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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

NCT06948214

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