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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| FCN-159 | Experimental | Experimental: FCN-159 Dosage form:tablet Specification: 1mg，4mg Dose: FCN-159 8 mg, orally, once daily Method of administration: Oral |
|
| placebo | Placebo Comparator | Experimental: placebo Dosage form:tablet Specification: 1mg，4mg Dose: placebo 8 mg, orally, once daily Method of administration: Oral |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Test group (Group A): FCN-159 8 mg, orally, once daily; | Drug | 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. |
| Measure | Description | Time Frame |
|---|---|---|
| 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. | Through study completion, an average of 2 years |
| Measure | Description | Time Frame |
|---|---|---|
| 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. | Through study completion, an average of 2 years |
| Duration of response (DOR) evaluated by BIRC and the investigator; |
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Inclusion Criteria:
Exclusion Criteria:
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:
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| Name | Affiliation | Role |
|---|---|---|
| Wenbin Li, MD | Beijing Tiantan Hospital | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Cancer Hospital Chinese Academy of Medical Sciences | Beijing | Beijing Municipality | China | |||
| Chinese Academy of Medical Sciences & Peking Union Medical College |
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|
| Control group (Group B): Placebo, orally, once daily; | Drug | 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. |
|
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. |
| 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. | 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. | Through study completion, an average of 2 years |
| Progression free survival (PFS) evaluated by BIRC and the investigator; | 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. | 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. | 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. | 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. | 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. | Through study completion, an average of 2 years |
| Beijing |
| Beijing Municipality |
| China |
| 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 |
| The First Hospial of China Medical University | Shenyang | Liaoning | China |
| Fudan University Shanghai Cancer center | Shanghai | Shanghai Municipality | China |
| West China Hospital，Sichuan University | Chengdu | Sichuan | China |
| Hangzhou First People's Hospital | Hangzhou | Zhejiang | China |
| Zhejiang Provincial People'S Hospital | Hanzhou | Zhejiang | China |
| ID | Term |
|---|---|
| D009456 | Neurofibromatosis 1 |
| D018318 | Neurofibroma, Plexiform |
| ID | Term |
|---|---|
| D017253 | Neurofibromatoses |
| D009455 | Neurofibroma |
| D018317 | Nerve Sheath Neoplasms |
| D009380 | Neoplasms, Nerve Tissue |
| D009370 | Neoplasms by Histologic Type |
| D009369 | Neoplasms |
| D009386 | Neoplastic Syndromes, Hereditary |
| D020752 | Neurocutaneous Syndromes |
| D009422 | Nervous System Diseases |
| D020271 | Heredodegenerative Disorders, Nervous System |
| D019636 | Neurodegenerative Diseases |
| D010523 | Peripheral Nervous System Diseases |
| D009468 | Neuromuscular Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D010524 | Peripheral Nervous System Neoplasms |
| D009423 | Nervous System Neoplasms |
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| ID | Term |
|---|---|
| D035061 | Control Groups |
| ID | Term |
|---|---|
| D015340 | Epidemiologic Research Design |
| D004812 | Epidemiologic Methods |
| D008919 | Investigative Techniques |
| D012107 | Research Design |
| D008722 | Methods |
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