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Targeted Therapy with Trametinib for Langerhans Cell Histiocytosis

This study is testing a medicine called trametinib for people with Langerhans Cell Histiocytosis (LCH), a rare condition affecting children. Trametinib is a "targeted therapy" that works by blocking specific signals (RAF) that can cause LCH cells to grow. The study aims to see how safe and effective trametinib is for newly diagnosed LCH or when LCH has come back or not responded to other treatments. Participants will take trametinib once a day, either as a pill or a liquid. The study will follow participants for up to six years to see how long they live, how long they live without the disease getting worse, and how long it takes for the disease to progress. This study is for people aged 1 to 30 years old. The current status of the study is unclear, and it plans to enroll 75 participants.

Study design
This is an interventional study, meaning participants will receive a specific treatment. It plans to enroll 75 participants, but the phase is not specified.
What's involved
Participants will be given trametinib once a day, either as oral tablets or a liquid formula concentrate.
Compensation
Not stated in the trial record.
Follow-up
Participants will be followed for up to six years to measure time to progression, progression-free survival, and overall survival.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT06582745

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

Recruiting
PHASE2Ages 1–30InterventionalTreatment
Cook Children's Health Care System
~75 participants
Updated 2026-04-07 on ClinicalTrials.gov
What's tested:Trametinib

At a glance

Recruiting sites
1 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Time to Progression (TTP)
Measured over Up to six years
+2 more outcomes measured

Conditions

Langerhans Cell Histiocytosis

Explore related trials

Langerhans Cell Histiocytosis

NCT06582745

Where you'd take part

This study runs at 1 site. They're the same protocol — you choose where, and that choice sets who your contact draft is addressed to.

  • Cook Children's Health Care System

    Fort Worth, Texasstudy coordinator listed

    Recruiting

Sites open and close at different times, so the status above is per site — it can differ from the study's overall status.

Study leadership

  • Anish Ray, MD · PRINCIPAL_INVESTIGATOR · Cook Children's Health Care System

Who to contact

Megan Gibbs, BSN, RN
[email protected]
(682) 885-2116
Email the study team

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Eligibility criteria

Inclusion

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 and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry, for the duration of study participation, and for 4 months after the last dose. Should a woman become pregnant or suspect she is pregnant while participating in this study, she should inform her treating physician immediately.
Ability to understand study procedures and to comply with them for the entire length of the study.

Exclusion

Patients diagnosed with Low-Risk True Skin Only or a Single Bone lesion that does not require treatment and will only be observed will not be eligible, with the exception of CNS-risk lesions/special site disease or functionally critical lesions:
CNS-risk/special site includes: Sphenoid, Mastoid, Orbital, zygomatic, ethmoid, maxillary, or temporal bones, the cranial fossa, pituitary gland or neurodegenerative disease, odontoid peg, vertebral lesion with intraspinal soft tissue extension
Functionally critical: A single lesion not described above which may cause "functionally critical anatomic abnormality" wherein attempts at local therapy would cause unacceptable morbidity. This can be at the discretion of the Principal Investigator.
Patients whose genetic testing reveals a class 3 MAP2K1 mutation:
I103\_K104del
E102\_I103del
L98\_K104delinsQ
L98\_I103del
I99\_K104del
Patients who present with jaundice at diagnosis.
Patients who are pregnant or breastfeeding are not eligible. Women of childbearing potential must receive a negative pregnancy test within 14 days of starting treatment or the patient will not be eligible.
Patients who are allergic to trametinib
Current drug or alcohol use or dependence that, in the opinion of the site investigator, would interfere with adherence to study requirements.
Inability or unwillingness of patient or parent/legally authorized representative to give written informed consent.

What this trial measures

  • Time to Progression (TTP)Up to six years

    TTP defined for each participant as the period of time from start of treatment to the manifestation of objective progression, but excluding occurrences of death.

  • Progression-free Survival (PFS)Up to six years

    PFS defined for each participant as the time from start of treatment to disease progression (recurrence) or death by any cause in the absence of progression.

  • Overall Survival (OS)Up to six years

    OS defined for each participant as the time from the date of first administration until the date of death from any cause. Participants not having an event at the time of analysis will be censored at the date they were last known to be alive.