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.
Carl Allen
Summary
This is a research study of a drug called cobimetinib in children and adults diagnosed with Langerhans cell histiocytosis (LCH), and other histiocytic disorders that has returned or does not respond to treatment. Cobimetinib blocks activation of a protein called Mitogen-activated protein kinase (MEK) that is part of incorrect growth signals in histiocytosis cells. Four different groups of patients will be enrolled.
Description
Histiocytic disorders are diseases caused by misfunctioning or buildup of particular immune cells called histiocytes. Many histiocytic disorders (LCH, juvenile xanthogranuloma (JXG), Erdheim-Chester disease (ECD), and Rosai-Dorfman Disease (RDD)) arises from blood cells that receive incorrect growth signals. These incorrect signals are caused by changes in genes (mutations) that lead to tissue damage (lesions) which causes disease. Some patients with LCH can develop neurodegeneration (LCH-ND) which is damage to neurons that results in reduced brain function, from LCH cells that go to the brain…
Eligibility
- Age range
- Not specified
- Sex
- All
- Healthy volunteers
- No
INCLUSION CRITERIA: Age at study entry * For Group 1: Participant must be at least 6 months of age and less than 21 years of age at the time of enrollment * For Group 2: Participant may be at least 6 months of age at the time of enrollment * For Group 3: Participant must be at least 6 months of age and less than 21 years of age at the time of enrollment * For Group 4: Participant must be 21 years of age or older at the time of enrollment * Participant must be able to take an enteral dose and formulation of medication. Study medication is only available as an oral suspension or tablet which m…
Interventions
- DrugCobimetinib
Cobimetinib will be administered at a maximal dose of 60 mg daily for patients \<18 years old and a flat dose of 40 mg daily for patients ≥18 years for 21 days on, then 7 days off, in a 28-day treatment cycle for a total of 12 cycles (approximately 12 months).
Locations (12)
- Phoenix Children's HospitalPhoenix, Arizona
- Arkansas Children's HospitalLittle Rock, Arkansas
- Children's Hospital of Orange CountyOrange, California
- UCSF Benioff Children's HospitalSan Francisco, California
- Children's National HospitalWashington D.C., District of Columbia
- John Hopkins University School of MedicineBaltimore, Maryland