A Phase 2 Study of Cobimetinib in People With Langerhans Cell Histiocytosis and Other Histiocytic Disorders

Full Title

A Phase 2 Study to Assess the Safety and Efficacy of Cobimetinib in Refractory Langerhans Cell Histiocytosis, LCH-Associated Neurodegenerative Disease, and Other Histiocytic Disorders

Purpose

Researchers are assessing the use of cobimetinib to treat Langerhans cell histiocytosis (LCH) and other histiocytic disorders. Histiocytic disorders are diseases caused by the abnormal function or buildup of immune cells called histiocytes. Cobimetinib works by blocking the activity of a protein called MEK. This protein leads to the abnormal growth and development of histiocytosis cells.

Cobimetinib is already used to treat adults with certain histiocytic disorders. In this study, researchers want to see if it works well in children and teens with LCH and other histiocytic disorders. Cobimetinib is taken orally (by mouth).

Contact

For more information or to see if your child can join this study, please call 833-MSK-KIDS.

Protocol
23-187
Phase
Phase II (phase 2)
Disease Status
Newly Diagnosed & Relapsed/Refractory
Co-Investigators
Diseases
ClinicalTrials.gov ID
NCT04079179