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

NCT05913037 | Active Not Recruiting Phase 3 DMC

• 1 other identifier: FCN-159-007
• Study Type: interventional
• Target: 167
• Locations: 1 country
• Sites: 15

Brief Summary

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

Conditions

Neurofibromatosis 1; Plexiform Neurofibroma; NF1

Outcome Measures

Primary Outcomes (1)

• 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

Secondary Outcomes (9)

• Objective response rate (ORR) evaluated by the investigator (REiNS criteria)

  Through study completion, an average of 2 years

• Duration of response (DOR) evaluated by BIRC and the investigator;

  Through study completion, an average of 2 years

• Disease control rate (DCR) evaluated by BIRC and the investigator;

  Through study completion, an average of 2 years

• Clinical benefit rate (CBR)evaluated by BIRC and the investigator;

  Through study completion, an average of 2 years

• Progression free survival (PFS) evaluated by BIRC and the investigator;

  Through study completion, an average of 2 years

Study Arms (2)

FCN-159

EXPERIMENTAL

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;

placebo

PLACEBO COMPARATOR

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;

Interventions

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.

FCN-159

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.

placebo

Eligibility Criteria

• Age: 18 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• ≥ 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.

You may not qualify if:

• 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:
• 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.

Sponsors & Collaborators

• Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd.: lead

Study Sites (15)

Cancer Hospital Chinese Academy of Medical Sciences

Beijing, Beijing Municipality, China

Location

Chinese Academy of Medical Sciences & Peking Union Medical College

Beijing, Beijing Municipality, China

Location

Plastic Surgery Hospital,Chinese Academy of Medical Sciences

Beijing, Beijing Municipality, China

Location

Nanfang Hospital, Southern Medical University

Guangzhou, Guangdong, China

Location

Sun Yat-sen University Cancer Center

Guangzhou, Guangdong, China

Location

Peking University Shenzhen Hospital

Shenzhen, Guangdong, China

Location

The Fourth Hospital of Hebei Medical University

Shijiazhuang, Hebei, China

Location

The Second Hospital of Hebei Medical University

Shijiazhuang, Hebei, China

Location

Renmin Hospital of Wuhan University

Wuhan, Hubei, China

Location

TongJi Hospital，TongJi Medical College Huazhong University of Science and Technology

Wuhan, Hubei, China

Location

The First Hospial of China Medical University

Shenyang, Liaoning, China

Location

Fudan University Shanghai Cancer center

Shanghai, Shanghai Municipality, China

Location

West China Hospital，Sichuan University

Chengdu, Sichuan, China

Location

Hangzhou First People's Hospital

Hangzhou, Zhejiang, China

Location

Zhejiang Provincial People'S Hospital

Hanzhou, Zhejiang, China

Location

MeSH Terms

Conditions

Neurofibromatosis 1; Neurofibroma, Plexiform

Interventions

Control Groups

Condition Hierarchy (Ancestors)

Neurofibromatoses; Neurofibroma; 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; Peripheral Nervous System Diseases; Neuromuscular Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Peripheral Nervous System Neoplasms; Nervous System Neoplasms

Intervention Hierarchy (Ancestors)

Epidemiologic Research Design; Epidemiologic Methods; Investigative Techniques; Research Design; Methods

Study Officials

• Wenbin Li, MD

  Beijing Tiantan Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 6, 2023
• First Posted: June 22, 2023
• Study Start: June 20, 2023
• Primary Completion: August 19, 2025
• Study Completion (Estimated): June 30, 2026
• Last Updated: March 6, 2025

Record last verified: 2025-02

Data Sharing

• IPD Sharing: Will not share

Locations

CN (15)