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Dordaviprone

Phase 3

H3 K27M | Small molecule | Oncology |Jazz Pharmaceuticals plc|Last Updated: Apr 16, 2026

Target and mechanism

Molecular targetClpP, DRD2
Target classMitochondrial Protease, Dopamine Receptor
ModalitySmall molecule

Also known as Dordaviprone (ONC201)

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLEDDMC
Total Trials1
Total Enrollment510

FDA Designations

No designations recorded

Clinical trial landscape

Dordaviprone · 1 trial · 2 indications

Phase 3 1
NCT05580562ONC201 in H3 K27M-mutant Diffuse Glioma Following Radiotherapy (the ACTION Study)H3 K27M
RECRUITING510 Analytics
PHASE3RECRUITING
ONC201 in H3 K27M-mutant Diffuse Glioma Following Radiotherapy (the ACTION Study)
H3 K27MUnlock trial analytics

Study Endpoints

Primary Endpoints

Overall survival (OS)
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.

Secondary Endpoints

Progression Free Survival (PFS) using RANO 2.0 Criteria for All Participants
From date of randomization until the date of first documented progression assessed up to approximately 44 months.
PFS Using RANO 2.0 Criteria for Participants with Measurable Contrast-Enhancing Disease
From date of randomization up to 44 months
Incidence of adverse events
From date of randomization up to 44 months
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Study Design & Arms

AllocationRANDOMIZED
MaskingTRIPLE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Dordaviprone Twice Weekly GroupEXPERIMENTAL -
Dordaviprone Once Weekly GroupEXPERIMENTAL -
Placebo GroupPLACEBO_COMPARATOR -

Interventions

NameTypeDescription
Dordaviprone (ONC201)DRUGParticipants ≥ 52.5 kg will receive 625 mg of dordaviprone (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.
Dordaviprone (ONC201) + PlaceboDRUGParticipants ≥ 52.5 kg will receive 625 mg of dordaviprone (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
PlaceboOTHERParticipants will receive placebo (same number of capsules as the dordaviprone dose) on dosing days
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Eligibility Criteria

SexALL
Healthy VolunteersNo
Study Sites162

Inclusion Criteria: 1. Able to understand the study procedures and agree to participate in the study by providing written informed consent (by participant or legally authorized representative), and assent when applicable. 2. Body weight ≥ 10 kg at time of randomization. 3. Histologically diagnosed ...

Countries:United StatesArgentinaAustraliaAustriaBrazilCanadaDenmarkGermanyHong KongIsraelItalyJapanNetherlandsSingaporeSouth KoreaSpainSwitzerlandUnited Kingdom
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Frequently asked questions about Dordaviprone

What is Dordaviprone used for?

Dordaviprone is an investigational small molecule being studied for the treatment of H3 K27M-mutant diffuse glioma, a type of brain tumor. It is currently in Phase 3 clinical development and has not been approved by regulatory authorities.

What does Dordaviprone target?

Dordaviprone targets ClpP, a mitochondrial protease, and DRD2, a dopamine receptor. By interacting with these molecular targets, the drug is being evaluated for its potential therapeutic effects in H3 K27M-mutant diffuse glioma.

Who makes Dordaviprone?

Dordaviprone is being developed by Jazz Pharmaceuticals plc, a biopharmaceutical company listed on the stock exchange under the ticker symbol JAZZ. The company is conducting clinical trials to evaluate the drug's safety and efficacy.

What phase is Dordaviprone in?

Dordaviprone is in Phase 3 clinical development. It is an investigational drug, meaning it has not yet been approved by the FDA or other regulatory bodies, and its safety and efficacy are still being evaluated in clinical trials.

What clinical trials is Dordaviprone in?

Dordaviprone is being studied in the ACTION Study, a Phase 3 clinical trial with the identifier NCT05580562. This randomized, double-blind, placebo-controlled trial is recruiting 510 participants with H3 K27M-mutant diffuse glioma following radiotherapy, across multiple countries.

Is Dordaviprone the same as ONC201?

Yes, Dordaviprone is also known as ONC201. Both names refer to the same investigational drug being developed by Jazz Pharmaceuticals for the treatment of H3 K27M-mutant diffuse glioma.