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NCT Number: NCT07024394

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

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.

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Key information

Conditions

Neurofibromatosis 1 Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Heredodegenerative Disorders, Nervous System NF1 Neoplasms Neoplasms by Histologic Type Neoplasms, Nerve Tissue Neoplastic Syndromes, Hereditary Nerve Sheath Neoplasms Nervous System Diseases Nervous System Neoplasms Neurocutaneous Syndromes Neurodegenerative Diseases Neurofibroma Neurofibroma, Plexiform Neurofibromatoses Neuromuscular Diseases Peripheral Nervous System Diseases Peripheral Nervous System Neoplasms Plexiform Neurofibroma

Age range

2 year–20 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1 / Phase 2

Primary location

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

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

Other names: FCN-159

Primary outcomes

  1. long term safty

    Time frame: Through study completion, about 5 years

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

Secondary outcomes

  1. Height in centimeters

    Time frame: Through study completion, about 5 years

    Height will be measured in centimeters, with subsequent plotting of height growth curves and calculation of growth velocity.

  2. Weight in kilograms

    Time frame: Through study completion, about 5 years

    Weight will be measured in kilograms, with subsequent plotting of weight growth curves.

  3. Tanner stage

    Time frame: Through study completion, about 5 years

    Tanner stage will be examined, and the mean age for each Tanner stage will be calculated.

  4. Progression-Free Survival (PFS)

    Time frame: Through study completion, about 5 years

    PFS assessed per REiNS (Response Evaluation in Neurofibromatosis and Schwannomatosis) criteria by inverstigator

  5. Objective response rate (ORR)

    Time frame: Through study completion, about 5 years

    ORR assessed per REiNS (Response Evaluation in Neurofibromatosis and Schwannomatosis) criteria by inverstigator.

  6. Changes in pain intensity

    Time frame: Through study completion, about 5 years

    Changes in pain intensity score relative to baseline will be assessed according to 11-point Numerical Rating Scale (NRS-11).NRS-11 quantifies pain intensity on a scale from 0 to 10, 0 represents "No pain" ,10 represents "The worst pain imaginable".

Study contacts

Contact information is provided by the study sponsor or research team.

Xiaojie Hu

CONTACT

[email protected]

+86 13701997136

Xiaoxi Lin

CONTACT

[email protected]

+86 18017681268

Sponsors and collaborators

Lead sponsor

Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd.

Industry

Registry information

Official study 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

Important dates

Study start
2025
Primary completion
2029
Study completion
2029
First posted
Jun 17, 2025
Registry last updated
Jun 17, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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