A Long-term Follow-up Study to Evaluate the Safety and Efficacy of Ruvometinib Tablets (FCN-159 Tablets) in Pediatric Participants With Neurofibromatosis Type 1
试验速览
- 阶段
- 1 期
- 状态
- 尚未招募
- 发起方
- 入组人数
- 65
- 试验地点
- 1
- 主要终点
- long term safty
研究概览
简要总结
FCN-159 (Luvometinib Tablets), an orally available and highly potent selective inhibitor of MEK1/2,demonstrated good tolerability and exhibited notable anti-tumor activity in pediatric pts with NF1-related PN in study NCT04954001.This study is a 5-year long-term follow-up of the FCN-159-002 study, involving all enrolled patients to further assess safety, growth and development effects, and treatment efficacy.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 2 Years 至 20 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients from the pediatric cohort of the FCN-159-002 study. Willing to participate in the long-term follow-up study and capable of understanding and voluntarily signing the informed consent form.
排除标准
- •Any clinically significant condition that, in the investigator's judgment, may interfere with study participation or compliance with safety requirements.
- •Patients unable to comply with visit-related requirements.
研究组 & 干预措施
FCN-159
Study Drug: FCN-159 Dosing Regimen: FCN-159 to be administered once daily until disease progression or study completion, whichever occurs first
干预措施: Luvometinib Tablets (Drug)
结局指标
主要结局
long term safty
时间窗: Through study completion, about 5 years
Adverse events during treatment of FCN-159 will be assessed by the investigators according to CTCAE 5.0.
次要结局
- Height in centimeters(Through study completion, about 5 years)
- Weight in kilograms(Through study completion, about 5 years)
- Tanner stage(Through study completion, about 5 years)
- Progression-Free Survival (PFS)(Through study completion, about 5 years)
- Objective response rate (ORR)(Through study completion, about 5 years)
- Changes in pain intensity(Through study completion, about 5 years)