FCN-159 in Adult Patients with Symptomatic, Inoperable Neurofibromatosis Type 1-Related Plexiform Neurofibromas
- Conditions
- Neurofibromatosis 1Plexiform NeurofibromaNF1
- Registration Number
- NCT05913037
- Lead Sponsor
- Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd.
- Brief 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.
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- All
- Target Recruitment
- 167
- ≥ 18 years old and ≤ 70 years old.
- Patients must be diagnosed with symptomatic NF1-related plexiform neurofibromas (PNs) and require systemic therapy at the investigator's discretion.
- Presence of measurable lesions, defined as ≥ 3 cm in length in at least one dimension, which can be evaluated for efficacy by MRI.
- Karnofsky performance status score ≥ 70.
- Patients with adequate organ and bone marrow functions.
-
NF1-related malignancies requiring chemotherapy, radiotherapy, or surgery, such as medium to high grade optic glioma or malignant peripheral nerve sheath tumor.
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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).
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Patients who cannot undergo MRI and/or have contraindications to MRI.
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Patients with previous or current retinal vein obstruction (RVO), retinal pigment epithelial detachment (RPED), glaucoma, and other abnormal ophthalmic examination with clinical significance.
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Interstitial pneumonia, including clinically significant radiation pneumonia.
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Cardiac function or combined diseases meet one of the following conditions:
- QTcF value of > 470 milliseconds; patients with risk factors for QTcF prolongation or patients receiving drugs that prolong the QTcF interval.
- Congestive heart failure per New York Heart Association (NYHA) classification ≥ Class 3.
- Arrhythmias with clinical significance.
- Known concurrent clinically significant coronary artery disease, cardiomyopathy, and severe valvular disease.
- LVEF < 50%.
- Patients with a heart rate of < 50 beats/min.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Primary Outcome Measures
Name Time Method 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.
- Secondary Outcome Measures
Name Time Method Duration of response (DOR) evaluated by BIRC and the investigator; Through study completion, an average of 2 years 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.
Progression free survival (PFS) evaluated by BIRC and the investigator; Through study completion, an average of 2 years 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.
Change from baseline in pain intensity score Through study completion, an average of 2 years 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 to response (TTR) evaluated by BIRC and the investigator; Through study completion, an average of 2 years TTR is defined as the time from date of randomization until the date of objective response by BIRC and investigator per REiNS criteria.
Objective response rate (ORR) evaluated by the investigator (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.
Clinical benefit rate (CBR)evaluated by BIRC and the investigator; Through study completion, an average of 2 years 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 to progression (TTP) evaluated by BIRC and the investigator; Through study completion, an average of 2 years TTP is defined as the time from randomization until date of disease progression by BIRC and investigator per REiNS criteria.
Change from baseline in appearance Through study completion, an average of 2 years Change in appearance from baseline for Arm A versus Arm B as assessed using a sponsor-customized 'appearance evaluation'PRO questionnaire, which is descriptive.
Disease control rate (DCR) evaluated by BIRC and the investigator; Through study completion, an average of 2 years 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.
Trial Locations
- Locations (15)
Cancer Hospital Chinese Academy of Medical Sciences
🇨🇳Beijing, Beijing, China
Chinese Academy of Medical Sciences & Peking Union Medical College
🇨🇳Beijing, Beijing, China
Plastic Surgery Hospital,Chinese Academy of Medical Sciences
🇨🇳Beijing, Beijing, 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
Scroll for more (5 remaining)Cancer Hospital Chinese Academy of Medical Sciences🇨🇳Beijing, Beijing, China