Plain-English translation of NCT04732065 on ClinicalTrials.gov ↗ · Source last updated · Translation generated · How we translate trials
Phase 1 — Testing in a small group (usually 20–80 people) to find a safe dose and watch for side effects.
This trial is testing a new medication called to treat serious brain and spinal cord tumors in children and young adults. The medication works in a new way — it targets two different pathways in cancer cells that are thought to help tumors grow. Depending on your situation, you might take the medication alone, or combine it with radiation therapy.
Diffuse midline gliomas and other brain tumors in children are very difficult to treat with current therapies, and many patients relapse or don't respond well. This trial exists to see if this new medication could offer hope by attacking these tumors through a different mechanism than standard treatments.
You likely qualify if…
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You would take the medication by mouth up to six times per week for treatment cycles lasting 28 days. Depending on your arm, you might receive this medication alone, or alongside daily radiation therapy given five days per week. The trial lasts up to 12 months (possibly extending to 24 months if you're benefiting), with regular visits to monitor how you're responding and check for any side effects. Some participants will also have tumor tissue samples tested to see how much of the medication reaches the tumor.
AI-generated summary from trial data · Aug 22, 2026 · Not medical advice
United States