Clinical Trials for Brain Tumor

DATO-BASE: A Phase 2 Trial of DATOpotamab-deruxtecan for Breast Cancer Brain metAstaSEs

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.

Active drug

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

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: This trial enrolls adults with HER2-negative metastatic breast cancer (ER-positive or triple-negative) with brain metastases or any HER2-negative subtype with leptomeningeal disease, who receive datopotamab deruxtecan, an anti-TROP2 antibody-drug conjugate delivering a topoisomerase I inhibitor. All participants must have CNS involvement, good performance status, and no major comorbidities; the drug is given IV every 3 weeks.

ClinicalTrials.gov ID: NCT06176261

Phase 1 / 2 Multicenter Study of the Safety, Pharmacokinetics, and Preliminary Efficacy of APL-101 in Subjects With Non-Small Cell Lung Cancer With c-Met EXON 14 Skip Mutations and c-Met Dysregulation Advanced Solid Tumors

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.

Active drug

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

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: The trial investigates APL-101, a selective c-MET receptor tyrosine kinase inhibitor, in adult patients with NSCLC exhibiting c-Met exon 14 skipping mutations, various solid tumors with MET alterations, and primary CNS tumors. It includes APL-101 monotherapy and combination therapy with EGFR inhibitors in cases of acquired MET amplification resistance.

ClinicalTrials.gov ID: NCT03175224

A Double-blind, Phase II Randomized Study of Brain-directed Stereotactic Radiation With or Without AGuIX Gadolinium-based Nanoparticles in the Management of Brain Metastases at Higher Risk of Local Recurrence With Radiation Alone

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.

Active drug

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

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 solid tumors and high-risk brain metastases (e.g., melanoma refractory to immunotherapy, GI primaries, HER2+ breast cancer, cystic or large lesions, or local recurrence after prior brain radiation) are randomized to stereotactic radiation with or without intravenous AGuIX gadolinium-based nanoparticles, which act as tumor-targeted radiosensitizers and MRI contrast agents.

ClinicalTrials.gov ID: NCT04899908

A Phase 2a Trial of Immune Modulation in Combination With Ultrasound-mediated Blood Brain Barrier Opening in Patients With Newly Diagnosed Glioblastoma

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.

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.

HealthScout AI summary: Adults with newly diagnosed, IDH-wild type, MGMT-unmethylated GBM post-radiotherapy (ECOG 0–2) receive Sonocloud-9–mediated blood–brain barrier opening every 3 weeks synchronized with liposomal doxorubicin plus balstilimab (anti–PD-1) and botensilimab (Fc‑enhanced anti–CTLA‑4). Excludes prior immunotherapy, multifocal/not-coverable or posterior fossa disease, active autoimmune disease, and other major comorbidities.

ClinicalTrials.gov ID: NCT05864534

A Multicenter, Phase II Trial of Relatlimab, Nivolumab, and Ipilimumab in Patients With Asymptomatic and Symptomatic Melanoma Brain Metastases

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.

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.

HealthScout AI summary: Adults with non-uveal melanoma and measurable untreated brain metastases (including asymptomatic ECOG 0–1 and symptomatic ECOG 0–2 with limited steroids) receive triple checkpoint blockade: nivolumab (PD-1 inhibitor) plus relatlimab (LAG-3 inhibitor) combined with ipilimumab (CTLA-4 inhibitor). Prior adjuvant/neoadjuvant PD-1/CTLA-4/LAG-3 allowed if >6 months; excludes leptomeningeal disease, prior whole-brain RT, active autoimmune disease requiring systemic therapy, and significant cardiac comorbidity.

ClinicalTrials.gov ID: NCT06712927

Phase I/II Study of Stereotactic Radiation and Sacituzumab Govitecan With Zimberelimab in the Management of Metastatic Triple Negative Breast Cancer With Brain Metastases (TARGET-TNBC)

Active drug

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

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: Eligible patients are adults with metastatic triple-negative breast cancer and measurable brain metastases who can undergo stereotactic radiosurgery; treatment consists of SRS followed by sacituzumab govitecan (an anti-Trop-2 antibody-drug conjugate) and zimberelimab (a PD-1 inhibitor immunotherapy). Prior taxane/anthracycline or SRS/FSRT therapy (if new lesions) is allowed; key exclusions are leptomeningeal disease, significant comorbidities, active autoimmune disease requiring immunosuppression, and prior topoisomerase I inhibitor use for brain metastases.

ClinicalTrials.gov ID: NCT06238921

A Phase I/II Study of Zotiraciclib for Recurrent Malignant Gliomas With Isocitrate Dehydrogenase 1 or 2 (IDH1 or IDH2) Mutations

Active drug

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

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 and adolescents (≥15) with recurrent IDH1/2-mutant diffuse glioma (WHO grade 2–4) after prior radiation and/or chemotherapy receive intermittent oral zotiraciclib monotherapy. Zotiraciclib is an investigational multi-kinase, CDK9-focused inhibitor that suppresses RNA Pol II–dependent transcription (e.g., MCL-1/MYC pathways); a surgical PD cohort gets a single pre-resection dose with post-op resumption.

ClinicalTrials.gov ID: NCT05588141

A Surgical Window of Opportunity Clinical Trial of Troriluzole in Recurrent IDH Wild-Type Glioblastoma

Active drug

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

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 IDH–wildtype glioblastoma (first/second recurrence) eligible for resection and with cortical involvement receive the glutamatergic modulator troriluzole (riluzole prodrug that reduces synaptic glutamate via presynaptic release inhibition, Na+ channel blockade, and EAAT2 upregulation) either as a short presurgical course with postoperative continuation or starting postoperatively. Designed to assess pharmacodynamic effects on neuronal/tumor glutamate biology and safety, with MRIs q8 weeks on treatment.

ClinicalTrials.gov ID: NCT06552260

Phase 1b/2 Trial of Defactinib and Avutometinib, With or Without Encorafenib, for the Treatment of Patients With Brain Metastases From Cutaneous Melanoma

Active drug

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

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 cutaneous melanoma and measurable brain metastases after immune checkpoint inhibitors, including BRAF V600E/K–mutant and non‑BRAF cohorts; requires at least one untreated 0.5–4 cm parenchymal lesion, ECOG 0–1, and known RAS/BRAF/NF1 status. Treatment is avutometinib (dual RAF/MEK “clamp”) plus defactinib (FAK/Pyk2 inhibitor), with an added encorafenib (BRAF inhibitor) dose‑finding and expansion for BRAF V600E/K disease.

ClinicalTrials.gov ID: NCT06194929

A Dose Escalation Study Using Eflornithine (DFMO) and AMXT 1501 Followed by a Randomized Controlled Trial of DFMO With or Without AMXT 1501 for Neuroblastoma, CNS Tumors, and Sarcomas

Active drug

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

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: Pediatric, adolescent, and young adult patients with relapsed/refractory neuroblastoma (randomized to DFMO with or without AMXT 1501) and single-arm cohorts for relapsed/refractory ETMR/ATRT, newly diagnosed DIPG post-RT, and relapsed/refractory Ewing sarcoma or osteosarcoma receive oral eflornithine (DFMO) with AMXT 1501. AMXT 1501 inhibits polyamine transport and DFMO irreversibly inhibits ornithine decarboxylase, aiming to block both polyamine uptake and synthesis.

ClinicalTrials.gov ID: NCT06465199