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 Open Clinical Trials by Disease Type (A-G)

Disease Status: Newly Diagnosed

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)

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 Open 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

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