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Phase 3 Study of LUM-201 in Children With Growth Hormone Deficiency
Phase 3 Study of LUM-201 in Children With Growth Hormone Deficiency
RecruitingPhase 3
Sponsor: Lumos Pharma
Conditions: Growth Hormone Deficiency (GHD)
Interventions: LUM-201, Matched Placebo (Capsules)
Countries: United States, Australia, Chile, New Zealand, Thailand, United Kingdom
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.
Eligibility overview
Sex: ALL
Age: 3 Years to 11 Years
Healthy volunteers: No
Study type: INTERVENTIONAL
Eligibility criteria
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
Locations (43)
- Birmingham, Alabama, United States
- Madera, California, United States
- Orange, California, United States
- Sacramento, California, United States
- San Diego, California, United States
- Centennial, Colorado, United States
- Greenwood Village, Colorado, United States
- Washington D.C., District of Columbia, United States
- Hollywood, Florida, United States
- Miami, Florida, United States
- Orlando, Florida, United States
- Tallahassee, Florida, United States
- Indianapolis, Indiana, United States
- Iowa City, Iowa, United States
- New Orleans, Louisiana, United States
- Minneapolis, Minnesota, United States
- Kansas City, Missouri, United States
- Las Vegas, Nevada, United States
- New Brunswick, New Jersey, United States
- Staten Island, New York, United States
- Oklahoma City, Oklahoma, United States
- Columbia, South Carolina, United States
- Amarillo, Texas, United States
- Corpus Christi, Texas, United States
- Fort Worth, Texas, United States
- San Antonio, Texas, United States
- Charlottesville, Virginia, United States
- Seattle, Washington, United States
- South Brisbane, Queensland, Australia
- Clayton, Victoria, Australia
- + 13 more on CT.gov