ONC201 in H3 K27M-mutant Diffuse Glioma Following Radiotherapy (the ACTION Study) (ACTION)
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|ClinicalTrials.gov Identifier: NCT05580562|
Recruitment Status : Recruiting
First Posted : October 14, 2022
Last Update Posted : September 26, 2023
|Condition or disease||Intervention/treatment||Phase|
|H3 K27M Glioma||Drug: ONC201 Drug: ONC201 + Placebo Other: Placebo||Phase 3|
Expanded Access : An investigational treatment associated with this study is available outside the clinical trial. More info ...
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||450 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Triple (Participant, Care Provider, Investigator)|
|Official 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|
|Actual Study Start Date :||January 23, 2023|
|Estimated Primary Completion Date :||August 2026|
|Estimated Study Completion Date :||August 2026|
|Experimental: ONC201 Twice Weekly Group||
Participants ≥ 52.5 kg will receive 625 mg of ONC201 (5 × 125-mg capsules) dosing days; participants < 52.5 kg will receive a dose (and corresponding number of capsules) scaled by body weight and rounded to 125-mg increments.
|Experimental: ONC201 Once Weekly Group||
Drug: ONC201 + Placebo
Participants ≥ 52.5 kg will receive 625 mg of ONC201 (5 × 125-mg capsules) or matching placebo on dosing days; participants < 52.5 kg will receive a dose (and corresponding number of capsules) scaled by body weight and rounded to 125-mg increments
|Placebo Comparator: Placebo Group||
Participants will receive placebo (same number of capsules as the ONC201 dose) on dosing days
- Overall survival (OS) [ Time Frame: From date of randomization until date of death from any cause, assessed up to approximately 44 months ]Overall Survival is defined as the time from randomization to death due to any cause.
- Progression free survival (PFS) as assessed by using RANO-HGG criteria [ Time Frame: From date of randomization until the date of first documented progression assessed up to approximately 44 months ]PFS is defined as time from randomization to disease progression (PD) or death.
- Incidence of adverse events [ Time Frame: From date of randomization up to 44 months ]Incidence of overall, treatment-related, Grade 3 or higher in severity, serious, fatal, those resulting in treatment discontinuation, and events of special interest
- Change from baseline in clinical laboratory parameters [ Time Frame: From date of randomization up to 44 months ]Percentage of participants with clinically significant laboratory results
- PFS using RANO-HGG criteria [ Time Frame: From date of randomization up to 44 months ]PFS using RANO-HGG criteria for participants with measurable contrast-enhancing disease
- Corticosteroid response [ Time Frame: From date of randomization up to 44 months ]Corticosteroid response will be measured by a confirmed 50% decrease in use of dexamethasone or equivalent
- Performance status response [ Time Frame: From date of randomization up to 44 months ]Performance status response will be measured by confirmed increase in Karnofsky Performance Status (KPS) or Lansky Performance Status (LPS)
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05580562
|Contact: Tarapore, PhDfirstname.lastname@example.org|