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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.
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.
Shares RACE for Children Act, Paediatric oncology roadmap: cooperative-group cures → engineered immunity → drugs developed for children first and the tags paediatric, cns.
Shares RACE for Children Act, Paediatric oncology roadmap: cooperative-group cures → engineered immunity → drugs developed for children first and the tags paediatric, cns.
Shares RACE for Children Act, Diffuse midline glioma, H3 K27-altered (including DIPG), Paediatric oncology roadmap: cooperative-group cures → engineered immunity → drugs developed for children first, Rare and paediatric cancers without markets and the tags paediatric, cns.
Shares Rare and paediatric cancers without markets and the tags paediatric, cns.
Shares Rare and paediatric cancers without markets and the tags paediatric, cns.
Shares Rare and paediatric cancers without markets and the tags paediatric, cns.
Shares the tags paediatric, cns.
Shares RACE for Children Act, Diffuse midline glioma, H3 K27-altered (including DIPG), Paediatric oncology roadmap: cooperative-group cures → engineered immunity → drugs developed for children first, Rare and paediatric cancers without markets and the tag paediatric.