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Clinical Trials in China / NCT05913037
Active, not recruiting Phase 3

FCN-159 in Adult Patients With Symptomatic, Inoperable Neurofibromatosis Type 1-Related Plexiform Neurofibromas

NCT05913037 · tracked via the Priya Life Science China tracker
Sponsor
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)

Trial sites (15)

FacilityCityRegionStatus
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

Other trials for the same condition

Official registry record

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.

View NCT05913037 on ClinicalTrials.gov ↗ ← All trials in China