Phase 3 Study of LUM-201 in Children With Growth Hormone Deficiency

Registered clinical trial record on ClinicalTrials.gov (NCT06948214). This describes a planned, ongoing, or completed study — it is NOT peer-reviewed results. Status: recruiting. Study type: interventional. Phase: Phase 3. Sponsor: Lumos Pharma. Conditions: Growth Hormone Deficiency (GHD). Interventions: LUM-201, Matched Placebo (Capsules). Summary: The OraGrowtH Phase 3 Trial 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 the LUM-201 predictive enrichment marker (LUM-201 PEM) strategy to select subjects likely to respond to therapy with daily oral LUM-201. Primary outcome measures: AHV after 12 months on LUM-201 compared to placebo. Eligibility: Inclusion Criteria: * 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 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. * Arm span to height ratio \> 2 SDs below the mean for age and sex * A medical or genetic condition that, in the opinion of the Investigator and/or MM, adds unwarranted risk to use of LUM-201 * 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 * Current inflammatory diseases requiring systemic corticosteroid treatment for \> 2 consecutive weeks within the last 3 months prior to the Screening Visit * Use of hormone replacement therapy for any hormone deficiency other than thyroid deficiency * Any ECG at the Screening Visit noted to have a clinically significant abnormality, as confirmed by the MM * Any subjects suspected of having past or present intracranial tumor growth as confirmed by brain imaging prior to the Screening or Day 1 Visit * Any subject suspected of having intracranial hypertension (IH) as confirmed by fundoscopy and other assessments * Any subject with serum alanine transaminase (ALT), aspartate transaminase (AST), or total bilirubin \> upper limit of normal (ULN) * Suspicion of absent pituitary function as evidenced by a maximal stimulated GH ≤ 3.0 ng/mL on any prior standard of care GH stimulation test completed within 12 months * Body weight ≤ 14.0 kg * BMI \< -2 or \> +2 SDs for age and sex based on WHO standards * Birth weight for gestational age \< 3rd percentile based on WHO standards * Treatment with medications known to be moderate or strong inhibitors or strong inducers of cytochrome P450 (CYP) 3A/4 * History of spinal, cranial, or total body irradiation * Attention deficit hyperactivity disorder (ADHD) diagnosis