Actively Recruiting

Phase 2
Age: 1Year - 30Years
All Genders
ID06582745

Targeted Approach to Langerhans Cell Histiocytosis Using MEK Inhibitor Trametinib for Pediatric Patients

Led by Cook Children's Health Care System · Updated on 2026-04-07

75

Participants Needed

1

Research Sites

26 weeks

Total Duration

On this page

AI-Summary

What this Trial Is About

This research aims to evaluate the safety and effectiveness of trametinib, a targeted therapy, in treating newly or recently diagnosed Langerhans Cell Histiocytosis (LCH) in pediatric patients. LCH is a rare disease affecting children, involving abnormal growth of certain immune cells, and can impact one or multiple organs. The study also explores genomic cancer testing to better understand the disease through advanced sequencing techniques. Participants will receive trametinib once daily in 28-day cycles, with dosing adjusted by the physician up to a maximum of 2 mg daily. Those who can swallow pills will take oral tablets, while others will receive a liquid formula based on weight. The treatment period lasts for two years, after which patients are followed for four additional years. Patients already on trametinib since January 2020 may join an observational group to monitor long-term outcomes. During the study, participants will undergo regular assessments including biopsies, imaging (CT, MRI, PET), heart function tests, and blood tests to monitor health and disease progression. Researchers will track outcomes such as time to disease progression, survival, and response to treatment over up to six years. Safety, side effects, and treatment adherence will be closely monitored throughout the study.

CONDITIONS

Brief Title

Targeted Approach to Langerhans Cell Histiocytosis (LCH) Using MEK Inhibitor, Trametinib

Who Can Participate

Age: 1Year - 30Years
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Patients with newly diagnosed or relapsed/refractory Langerhans Cell Histiocytosis (LCH)
  • Diagnosis confirmed by biopsy before treatment
  • Adequate cardiac function shown by recent echocardiogram or EKG
  • Performance status with ECOG score 0, 1, or 2; Karnofsky 60% for patients over 16 years, Lansky 50% for 16 years and younger
  • Adequate organ and marrow function including neutrophil count 600/bcL, platelets 60x103/bcL, bilirubin, liver enzymes, creatinine, and hemoglobin levels
  • Women of childbearing potential and men agree to use effective contraception during study and 4 months after
  • Ability to understand and comply with study procedures
Not Eligible

You will not qualify if you...

  • Patients with low-risk single skin or bone lesion not requiring treatment, except CNS-risk or functionally critical lesions
  • Patients with specific MAP2K1 genetic mutations (I103_K104del, E102_I103del, L98_K104delinsQ, L98_I103del, I99_K104del)
  • Patients presenting with jaundice at diagnosis
  • Pregnant or breastfeeding patients
  • Allergy to trametinib
  • Current drug or alcohol use interfering with study adherence
  • Inability or unwillingness to provide informed consent

AI-Screening

AI-Powered Screening

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Your Study Journey

Screening

Duration - 2 to 4 weeks

Participants are screened for eligibility to participate in the trial.

Treatment

Duration - Minimum of two years with possible extension beyond two years

Participants receive trametinib once daily in 28-day cycles for a minimum of two years. Dosing and duration may be adjusted based on clinical judgment and participant needs.

Daily dosing with regular clinical visits during treatment

Follow-up

Duration - Up to four years after treatment ends

Participants are followed for up to four years after completing treatment to monitor health and disease status.

Regular follow-up visits over four years

Trial Site Locations

Total: 1 location

1

Cook Children's Health Care System

Fort Worth, Texas, United States, 76104

Actively Recruiting

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Research Team

M

Megan Gibbs, BSN, RN

A

Angelica Gonzalez, PhD

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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Published Research Related To This Trial

Mutually exclusive recurrent somatic mutations in MAP2K1 and BRAF support a central role for ERK activation in LCH pathogenesis.

Rikhia Chakraborty, Oliver A Hampton, Xiaoyun Shen...

https://pubmed.ncbi.nlm.nih.gov/25202140

Improved outcome in the treatment of pediatric multifocal Langerhans cell histiocytosis: Results from the Japan Langerhans Cell Histiocytosis Study Group-96 protocol study.

Akira Morimoto, Satoshi Ikushima, Naoko Kinugawa...

https://pubmed.ncbi.nlm.nih.gov/16804933