Brain Tumor Institute

Brain Tumor Clinical Trials

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The Brain Tumor Institute at Children’s National is leading the way to find new and better treatments for children. Our clinical trials give families the chance to try different options that may help their child and others. We understand this is a big decision and our team is here to support you. For more information about the trials listed below, contact research nurse coordinators Julia Mascia and Julia Batarseh, or call 202-476-2907.

Contact by Email

Email Julia Mascia Email Julia Batarseh

Contact by Phone

Call 202-476-2907

Explore Clinical Trials by Disease Type (A-G)

Disease Status: Recurrent

CONNECT1905: Phase 2 Study of Systemic IL-6 Receptor Antagonist ACTEMRA® (tocilizumab) for the treatment of Pediatric Adamantinomatous Craniopharyngioma

ClinicalTrials.gov Identifier: NCT05233397

Description:

In this Phase II, tociluzumab will be used to treat pediatric patients diagnosed with recurrent Adamantinomatous Craniopharyngioma including patients who have undergone surgery and/or radiation therapy.

Key Eligibility Criteria:

Stratum 1: Patients ≥ 12 months and ≤ 39 years of age with recurrent or progressive adamantinomatous craniopharyngioma at least 6 months post completion of radiation therapy
Stratum 2: Patients ≥ 12 months and ≤ 39 years of age with recurrent or progressive adamantinomatous craniopharyngioma who have undergone surgery but have NOT previously undergone irradiation
Progressive disease is allowed but not required

Sponsor:

Nationwide Children's Hospital

Investigator:

Eugene Hwang, MD

CONNECT2108: Phase 2 Study of the MEK inhibitor MEKTOVI® (binimetinib) for the Treatment of Pediatric Adamantinomatous Craniopharyngioma

ClinicalTrials.gov Identifier: NCT05286788

Description

Adamantinomatous Craniopharyngioma (ACP) is a highly debilitating pediatric brain tumor that lacks medical anti-tumor therapies. Current therapy, which depends largely on surgery and radiation, is associated with poor quality of life and becomes more challenging and risky in the setting of recurrent disease. MEKTOVI (binimetinib) is an oral, highly selective reversible inhibitor of mitogen-activated extracellular signal regulated kinase 1 (MEK1) and MEK2 that is being evaluated in pediatric patients diagnosed with recurrent Adamantinomatous Craniopharyngioma (ACP), including patients who have undergone surgery and/or radiation therapy.

Key Eligibility Criteria

Patients aged >1 year and <25 years and must have progressive or recurrent ACP and have recovered or stabilized from the acute toxic effects of prior treatments
Progressive disease is allowed but not required

Sponsor

Nationwide Children’s Hospital

Investigator

Eugene I. Hwang, M.D.

Disease Type

Recurrent Adamantinomatous Craniopharyngioma (ACP)

Explore Clinical Trials by Disease Type (H-Z)

Disease Status: Newly Diagnosed

CONNECT1903: A Pilot and Surgical Study of Larotrectinib for Treatment of Children with Newly Diagnosed High-Grade Glioma with NTRK Fusion

ClinicalTrials.gov Identifier: NCT04655404

Description:

This study will evaluate disease status in children that have been newly diagnosed with high-grade glioma with NTRK fusion after 2 cycles of the medication (Larotrectinib) have been given. The study will also evaluate the safety of larotrectinib when given with chemotherapy or post-focal radiation therapy.

Key Eligibility Criteria:

Patients ≤ 21 years of age with newly diagnosed high grade glioma with NTRK fusion

Sponsor:

Nationwide Children's Hospital

Investigator:

Lindsay Kilburn, MD

UPCOMING: PNOC-020: A Phase I/II Study of RNA-lipid Particle (RNA-LP) Vaccines for Newly Diagnosed Pediatric High-Grade Gliomas (pHGG) and Adult Glioblastoma (GBM)

ClinicalTrials.gov Identifier: NCT04573140

Description:

The primary objective will be to demonstrate the manufacturing feasibility and safety, and to determine the maximum tolerated dose (MTD) of RNA-LP vaccines in (Stratum 1) adult patients with newly diagnosed GBM (MGMT low level or unmethylated in adults only) and (Stratum 2) in pediatric patients with newly diagnosed HGG (pHGG).

