Trial Title:
FCN-159 in Adult Patients With Symptomatic, Inoperable Neurofibromatosis Type 1-Related Plexiform Neurofibromas
NCT ID:
NCT05913037
Condition:
Neurofibromatosis 1
Plexiform Neurofibroma
NF1
Conditions: Official terms:
Neurofibromatoses
Neurofibromatosis 1
Neurofibroma
Neurofibroma, Plexiform
Study type:
Interventional
Study phase:
Phase 3
Overall status:
Recruiting
Study design:
Allocation:
Randomized
Intervention model:
Parallel Assignment
Primary purpose:
Treatment
Masking:
Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Intervention:
Intervention type:
Drug
Intervention name:
Test group (Group A): FCN-159 8 mg, orally, once daily;
Description:
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.
Arm group label:
FCN-159
Intervention type:
Drug
Intervention name:
Control group (Group B): Placebo, orally, once daily;
Description:
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.
Arm group label:
placebo
Summary:
A study to evaluate the efficacy of FCN-159 in adult patients with symptomatic,
inoperable neurofibromatosis type 1-related plexiform neurofibromas.
Detailed description:
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.
Criteria for eligibility:
Criteria:
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.
Gender:
All
Minimum age:
18 Years
Maximum age:
70 Years
Healthy volunteers:
No
Locations:
Facility:
Name:
Cancer Hospital Chinese Academy of Medical Sciences
Address:
City:
Beijing
Country:
China
Status:
Recruiting
Contact:
Last name:
Li Ning, MD
Phone:
18614049602
Email:
lining@cicams.ac.cn
Investigator:
Last name:
Li Ning, MD
Email:
Principal Investigator
Facility:
Name:
Chinese Academy of Medical Sciences & Peking Union Medical College
Address:
City:
Beijing
Country:
China
Status:
Recruiting
Contact:
Last name:
Ma Wenbin, MD
Phone:
13701364566
Email:
Mawb2001@hotmail.com
Investigator:
Last name:
Ma Wenbin, MD
Email:
Principal Investigator
Facility:
Name:
Plastic Surgery Hospital,Chinese Academy of Medical Sciences
Address:
City:
Beijing
Country:
China
Status:
Recruiting
Contact:
Last name:
Yan Yan, MD
Phone:
13661348184
Email:
yanyan201606@163.com
Investigator:
Last name:
Yan Yan, MD
Email:
Principal Investigator
Facility:
Name:
Nanfang Hospital, Southern Medical University
Address:
City:
Guangzhou
Country:
China
Status:
Recruiting
Contact:
Last name:
Kang Zeng, MD
Phone:
13178850988
Email:
npfkzk@163.com
Investigator:
Last name:
Kang Zeng, MD
Email:
Principal Investigator
Facility:
Name:
Sun Yat-sen University Cancer Center
Address:
City:
Guangzhou
Country:
China
Status:
Recruiting
Contact:
Last name:
Chen Zhongping, MD
Phone:
13500002457
Email:
chenzhp@sysucc.org.cn
Investigator:
Last name:
Chen Zhongping, MD
Email:
Principal Investigator
Facility:
Name:
Peking University Shenzhen Hospital
Address:
City:
Shenzhen
Country:
China
Status:
Recruiting
Contact:
Last name:
Chen Baodong, MD
Phone:
13602593425
Email:
623564186@qq.com
Investigator:
Last name:
Chen Baodong, MD
Email:
Principal Investigator
Facility:
Name:
The Fourth Hospital of Hebei Medical University
Address:
City:
Shijiazhuang
Country:
China
Status:
Recruiting
Contact:
Last name:
Zhao Zongmao, MD
Phone:
13930466038
Email:
zzm69@163.com
Investigator:
Last name:
Zhang Yan, MD
Email:
Principal Investigator
Investigator:
Last name:
Zhao Zongmao, MD
Email:
Principal Investigator
Facility:
Name:
The Second Hospital of Hebei Medical University
Address:
City:
Shijiazhuang
Country:
China
Status:
Recruiting
Contact:
Last name:
Li Yanlin, MD
Phone:
15130119920
Email:
lyldoctor@sina.com
Investigator:
Last name:
Li Yanlin, MD
Email:
Principal Investigator
Facility:
Name:
Renmin Hospital of Wuhan University
Address:
City:
Wuhan
Country:
China
Status:
Recruiting
Contact:
Last name:
Lei Tiechi, MD
Phone:
15337173507
Email:
tiechilei@126.com
Investigator:
Last name:
Lei Tiechi, MD
Email:
Principal Investigator
Facility:
Name:
TongJi Hospital，TongJi Medical College Huazhong University of Science and Technology
Address:
City:
Wuhan
Country:
China
Status:
Recruiting
Contact:
Last name:
Shu Kai, MD
Phone:
13720373328
Email:
kshu@tjh.tjmu.edu.cn
Investigator:
Last name:
Shu Kai, MD
Email:
Principal Investigator
Facility:
Name:
The First Hospial of China Medical University
Address:
City:
Shenyang
Country:
China
Status:
Recruiting
Contact:
Last name:
Gao Xinghua, MD
Phone:
13940152467
Email:
gaobarry@hotmail.com
Investigator:
Last name:
Gao Xinghua, MD
Email:
Principal Investigator
Facility:
Name:
Fudan University Shanghai Cancer center
Address:
City:
Shanghai
Country:
China
Status:
Recruiting
Contact:
Last name:
Huang Wending, MD
Phone:
18616182390
Email:
orienthwd@163.com
Investigator:
Last name:
Huang Wending, MD
Email:
Principal Investigator
Facility:
Name:
West China Hospital，Sichuan University
Address:
City:
Chengdu
Country:
China
Status:
Recruiting
Contact:
Last name:
Liu Yanhui, MD
Phone:
18980601509
Email:
yhliu2001@gmail.com
Investigator:
Last name:
Liu Yanhui, MD
Email:
Principal Investigator
Facility:
Name:
Hangzhou First People's Hospital
Address:
City:
Hangzhou
Country:
China
Status:
Recruiting
Contact:
Last name:
Wu Liming, MD
Phone:
13750837205
Email:
18957118053@163.com
Investigator:
Last name:
Wu Liming, MD
Email:
Principal Investigator
Facility:
Name:
Zhejiang Provincial People'S Hospital
Address:
City:
Hanzhou
Country:
China
Status:
Recruiting
Contact:
Last name:
Wu Sufan, MD
Phone:
13857121888
Email:
sufanwu@163.com
Investigator:
Last name:
Wu Sufan, MD
Email:
Principal Investigator
Start date:
June 20, 2023
Completion date:
June 30, 2026
Lead sponsor:
Agency:
Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd.
Agency class:
Industry
Source:
Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd.
Record processing date:
ClinicalTrials.gov processed this data on November 12, 2024
Source: ClinicalTrials.gov page:
https://clinicaltrials.gov/ct2/show/NCT05913037
