Phase 2 Study of Cobimetinib for Treating Refractory Langerhans Cell Histiocytosis, LCH-Associated Neurodegenerative Disease, and Other Histiocytic Disorders in Children and Adults

What this Trial Is About

Researchers are studying a drug called cobimetinib in children and adults who have Langerhans cell histiocytosis LCH or other histiocytic disorders that have returned or do not respond to treatment. These disorders involve immune cells called histiocytes that grow abnormally due to genetic mutations, causing damage to tissues and organs. Some patients with LCH may develop neurodegeneration, which affects brain function. This phase 2 study aims to assess whether cobimetinib, which blocks a protein involved in abnormal cell growth, is safe and effective in treating these conditions, including patients with a specific mutation called BRAF-V600E. The study includes four groups of patients based on age and type of histiocytic disorder. Cobimetinib will be given orally as a tablet or suspension once daily for 21 days followed by 7 days off, making a 28-day treatment cycle. Children under 18 will receive up to 60 mg daily, and adults 18 or older will receive 40 mg daily. Participants will receive up to 12 cycles of treatment, approximately 12 months in total. Participants will be monitored regularly through clinical evaluations, imaging, and laboratory tests to measure overall response rates after 12 months using modified RECIST criteria. Researchers will also assess progression-free survival and record any side effects or adverse events during the study. Safety and effectiveness data will be collected throughout the treatment period to understand how patients respond to cobimetinib. The entire participation time is about one year of treatment and follow-up.

Cobimetinib in Refractory Langerhans Cell Histiocytosis (LCH), and Other Histiocytic Disorders