Key Eligibility Criteria:

Patients age > 3 and < 25 years with newly diagnosed high grade glioma, prior to surgical resection and radiation

Sponsor:

University of Florida

Investigator:

Lindsay Kilburn, MD

UPCOMING: CONNECT 1906: Phase 2 trial of a novel peptide vaccine (PEP-CMV) targeting CMV antigen for newly diagnosed pediatric high-grade glioma and diffuse intrinsic pontine glioma and recurrent medulloblastoma

ClinicalTrials.gov Identifier: NCT05096481

Description:

This study will address the question of whether targeting CMV antigens with PEP-CMV can serve as a novel immunotherapeutic approach in pediatric patients with newly diagnosed high-grade glioma (HGG) or diffuse intrinsic pontine glioma (DIPG) as well as recurrent medulloblastoma (MB).

Key Eligibility Criteria:

Patients aged ≥3 and ≤39 years with recurrent/progressive medulloblastoma, newly diagnosed high grade glioma, or newly diagnosed diffuse intrinsic pontine glioma (biopsy not required)

Sponsor:

Nationwide Children's Hospital

Investigator:

Eugene Hwang, MD

UPCOMING: CONNECT: TarGeT Screening: Targeted Pediatric High-Grade Glioma Therapy

ClinicalTrials.gov Identifier: NCT05839379

Official title:

Targeted Pediatric High-Grade Glioma Therapy

Description:

The goal of this study is to perform genetic sequencing on brain tumors from children, adolescents, and young adult patients who have been newly diagnosed with a high-grade glioma. This molecular profiling will decide if patients are eligible to participate in a subsequent treatment-based clinical trial based on the genetic alterations identified in their tumor.

Key Eligibility Criteria:

Patients ≥12 months and ≤39 years with newly diagnosed high-grade glioma (HGG), including diffuse intrinsic pontine glioma (DIPG) who have had a biopsy

Sponsor:

Nationwide Children's Hospital

Investigator:

Eugene Hwang, MD

UPCOMING: CONNECT: TarGeT-A: Phase 2 Study of Ribociclib and Everolimus Following Radiotherapy in Pediatric and Young Adult Patients Newly Diagnosed with HighGrade Glioma (HGG), including Diffuse Intrinsic Pontine Glioma (DIPG), which Harbor Alterations of the Cell Cycle and/or PI3K/mTOR Pathway

*For patients with PI3K/mTOR alterations

ClinicalTrials.gov ID: NCT05843253

Description:

The goal of this study is to determine the efficacy of the study drugs ribociclib and everolimus to treat pediatric and young adult patients newly diagnosed with a high-grade glioma (HGG), including DIPG, that have genetic changes in pathways (cell cycle, PI3K/mTOR) that these drugs target.

Key Eligibility Criteria:

For patients with PI3K/mTOR alterations

Sponsor:

Nationwide Children's Hospital

Investigator:

Eugene Hwang, MD

UPCOMING: LCH24C02: A Pilot Study Evaluating the Toxicity and Clinical Benefit of Mitogen-activated Protein Kinase (MAPK) Pathway Inhibition Combined with Programmed Cell Death-1 Checkpoint Blockade (anti-PD1) for the Treatment of BRAF-altered Pediatric Gliomas

*For patients with BRAF or NF-1 alterations

ClinicalTrials.gov Identifier: NCT06712875

Description:

This is a pilot study evaluating the toxicity and early efficacy of dabrafenib and/or trametinib combined with nivolumab for the treatment of BRAF-altered or NF altered gliomas.

