NCT07039760

Asciminib With or Without Sildenafil for Brain Tumors

An Early Phase 1 Study of Asciminib With or Without Sildenafil for Brain Tumors

• Status: Not Yet Recruiting
• Phase: EARLY_Phase 1
• Study type: Interventional
• Enrollment: 12 (estimated)
• Sponsor: Washington University School of Medicine · Academic / Other
• Sex: All
• Age: 6 Years – 25 Years
• Healthy volunteers: Not accepted

Summary

Dissemination of medulloblastoma is an independent risk factor of poor prognosis. Dissemination of medulloblastoma at recurrence is nearly universally fatal. ABL1 and 2 have been recently found to mediate the dissemination of medulloblastoma. Genetically inactivating ABL1 and 2 resulted in decreased leptomeningeal medulloblastoma and improved overall survival (OS) in rodent models. Asciminib is an FDA approved for the treatment of chronic myeloid leukemia and is well tolerated, likely due to its specificity for ABL1 and ABL2. Asciminib is a P-glycoprotein (P-gp) substrate and thus may be susceptible to being pumped out of tumor cells and brain endothelial cells. It is unclear if asciminib can enter the central nervous system (CNS) and brain tumors in adequate concentration to have anti-tumor effects.

Conditions

• Brain Tumor

Interventions

Type	Name	Description
DRUG	Asciminib	Commercially available stock
DRUG	Sildenafil	Commercially available stock
PROCEDURE	Surgical resection or biopsy	Standard of care

Timeline

• Start date: 2026-04-30
• Primary completion: 2027-10-31
• Completion: 2027-11-30
• First posted: 2025-06-26
• Last updated: 2026-03-11

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study