RECRUITING Phase 3 ClinicalTrials.gov

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

Sponsor: Lumos Pharma • ClinicalTrials.gov ID: NCT06948214

Registry updated: 2026-10-19

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Description

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.

Interventions

LUM-201 (Drug)
1.6 mg/kg/day, administered orally once daily
Matched Placebo (Capsules) (Other)
Administered orally once daily

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

Conditions

Growth Hormone Deficiency (GHD)

Locations

Lumos Pharma Investigational Site — Birmingham, Alabama, United States
Lumos Pharma Investigational Site — Madera, California, United States
Lumos Pharma Investigational Site — Orange, California, United States
Lumos Pharma Investigational Site — Sacramento, California, United States
Lumos Pharma Investigational Site — San Diego, California, United States
Lumos Pharma Investigational Site — Centennial, Colorado, United States
Lumos Pharma Investigational Site — Greenwood Village, Colorado, United States
Lumos Pharma Investigational Site — Washington D.C., District of Columbia, United States
Lumos Pharma Investigational Site — Hollywood, Florida, United States
Lumos Pharma Investigational Site — Jacksonville, Florida, United States
Lumos Pharma Investigational Site — Miami, Florida, United States
Lumos Pharma Investigational Site — Orlando, Florida, United States
Lumos Pharma Investigational Site — Tallahassee, Florida, United States
Lumos Pharma Investigational Site — Savannah, Georgia, United States
Lumos Pharma Investigational Site — Indianapolis, Indiana, United States
Lumos Pharma Investigational Site — Iowa City, Iowa, United States
Lumos Pharma Investigational Site — New Orleans, Louisiana, United States
Lumos Pharma Investigational Site — Minneapolis, Minnesota, United States
Lumos Pharma Investigational Site — Kansas City, Missouri, United States
Lumos Pharma Investigational Site — Las Vegas, Nevada, United States
Lumos Pharma Investigational Site — New Brunswick, New Jersey, United States
Lumos Pharma Investigational Site — Staten Island, New York, United States
Lumos Pharma Investigational Site — Columbus, Ohio, United States
Lumos Pharma Investigational Site — Oklahoma City, Oklahoma, United States
Lumos Pharma Investigational Site — Columbia, South Carolina, United States
Lumos Pharma Investigational Site — Amarillo, Texas, United States
Lumos Pharma Investigational Site — Corpus Christi, Texas, United States
Lumos Pharma Investigational Site — Fort Worth, Texas, United States
Lumos Pharma Investigational Site — San Antonio, Texas, United States
Lumos Pharma Investigational Site — Charlottesville, Virginia, United States
Lumos Pharma Investigational Site — Seattle, Washington, United States
Lumos Pharma Investigational Site — San Miguel de Tucumán, Tucumán Province, Argentina
Children's Health Queensland Hospital and Health Service — South Brisbane, Queensland, Australia
Lumos Pharma Investigational Site — Clayton, Victoria, Australia
Lumos Pharma Investigational Site — Parkville, Victoria, Australia
Lumos Pharma Investigational Site — Santiago, Santiago Metropolitan, Chile
Lumos Pharma Investigational Site — Mumbai, Maharashtra, India
Lumos Pharma Investigational Site — Nashik, Maharashtra, India
Lumos Pharma Investigational Site — Jaipur, Rajasthan, India
Lumos Pharma Investigational Site — Madurai, Tamil Nadu, India
Lumos Pharma Investigational Site — Chandigarh, India
University of Auckland (Liggins Institute, Maurice and Nessie Paykel Clinical Research Unit) — Auckland, New Zealand
Wellington Regional Hospital — Wellington, New Zealand
Lumos Pharma Investigational Site — Bangkok, Bangkok, Thailand
Lumos Pharma Investigational Site — Bangkok Noi, Bangkok, Thailand
Lumos Pharma Investigational Site — Pathum Wan, Bangkok, Thailand
Lumos Pharma Investigational Site — Muang, Changwat Khon Kaen, Thailand
Lumos Pharma Investigational Site — Hat Yai, Changwat Songkhla, Thailand
Lumos Pharma Investigational Site — Muang, Chiang Mai, Thailand
Lumos Pharma Investigational Site — Liverpool, England, United Kingdom
Lumos Pharma Investigational Site — London, England, United Kingdom
Lumos Pharma Investigational Site — London, England, United Kingdom
Lumos Pharma Investigational Site — London, England, United Kingdom
Lumos Pharma Investigational Site — Manchester, England, United Kingdom
Lumos Pharma Investigational Site — Sheffield, England, United Kingdom

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Official Source

Data sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

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