Neurofibromatosis 1, NF1, Plexiform Neurofibroma
Conditions
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.
Interventions
DRUGLuvometinib 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
Sponsors
Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
2 Years to 20 Years
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| long term safty | 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
| Measure | Time frame | Description |
|---|---|---|
| Weight in kilograms | Through study completion, about 5 years | Weight will be measured in kilograms, with subsequent plotting of weight growth curves. |
| Tanner stage | Through study completion, about 5 years | Tanner stage will be examined, and the mean age for each Tanner stage will be calculated. |
| Height in centimeters | Through study completion, about 5 years | Height will be measured in centimeters, with subsequent plotting of height growth curves and calculation of growth velocity. |
| Objective response rate (ORR) | Through study completion, about 5 years | ORR assessed per REiNS (Response Evaluation in Neurofibromatosis and Schwannomatosis) criteria by inverstigator. |
| Changes in pain intensity | 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. |
| Progression-Free Survival (PFS) | Through study completion, about 5 years | PFS assessed per REiNS (Response Evaluation in Neurofibromatosis and Schwannomatosis) criteria by inverstigator |
Countries
China
Contacts
Primary ContactXiaojie Hu
Backup ContactXiaoxi Lin
Outcome results
None listed