Clinical Trials for Brain Tumor

Phase I Study of Autologous T Lymphocytes Expressing GD2-specific Chimeric Antigen and Constitutively Active IL-7 Receptors for the Treatment of Patients With GD2-expressing Brain Tumors (GAIL-B)

High burden on patient

The trial has a high burden on patients. An example would be a phase 1 trial with extra tumor biopsies and frequent pharmacokinetic blood draws.

Started >3 years ago

The trial was activated more than 3 years ago. This is not always bad but can indicate that the trial has less current treatments, or has had trouble getting enough patients to enroll due to other issues.

HealthScout AI summary: Pediatric and young adult patients (12 months–22 years) with GD2-expressing CNS tumors—including diffuse midline glioma/high-grade glioma and select other high-grade brain tumors—receive lymphodepletion (cyclophosphamide/fludarabine) followed by autologous GD2-directed CAR T cells engineered with a constitutively active IL‑7 receptor (C7R) to enhance persistence. Cohort 1 gets initial IV CAR T then intracerebroventricular dosing via Ommaya/VP shunt; Cohort 2 (recurrent/progressive pontine HGG or H3K27-altered DMG) receives IV only.

ClinicalTrials.gov ID: NCT04099797

SJ901: Phase 1/2 Evaluation of Single Agent Mirdametinib (PD-0325901), a Brain-Penetrant MEK1/2 Inhibitor, for the Treatment of Children, Adolescents, and Young Adults With Low-Grade Glioma

High burden on patient

The trial has a high burden on patients. An example would be a phase 1 trial with extra tumor biopsies and frequent pharmacokinetic blood draws.

Started >3 years ago

The trial was activated more than 3 years ago. This is not always bad but can indicate that the trial has less current treatments, or has had trouble getting enough patients to enroll due to other issues.

HealthScout AI summary: Enrolling patients aged 2 to <25 years with centrally confirmed MAPK-activated WHO grade I–II pediatric low-grade glioma (including eligible glioneuronal/neuroepithelial tumors), across newly diagnosed and recurrent/progressive settings, with measurable/evaluable disease; excludes BRAF V600, NTRK/ALK/ROS1 fusions, IDH1/2, and significant ocular/cardiac/hepatic/pulmonary comorbidity. Investigational therapy is oral single-agent mirdametinib, a selective brain-penetrant MEK1/2 inhibitor targeting MAPK/ERK signaling, given BID in 28-day cycles; cohorts include MEK inhibitor–naïve and previously MEK-exposed patients.

ClinicalTrials.gov ID: NCT04923126

A Phase I Study of RNA-Lipid Particle (RNA-LP) Vaccines for Recurrent Adult Glioblastoma (GBM)

No known activity

No investigational drug(s) in this trial have shown anticancer activity yet in clinical trials

High burden on patient

The trial has a high burden on patients. An example would be a phase 1 trial with extra tumor biopsies and frequent pharmacokinetic blood draws.

HealthScout AI summary: Adults with recurrent supratentorial GBM post–surgery and temozolomide (KPS ≥60) receive intravenous RNA–lipid particle vaccines: three priming doses of pp65 mRNA-LP (targets CMV pp65, LAMP-enhanced MHC II presentation) followed by monthly doses combining pp65 mRNA with autologous tumor RNA; randomized to start priming before vs after biopsy/resection. Excludes prior bevacizumab and significant immunosuppression/autoimmunity; aims to assess feasibility, safety, and MTD.

ClinicalTrials.gov ID: NCT06389591

A Dual Phase 1/2, Investigator Initiated Study to Determine the Maximum Tolerated Dose, Safety, and Efficacy of 186Rhenium Nanoliposomes (186RNL) in Recurrent Glioma (CTRC# 12-02)

High burden on patient

The trial has a high burden on patients. An example would be a phase 1 trial with extra tumor biopsies and frequent pharmacokinetic blood draws.

Started >3 years ago

The trial was activated more than 3 years ago. This is not always bad but can indicate that the trial has less current treatments, or has had trouble getting enough patients to enroll due to other issues.

HealthScout AI summary: Adults with recurrent high-grade glioma (IDH-mutant grade 3/4 astrocytoma or IDH-wildtype glioblastoma) after standard therapy, with tumors suitable for convection-enhanced delivery and meeting RANO progression and performance criteria. Single intratumoral infusion of 186Rhenium nanoliposomes via CED, a beta-emitting radiotherapeutic encapsulated in nanoliposomes to deliver localized high-dose radiation with imageable distribution (SPECT); excludes multifocal/leptomeningeal disease, infratentorial tumors, recent bevacizumab or radiation, and lesions risking ventricular/subarachnoid leak.

ClinicalTrials.gov ID: NCT01906385

Phase 1 Study of B7-H3, EGFR806, HER2, And IL13-Zetakine (Quad) CAR T Cell Locoregional Immunotherapy For Pediatric Diffuse Intrinsic Pontine Glioma, Diffuse Midline Glioma, And Recurrent Or Refractory Central Nervous System Tumors

No known activity

No investigational drug(s) in this trial have shown anticancer activity yet in clinical trials

High burden on patient

The trial has a high burden on patients. An example would be a phase 1 trial with extra tumor biopsies and frequent pharmacokinetic blood draws.

