brain Tumor Consortium

Investigator

Eugene Hwang, M.D.

Disease Type

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

ClinicalTrials.gov Identifier: NCT03690869

Description

Uses the investigational agent REGN2810, a human monoclonal antibody targeting the PD-1 receptor shown to be present in many High Grade Gliomas.

Key Eligibility Criteria

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

Sponsor

Pacific Neuro-Oncology Consortium

Investigator

Lindsay Kilburn, M.D.

Disease Type

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

ClinicalTrials.gov Identifier: NCT03652545

Description

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).

Key Eligibility Criteria

High risk CNS tumors
Ages 6 months to 80 years

Sponsor

Catherine Bollard, M.D. and Children's National Hospital

Investigator

Eugene Hwang, M.D.

Disease Type

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

ClinicalTrials.gov Identifier: NCT03605550

Description

Uses investigational agent PTC-596 in combination with radiotherapy for patients with High grade brain tumors.

Key Eligibility Criteria

Newly diagnosed diffuse intrinsic pontine glioma, high grade glioma H3K27 mutant diffuse midline glioma, anaplastic astrocytoma, glioblastoma
Ages ≥ 12 months to ≤ 21 years

Sponsor

Nationwide Children's Hospital

Investigator

Eugene Hwang, M.D.

Disease Type

High grade glioma, diffuse intrinsic pontine glioma