Clinical Trials for Brain Tumor

UAB 2507 - Randomized Phase II Study of Differential Margins in Single Isocenter Radiosurgery of 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.

HealthScout AI summary: Adults with up to ~20 brain metastases (each ≤4 cm), including intact lesions or postoperative cavities and even after prior WBRT, are randomized to linac-based single-isocenter SRS using either 0 mm or 2 mm PTV margins. The trial compares local control and grade ≥3 CNS toxicity at 12 months with standard SRS dosing (16–20 Gy ×1 for <2 cm; 27 Gy in 3 fractions for 2–4 cm); no investigational drugs are used.

ClinicalTrials.gov ID: NCT06857006

Targeting Pediatric Brain Tumors and Relapsed/Refractory Solid Tumors With Sodium Glucose Cotransporter 2 Inhibitors (SGLT2i)

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.

HealthScout AI summary: Children and young adults (6–21) with recurrent primary brain tumors lacking curative options or relapsed/refractory solid tumors after ≥2 prior lines receive standard chemotherapy plus dapagliflozin, an SGLT2 inhibitor (metabolic modulation via reduced glucose/insulin and ketone shift) to assess safety/feasibility and preliminary activity. Regimens include carmustine+dapagliflozin for brain tumors and topotecan/cyclophosphamide+dapagliflozin for solid tumors, with age-based dapagliflozin dosing and close metabolic/renal monitoring due to risks such as euglycemic ketoacidosis and infections.

ClinicalTrials.gov ID: NCT05521984

A Phase II Study of Tucatinib and Ado-trastuzumab Emtansine (T-DM1) in Patients With HER2-positive Metastatic Solid Tumors and Metastases to Brain (TUCATEMEB)

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.

HealthScout AI summary: Adults with HER2-positive metastatic solid tumors (including ERBB2-mutant) and active/progressing brain metastases not needing immediate local CNS therapy receive tucatinib (selective HER2 TKI with CNS penetration) plus ado-trastuzumab emtansine (HER2-directed antibody–drug conjugate) in 21-day cycles after prior HER2 therapy as applicable. Aims to assess intracranial response and safety; excludes patients with large/unstable brain lesions or needing urgent local therapy.

ClinicalTrials.gov ID: NCT05673928

EXCLAIM: Exploring Combined Local and Systemic Approaches In Brain Metastasis: a Multi-cohort Randomized Phase II Study Evaluating Initial Response to Systemic Therapy and Subsequent Integration of Stereotactic Radiosurgery in Patients With Low-risk Brain Metastases and Central Nervous System-active Systemic Therapy Options

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.

HealthScout AI summary: Adults with metastatic solid tumors and low-risk, untreated brain metastases (ECOG 0–1) start CNS-active standard systemic therapy; responders with non-complete response are randomized to consolidative stereotactic radiosurgery (SRS) to all residual lesions versus observation, while those with CNS progression are randomized to SRS to progressing lesions alone versus SRS to progressing lesions plus consolidative SRS to all residual lesions. Excludes leptomeningeal disease, SCLC, hematologic malignancies, large (>3 cm) or hemorrhagic lesions, prior WBRT, and certain oncogene-driven NSCLC starting highly CNS-active targeted therapy.

ClinicalTrials.gov ID: NCT06649058

Phase I Study of Cellular Immunotherapy Using Memory Enriched T Cells Lentivirally Transduced to Express an IL13Rα2-Targeting, Hinge-Optimized, 41BB-Costimulatory Chimeric Receptor and a Truncated CD19 for Children With Recurrent/Refractory Malignant Brain Tumors

Active drug

Investigational drug(s) in this trial have shown anticancer activity in clinical trials

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.

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 patients with recurrent or refractory IL13Rα2-positive malignant brain tumors receive lymphodepleting cyclophosphamide/fludarabine followed by repeated intraventricular infusions of autologous IL13Rα2-targeted CAR T cells (IL-13 mutein binder, 4-1BB/CD3ζ; includes truncated CD19). Locoregional delivery aims to enhance CNS exposure while monitoring safety, CAR T persistence, and preliminary antitumor activity.

