Actively Recruiting
Cobimetinib in Refractory Langerhans Cell Histiocytosis (LCH), and Other Histiocytic Disorders
Led by Carl Allen · Updated on 2025-09-18
90
Participants Needed
12
Research Sites
449 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
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.
CONDITIONS
Official 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: At least 6 months and less than 21 years old at enrollment
- For Group 2: At least 6 months old at enrollment
- For Group 3: At least 6 months and less than 21 years old at enrollment
- For Group 4: 21 years or older at enrollment
- Able to take medication by mouth or enteral route (oral suspension, tablet, nasogastric or gastric tube)
- Biopsy-proven Langerhans cell histiocytosis (LCH) with failure of at least front-line therapy and evaluable disease, or diagnosis of LCH-associated neurodegenerative disease with recent progression, or biopsy-proven juvenile xanthogranuloma (JXG), Erdheim-Chester disease (ECD), Rosai-Dorfman disease (RDD), histiocytic sarcoma, or other histiocytic lesions with active disease
- Karnofsky performance score ≥ 50% for patients >16 years and Lansky score ≥ 50% for patients ≤16 years
- Adequate blood counts: ANC ≥ 0.75 x 10^9/L without growth factors, platelet count ≥ 75 x 10^9/L without transfusion in past 7 days (or with transfusion support if marrow disease), hemoglobin ≥ 8 g/dL without transfusion in past 7 days (or with transfusion support if marrow disease)
- Adequate kidney function by creatinine clearance or serum creatinine limits by age and gender
- Adequate liver function: bilirubin ≤1.5 x ULN, AST and ALT ≤ 3x ULN (or ≤ 5x ULN if liver involved), serum albumin ≥ 2 g/dL
- For liver disease caused by histiocytic disorder, may enroll with abnormal liver tests if documented
- Adequate heart function with fractional shortening ≥ 30% or ejection fraction ≥ 50% by echocardiogram or MUGA within 28 days prior to enrollment
- Female patients of childbearing potential must have negative pregnancy test and agree to contraceptive measures; males with partners of childbearing potential must agree to contraception during treatment and for 3 months after
You will not qualify if you...
- Use of strong CYP3A4 inducers or inhibitors within 14 days prior to enrollment, including certain antibiotics, antifungals, grapefruit juice, or St. John's wort
- Prior chemotherapy, immunotherapy, radiotherapy, or targeted therapy within specified timeframes prior to enrollment (e.g., radiation within 14 days, investigational therapy within 4 weeks, stem cell transplant within 90 days)
- Prior treatment with cobimetinib
- Recent use of hematopoietic growth factors within 7-14 days before enrollment
- Use of hormonal therapy (except hormone replacement or oral contraceptives), immunotherapy, biologics, investigational or herbal cancer therapy within 28 days or less than 5 half-lives prior to enrollment
- Use of anticoagulants within 7 days prior to enrollment for patients with brain tumors
- Corticosteroid therapy above 0.5 mg/kg/day or unstable dosing
- Taking anticoagulants or having bleeding disorders unrelated to histiocytic disease
- Other active cancers or history of secondary malignancy
- Severe nausea, vomiting, malabsorption, biliary shunt
- Active infections within 28 days prior to enrollment
- Recent major surgery or traumatic injury within 28 days
- History of significant bowel resection or malabsorption
- History of pneumonitis
- Significant eye conditions or risk factors for retinal vein occlusion
- Prior solid organ transplantation
- Any disease or condition deemed unsafe by the investigator
- Significant cardiac problems including unstable arrhythmias, angina, heart failure, recent heart attack
- Known chronic HIV infection
- Recent or significant CNS hemorrhage
- Pregnant or breastfeeding women
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
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
CONTACT
O
Olive Eckstein, MD
CONTACT
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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