Not Yet RecruitingEarly Phase 1

Asciminib With or Without Sildenafil for Brain Tumors

United States12 participantsStarted 2026-04-30

Plain-language 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.

Who can participate

Age range6 Years – 25 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * Ages 6-25 years old, inclusive. * Radiographic evidence of a recurrent/progressive brain tumor. * Tumor must be predominantly in an intraparenchymal location. * Deemed operable (able to be resected or have an open or stereotactic needle biopsy) by treating neurosurgeon. * Karnofsky/Lansky Performance Status of ≥ 60. Patients who are unable to walk because of paralysis but who are up in a wheelchair will be considered ambulatory for the purposes of the performance score. * Bone Marrow: * ANC (Absolute neutrophil count) ≥ 1000/µl (unsupported). * Platelets ≥ 100,000/µl (may be supported by transfusion). * Hemoglobin \> 8 g/dL (may be supported by transfusion). * Renal: * Serum creatinine ≤ upper limit of institutional normal. * Hepatic: * Bilirubin ≤ 1.5 times upper limit of normal for age. * ALT (SGPT) ≤ 3 times institutional upper limit of normal for age. * AST (SGOT) ≤ 3 times institutional upper limit of normal for age. * Ability to understand and willingness to sign an IRB approved written informed consent document. Legally authorized representatives may sign and give informed consent on behalf of study participants. All patients and/or their parents or legal guardians must sign an IRB approved written informed consent document. Exclusion Criteria: * Tumors suspected to be pituitary tumors or tumors of the meninges. * Diagnosis of atypical teratoid rhabdoid tumor (ATRT) or diagnosis of pilocytic astrocytoma (PA). * Unable to take t…

What they're measuring

Tumor:plasma ratio of asciminib

Timeframe: At time of surgical resection or biopsy (day 1)

Tumor:plasma ratio of asciminib with sildenafil

Timeframe: At time of surgical resection or biopsy (day 1)

Trial details

NCT IDNCT07039760

SponsorWashington University School of Medicine

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2027-10-31

Contact for this trial

Eric Thompson, M.D.

314-454-4707 pedshemonctrialreferral@wustl.edu