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

Completed · Phase 2

Conditions studied: Growth Hormone Deficiency

In brief

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.

Key facts

Study ID

NCT04614337

Run by

Lumos Pharma

People needed

104

Starts

2020-12-31

Expected to finish

2024-09-04

Last updated by the study team

2026-06-17

Who can join

Age: 3 and older, up to 12. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• 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).

Where it is running

• Center of Excellence in Diabetes and Endocrinology — Sacramento, California, United States
• Rady Children's Hospital — San Diego, California, United States
• Pediatric Endocrine Associates — Greenwood Village, Colorado, United States
• Children's National Hospital — Washington D.C., District of Columbia, United States
• Atlanta Diabetes Associates — Atlanta, Georgia, United States
• Indiana University School of Medicine — Indianapolis, Indiana, United States
• University of Iowa — Iowa City, Iowa, United States
• Novak Center For Childrens Health — Louisville, Kentucky, United States
• UMass Memorial Medical Center — Worcester, Massachusetts, United States
• M Health, Fairview Pediatric Specialty Clinics- Discovery Clinic — Minneapolis, Minnesota, United States
• Children's Minnesota — Saint Paul, Minnesota, United States
• The Children's Mercy Hospital — Kansas City, Missouri, United States
• UBMD Pediatrics — Buffalo, New York, United States
• The Mount Sinai Hospital — Mount Sinai, New York, United States
• NYU Grossman School of Medicine — New York, New York, United States
• Nationwide Children's Hospital — Columbus, Ohio, United States
• University of Oklahoma Health Sciences Center, Pediatric Diabetes and Endocrinology — Oklahoma City, Oklahoma, United States
• Penn State College of Medicine — Hershey, Pennsylvania, United States
• The Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States
• Children's Hospital of Pittsburgh of UPMC — Pittsburgh, Pennsylvania, United States
• Medical University of South Carolina — Charleston, South Carolina, United States
• Texas Tech University Health Sciences Center — Amarillo, Texas, United States
• Cook Children's Medical Center — Fort Worth, Texas, United States
• Diabetes & Glandular Disease Clinic, P.A. — San Antonio, Texas, United States
• University of Virginia Health System — Charlottesville, Virginia, United States

Full record on ClinicalTrials.gov

Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.