Asciminib With or Without Sildenafil for Brain Tumors

Launched by WASHINGTON UNIVERSITY SCHOOL OF MEDICINE · Jun 17, 2025

Keywords

ClinConnect Summary

This clinical trial is studying a new treatment approach for brain tumors, specifically for patients whose tumors have come back or grown despite previous treatments. The study is testing a drug called asciminib, which is already approved for a type of blood cancer but is now being explored to see if it can help control brain tumors by targeting certain proteins involved in tumor spread. The trial will also look at whether adding another medication, sildenafil (commonly used for other conditions), makes a difference. Researchers want to find out if asciminib can safely reach the brain and affect tumor growth.

Young people between the ages of 6 and 25 with a certain type of brain tumor that can be safely reached by surgery or biopsy may be eligible to join. Participants will need to have a good overall health status and meet specific blood and organ function requirements. Those who join will be carefully monitored before, during, and after treatment to assess safety and how well the drug works. It’s important to know that this study is still in an early phase, meaning its main goal is to see if the treatment is safe and to learn how it behaves in the body, rather than to prove it definitely works yet. Families interested in this trial should discuss with their doctors to see if it might be a suitable option.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • Ages 6-25 years old.
• • 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.
• • Unable to take tablets orally
• • Pregnant and/or breastfeeding. Subjects of childbearing potential must have a negative serum or urine pregnancy test within 10 days prior to Day 1.
• • Active infection requiring treatment or an unexplained febrile (\> 101.5o F) illness.
• • Known immunosuppressive disease or human immunodeficiency virus infection.
• • Any active renal, cardiac (congestive cardiac failure, myocardial infarction, myocarditis), or pulmonary disease.
• • Any clinically significant unrelated systemic illness (serious infections or significant cardiac, pulmonary, hepatic or other organ dysfunction).
• • Inability to return for follow-up visits or obtain follow-up studies required to assess toxicity to therapy.