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

Actively Recruiting

Phase 3

Age: 3Years - 11Years

All Genders

NCT06948214

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

Led by Lumos Pharma · Updated on 2026-05-12

150

Participants Needed

Research Sites

91 weeks

Total Duration

AI-Summary

What this Trial Is About

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.

CONDITIONS

Official Title

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

Who Can Participate

Age: 3Years - 11Years

All Genders

Eligibility Criteria

You may qualify if you...

Subjects must be naive to treatment and prepubertal
Maximal growth hormone response of less than 10 ng/mL from 2 prior GH stimulation tests done within the last 12 months
Height at least 2.0 standard deviations below the mean for age and sex
Morning or random cortisol level of at least 7.0 micrograms per deciliter
Age 3.0 years or older and up to 10.0 years for girls, up to 11.0 years for boys
Baseline height velocity below the 25th percentile for age and sex based on at least 6 months of growth data
Bone age delay of at least 12 months compared to chronological age
Girls must have genetic testing ruling out Turner syndrome and negative SHOX gene testing if available
Normal thyroid function or documented successful treatment for hypothyroidism for at least 3 months before Day 1
Baseline IGF-1 standard deviation score of -1.0 or lower

You will not qualify if you...

Any medical or genetic condition that can cause short stature or limit response to growth hormone treatment, as judged by investigators
Arm span to height ratio more than 2 standard deviations below the mean for age and sex
Medical or genetic condition posing extra risk for LUM-201 use, as judged by investigators
Use of medications that may cause short stature or limit growth hormone response
Current inflammatory diseases needing systemic corticosteroids for more than 2 weeks within the last 3 months
Use of hormone replacement therapy for hormone deficiencies other than thyroid
Clinically significant abnormal ECG at screening
Past or present intracranial tumor confirmed by brain imaging
Suspected intracranial hypertension confirmed by fundoscopy or other tests
Elevated liver enzymes or bilirubin above normal limits
Suspected absent pituitary function with maximal stimulated GH 3.0 ng/mL or less on prior tests
Body weight 14.0 kg or less
Body mass index below -2 or above +2 standard deviations for age and sex
Birth weight below 3rd percentile for gestational age
Treatment with medications that strongly affect cytochrome P450 3A/4 enzymes
History of spinal, cranial, or total body irradiation
Diagnosis of attention deficit hyperactivity disorder (ADHD)

AI-Screening