Key Eligibility Criteria:

Cohort A: Patients ≥1 and ≤26 years of age with a recurrent or progressive low grade glioma harboring a KIAA1549-BRAF fusion, or a newly diagnosed high grade glioma harboring a KIAA1549-BRAF fusion

Cohort B: Patients ≥1 and ≤26 years of age with NF-1 associated or altered low-grade glioma that is recurrent or progressive, high-grade glioma that is newly diagnosed or recurrent, or transforming glioma that is newly diagnosed or recurrent

Sponsor:

Ann & Robert H Lurie Children's Hospital of Chicago

Investigator:

Lindsay Kilburn, MD

Disease Status: Recurrent

PBTC-048: Feasibility Trial of Optune for Children with Recurrent or Progressive Supratentorial High-Grade Glioma or Ependymoma, and Feasibility and Efficacy Trial of Optune in Conjunction with Radiation Therapy for children with Newly Diagnosed DIPG

ClinicalTrials.gov Identifier: NCT03033992

Description

The Optune Device produce intermediate frequency electric fields impair the growth of tumor cells through the arrest of cell division and inducing apoptosis.

Key Eligibility Criteria

Stratum 1 for patients with supratentorial high grade glioma or ependymoma
Stratum 2 for patients with newly diagnosed DIPG both for ages 5-21
For stratum 2 patients must not have had any prior antitumor therapy or radiation

Sponsor

Pediatric Brain Tumor Consortium

Investigator

Eugene Hwang, MD

Disease Type

Recurrent or progressive supratentorial high grade glioma or ependymoma, and or patients with newly diagnosed DIPG

PBTC-049: Phase 1 study of Savolitinib in Recurrent, Progressive or Refractory Medulloblastoma, High Grade Glioma, DIPG and CNS Tumors Harboring MET Aberrations

ClinicalTrials.gov Identifier: NCT03598244

Description

This trial studies the side effects and best dose of volitinib (savolitinib) in treating patients with primary central nervous system (CNS) tumors that have come back (recurrent) or does not respond to treatment (refractory).

Key Eligibility Criteria

Confirmed diagnosis of a primary CNS tumor (medulloblastoma, high-grade glioma, or diffuse intrinsic pontine glioma [DIPG]) that is recurrent, refractory, or progressive
Evidence of genetic activation of the MET pathway, regardless of histology
Patients must be > 5 years and ≤ 21 years of age

Sponsor

National Cancer Institute (NCI)

Investigator

Eugene I. Hwang, MD

Disease Type

Primary central nervous system (CNS) tumors (medulloblastoma, high-grade glioma, or diffuse intrinsic pontine glioma [DIPG])

PBTC-060: A 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

ClinicalTrials.gov Identifier: NCT04978727

Description

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)

Key Eligibility Criteria

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
Patients must have received their last dose of known myelosuppressive anticancer therapy at least 21 days prior to enrollment

Sponsor

Pediatric Brain Tumor Consortium

Investigator

Eugene Hwang, MD

Disease Type

Progressive or Relapsed Medulloblastoma, High Grade Glioma, Ependymoma and Newly Diagnosed Diffuse Intrinsic Pontine Glioma

PEPN2111: 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, MD, PhD

Disease Type

Solid tumors, lymphoma

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:

This phase I trial studies the side effects of nivolumab before and after surgery in treating children and young adults with high grade glioma that has come back (recurrent) or is increasing in scope or severity (progressive). Immunotherapy with monoclonal antibodies, such as nivolumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread.

