Asciminib With or Without Sildenafil for Brain Tumors (NCT07039760) | Clinical Trial Compass
Not Yet RecruitingEarly Phase 1
Asciminib With or Without Sildenafil for Brain Tumors
United States12 participantsStarted 2026-08-31
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 range
6 Years – 25 Years
Sex
ALL
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…
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Tumor:plasma ratio of asciminib
Timeframe: At time of surgical resection or biopsy (day 1)
2
Tumor:plasma ratio of asciminib with sildenafil
Timeframe: At time of surgical resection or biopsy (day 1)