Clinical Trials for Brain Tumor

A Phase I Study of Intra-Tumoral Injections of Ex Vivo Expanded Natural Killer Cells in Children and Young Adults With Recurrent or Progressive Malignant Brain Tumors

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: Children and young adults (1–39 years) with recurrent or progressive malignant supratentorial brain tumors undergoing resection and Ommaya placement receive weekly intratumoral infusions of universal donor, ex vivo–expanded, TGFβ-imprinted natural killer cells for up to three 28‑day cycles. The NK cells are engineered ex vivo to resist TGFβ-mediated immunosuppression and enhance antitumor cytotoxicity; key exclusions include need for ventricular/brainstem access, uncontrolled infection, significant comorbidities, and immunosuppression.

ClinicalTrials.gov ID: NCT05887882

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

Investigational drug late phase

The trial is testing an investigational drug and is at least phase 2, suggesting that the drug has shown promise in phase 1 studies and might have a high chance of working well compared to other investigational drugs.

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.

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: Children, adolescents, and young adults (2–39 years) with diffuse midline glioma/DIPG at diagnosis, post-radiation, or first progression receive ONC201 (dordaviprone; DRD2/3 antagonist and ClpP activator activating the integrated stress response) as backbone therapy, with cohorts optimizing ONC201 dosing (including with radiation/ re-irradiation) or combining ONC201 with targeted agents (e.g., paxalisib [PI3K/mTOR inhibitor] or alteration-guided therapies for BRAFV600E, PDGFRA, FGFR1, NF1). Allocation is nonrandomized and molecularly guided in active cohorts.

ClinicalTrials.gov ID: NCT05009992

Phase I Study of the Safety of Intracavitary Photodynamic Therapy (PDT) of the Brain Bordering Resected Recurrent Glioblastoma or Gliosarcoma Using Intravenous Photobac® and a Balloon Light Applicator

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, surgically resectable glioblastoma or gliosarcoma after standard chemoradiation receive intravenous Photobac, a bacteriopurpurin-based photosensitizer designed to cross the BBB and preferentially accumulate in tumor, followed by fixed-dose 787 nm intracavitary light via a balloon applicator after resection. Dose-escalation evaluates safety/DLTs and MTD, with exploratory PK, tissue drug levels, and preliminary efficacy.

ClinicalTrials.gov ID: NCT05363826

Phase 1 Study of Locoregional Injections of Ex Vivo Expanded Natural Killer Cells in Children and Young Adults With Recurrent, Progressive, or Refractory Brain Tumors

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: Children and young adults with recurrent, progressive, or refractory primary CNS tumors (excluding DMG/DIPG) receive repeated locoregional infusions of universal-donor, TGFβ-imprinted natural killer cells via Ommaya reservoir or programmable VP shunt. The NK product is engineered ex vivo to resist TGFβ-mediated suppression and maintain cytotoxicity in TGFβ-rich brain tumor microenvironments; measurable disease is not required but patients must be eligible for intratumoral or intraventricular access.

ClinicalTrials.gov ID: NCT04254419

ViCToRy: Vorasidenib in Combination With Tumor Specific Peptide Vaccine for Recurrent IDH1 Mutant Lower Grade Gliomas

Moderate burden on patient

The trial has a moderate burden on patients. An example would be a trial testing an IV drug, with scans every 8 weeks which would be more frequent than standard of care.

No known activity

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

HealthScout AI summary: Adults with first recurrence of WHO grade 2–3 IDH1 R132H–mutant, predominantly nonenhancing glioma (KPS ≥70) receive oral vorasidenib (brain‑penetrant mutant IDH1/2 inhibitor) plus PEPIDH1M, an intradermal peptide vaccine targeting the IDH1 R132H neoepitope with GM‑CSF/Montanide and Td priming. Excludes prior mutant IDH therapy and measurable enhancing disease >1×1 cm; primary endpoints focus on safety and PFS.

ClinicalTrials.gov ID: NCT05609994

Pilot Study of Laser Interstitial Thermal Therapy Followed By Hypofractionated Radiation Therapy for Treatment of Recurrent Gliomas.

Moderate burden on patient

The trial has a moderate burden on patients. An example would be a trial testing an IV drug, with scans every 8 weeks which would be more frequent than standard of care.

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 ≥22 with previously irradiated WHO grade II–IV gliomas with radiographic recurrence/progression (measurable enhancing lesion ≤6 cm, KPS ≥60) undergo MRI-guided laser interstitial thermal therapy (LITT) for cytoreduction/biopsy confirmation followed by hypofractionated re-irradiation (35 Gy in 10 fractions). Excludes infratentorial or leptomeningeal disease and prior re-irradiation; aims to assess feasibility, safety, and early efficacy.

ClinicalTrials.gov ID: NCT04181684

A Randomized Phase III Trial of Pre-Operative Compared to Post-Operative Stereotactic Radiosurgery in Patients With Resectable Brain Metastases

Moderate burden on patient

The trial has a moderate burden on patients. An example would be a trial testing an IV drug, with scans every 8 weeks which would be more frequent than standard of care.

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 1–4 resectable brain metastases from non-CNS primaries (one 2–5 cm lesion requiring resection; KPS ≥60) are randomized to standard single-fraction SRS either preoperatively (within 7 days before surgery) or postoperatively (to the cavity within 10–30 days). Systemic therapy is generally allowed except cytotoxic/TKI within ±3 days of SRS; key exclusions include prior RT to the index site, leptomeningeal disease, and small cell/germ cell/lymphoma histologies.

ClinicalTrials.gov ID: NCT05438212

Intracavitary Carrier-embedded Cs131 Brachytherapy for Recurrent Brain Metastases: a Randomized Phase II Study

Moderate burden on patient

The trial has a moderate burden on patients. An example would be a trial testing an IV drug, with scans every 8 weeks which would be more frequent than standard of care.

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 previously irradiated recurrent brain metastases undergoing planned gross/near-total resection are randomized to surgery alone versus surgery plus intracavitary Cs-131 seed brachytherapy. The investigational approach uses low-energy, short–half-life cesium-131 to deliver high local radiation to the resection cavity to sterilize residual disease while sparing normal tissue.

ClinicalTrials.gov ID: NCT04690348

Arginine With Whole Brain Radiation Therapy for the Treatment of Brain Metastases

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 brain metastases from any solid tumor scheduled for standard-fractionation WBRT are randomized to receive L-arginine either intravenously or orally prior to each fraction, without concurrent systemic therapy. L-arginine, a nitric oxide synthase substrate that may enhance radiosensitivity via improved tumor oxygenation and immune modulation, is being evaluated for pharmacokinetics, safety, and physiologic effects to inform dosing and route.

ClinicalTrials.gov ID: NCT06328686

Window of Opportunity Assessment of [Fam-]Trastuzumab DERuxtecan-nxki (T-DXd) Brain Tumor Penetration and Efficacy (WOnDER-BT)

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 resectable brain tumors, including HER2-expressing or ERBB2-mutant brain metastases (HER2-high, HER2-low, or HER2-mutant) and HER2-expressing recurrent glioblastoma; includes patients with or without prior T-DXd. Patients receive perioperative trastuzumab deruxtecan (HER2-directed antibody-drug conjugate delivering a topoisomerase I inhibitor) to assess intratumoral penetration and preliminary efficacy.

ClinicalTrials.gov ID: NCT06058988