Key Eligibility Criteria:

Patients > 6 months and < 22 years with recurrent or progressive HGG (WHO grade III or grade IV) who are candidates for surgical tumor debulking

Sponsor:

Sabine Mueller, MD, PhD

Investigator:

Lindsay Kilburn, MD

PNOC-021: A Phase I Trial Evaluating the Combination of Trametinib and Everolimus in Pediatric and Young Adult Patients with Recurrent Low Grade Gliomas and High 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 Neuro-Oncology Consortium

Investigator

Lindsay Kilburn, MD

Disease Type

Low grade glioma
High grade glioma

UPCOMING: PBTC-061: Phase II Clinical Trial of HSV G207 with a Single 5 Gy Radiation Dose in Children with Recurrent High-Grade Glioma

ClinicalTrials.gov Identifier: NCT04482933

Description:

This study is a clinical trial to assess the efficacy and confirm the safety of intratumoral inoculation of G207 (an experimental virus therapy) combined with a single 5 Gy dose of radiation in recurrent/progressive pediatric high-grade gliomas.

Key Eligibility Criteria:

Patients ≥ 3 years at initial diagnosis but < 22 years with high grade glioma after their first progression following prior surgery and radiotherapy

Sponsor:

Pediatric Brain Tumor Consortium

Investigator:

Eugene Hwang, MD

UPCOMING: PNOC-023: Open label Phase 1 and Target Validation study of ONC206 in Children and Young Adults with Newly Diagnosed or Recurrent Diffuse Midline Glioma (DMG), and Other Recurrent Primary Malignant Central Nervous System (CNS) Tumors o Description: This phase I trial studies the effects and best dose of ONC206 alone or in combination with radiation therapy in treating patients with diffuse midline gliomas that is newly diagnosed or has come back (recurrent) or other recurrent primary malignant CNS tumors

*Biopsy required

ClinicalTrials.gov Identifier: NCT04732065

Description:

This phase I trial studies the effects and best dose of ONC206 alone or in combination with radiation therapy in treating patients with diffuse midline gliomas that is newly diagnosed or has come back (recurrent) or other recurrent primary malignant CNS tumors.

Key Eligibility Criteria:

Arm A: Children and young adults with DMG, H3K27 altered (Dose escalation: 2-21 years of age; Dose expansion: 2 years of age and above) who completed at least one line of prior therapy
Arm B: Newly diagnosed children and young adults (Dose escalation: 2-21 years of age; Dose expansion: 2 years of age and above) with a diagnosis of DMG, H3K27 altered are eligible, including spinal cord DMGs
Arm C: Children and young adults with DMGs (Dose escalation: 2-21 years of age; Dose expansion: 2 years of age and above) who have evidence of progression but have not been treated for this progression and are recommended to get re-irradiation
Arm D: Children and young adults with recurrent primary malignant CNS tumors (Dose escalation: 2-21 years of age; Dose expansion: 2 years of age and above) who have evidence of progression but have not been treated for this progression

Sponsor:

Sabine Mueller, MD, PhD

Investigator:

Lindsay Kilburn, MD

UPCOMING: LCH24C02: A Pilot Study Evaluating the Toxicity and Clinical Benefit of Mitogen-activated Protein Kinase (MAPK) Pathway Inhibition Combined with Programmed Cell Death-1 Checkpoint Blockade (anti-PD1) for the Treatment of BRAF-altered Pediatric Gliomas

*For patients with BRAF or NF-1 alterations

ClinicalTrials.gov Identifier: NCT06712875

Description:

This is a pilot study evaluating the toxicity and early efficacy of dabrafenib and/or trametinib combined with nivolumab for the treatment of BRAF-altered or NF altered gliomas.

Key Eligibility Criteria:

Cohort A: Patients ≥1 and ≤26 years of age with a recurrent or progressive low grade glioma harboring a KIAA1549-BRAF fusion, or a newly diagnosed high grade glioma harboring a KIAA1549-BRAF fusion

Cohort B: Patients ≥1 and ≤26 years of age with NF-1 associated or altered low-grade glioma that is recurrent or progressive, high-grade glioma that is newly diagnosed or recurrent, or transforming glioma that is newly diagnosed or recurrent

Sponsor:

Ann & Robert H Lurie Children's Hospital of Chicago

Investigator:

Lindsay Kilburn, MD