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ACT001 (NCT06838676)

ACT001 for the Treatment of Diffuse Intrinsic Pontine Gliomas and H3K27-altered High Grade Gliomas: a one-minute explainerNCT068386761 min

ACT001 trial for Diffuse Intrinsic Pontine Gliomas (DIPG) (NCT06838676). This is a short film with English narration. The transcript is on this page, and the written brief is separate.

What the film says

Read the transcript

This trial is for patients aged 1–39 with newly diagnosed DIPG, a brainstem tumor, without metastatic disease. Treatment starts 28–35 days after radiation. Enrollment for recurrent or refractory tumors is paused.

The idea: oral ACT001 blocks PAI-1-linked growth signals and other tumor pathways. Patients take it twice daily in 28-day cycles, usually for up to two years. Biopsy is not required for typical DIPG imaging.

This phase 2 study plans 60 patients. Nationwide Children’s Hospital sponsors it, with seven recruiting US sites. Researchers assess survival and safety.

Public record; not medical advice.

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