NCT05913037进行中（未招募）3 期
A Randomized, Double-Blind, Placebo-Controlled, Multi-center Phase III Clinical Study to Evaluate the Efficacy and Safety of FCN-159 in Adult Patients With Symptomatic, Inoperable Neurofibromatosis Type 1-Related Plexiform Neurofibromas
Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd.15 个研究点 分布在 1 个国家目标入组 167 人开始时间: 2023年6月20日最近更新:
适应症
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 167
- 试验地点
- 15
- 主要终点
- Objective response rate (ORR) evaluated by BIRC (Response evaluation in Nerufibromatosis and Schwannomatosis, REiNS criteria)
研究概览
简要总结
A study to evaluate the efficacy of FCN-159 in adult patients with symptomatic, inoperable neurofibromatosis type 1-related plexiform neurofibromas.
详细描述
This is a randomized, double-blind, placebo-controlled, multi-center phase III clinical study to evaluate the efficacy and safety of FCN-159 in adult patients with symptomatic, inoperable neurofibromatosis type 1-related plexiform neurofibromas.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 18 Years 至 70 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •≥ 18 years old and ≤ 70 years old.
- •Patients must be diagnosed with symptomatic NF1-related plexiform neurofibromas (PNs) and require systemic therapy at the investigator's discretion.
- •Presence of measurable lesions, defined as ≥ 3 cm in length in at least one dimension, which can be evaluated for efficacy by MRI.
- •Karnofsky performance status score ≥
- •Patients with adequate organ and bone marrow functions.
排除标准
- •NF1-related malignancies requiring chemotherapy, radiotherapy, or surgery, such as medium to high grade optic glioma or malignant peripheral nerve sheath tumor.
- •Patients with a history of or concurrently with other malignancies (excluding cured non-melanoma skin basal cell carcinoma, breast cancer in situ or cervical cancer in situ, and other malignancies without evidence of disease within 5 years).
- •Patients who cannot undergo MRI and/or have contraindications to MRI.
- •Patients with previous or current retinal vein obstruction (RVO), retinal pigment epithelial detachment (RPED), glaucoma, and other abnormal ophthalmic examination with clinical significance.
- •Interstitial pneumonia, including clinically significant radiation pneumonia.
- •Cardiac function or combined diseases meet one of the following conditions:
- •QTcF value of > 470 milliseconds; patients with risk factors for QTcF prolongation or patients receiving drugs that prolong the QTcF interval.
- •Congestive heart failure per New York Heart Association (NYHA) classification ≥ Class
- •Arrhythmias with clinical significance.
- •Known concurrent clinically significant coronary artery disease, cardiomyopathy, and severe valvular disease.
- •LVEF < 50%.
- •Patients with a heart rate of < 50 beats/min.
结局指标
主要结局
Objective response rate (ORR) evaluated by BIRC (Response evaluation in Nerufibromatosis and Schwannomatosis, REiNS criteria)
时间窗: Through study completion, an average of 2 years
ORR is defined as the proportion of patients who have a confirmed complete response or confirmed partial response as determined by ICR per REiNS criteria.
次要结局
- Duration of response (DOR) evaluated by BIRC and the investigator;(Through study completion, an average of 2 years)
- Progression free survival (PFS) evaluated by BIRC and the investigator;(Through study completion, an average of 2 years)
- Change from baseline in pain intensity score(Through study completion, an average of 2 years)
- Time to response (TTR) evaluated by BIRC and the investigator;(Through study completion, an average of 2 years)
- Objective response rate (ORR) evaluated by the investigator (REiNS criteria)(Through study completion, an average of 2 years)
- Clinical benefit rate (CBR)evaluated by BIRC and the investigator;(Through study completion, an average of 2 years)
- Time to progression (TTP) evaluated by BIRC and the investigator;(Through study completion, an average of 2 years)
- Change from baseline in appearance(Through study completion, an average of 2 years)
- Disease control rate (DCR) evaluated by BIRC and the investigator;(Through study completion, an average of 2 years)
研究者
研究点 (15)
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