[Translation] A randomized, double-blind, placebo-controlled, multicenter phase III clinical study evaluating the efficacy and safety of FCN-159 in adult patients with symptomatic, inoperable neurofibromatosis type 1-associated plexiform neurofibromas sex
主要目的:
根据盲态独立评审委员会(Blinded Independent Review Committee,BIRC)评估的客观缓解率(Objective response rate,ORR),评价FCN 159对比安慰剂治疗存在症状、不能手术的NF1相关的丛状神经纤维瘤成人患者的有效性。
次要目的:
评估FCN-159对比安慰剂治疗存在症状、不能手术的NF1相关的丛状神经纤维瘤成人患者的其他影像学疗效指标、疼痛和外观改善情况。
进一步评估FCN-159治疗存在症状、不能手术的NF1相关的丛状神经纤维瘤成人患者的安全性。
探索性目的:
进一步评估NF1成人患者口服FCN-159的药物代谢动力学(Pharmacokinetics,PK)特征。
评估FCN-159对于存在症状、不能手术的NF1相关的丛状神经纤维瘤成人患者的生活质量的改善情况。
利用患者报告量表进一步评估FCN-159治疗存在症状、不能手术的NF1相关的丛状神经纤维瘤成人患者的长期安全性。
[Translation] main purpose:
To evaluate FCN 159 versus placebo in adult patients with symptomatic, inoperable NF1-related plexiform neurofibroma, according to the objective response rate (ORR) assessed by the Blinded Independent Review Committee (BIRC). effectiveness.
Secondary purpose:
To assess improvement in other radiographic efficacy measures, pain, and appearance in adult patients with symptomatic, inoperable NF1-related plexiform neurofibroma treated with FCN-159 versus placebo.
To further evaluate the safety of FCN-159 in adult patients with symptomatic, inoperable NF1-related plexiform neurofibroma.
Exploratory purpose:
To further evaluate the pharmacokinetics (PK) characteristics of oral FCN-159 in adult patients with NF1.
To assess the improvement in quality of life of FCN-159 in adult patients with symptomatic, inoperable NF1-related plexiform neurofibroma.
The long-term safety of FCN-159 in adult patients with symptomatic, inoperable NF1-related plexiform neurofibroma was further assessed using a patient-report scale.