Clinical Trials for Brain Tumor

Adopting a Functional Precision Medicine Approach For Individualized Pediatric Cancer Treatments

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, adolescents, and young adults (≤21 years) with relapsed or refractory solid or hematologic malignancies and accessible tumor tissue undergo ex vivo drug sensitivity testing integrated with genomic profiling to guide individualized therapy selection. Treating physicians may use matched, typically approved monotherapies or combinations based on predicted sensitivity; outcomes are compared with contemporaneous patients receiving non–FPM standard-of-care therapy.

ClinicalTrials.gov ID: NCT05857969

Phase 1b Study of the Safety of Concurrent Cabozantinib and Nivolumab With Radiation Therapy for Brain Metastases in Patients With Metastatic Renal Cell Carcinoma

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/unresectable RCC (clear cell or most non–clear cell; measurable brain metastasis requiring SRS) receive concurrent cabozantinib (oral multikinase inhibitor of MET/VEGFR2/AXL) plus nivolumab (PD‑1 inhibitor) with stereotactic radiosurgery. Key exclusions include prior cabozantinib for RCC, recent VEGF TKI or brain RT, need for whole-brain RT, significant autoimmune/cardiovascular disease, uncontrolled hypertension, high bleeding risk, and active infections.

ClinicalTrials.gov ID: NCT06132945

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

Pilot Trial of Spectroscopic MRI-guided, Dose-Escalated Proton Radiation Therapy and Bevacizumab for Recurrent Glioblastoma

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 glioblastoma or gliosarcoma after prior standard brain RT (≤63 Gy), ECOG 0–2, single measurable lesion ≤6 cm, receive spectroscopic MRI-guided focal proton re-irradiation (IMPT, 35 Gy/10 fx or 40 Gy/10 fx with SIB) combined with bevacizumab (anti–VEGF-A monoclonal antibody) started before RT. Excludes multifocal disease, prior re-irradiation or bevacizumab, and lesions in regions prone to sMRI artifact.

ClinicalTrials.gov ID: NCT05284643

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

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 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

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: 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