Biological Medicine for Diffuse Intrinsic Pontine Glioma (DIPG) Eradication 2.0

The BIOMEDE 2.0 study is the second stage of the BIOMEDE multi-arm, multistage rolling programme (adaptive platform protocol). It is a multicenter, randomized, open-label, controlled phase-3 trial evaluating efficacy of ONC201 in comparison with everolimus (primary objective based on internal comparison) and subsequently to historical controls. Two treatment groups will be compared. A switch between treatment groups is allowed after confirmation of the disease progression (real-time central review blinded to the treatment arm allocation). Study treatment will be continued until centrally confirmed disease progression (either radiologically or histologically), unacceptable toxicity or consent withdrawal. The final conclusion of the trial will be successful for ONC201, if ONC201 is found significantly superior to everolimus in terms of centrally-reviewed PFS (Progression-free survival) from randomization (internal comparison) either overall, considering ND-DMG and DIPG-patients together, or in the subgroup of ND-DMG patients alone. In other cases, Everolimus will remain the standard arm unless it appears associated with an excess of toxicity compared to ONC201 which could then be discussed as a new standard.

NCT Number	NCT05476939
Lead Sponsor	Gustave Roussy, Cancer Campus, Grand Paris Collaborators: Innovative Therapies For Children with Cancer Consortium, Ministry of Health, France, Jazz Pharmaceuticals
Conditions	Diffuse Intrinsic Pontine Glioma, Diffuse Midline Glioma, H3 K27M-Mutant, Diffuse Midline Glioma, H3K27-altered
Enrollment	433 participants
Start Date	2022-09-29
Primary Completion	2028-09 (estimated)
Study Completion	2031-09 (estimated)
Updated on ClinicalTrials.gov	2026-08-13