Targeted Approach to Langerhans Cell Histiocytosis (LCH) Using MEK Inhibitor, Trametinib (NCT06582745) | Clinical Trial Compass
RecruitingPhase 2
Targeted Approach to Langerhans Cell Histiocytosis (LCH) Using MEK Inhibitor, Trametinib
United States75 participantsStarted 2024-06-24
Plain-language summary
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.
Who can participate
Age range1 Year – 30 Years
SexALL
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Inclusion Criteria:
* Diagnosis/disease status:
* Patients with newly diagnosed Langerhans cell histiocytosis (LCH) OR
* Patients with relapsed or refractory disease OR
* Patients with newly diagnosed or relapsed/refractory disease who are receiving the liquid formula of trametinib OR
* Patients who have been receiving trametinib as a treatment for LCH since January 1, 2020 may be included in the observational chart review to track long-term follow-up. Eligibility for chart review cohort will include receiving trametinib as treatment.
* Diagnosis confirmed with biopsy prior to start of treatment
* Patient must have adequate cardiac function evident through Echocardiogram (ECHO) and Electrocardiogram (EKG) within 30 days of starting treatment.
* Shortening fraction of ≥ 27% by echocardiogram or
* Ejection fraction of ≥ 50% by gated radionuclide study
* QTC \< 480 msec
* Performance status: Patients must have a performance status corresponding to ECOG scores of 0, 1, or 2. Use Karnofsky ≥ 50% for patients \> 16 years of age and Lansky ≥50% for patients ≤16 years of age.
* Adequate organ and marrow function as defined below:
* Absolute Neutrophil count ≥ 1,500/μL
* Platelets ≥ 100x103/μL
* Total bilirubin ≤ 1.5X ULN for age
* AST/ALT ≤ 2.5 X ULN for age
* Serum creatinine based on age/gender
* Hemoglobin ≥ 8 g/dL
* Patients with bone marrow disease must have hemoglobin ≥ 8 g/dL with transfusion support allowed
* Women of childbearing potential …