A study to evaluate the efficacy of FCN-159 in adult patients with symptomatic, inoperable neurofibromatosis type 1-related plexiform neurofibromas.
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.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
167
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.
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.
Cancer Hospital Chinese Academy of Medical Sciences
Beijing, Beijing Municipality, China
Chinese Academy of Medical Sciences & Peking Union Medical College
Beijing, Beijing Municipality, China
Objective response rate (ORR) evaluated by BIRC (Response evaluation in Nerufibromatosis and Schwannomatosis, REiNS criteria)
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.
Time frame: Through study completion, an average of 2 years
Objective response rate (ORR) evaluated by the investigator (REiNS criteria)
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.
Time frame: Through study completion, an average of 2 years
Duration of response (DOR) evaluated by BIRC and the investigator;
DOR is defined as the time from the date of first documented response (which is subsequently confirmed) until progression by BIRC and the investigator per REiNS criteria or death due to any cause.
Time frame: Through study completion, an average of 2 years
Disease control rate (DCR) evaluated by BIRC and the investigator;
DCR is defined as the proportion of patients who have a confirmed complete response or confirmed partial response or stable disease as determined by BIRC and the investigator per REiNS criteria.
Time frame: Through study completion, an average of 2 years
Clinical benefit rate (CBR)evaluated by BIRC and the investigator;
CBR is defined as the proportion of patients who have a confirmed complete response or confirmed partial response or stable disease\>48 weeks as determined by BIRC and the investigator per REiNS criteria.
Time frame: Through study completion, an average of 2 years
Progression free survival (PFS) evaluated by BIRC and the investigator;
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Plastic Surgery Hospital,Chinese Academy of Medical Sciences
Beijing, Beijing Municipality, China
Nanfang Hospital, Southern Medical University
Guangzhou, Guangdong, China
Sun Yat-sen University Cancer Center
Guangzhou, Guangdong, China
Peking University Shenzhen Hospital
Shenzhen, Guangdong, China
The Fourth Hospital of Hebei Medical University
Shijiazhuang, Hebei, China
The Second Hospital of Hebei Medical University
Shijiazhuang, Hebei, China
Renmin Hospital of Wuhan University
Wuhan, Hubei, China
TongJi Hospital，TongJi Medical College Huazhong University of Science and Technology
Wuhan, Hubei, China
...and 5 more locations
PFS is defined as the time from randomization until date of disease progression by BIRC and investigator per REiNS criteria or death due to any cause.
Time frame: Through study completion, an average of 2 years
Time to progression (TTP) evaluated by BIRC and the investigator;
TTP is defined as the time from randomization until date of disease progression by BIRC and investigator per REiNS criteria.
Time frame: Through study completion, an average of 2 years
Time to response (TTR) evaluated by BIRC and the investigator;
TTR is defined as the time from date of randomization until the date of objective response by BIRC and investigator per REiNS criteria.
Time frame: Through study completion, an average of 2 years
Change from baseline in pain intensity score
Difference in mean change from baseline in overall tumor and target PN pain intensity score between Arm A and Arm B as assessed by the 11-point Numerical Rating Scale (NRS-11),which uses the range 0-10,higher scores mean worse outcome.
Time frame: Through study completion, an average of 2 years
Change from baseline in appearance
Change in appearance from baseline for Arm A versus Arm B as assessed using a sponsor-customized 'appearance evaluation'PRO questionnaire, which is descriptive.
Time frame: Through study completion, an average of 2 years