Could a leukemia drug stop deadly brain tumor spread?
NCT ID NCT07039760
First seen Jun 25, 2026 · Last updated Sep 11, 2026 · Updated 4 times
Summary
This early study tests whether asciminib, a drug already approved for leukemia, can get into brain tumors in children and young adults (ages 6-25) with recurrent brain tumors. Some participants will also get sildenafil to see if it helps the drug enter the tumor. The main goal is to measure drug levels in tumor tissue after surgery, not to treat the tumor itself.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- asciminib with or without sildenafil
- What this could lead to
- If it works, this could point toward a way to treat brain tumors that have spread, by helping the drug reach the tumor in higher amounts.
- What could go wrong
- This is a very early, small study (12 people) that only measures drug levels in the tumor, not whether the drug actually shrinks tumors or improves survival. It may not lead to a treatment.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Early phase 1
The earliest testing in people: a first look at safety, in a very small group.
- Participants
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About 12 people
The number the study aims to enrol. It can still change while the study runs.
- Expected to start
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Oct 2026
An estimate. Start dates often move.
- Expected to finish
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May 2028
An estimate. End dates often move.
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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18 to 39 years
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: * Ages 18-39 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. * ECOG Performance Status of ≥ 2. 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. * Tumors that are suspected of a non-CNS primary location or history of non-CNS malignancy). * 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.
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Genom att skicka in godkänner du våra Användarvillkor
Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The study's own enquiry address
This study publishes an address for enquiries. See it below .
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The places running it
1 site. The list below names each one and where it is.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Show contact details
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Genom att skicka in godkänner du våra Användarvillkor
Study contacts
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Contact
Email: •••••@•••••
Locations
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Washington University School of Medicine/St. Louis Children's Hospital
St Louis, Missouri, 63110, United States
Contact Email: •••••@•••••
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