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

Part of paid clinical trials in Fort Worth, Texas.

Sponsor
Cook Children's Health Care System
Study ID
NCT06582745
Phase
PHASE2
Status
Recruiting
Apply to this trial

Conditions

  • Langerhans Cell Histiocytosis

Eligibility Criteria

Sex
ALL
Age
1 Year - 30 Years
Healthy Volunteers
Not accepted

Interventions

  • Trametinib — DRUG
    Participants will be given trametinib as a single agent once a day dosing, with a maximum dose of 2kg. Participants who are able to swallow pills will be given oral tablets. Participants unable to swallow pills will be dispensed the liquid formula concentrate at 0.05mg/mL with dosing of 0.015mg/kg.

Study Details

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.

Key Dates

Start date
Jun 24, 2024
Status verified
Apr 2026
Primary completion
Jun 30, 2039
Completion
Dec 31, 2039

Study Design

Enrollment
75 participants (estimated)
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT

Arms

  • Experimental: Prospective Treatment
    Trametinib will be administered in 28-day cycles, given once daily or adjusted as per clinical judgment of the treating physician, with a maximum dosage of 2mg daily. Patients will be followed for 4 years after receiving treatment for two years. Patients may continue on the same treatment beyond two years if they and their treating physicians agree to do so in the best interest of the patient.
  • No Intervention: Observation Only
    Patients who have been receiving trametinib as a treatment for LCH since January 1, 2020 may be included in an observational chart review to track long-term follow-up.

Primary Outcome Measure

Time to Progression (TTP) [ Time Frame: Up to six years ]

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
Cook Children's Health Care SystemFort WorthTexas76104
Megan Gibbs, BSN, RN
[email protected]
682-885-2116
Anish Ray, MD (PRINCIPAL_INVESTIGATOR)

Find similar trials in Fort Worth, TX

By research site
Cook Children's Health Care System· Fort Worth, TX

Related Studies