Dabrafenib or Trametinib plus Clofarabine for high-risk or relapsed pediatric Langerhans cell histiocytosis

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This is a Phase 2 interventional program using either dabrafenib or trametinib (MAPK pathway inhibitors) combined with clofarabine (a nucleoside analogue) to treat children with high‑risk or relapsed LCH, especially those with liver, spleen, or hematopoietic involvement. Eligible patients are 0–18 years old with CD1a+/CD207+ LCH who have had disease progression or reactivation after prior chemotherapy or targeted therapy and meet organ‑function and performance criteria. Patients will receive the combination therapy with regular monitoring for response, toxicity, and predefined exit criteria such as progression at three months or severe clofarabine toxicity. The work is conducted in a real‑world clinical setting at West China Second University Hospital with consented follow‑up and safety oversight.

Why it matters

Who can join: the full rules

Inclusion Criteria:

1. Children aged 0-18 with LCH (CD1a+/CD207+);
2. Initial LCH diagnosis with hematopoietic, liver, or spleen involvement;
3. LCH patients with disease progression or reactivation after chemotherapy (e.g., prednisone, vincristine, cytarabine, clarithromycin) or targeted therapy;
4. Consent to treatment and follow-up;
5. ECOG score ≥ 2, Lansky score ≥ 50, organ function suitable for chemotherapy.

Exclusion Criteria:

1. Other underlying diseases (e.g., primary immunodeficiency, heart/kidney failure, hepatitis, HIV, organ transplant);
2. Secondary tumor;
3. Recent chemotherapy, radiotherapy, or MAPK inhibitor use with lingering adverse effects;
4. Ongoing nephrotoxic drug use;
5. Refusal to consent.

Exit Criteria:

1. Allergies to dabrafenib or trametinib and clofarabine；
2. Disease progression after 3 months on dabrafenib or trametinib；
3. Severe toxic side effects from clofarabine (grade 4 non-infectious non-hematological toxicity, SIRS, capillary leak syndrome)； 4）The doctor recommends halting the current treatment plan for the patient's benefit.

Where it's running

• Department of Pediatric Hematology and Oncology, West China Second University Hospital, Sichuan University, Chengdu, China
  Chengdu, Sichuan, China Recruiting

Study contacts

• Study coordinator: Xue Tang, MD
• Email: txily0912@126.com
• Phone: 86+18280145819

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.