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

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ClinicalTrials.gov Identifier: NCT05913037
Recruitment Status : Recruiting
First Posted : June 22, 2023
Last Update Posted : March 15, 2024
Sponsor:
Information provided by (Responsible Party):
Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd.

Study Description
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Brief Summary:
A study to evaluate the efficacy of FCN-159 in adult patients with symptomatic, inoperable neurofibromatosis type 1-related plexiform neurofibromas.

Condition or disease Intervention/treatment Phase
Neurofibromatosis 1 Plexiform Neurofibroma NF1 Drug: Test group (Group A): FCN-159 8 mg, orally, once daily; Drug: Control group (Group B): Placebo, orally, once daily; Phase 3

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.
Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 162 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: 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
Actual Study Start Date : June 20, 2023
Estimated Primary Completion Date : June 30, 2025
Estimated Study Completion Date : June 30, 2026

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Neurofibromatosis

Arms and Interventions
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Arm Intervention/treatment
Experimental: FCN-159
Experimental: FCN-159 Dosage form:tablet Specification: 1mg，4mg Dose: FCN-159 8 mg, orally, once daily Method of administration: Oral
 Drug: 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.
Placebo Comparator: placebo
Experimental: placebo Dosage form:tablet Specification: 1mg，4mg Dose: placebo 8 mg, orally, once daily Method of administration: Oral
 Drug: 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.


Outcome Measures
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Primary Outcome Measures :
  1. Objective response rate (ORR) evaluated by BIRC (Response evaluation in Nerufibromatosis and Schwannomatosis, REiNS criteria) [ Time Frame: 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 :
  1. Objective response rate (ORR) evaluated by the investigator (REiNS criteria) [ Time Frame: 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.

  2. Duration of response (DOR) evaluated by BIRC and the investigator; [ Time Frame: 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.

  3. Disease control rate (DCR) evaluated by BIRC and the investigator; [ Time Frame: 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.

  4. Clinical benefit rate (CBR)evaluated by BIRC and the investigator; [ Time Frame: 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.

  5. Progression free survival (PFS) evaluated by BIRC and the investigator; [ Time Frame: 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.

  6. Time to progression (TTP) evaluated by BIRC and the investigator; [ Time Frame: 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.

  7. Time to response (TTR) evaluated by BIRC and the investigator; [ Time Frame: 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.

  8. Change from baseline in pain intensity score [ Time Frame: 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.

  9. Change from baseline in appearance [ Time Frame: 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.


Eligibility Criteria
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Ages Eligible for Study:   18 Years to 70 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05913037


Contacts
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Contact: Wenbin Li, MD 15301377998 Neure55@126.com

Locations
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China, Beijing
Cancer Hospital Chinese Academy of Medical Sciences Recruiting
Beijing, Beijing, China
Contact: Li Ning, MD    18614049602    lining@cicams.ac.cn   
Principal Investigator: Li Ning, MD         
Chinese Academy of Medical Sciences & Peking Union Medical College Recruiting
Beijing, Beijing, China
Contact: Ma Wenbin, MD    13701364566    Mawb2001@hotmail.com   
Principal Investigator: Ma Wenbin, MD         
Plastic Surgery Hospital,Chinese Academy of Medical Sciences Recruiting
Beijing, Beijing, China
Contact: Yan Yan, MD    13661348184    yanyan201606@163.com   
Principal Investigator: Yan Yan, MD         
China, Guangdong
Nanfang Hospital, Southern Medical University Recruiting
Guangzhou, Guangdong, China
Contact: Kang Zeng, MD    13178850988    npfkzk@163.com   
Principal Investigator: Kang Zeng, MD         
Sun Yat-sen University Cancer Center Recruiting
Guangzhou, Guangdong, China
Contact: Chen Zhongping, MD    13500002457    chenzhp@sysucc.org.cn   
Principal Investigator: Chen Zhongping, MD         
Peking University Shenzhen Hospital Recruiting
Shenzhen, Guangdong, China
Contact: Chen Baodong, MD    13602593425    623564186@qq.com   
Principal Investigator: Chen Baodong, MD         
China, Hebei
The Fourth Hospital of Hebei Medical University Recruiting
Shijiazhuang, Hebei, China
Contact: Zhao Zongmao, MD    13930466038    zzm69@163.com   
Principal Investigator: Zhang Yan, MD         
Principal Investigator: Zhao Zongmao, MD         
The Second Hospital of Hebei Medical University Recruiting
Shijiazhuang, Hebei, China
Contact: Li Yanlin, MD    15130119920    lyldoctor@sina.com   
Principal Investigator: Li Yanlin, MD         
China, Hubei
Renmin Hospital of Wuhan University Recruiting
Wuhan, Hubei, China
Contact: Lei Tiechi, MD    15337173507    tiechilei@126.com   
Principal Investigator: Lei Tiechi, MD         
TongJi Hospital，TongJi Medical College Huazhong University of Science and Technology Recruiting
Wuhan, Hubei, China
Contact: Shu Kai, MD    13720373328    kshu@tjh.tjmu.edu.cn   
Principal Investigator: Shu Kai, MD         
China, Liaoning
The First Hospial of China Medical University Recruiting
Shenyang, Liaoning, China
Contact: Gao Xinghua, MD    13940152467    gaobarry@hotmail.com   
Principal Investigator: Gao Xinghua, MD         
China, Shanghai
Fudan University Shanghai Cancer center Recruiting
Shanghai, Shanghai, China
Contact: Huang Wending, MD    18616182390    orienthwd@163.com   
Principal Investigator: Huang Wending, MD         
China, Sichuan
West China Hospital，Sichuan University Recruiting
Chengdu, Sichuan, China
Contact: Liu Yanhui, MD    18980601509    yhliu2001@gmail.com   
Principal Investigator: Liu Yanhui, MD         
China, Zhejiang
Hangzhou First People's Hospital Recruiting
Hangzhou, Zhejiang, China
Contact: Wu Liming, MD    13750837205    18957118053@163.com   
Principal Investigator: Wu Liming, MD         
Zhejiang Provincial People'S Hospital Recruiting
Hanzhou, Zhejiang, China
Contact: Wu Sufan, MD    13857121888    sufanwu@163.com   
Principal Investigator: Wu Sufan, MD         
Sponsors and Collaborators
Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd.
Investigators
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Principal Investigator: Wenbin Li, MD Beijing Tiantan Hospital
More Information
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Responsible Party: Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd.
ClinicalTrials.gov Identifier: NCT05913037    
Other Study ID Numbers: FCN-159-007
First Posted: June 22, 2023    Key Record Dates
Last Update Posted: March 15, 2024
Last Verified: March 2024

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Neurofibromatoses
Neurofibromatosis 1
Neurofibroma
Neurofibroma, Plexiform
Nerve Sheath Neoplasms
Neoplasms, Nerve Tissue
Neoplasms by Histologic Type
Neoplasms
Neoplastic Syndromes, Hereditary
 Neurocutaneous Syndromes
Nervous System Diseases
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Peripheral Nervous System Diseases
Neuromuscular Diseases
Peripheral Nervous System Neoplasms
Nervous System Neoplasms