Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd.
Phase
Phase 3
Started
2023-06-20
Last updated
2025-03-06
Condition(s) studied
Neurofibromatosis 1Plexiform NeurofibromaNF1
Investigational drug(s) / intervention(s)
Test group (Group A): FCN-159 8 mg, orally, once daily;Control group (Group B): Placebo, orally, once daily;
Test group (Group A): FCN-159 8 mg, orally, once daily;: After completing all screening visit items, qualified patients will be randomly assigned to the test group (Group A) or control group (Group B) in a 2:1 ratio, and receive FCN-159 or placebo within 3 days after randomization.
Control group (Group B): Placebo, orally, once daily;: After completing all screening visit items, qualified patients will be randomly assigned to the test group (Group A) or control group (Group B) in a 2:1 ratio, and receive FCN-159 or placebo within 3 days after randomization.
Study summary
A study to evaluate the efficacy of FCN-159 in adult patients with symptomatic, inoperable neurofibromatosis type 1-related plexiform neurofibromas.
Eligibility
Sex
ALL
Min age
18 Years
Max age
70 Years
Healthy volunteers
No
Inclusion Criteria:
1. ≥ 18 years old and ≤ 70 years old.
2. Patients must be diagnosed with symptomatic NF1-related plexiform neurofibromas (PNs) and require systemic therapy at the investigator's discretion.
3. Presence of measurable lesions, defined as ≥ 3 cm in length in at least one dimension, which can be evaluated for efficacy by MRI.
4. Karnofsky performance status score ≥ 70.
5. Patients with adequate organ and bone marrow functions.
Exclusion Criteria:
1. NF1-related malignancies requiring chemotherapy, radiotherapy, or surgery, such as medium to high grade optic glioma or malignant peripheral nerve sheath tumor.
2. 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).
3. Patients who cannot undergo MRI and/or have contraindications to MRI.
4. Patients with previous or current retinal vein obstruction (RVO), retinal pigment epithelial detachment (RPED), glaucoma, and other abnormal ophthalmic examination with clinical significance.
5. Interstitial pneumonia, including clinically significant radiation pneumonia.
6. Cardiac function or combined diseases meet one of the following conditions:
1. QTcF value of \> 470 milliseconds; patients with risk factors for QTcF prolongation or patients receiving drugs that prolong the QTcF interval.
2. Congestive heart failure per New York Heart Association (NYHA) classification ≥ Class 3.
3. Arrhythmias with clinical significance.
4. Known concurrent clinically significant coronary artery disease, cardiomyopathy, and severe valvular disease.
5. LVEF \< 50%.
6. Patients with a heart rate of \< 50 beats/min.
Primary outcome measure(s)
Objective response rate (ORR) evaluated by BIRC (Response evaluation in Nerufibromatosis and Schwannomatosis, REiNS criteria) — 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.
Trial sites (15)
Facility
City
Region
Status
Cancer Hospital Chinese Academy of Medical Sciences
Beijing
Beijing Municipality
Chinese Academy of Medical Sciences & Peking Union Medical College
Beijing
Beijing Municipality
Plastic Surgery Hospital,Chinese Academy of Medical Sciences
Beijing
Beijing Municipality
Nanfang Hospital, Southern Medical University
Guangzhou
Guangdong
Sun Yat-sen University Cancer Center
Guangzhou
Guangdong
Peking University Shenzhen Hospital
Shenzhen
Guangdong
The Fourth Hospital of Hebei Medical University
Shijiazhuang
Hebei
The Second Hospital of Hebei Medical University
Shijiazhuang
Hebei
Renmin Hospital of Wuhan University
Wuhan
Hubei
TongJi Hospital,TongJi Medical College Huazhong University of Science and Technology
Wuhan
Hubei
The First Hospial of China Medical University
Shenyang
Liaoning
Fudan University Shanghai Cancer center
Shanghai
Shanghai Municipality
West China Hospital,Sichuan University
Chengdu
Sichuan
Hangzhou First People's Hospital
Hangzhou
Zhejiang
Zhejiang Provincial People'S Hospital
Hanzhou
Zhejiang
More Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd. trials in China
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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