HealthScout AI summary: Pediatric and young adult patients (1–26 years) with DIPG after radiotherapy, non-pontine DMG, or other recurrent/refractory CNS tumors receive intraventricular, fractionated infusions of an autologous multi-antigen CAR T-cell product (SC-CAR4BRAIN) targeting B7-H3, EGFR806 (conformation-specific EGFR), HER2, and IL13Rα2 (IL13-zetakine). Locoregional delivery via ventricular reservoir aims to enhance CNS tumor targeting while limiting systemic exposure.

ClinicalTrials.gov ID: NCT05768880

A Phase 0/1 Pharmacokinetic and Pharmacodynamics and Safety and Tolerability Study of Letrozole in Combination With Standard Therapy in Recurrent High Grade Gliomas

High burden on patient

The trial has a high burden on patients. An example would be a phase 1 trial with extra tumor biopsies and frequent pharmacokinetic blood draws.

Started >3 years ago

The trial was activated more than 3 years ago. This is not always bad but can indicate that the trial has less current treatments, or has had trouble getting enough patients to enroll due to other issues.

HealthScout AI summary: Adults with recurrent high-grade glioma (ECOG 0–2) receive oral letrozole, an aromatase inhibitor aimed at achieving brain/tumor penetration, with the expansion cohort treating with letrozole 15 mg daily plus metronomic temozolomide 50 mg/m2 daily. Initial cohorts required planned resection/biopsy for PK assessment; the expansion enrolls patients with measurable recurrence without a surgery requirement.

ClinicalTrials.gov ID: NCT03122197

Phase I/II Study of Laser Interstitial Thermotherapy (LITT) Combined With Checkpoint Inhibitor for Recurrent GBM (RGBM)

High burden on patient

The trial has a high burden on patients. An example would be a phase 1 trial with extra tumor biopsies and frequent pharmacokinetic blood draws.

Started >3 years ago

The trial was activated more than 3 years ago. This is not always bad but can indicate that the trial has less current treatments, or has had trouble getting enough patients to enroll due to other issues.

HealthScout AI summary: Adults with recurrent supratentorial glioblastoma or gliosarcoma after standard chemoradiation, with unifocal/unilateral disease suitable for LITT (≤6 cm), receive stereotactic biopsy and LITT plus pembrolizumab (anti–PD‑1) given either 7 days before or 14/35 days after LITT, then every 3 weeks. Excludes prior PD‑1/PD‑L1 therapy and active autoimmune disease; aims to define optimal sequencing and assess antitumor activity versus historical controls.

ClinicalTrials.gov ID: NCT03277638

Phase I/II Trial of Super-selective Intra-arterial Repeated Infusion of Cetuximab for the Treatment of Newly Diagnosed Glioblastoma

High burden on patient

The trial has a high burden on patients. An example would be a phase 1 trial with extra tumor biopsies and frequent pharmacokinetic blood draws.

Started >3 years ago

The trial was activated more than 3 years ago. This is not always bad but can indicate that the trial has less current treatments, or has had trouble getting enough patients to enroll due to other issues.

HealthScout AI summary: Adults with newly diagnosed, EGFR-overexpressing glioblastoma receive super-selective intra-arterial cetuximab after mannitol blood–brain barrier disruption at set postoperative intervals, in addition to standard Stupp chemoradiation. Cetuximab is an anti-EGFR IgG1 monoclonal antibody that blocks EGFR signaling and may mediate ADCC.

ClinicalTrials.gov ID: NCT02861898

Phase I/II Trial Of Repeated Super-selective Intraarterial Cerebral Infusion Of Bevacizumab (Avastin) for Treatment of Relapsed/Refractory Glioblastoma Multiforme and Anaplastic Astrocytoma.

High burden on patient

The trial has a high burden on patients. An example would be a phase 1 trial with extra tumor biopsies and frequent pharmacokinetic blood draws.

Started >3 years ago

The trial was activated more than 3 years ago. This is not always bad but can indicate that the trial has less current treatments, or has had trouble getting enough patients to enroll due to other issues.

HealthScout AI summary: Adults with relapsed/refractory high-grade glioma (GBM, anaplastic astrocytoma/oligoastrocytoma), KPS ≥70, receive super-selective intraarterial bevacizumab 15 mg/kg after osmotic BBB disruption with IA mannitol, repeated at progression. One arm also adds standard biweekly IV bevacizumab 10 mg/kg between IA treatments; bevacizumab is an anti–VEGF-A monoclonal antibody inhibiting angiogenesis.

ClinicalTrials.gov ID: NCT01269853

A First-in-human Phase I Single-agent Dose-escalation, Food Effect and Dose Expansion Study of Oral ONC206 in Recurrent and Rare Primary Central Nervous System Neoplasms

No known activity

No investigational drug(s) in this trial have shown anticancer activity yet in clinical trials

High burden on patient

The trial has a high burden on patients. An example would be a phase 1 trial with extra tumor biopsies and frequent pharmacokinetic blood draws.

Started >3 years ago

The trial was activated more than 3 years ago. This is not always bad but can indicate that the trial has less current treatments, or has had trouble getting enough patients to enroll due to other issues.

HealthScout AI summary: Adults with recurrent or rare primary CNS tumors (e.g., GBM, diffuse midline glioma, ependymoma, medulloblastoma, atypical/anaplastic meningioma) after standard therapy receive oral ONC206 monotherapy. ONC206 is an imipridone that non-competitively antagonizes DRD2/DRD3 and allosterically hyperactivates mitochondrial ClpP, aiming to trigger integrated stress response and apoptosis; dose-escalation with food-effect/PK assessment and potential expansion at RP2D.

ClinicalTrials.gov ID: NCT04541082