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

Launched by SHANGHAI FOSUN PHARMACEUTICAL INDUSTRIAL DEVELOPMENT CO. LTD. · Jun 9, 2025

Keywords

ClinConnect Summary

This clinical trial is a long-term follow-up study looking at the safety and effectiveness of a medicine called FCN-159 (also known as Luvometinib) for children with a condition called Neurofibromatosis Type 1 (NF1). NF1 can cause tumors called plexiform neurofibromas to grow on nerves, which can be painful or cause other problems. FCN-159 is a pill that was shown to be well-tolerated and helped shrink these tumors in an earlier study. This new study will follow patients for up to five years to see how well the medicine works over time and to check on their growth and overall health.

Children who took part in the earlier FCN-159 study may be eligible to join this follow-up if they and their families agree to continue and can commit to regular visits. The study is open to all genders, and the main requirement is being part of the original pediatric group. Participants can expect regular check-ups to monitor their health and how the medicine is working. It’s important to note that this study is not yet recruiting, and children with other serious health issues that might affect their ability to participate may not be eligible. This long-term study aims to better understand the ongoing safety and benefits of FCN-159 for kids living with NF1.

Gender

ALL

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.