The ACTION Study - ONC201 in H3 K27M-mutant Diffuse Glioma Following Radiotherapy
Research type
Research Study
Full title
ONC201 for the Treatment of Newly Diagnosed H3 K27M-mutant Diffuse Glioma Following Completion of Radiotherapy: A Randomized, Double-Blind, Placebo-Controlled, Multicenter Study
IRAS ID
1006536
Contact name
ONC201-108 Clinical Trial Manager
Contact email
Sponsor organisation
Chimerix, Inc.
Clinicaltrials.gov Identifier
Research summary
This study is being done to learn how well a new drug called ONC201 works to treat a kind of brain tumour (“H3 K27M-mutant diffuse glioma”) and what side effects people have when they take it. After surgery and radiation, the cancer will come back in most people. There are currently no approved drugs for this type of brain tumour, so it is important to study new drugs. \nThe main goals of the study are to learn if ONC201 helps people with this kind of brain tumour live longer overall and live longer before the glioma gets worse. Other goals are to learn more about ONC201 side effects, if ONC201 improves quality of life, and if ONC201 lowers the dose of steroids needed.\nStudy visits will be about every 4 weeks for a year, then about every 8 weeks. MR brain scans will be done about every 8 weeks to monitor tumour size. Other procedures include physical exams, blood pressure, heart rate, heart rhythm (ECG), blood tests, and quality of life questions.\nThe study drug (ONC201 and placebo) is a capsule to take by mouth (swallow) on 2 days of every week. People in this study will be split into one of the following 3 groups by chance (randomly):\n•\tONC201 on 2 days of every week \n•\tONC201 on 1 day of every week and a placebo on 1 day of every week \n•\tPlacebo on 2 days of every week. \nPeople in the study may be able to keep taking study drug if their disease does not get worse and they do not have unacceptable side effects. After stopping study drug, people will still follow-up with the study centre about every 2 to 3 months.\nThis study is sponsored by Chimerix, Inc.\nParticipation in this study is completely voluntary.
REC name
South West - Central Bristol Research Ethics Committee
REC reference
22/SW/0169
Date of REC Opinion
20 Feb 2023
REC opinion
Further Information Favourable Opinion