Cobimetinib for BRAF-wild-type or Mutated Histiocytoses

• Status: Active, not recruiting
• Trial end date: 2022-06-22
• Target enrollment: 0
• Participant gender: All

Summary

COBRAH is a randomized double-blind 2-steps controlled superiority trial, with 2 parallel groups.

Patients will be randomly assigned in a 2:1 ratio to receive Cobimetinib orally or placebo during the first 12-weeks step, allowing the determination of the primary criteria.

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Assistance Publique - Hôpitaux de Paris

Criteria

Inclusion Criteria:

• Eligible patients should be at least 18 years of age,

• Have a histologically confirmed L or R group histiocytoses without BRAFV600E mutation detected with the use of a real-time polymerase chain reaction or with BRAFV600E mutation AND a contra-indication to BRAF inhibitors

• Have a measurable disease according to the PERCIST criteria with presence of at least one severe organ involvement (heart, vascular, central nervous system) OR a multisystemic disease with ≥3 organ involvement AND failure of a first-line treatment or contra-indication to these treatments,

• Accepting effective contraception during treatment duration (men and women childbearing potential) and 3 months after.

• Signed informed consent

Exclusion Criteria:

• Patients with severe hepatic, renal and cardiac outcomes

• Patients with myopathies at baseline

• Patients with retinal detachment at baseline

• Patients with inherited disorders of galactose intolerance, Lapp lactase deficiency or glucose-galactose malabsorption

• Patients with high bleeding risk.

• Allergies to iodized contrast media

• Simultaneous participation in another medical research

• Pregnancy or breast-feeding.

• No affiliation to the French Health Care System "sécurité sociale" OR no affiliation of European Health within the scope of Regulations (EEC) n° 1408/71 and 574/72 coordinating social security systems.