Follow-up Study to Evaluate the Safety and Efficacy of FCN-159 in Pediatric Participants With Neurofibromatosis Type 1

NCT07024394 | Not Yet Recruiting Phase 1 FDA Drug

• 1 other identifier: FCN-159-002-LT
• Study Type: interventional
• Target: 65
• Locations: 1 country
• Sites: 1

Brief Summary

FCN-159 (Luvometinib Tablets), an orally available and highly potent selective inhibitor of MEK1/2,demonstrated good tolerability and exhibited notable anti-tumor activity in pediatric pts with NF1-related PN in study NCT04954001.This study is a 5-year long-term follow-up of the FCN-159-002 study, involving all enrolled patients to further assess safety, growth and development effects, and treatment efficacy.

Conditions

Neurofibromatosis 1; Plexiform Neurofibroma; NF1

Outcome Measures

Primary Outcomes (1)

• long term safty

  Adverse events during treatment of FCN-159 will be assessed by the investigators according to CTCAE 5.0.

  Through study completion, about 5 years

Secondary Outcomes (6)

• Height in centimeters

  Through study completion, about 5 years

• Weight in kilograms

  Through study completion, about 5 years

• Tanner stage

  Through study completion, about 5 years

• Progression-Free Survival (PFS)

  Through study completion, about 5 years

• Objective response rate (ORR)

  Through study completion, about 5 years

Study Arms (1)

FCN-159

EXPERIMENTAL

Study Drug: FCN-159 Dosing Regimen: FCN-159 to be administered once daily until disease progression or study completion, whichever occurs first

Drug: Luvometinib Tablets

Interventions

Luvometinib Tablets DRUG

5mg/m² (Maximum dose does not exceed 8mg, the recommended oral dose for adults), orally, once daily, until disease progression or study completion, whichever occurs first

Also known as: FCN-159

FCN-159

Eligibility Criteria

• Age: 2 Years - 20 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Patients from the pediatric cohort of the FCN-159-002 study. Willing to participate in the long-term follow-up study and capable of understanding and voluntarily signing the informed consent form.

You may not qualify if:

• Any clinically significant condition that, in the investigator's judgment, may interfere with study participation or compliance with safety requirements.
• Patients unable to comply with visit-related requirements.

Sponsors & Collaborators

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

Study Sites (1)

Shanghai Ninth People's Hospital

Shanghai, Shanghai Municipality, 200011, China

Location

MeSH Terms

Conditions

Neurofibromatosis 1; Neurofibroma, Plexiform

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

Central Study Contacts

Xiaojie Hu

CONTACT

+86 13701997136; hxjie2002@hotmail.com

Xiaoxi Lin

CONTACT

+86 18017681268; linxiaoxi@126.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 3, 2025
• First Posted: June 17, 2025
• Study Start: June 12, 2025
• Primary Completion (Estimated): September 23, 2029
• Study Completion (Estimated): September 23, 2029
• Last Updated: June 17, 2025

Record last verified: 2025-06

Locations

CN (1)