Real-world Study of Darafenib or Trametinib and Clofarabine for High-risk/Recurrent/Refractory Langerhans Cell Histiocytosis in Children

Launched by WEST CHINA SECOND UNIVERSITY HOSPITAL · Jun 7, 2025

Keywords

ClinConnect Summary

This clinical trial is studying the effectiveness and safety of two medications, dabrafenib or trametinib combined with clofarabine, for children with a rare condition called Langerhans Cell Histiocytosis (LCH). LCH can be serious, especially when it affects important organs like the liver or spleen, and some children may not respond to standard treatments. The trial is open to children aged 0-18 who have LCH that has not improved after previous treatments or who have a high-risk form of the disease. Participants will receive these medications and will be monitored closely to see how well they work and if there are any side effects.

If you decide to participate, you can expect regular check-ups and assessments to track your child's progress and health. The goal of this trial is to find better treatment options for high-risk LCH, especially for those who have not responded well to other therapies. It’s important to know that while the medications are being tested for safety and effectiveness, there may be some risks involved, and the doctors will make sure to provide the best care throughout the study.

Gender

ALL

Eligibility criteria

• 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.