NCT05997602

To Evaluate the Efficacy, Safety, and PK Characteristics of FCN-159 in Pediatric Patients With Refractory/Recurrent LCH

A Multi-center, Open-label, Single Arm Phase 2 Study to Evaluate the Efficacy, Safety, and Pharmacokinetic Characteristics of FCN-159 in Pediatric Patients With Refractory/Recurrent Langerhans Cell Histiocytosis

• Status: Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 56 (estimated)
• Sponsor: Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd. · Industry
• Sex: All
• Age: 2 Years – 16 Years
• Healthy volunteers: Not accepted

Summary

This is a rare disease, single-arm, open-label,multi-center, non-randomized Phase 2 clinical study to evaluate the efficacy, safety, and pharmacokinetic characteristics of FCN-159 monotherapy in pediatric patients with refractory/recurrent Langerhans cell histiocytosis (LCH).

Detailed description

This is a rare disease, single-arm, open-label,multi-center, non-randomized Phase 2 clinical study to evaluate the efficacy, safety, and pharmacokinetic characteristics of FCN-159 monotherapy in pediatric patients with refractory/recurrent Langerhans cell histiocytosis (LCH). Approximately 56 pediatric patients will be enrolled in this study.The study included screening period, treatment period and follow-up period. Subjects will receive FCN-159 5mg/m² (NMT 8mg, the recommended oral dose for adults), orally, once daily, continuously for 28 days per cycle. Subjects will be treated until disease progression (PD), intolerable toxicity, withdrawal of consent, death, or other protocol-specified reasons. According to the PET Response Criteria and Langerhans Cell Histiocytosis Evaluation and Treatment Guidelines, tumor assessment will be performed until disease progression, death, and withdrawal of information Intention, loss of follow-up, initiation of new antitumor therapy, or study termination.Efficacy evaluation will be performed by the investigator and the Independent Review Committee (IRC), respectively.

Conditions

• Langerhans Cell Histiocytosis
• LCH

Interventions

Type	Name	Description
DRUG	FCN-159	5mg/m² (Maximum dose does not exceed 8mg, the recommended oral dose for adults), orally, once daily, continuously for 28 days per cycle.

Timeline

• Start date: 2023-09-28
• Primary completion: 2025-05-14
• Completion: 2027-05-14
• First posted: 2023-08-18
• Last updated: 2025-01-24

Locations

11 sites across 1 country: China