ClinicalTrials.gov ID: NCT04510051

Phase II Study of Nivolumab in Combination With Relatlimab in Patients With Active Melanoma 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.

HealthScout AI summary: Adults with melanoma and at least one measurable, untreated brain metastasis (0.5–3 cm), ECOG 0–1, and no steroid-requiring neurologic symptoms receive fixed-dose nivolumab (PD-1 inhibitor) plus relatlimab (LAG-3 inhibitor) IV every 28 days. Prior limited SRT/excision allowed if non-irradiated measurable disease remains; excludes leptomeningeal disease, lesions >3 cm, prior metastatic PD-1 therapy, and significant autoimmune/infectious comorbidities.

ClinicalTrials.gov ID: NCT05704647

Phase 1 Study of B7-H3-Specific CAR T Cell Locoregional Immunotherapy for Diffuse Intrinsic Pontine Glioma/Diffuse Midline Glioma and Recurrent or Refractory Pediatric Central Nervous System Tumors

Active drug

Investigational drug(s) in this trial have shown anticancer activity in clinical trials

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.

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: Enrolls children and young adults (1–26 years) with DIPG/DMG after standard therapy or with recurrent/refractory primary CNS tumors lacking options, requiring an indwelling CNS catheter. Investigational treatment is autologous B7-H3 (CD276)–targeted CAR T cells (SCRI-CARB7H3/BCB-276) delivered locoregionally via tumor cavity or intraventricular catheter without lymphodepletion, with dosing over two courses and safety/feasibility as primary endpoints.

ClinicalTrials.gov ID: NCT04185038

A Phase 0, Single-center, Open-label, Dose-escalating Trial Using Super-selective Intra-arterial Infusion of a Single Dose of Temsirolimus for the Treatment of Recurrent High-grade Glioma

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.

HealthScout AI summary: Adults with recurrent, resectable WHO grade 3–4 frontal lobe glioma that is mTOR‑pathway positive (e.g., PTEN loss or PI3K/AKT/mTOR alterations) after prior Stupp regimen receive a single pre-resection dose of temsirolimus given via super-selective intra-arterial infusion or standard IV. Temsirolimus is an mTORC1 inhibitor; the study compares intratumoral drug exposure and target inhibition (pS6) between delivery routes.

ClinicalTrials.gov ID: NCT05773326

Phase II Trial of the PARP Inhibitor Niraparib and PD-1 Inhibitor Dostarlimab in Patients With Advanced Cancers With Active Progressing Brain Metastases (STARLET)

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.

HealthScout AI summary: Adults with advanced cancers and actively progressing, measurable, untreated asymptomatic brain metastases (including BRCA1/2-mutated, other HRR-aberrant tumors, SCLC, NSCLC, and TNBC) receive niraparib (PARP inhibitor exploiting HRD/synthetic lethality) plus dostarlimab (PD‑1 inhibitor) to assess intracranial response and durability; prior PD‑1/L1 allowed, prior full‑dose PARP monotherapy excluded. Requires ECOG 0–2, adequate organ function, no leptomeningeal disease, and no need for immediate local therapy or steroids.

ClinicalTrials.gov ID: NCT05700721

Phase 1 Trial of Engineered HSV G207 in Children With Recurrent or Refractory Cerebellar Brain Tumors

Active drug

Investigational drug(s) in this trial have shown anticancer activity in clinical trials

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.

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 (3 to <22 years) with recurrent or progressive malignant cerebellar brain tumors (e.g., medulloblastoma, high‑grade glioma/GBM, ependymoma, ATRT, PNET, germ cell tumor) with surgically accessible lesions ≤3 cm receive intratumoral G207, an engineered, tumor‑selective oncolytic HSV‑1 (γ34.5 deletions, UL39 inactivation; TK‑retained), with later cohorts also receiving a single 5‑Gy focal radiation dose within 24 hours. Key exclusions include diffuse multifocal disease (≥3 lobes), need for ventricular/brainstem inoculation or access, active infection/immunosuppression, and concurrent anticancer therapy.

ClinicalTrials.gov ID: NCT03911388