A Multicenter, 12-Month, Randomized, Double Blind, Placebo-Controlled Phase 3 Efficacy and Safety Study of Daily Oral LUM-201 in Naïve-to-Treatment, Prepubertal Children With Growth Hormone Deficiency (GHD)
This is a multi-national trial. The goals of the trial are to study LUM-201 as a treatment for Pediatric Growth Hormone Deficiency (PGHD) in naive to treatment children and validate a predictive enrichment marker (PEM) strategy to select subjects likely to respond to therapy with LUM-201.
• Subjects must be naïve to treatment and prepubertal
• Subjects must have a maximal GH response of \< 10 ng/mL from 2 prior GH stimulation tests conducted within the preceding 12 months
• Impaired height defined as ≥ 2.0 standard deviations (SDs) below the mean height for chronological age and sex
• Morning or random cortisol level of ≥ 7.0 μg/dL
• ≥ 3.0 years and age ≤ 10.0 years for girls and ≤ 11.0 years for boys
• Baseline height velocity (HV) based on ≥ 6 months of growth assessments \< 25th percentile for age and sex
• Bone Age delay of ≥ 12 months compared to the chronological age
• In girls, have genetic testing results to rule out Turner syndrome. If SHOX genetic testing results are available, they need to be negative.
• Have normal thyroid function. Subjects diagnosed with hypothyroidism must have documented successful treatment for at least 3 months prior to Day 1
• Baseline IGF-1 standard deviation score (SDS) ≤ -1.0