Phase 2 Study of LUM-201 in Children With Growth Hormone Deficiency (OraGrowtH210 Trial)

This is a multi-national trial. The goals of the trial are to study LUM-201 as a possible treatment for Pediatric Growth Hormone Deficiency (PGHD) and investigate a predictive enrichment marker (PEM) strategy to select subjects likely to respond to therapy with LUM-201.

Inclusion Criteria: * Have an established diagnosis of idiopathic PGHD as determined by standard diagnostic criteria. Eligible subjects must be naïve-to-treatment and be prepubertal. * Morning cortisol ≥ 7 µg/dL or stimulated cortisol ≥ 14 µg/dL. * At Screening, be ≥ 3.0 years and ≤ 11.0 years for girls and ≤ 12.0 years for boys. * Have HT-SDS ≤ -2.0 or HT-SDS ≥ 2 SD below mean parental HT-SDS. * Have a baseline height velocity \< 5.5 cm/year based on at least 6 months of growth. * Have a bone age delayed by ≥ 6 months with respect to chronological age. * Have prepubertal status as evidenced by Tanner Stage I breast development in girls and testicular volume \< 4.0 mL in boys. * 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 30 days prior to Day 1. Exclusion Criteria: * Any medical or genetic condition which, in the opinion of the Investigator or Medical Monitor (MM), can be an independent cause of short stature and/or limit the response to exogenous growth factor treatment. (Examples: diabetes, idiopathic short stature). * A medical or genetic condition that, in the opinion of the Investigator and/or MM, adds unwarranted risk to use of LUM-201 or rhGH. * Use of any medication that, in the opinion of the Investigator and/or MM, can independently cause short stature or limit the response to exogenous growth factors (Example: glucocorticoids). * Evidence or history of an intracranial mass (e.g., pituitary tumor, craniopharyngioma). * Suspicion of absent pituitary function as evidenced by a maximal stimulated GH ≤ 3 ng/mL on two prior standard of care GH stimulation tests, or pituitary deficiencies beyond GH and thyroid function. * Malnutrition as evidenced by medical history or a body weight \< 3rdth percentile for current height. * BMI \> 95th percentile. * Gestational age-adjusted birth weight \< 5th percentile (small for gestational age). * History of spinal, cranial, or total body irradiation. * Treatment with medications known to act as moderate or strong inhibitors or strong inducers of CYP3A/4, or with medications known to act as strong inhibitors of P-glycoprotein (P-gp) or potent substrates of P-gp or Multidrug and toxin extrusion protein 1 (MATE1).