Asciminib With or Without Sildenafil for Brain Tumors

Conditions

• Brain Tumor

Eligibility Criteria

Sex

ALL

Age

6 Years - 25 Years

Healthy Volunteers

Not accepted

Interventions

• Asciminib — DRUG
  Commercially available stock
• Sildenafil — DRUG
  Commercially available stock
• Surgical resection or biopsy — PROCEDURE
  Standard of care

Study Details

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.

Key Dates

Start date

Aug 31, 2026

Status verified

Jun 2026

Primary completion

Feb 28, 2028

Completion

Mar 31, 2028

Study Design

Enrollment

12 participants (estimated)

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Arms

• Experimental: Group A: Asciminib
  Patients will receive 1.3 mg/kg oral asciminib 12 +/- 1.5 hours prior to a second dose of 1.3 mg/kg oral asciminib. Surgery or biopsy will take place 3 +/- 1.5 hours after last dose.
• Experimental: Group B: Asciminib + Sildenafil
  Patients will receive 1.3 mg/kg oral asciminib plus 20 mg sildenafil (10 mg if \< 20 kg) 12 +/- 1.5 hours prior to a second dose of 1/3 mg/kg asciminib plus 20 mg (10 mg if \< 20 kg) sildenafil. Surgery or biopsy will take place 3 +/- 1.5 hours after last dose.

Primary Outcome Measure

Tumor:plasma ratio of asciminib [ Time Frame: At time of surgical resection or biopsy (day 1) ]

Central Contacts

• Eric Thompson, M.D.
  314-454-4707
  [email protected]

Locations (1)

Find similar trials in St Louis, MO

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