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Not yet recruiting NCT07024394

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

Phase I / Phase II Interventional Neurofibromatosis 1 Plexiform Neurofibroma NF1

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: Luvometinib Tablets.
Who it may be relevant to
Registry conditions: Neurofibromatosis 1, Plexiform Neurofibroma, NF1. Basic parameters: 2 years — 20 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
China
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

A Long-term Follow-up Study to Evaluate the Safety and Efficacy of Ruvometinib Tablets (FCN-159 Tablets) in Pediatric Participants With Neurofibromatosis Type 1

Overview

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.

Interventions

  • Drug Luvometinib Tablets
    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

Primary outcome measures

  • long term safty [Time frame: Through study completion, about 5 years]
Secondary outcome measures (6)
  • Height in centimeters [Time frame: Through study completion, about 5 years]
  • Weight in kilograms [Time frame: Through study completion, about 5 years]
  • Tanner stage [Time frame: Through study completion, about 5 years]
  • Progression-Free Survival (PFS) [Time frame: Through study completion, about 5 years]
  • Objective response rate (ORR) [Time frame: Through study completion, about 5 years]
  • Changes in pain intensity [Time frame: Through study completion, about 5 years]

Eligibility criteria

Inclusion criteria

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.

Exclusion criteria

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.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
N/A
Model
Single group
Masking
Open label
Primary purpose
Treatment

Study locations

China · 1 center
  • Shanghai Ninth People's Hospital — Shanghai

Identifiers

NCT: NCT07024394 · FCN-159-002-LT

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