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

Langerhans Cell Histiocytosis Clinical Trial

Official title: 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

Clinical Trial 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).

Clinical Trial 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. ;

Related Conditions & MeSH terms

• Histiocytosis
• Histiocytosis, Langerhans-Cell
• Langerhans Cell Histiocytosis

• NCT number: NCT05997602
• Study type: Interventional
• Source: Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd.
• Contact: Rui Zhang, MD
• Phone: 18611106187
• Email: ruizh1973@126.com
• Status: Recruiting
• Phase: Phase 2
• Start date: September 28, 2023
• Completion date: July 14, 2026