ACTION
ACTION is the first placebo-controlled phase 3 trial ever run in diffuse midline glioma, the childhood brain-stem tumour that radiotherapy alone has never cured. It asks whether taking dordaviprone after radiotherapy lengthens life.
Overview
Randomised, double-blind, placebo-controlled phase 3 in patients aged 2 years and older with newly diagnosed H3 K27M-mutant diffuse glioma who have completed front-line radiotherapy; participants are randomised to dordaviprone once weekly, dordaviprone on two consecutive days weekly, or placebo. Primary endpoints are overall survival and progression-free survival. Dordaviprone (ONC201) is an imipridone that antagonises DRD2 and activates the mitochondrial protease ClpP, with activity concentrated in H3 K27M-mutant tumours in earlier single-arm studies; the FDA granted accelerated approval in August 2025 for recurrent H3 K27M-mutant diffuse midline glioma on the basis of pooled single-arm response data, with ACTION as the confirmatory trial. Jazz Pharmaceuticals acquired Chimerix in 2025 and now sponsors the study. Recruiting across North America, Europe, Asia and Australia; estimated enrolment 510.
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not linked directly; found by shared links- TrialFIREFLY-1
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- CancerPaediatric low-grade glioma
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- InstitutionACCELERATE
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- CancerCraniopharyngioma
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- CancerAtypical teratoid/rhabdoid tumour (ATRT)
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- InstitutionInnovative Therapies for Children with Cancer (ITCC)
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