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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
65
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
Shanghai Ninth People's Hospital
Shanghai, Shanghai Municipality, China
long term safty
Adverse events during treatment of FCN-159 will be assessed by the investigators according to CTCAE 5.0.
Time frame: Through study completion, about 5 years
Height in centimeters
Height will be measured in centimeters, with subsequent plotting of height growth curves and calculation of growth velocity.
Time frame: Through study completion, about 5 years
Weight in kilograms
Weight will be measured in kilograms, with subsequent plotting of weight growth curves.
Time frame: Through study completion, about 5 years
Tanner stage
Tanner stage will be examined, and the mean age for each Tanner stage will be calculated.
Time frame: Through study completion, about 5 years
Progression-Free Survival (PFS)
PFS assessed per REiNS (Response Evaluation in Neurofibromatosis and Schwannomatosis) criteria by inverstigator
Time frame: Through study completion, about 5 years
Objective response rate (ORR)
ORR assessed per REiNS (Response Evaluation in Neurofibromatosis and Schwannomatosis) criteria by inverstigator.
Time frame: Through study completion, about 5 years
Changes in pain intensity
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".
Time frame: Through study completion, about 5 years
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