RecruitingPhase 2NCT04079179

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

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.


Sponsor

Carl Allen

Enrollment

90 participants

Start Date

Apr 19, 2021

Study Type

INTERVENTIONAL

Conditions

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.


Eligibility

Plain Language Summary

Simplified for easier understanding

This study is testing cobimetinib — a drug that blocks a specific protein involved in cancer cell growth — in patients with Langerhans cell histiocytosis (LCH) and other rare immune cell cancers that have not responded to prior treatment. LCH is a rare disease where abnormal immune cells accumulate and damage organs. **You may be eligible if...** - You have been diagnosed with LCH confirmed by biopsy, or another related histiocytic disorder - Your disease has come back or not responded to at least one prior line of treatment - You are at least 6 months old (age requirements vary by study group) - You are able to take oral medication (pill or liquid suspension, including through a feeding tube) - Adults 21 and older may qualify for Group 4 **You may NOT be eligible if...** - You have not had prior treatment that has failed - You are unable to take oral or enteral (tube-fed) medication - You do not meet the specific age criteria for the group you would be enrolled in Talk to your doctor to see if this trial is right for you.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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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 Hospital

Phoenix, Arizona, United States

Arkansas Children's Hospital

Little Rock, Arkansas, United States

Children's Hospital of Orange County

Orange, California, United States

UCSF Benioff Children's Hospital

San Francisco, California, United States

Children's National Hospital

Washington D.C., District of Columbia, United States

John Hopkins University School of Medicine

Baltimore, Maryland, United States

Dana Farber Cancer Institute, Boston Children's

Boston, Massachusetts, United States

Memorial Sloan Kettering Cancer Center

New York, New York, United States

NACHO Consortium

Memphis, Tennessee, United States

Children's Medical Center- UTSW

Dallas, Texas, United States

Texas Children's Hospital

Houston, Texas, United States

University of Wisconsin-American Family Children's Hospital

Madison, Wisconsin, United States

View Full Details on ClinicalTrials.gov

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NCT04079179


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