Study Objective: This is a clinical trial of a new medication to treat patients with Erdheim-Chester disease whose ECD tissue (1) does NOT have the BRAF mutation or (2) whose tissue DOES have the BRAF mutation, but they do not have access to a BRAF inhibitor or they have taken a BRAF inhibitor and cannot tolerate the medication.
Sponsor: Genentech and Memorial Sloan Kettering Cancer Center
Trial Contact Information: Eli L. Diamond, MD (212) 610-0243 or [email protected]
Requested Patient Involvement: Any patient with ECD who is BRAF-negative or who is BRAF positive and cannot acquire or tolerate a BRAF inhibitor. Patients with certain eye or heart problems may not be eligible and screening will be required (but can often be done close to home). The study requires monthly visits to MSKCC although after one year on the trial these can be every two months.
Known Centers Accepting ECD patients in the trial: Memorial Sloan-Kettering, New York City, NY USA
ClinicalTrials.gov Identifier: NCT02649972