Brain Tumor Clinical Trials

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

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

ClinicalTrials.gov Identifier: NCT04775485

Description

• This is a Phase 2, multi-center, open label study of patient aged 6 months to 25 years with recurrent or progressive LGG with a known activating BRAF mutation. Patients will be treated with once weekly Day101 (an oral tablet or liquid suspension) for approximately 2 years, while undergoing routine clinic visits, labs, imaging, and other assessments.

Key Eligibility Criteria

• Patients must be > 6 months but < 25 years with a relapsed or progressive LGG with known activating BRAF alteration.

Sponsor

• Pediatric Neuro-Oncology Consortium

Investigator

• Lindsay Kilburn, M.D.

Disease Type

• Recurrent or progressive LGG with a known activating BRAF mutation

ClinicalTrials.gov Identifier: NCT05566795

Description

• 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

Key Eligibility Criteria

• Low grade gliomas who has not been treated prior
• Tumor must harbor a RAF alteration

Sponsor

• Day101 Pharmaceuticals

Investigator

• Lindsay Kilburn, M.D.

Disease Type

• Low grade glioma

ClinicalTrials.gov Identifier: NCT04617002

Description

• This is an intermediate-size expanded access protocol to provide ONC201 (dordaviprone) to patients with H3 K27M-mutant and/or midline gliomas who cannot access ONC201 (dordaviprone) through clinical trials. This study has multiple arms where patients can be enrolled depending on disease status and prior therapies received.

Key Eligibility Criteria

• Meetings one of the following criteria:
  
  • Diffuse intrinsic pontine glioma (DIPG)
  • A primary spinal glioma that is positive for the H3 K27M mutation
  • A diffuse glioma that is positive for the H3 K27M mutation
  • A H3 K27M-mutant glioma or a midline glioma of unknown H3 K27M mutational status who received ONC201 and/or ONC206 from an alternative (non-Chimerix) source prior to 30 November 2023
  • A H3 K27M-mutant diffuse glioma or a midline glioma with unknown H3 K27M mutational status who have completed frontline radiotherapy at least 6 weeks prior to 30 November 2023.
• Patients who qualify for ongoing ONC201 or ONC206 clinical trials are ineligible for this trial. 

Sponsor

• Chimerix

Investigator

• Lindsay Kilburn, M.D.

Disease Type

• H3 K27M-mutant and/or midline 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

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 glioma (DMG)

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

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

ClinicalTrials.gov Identifier: NCT03598244

Description

• Savolitinib is a small molecule inhibitor of c-MET kinase and has been found to decrease tumor cell growth

Key Eligibility Criteria

• Patients must have evaluable disease
• Ages 5-21
• Patients must harbor a MET pathway mutation, fusion or amplification to be eligible 

Sponsor

• Pediatric Brain Tumor Consortium

Investigator

• Eugene Hwang, M.D.

Disease Type

• CNS tumors with MET aberrations

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, M.D.

Disease Type

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

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, M.D.

Disease Type

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

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

ClinicalTrials.gov Identifier: NCT03904862

Description

• 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).

Key Eligibility Criteria

• 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

Sponsor

• Pediatric Brain Tumor Consortium

Investigator

• Eugene Hwang, M.D.

Disease Type

• Sonic HedgeHog Medulloblastoma

ClinicalTrials.gov Identifier: NCT04903080

Description

Ependymoma is the third most common central nervous system (CNS) tumor in children and is associated with poor long-term survival. This study evaluates the safety profile of a type of immune therapy called HER2 CAR T cells (short for HER2 chimeric antigen receptor T cells). In addition to looking for side effects, we will study how well this treatment works against ependymoma that has come back after treatment (recurrent) or has not responded well to treatment (progressive) in children.

Key Eligibility Criteria

• Diagnosis of ependymoma that is recurrent or progressive
• Patient must have received standard of care therapy including maximal safe surgical resection followed by local adjuvant radiation therapy prior to enrollment
• Patient must be ≥ 1 but ≤ 21 years of age at the time of screening consent.

Sponsor

Pediatric Brain Tumor Consortium (PBTC)

Investigator

Eugene I. Hwang, M.D.

Disease Type

Refractory or Recurrent Ependymoma

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, M.D.

Disease Type

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

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

ClinicalTrials.gov Identifier: NCT05286801

Description

• 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.

Key Eligibility Criteria

• Patients must have SMARCB1 (INI1) or SMARCA4 deficient tumors
• Part A age: ≥ 12 years  and < 18 years
• Part B age: ≥ 18 years

Sponsor

• Children's Oncology Group 

Investigator

• AeRang Kim, M.D.

Disease Type

• Renal medullary carcinoma, malignant rhabdoid tumor (extra-CNS), atypical teratoid rhabdoid tumor (CNS), poorly differentiated chordoma, epithelioid sarcoma, other SMARCB1 or SMARCA4 deficient tumors

ClinicalTrials.gov Identifier: NCT05359237

Description

• 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.

Key Eligibility Criteria

• Age: ≤ 12 years  
• Newly diagnosed and relapsed cancer diagnosis that is being treated with vincristine at the 1.5 mg/m2 dose level

Sponsor

• Children's Oncology Group

Investigator

• AeRang Kim, M.D.

Disease Type

• Any

ClinicalTrials.gov Identifier: NCT05057702

Description

• The current study will use a new treatment approach based on the molecular characteristics of each participant’s tumor.

Key Eligibility Criteria

• Participants must undergo surgery and have surgically accessible disease
• Participants must have recurrent medulloblastoma
• Participants must have had at leapt one prior therapy

Sponsor

• Pacific NeuroOncology Consortium

Investigator

• Lindsay Kilburn, M.D.

Disease Type

• Recurrent medulloblastoma

ClinicalTrials.gov Identifier: NCT04337177

Description

• 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

Key Eligibility Criteria

• Recurrent Pediatric Solid Tumors
•  ≥ 1 year of age or ≤ 30 years of age

Sponsor

• Valent Technologies LLC

Investigator

• AeRang Kim, M.D.

Disease Type

• Solid tumors, including CNS tumors