Actively Recruiting
Targeted Approach to Langerhans Cell Histiocytosis (LCH) Using MEK Inhibitor, Trametinib
Led by Cook Children's Health Care System · Updated on 2026-04-07
75
Participants Needed
1
Research Sites
805 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
The purpose of this Phase II clinical trial is to establish the safety and effectiveness of trametinib, a targeted therapy, for the treatment of newly or recently diagnosed Langerhans Cell Histiocytosis (LCH) among pediatric patients.
CONDITIONS
Official Title
Targeted Approach to Langerhans Cell Histiocytosis (LCH) Using MEK Inhibitor, Trametinib
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Patients with newly diagnosed or relapsed/refractory Langerhans Cell Histiocytosis (LCH)
- Diagnosis confirmed with biopsy before treatment start
- Adequate heart function shown by echocardiogram or electrocardiogram within 30 days before treatment
- Performance status of ECOG 0, 1, or 2; Karnofsky score 60; or Lansky score 60 depending on age
- Adequate organ and marrow function with specific blood count and chemistry levels
- Use of effective contraception by women of childbearing potential and men during and 4 months after study participation
- Ability to understand and follow study procedures throughout the study
- Patients receiving trametinib since January 1, 2020, may join for observational follow-up
You will not qualify if you...
- Patients with low-risk skin-only or single bone lesion LCH not requiring treatment, except for certain high-risk or critical site lesions
- Patients with specific class 3 MAP2K1 mutations (I103_K104del, E102_I103del, L98_K104delinsQ, L98_I103del, I99_K104del)
- Patients with jaundice at diagnosis
- Pregnant or breastfeeding patients; women must have a negative pregnancy test before treatment
- Allergy to trametinib
- Current drug or alcohol dependence affecting study adherence
- Inability or unwillingness to provide informed consent
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
Cook Children's Health Care System
Fort Worth, Texas, United States, 76104
Actively Recruiting
Research Team
M
Megan Gibbs, BSN, RN
CONTACT
A
Angelica Gonzalez, PhD
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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