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About
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. Study treatment will be continued until centrally confirmed disease progression (either radiologically or histologically), unacceptable toxicity or consent withdrawal. A switch between treatment groups is allowed after confirmation of the disease progression (real-time central review blinded to the treatment arm allocation).
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.
Enrollment
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Inclusion and exclusion criteria
Eligibility criteria for the inclusion (registration) in BIOMEDE 2.0 study:
Diagnosis Criteria:
Eligible for a biopsy, or biopsy material available for the biomarker assessment.
Age > 6 months, with no upper age limit. Children between 6 months and 3 years will be discussed on a case by case basis for inclusion in the study for the feasibility of the stereotactic biopsy.
Eligible for cerebral or craniospinal radiotherapy.
Tumor at diagnosis: no prior chemotherapy for the present cancer; no prior cerebral radiation therapy even for another neoplasm. Surgery is allowed when performed for diagnostic or therapeutic purpose.
Metastatic diseases or spinal tumors allowed; in this case, patients would receive craniospinal or spinal radiotherapy and medical treatment (everolimus or ONC201) will be postponed and only started after the end of radiotherapy.
Patients must be affiliated to a social security system or beneficiary of the same according to local requirements.
Written informed consent from parents/legal representative, patient, and age-appropriate assent before any study-specific procedures are conducted according to local, regional or national guidelines.
Non eligibility criteria for the inclusion (registration) in BIOMEDE 2.0 study:
Eligibility criteria for the randomization in BIOMEDE 2.0 study:
Non Eligibility criteria for the randomization in BIOMEDE 2.0 study:
Current organ toxicity > grade 2 according to the NCI-CTCAE version 5.0 especially cardiovascular or renal disease (including but not limited to: congenital long QT syndrome, nephrotic syndrome, glomerulopathy, uncontrolled high blood pressure despite adequate treatment).
ONC201 administration should be avoided for patients with:
In this case, patients will be treated in the Everolimus arm without randomization (except if contra-indication to Everolimus).
Primary purpose
Allocation
Interventional model
Masking
409 participants in 2 patient groups
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Central trial contact
Anne-Sophie BLANC, PharmD; Jacques GRILL, MD, PhD
Data sourced from clinicaltrials.gov
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