Actively Recruiting
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
Led by Carl Allen · Updated on 2025-09-18
90
Participants Needed
12
Research Sites
208 weeks
Total Duration
On this page
Sponsors
C
Carl Allen
Lead Sponsor
B
Baylor College of Medicine
Collaborating Sponsor
AI-Summary
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.
CONDITIONS
Brief Title
Cobimetinib in Refractory Langerhans Cell Histiocytosis (LCH), and Other Histiocytic Disorders
Who Can Participate
Eligibility Criteria
You may qualify if you...
- For Group 1: Age at least 6 months and less than 21 years at enrollment
- For Group 2: Age at least 6 months at enrollment
- For Group 3: Age at least 6 months and less than 21 years at enrollment
- For Group 4: Age 21 years or older at enrollment
- Able to take medication by mouth or enteral route (tablet or suspension)
- Biopsy proven Langerhans cell histiocytosis (LCH) or diagnosis of LCH-associated neurodegenerative disease with recent progression
- Biopsy proven juvenile xanthogranuloma, Erdheim-Chester disease, Rosai-Dorfman disease, histiocytic sarcoma, or other histiocytic lesions with active disease
- Karnofsky performance status ≥ 50% for patients older than 16 years, Lansky performance status ≥ 50% for patients 16 years or younger
- Adequate blood, kidney, liver, and heart function according to defined laboratory and clinical criteria
- Negative pregnancy test for females of childbearing potential and agreement to use effective birth control methods during and after the study
You will not qualify if you...
- Use of strong CYP3A4 inducers or inhibitors within 14 days before enrollment
- Recent chemotherapy, immunotherapy, radiotherapy, or targeted therapy within defined time frames
- Prior treatment with cobimetinib
- Use of hematopoietic growth factors or anticoagulants within specified periods
- Active infection or unresolved toxicity from previous treatments
- Major surgery or significant injury within 28 days before enrollment
- History of significant bowel malabsorption or pneumonitis
- Significant eye conditions or risk factors for retinal vein occlusion
- History of solid organ transplantation
- Other medical or psychological conditions that increase risk
- Clinically significant heart problems including unstable arrhythmias, angina, heart failure, or recent heart attack
- Known HIV infection
- Recent central nervous system hemorrhage
- Pregnant or breastfeeding females
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person) for screening and enrollment
Duration - Approximately 12 months
Participants receive cobimetinib daily in 28-day cycles, with 21 days on medication followed by 7 days off, for up to 12 cycles (approximately 12 months).
Visits occur during each 28-day treatment cycle for monitoring and medication management
Trial Site Locations
Total: 12 locations
1
Phoenix Children's Hospital
Phoenix, Arizona, United States, 85016
Actively Recruiting
2
Arkansas Children's Hospital
Little Rock, Arkansas, United States, 72202
Actively Recruiting
3
Children's Hospital of Orange County
Orange, California, United States, 92868
Actively Recruiting
4
UCSF Benioff Children's Hospital
San Francisco, California, United States, 94158
Actively Recruiting
5
Children's National Hospital
Washington D.C., District of Columbia, United States, 20010
Actively Recruiting
6
John Hopkins University School of Medicine
Baltimore, Maryland, United States, 21287
Actively Recruiting
7
Dana Farber Cancer Institute, Boston Children's
Boston, Massachusetts, United States, 02215
Actively Recruiting
8
Memorial Sloan Kettering Cancer Center
New York, New York, United States, 10065
Actively Recruiting
9
NACHO Consortium
Memphis, Tennessee, United States, 38105
Not Yet Recruiting
10
Children's Medical Center- UTSW
Dallas, Texas, United States, 75235
Actively Recruiting
11
Texas Children's Hospital
Houston, Texas, United States, 77030
Actively Recruiting
12
University of Wisconsin-American Family Children's Hospital
Madison, Wisconsin, United States, 53792
Terminated
Research Team
C
Carl E Allen, MD, PhD
O
Olive Eckstein, MD
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
4
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