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

NCT ID: NCT05913037

Last Updated: 2025-03-06

Basic Information

• Recruitment Status: ACTIVE_NOT_RECRUITING
• Clinical Phase: PHASE3
• Total Enrollment: 167 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2023-06-20
• Study Completion Date: 2026-06-30

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.

Conditions

Neurofibromatosis 1; Plexiform Neurofibroma; NF1

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

FCN-159

Experimental: FCN-159 Dosage form:tablet Specification: 1mg，4mg Dose: FCN-159 8 mg, orally, once daily Method of administration: Oral

Group Type: EXPERIMENTAL

Test group (Group A): FCN-159 8 mg, orally, once daily;

Intervention Type: 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

Experimental: placebo Dosage form:tablet Specification: 1mg，4mg Dose: placebo 8 mg, orally, once daily Method of administration: Oral

Group Type: PLACEBO_COMPARATOR

Control group (Group B): Placebo, orally, once daily;

Intervention Type: 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.

Interventions

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.

Intervention Type: 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.

Intervention Type: DRUG

Eligibility 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.

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 70 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Wenbin Li, MD

Role: PRINCIPAL_INVESTIGATOR

Beijing Tiantan Hospital

Locations

Cancer Hospital Chinese Academy of Medical Sciences

Beijing, Beijing Municipality, China

Chinese Academy of Medical Sciences & Peking Union Medical College

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

Countries

China

Other Identifiers

FCN-159-007

Identifier Type: -

Identifier Source: org_study_id