This is a Multi-national Trial. The Goal of the Trial is to Offer Subjects Who Complete 12 Months in the LUM-201-10 Phase 3 Trial up to an Additional 36 Months of Treatment of LUM-201 While Evaluating Safety and Tolerability of LUM-201.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
150
1.6 mg/kg/day, administered orally once daily
To evaluate the long-term safety and tolerability of LUM-201
* The number of subjects with at least 1 (serious) treatment-related adverse event (TEAE). * The number of subjects with at least 1 suspected unexpected serious adverse reaction. * The number of subjects with clinically significant abnormalities related to trial laboratory tests or electrocardiograms (ECGs). * The number of subjects with at least 1 adverse event of special interest (AESI). * Annual change from baseline in body weight standard deviation score (SDS). * Annual change from baseline in body mass index (BMI) SDS.
Time frame: Day 1 - Month 36
To evaluate the long-term effect of LUM-201 on growth.
* Annual height velocity. * Annual change in height standard deviation score (SDS). * Annual change in body mass index (BMI). * Annual change in BMI SDS. * Annual change in bone age (BA)/chronological age (CA) ratio.
Time frame: Day 1 - Month 36
To evaluate the long-term effect of LUM-201 on pharmacodynamic (PD) markers.
* Annual change in insulin-like growth factor (IGF)-1 SDS. * Annual change in insulin-like growth binding protein 3 (IGFBP-3) SDS.
Time frame: Day 1 - Month 36
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