What is LUM-201 studying for growth hormone deficiency in children?

LUM-201 is being studied as a daily oral treatment for children with growth hormone deficiency who have not received prior therapy. This Phase 3 trial evaluates how LUM-201 affects growth compared to a placebo. Lumos Pharma is leading the research to better understand which children respond to this treatment.

Who may be eligible for this growth hormone deficiency clinical trial?

Children aged 3 to 11 years who have not been treated before and are prepubertal may be eligible. They must have confirmed growth hormone deficiency and impaired height growth. Both boys and girls can participate. Please review eligibility criteria for more details.

Is there any cost to participate in this growth hormone deficiency clinical trial?

Study-related care, including the study drug or procedure and all trial visits, is provided at no cost. However, personal travel and other expenses related to participating may vary and are not guaranteed to be covered.

Actively Recruiting

Phase 3

Age: 3Years - 11Years

All Genders

ID06948214

Phase 3 Study of Daily Oral LUM-201 for Treating Growth Hormone Deficiency in Prepubertal Children Aged 3 to 11 Years

Led by Lumos Pharma · Updated on 2026-08-06

150

Participants Needed

Research Sites

4 weeks

Total Duration

AI-Summary

What this Trial Is About

Researchers are evaluating LUM-201 as a treatment for Pediatric Growth Hormone Deficiency PGHD in children who have not previously received treatment. This Phase 3 trial aims to validate the LUM-201 predictive enrichment marker strategy to identify children most likely to benefit from daily oral LUM-201 therapy. The study focuses on prepubertal children with impaired growth caused by growth hormone deficiency. Participants will be randomly assigned to receive either LUM-201 or a matching placebo by mouth once daily. LUM-201 is given at a dose of 1.6 mgkgday. This randomized, double-blind, placebo-controlled study lasts for 12 months. The trial compares annualized height velocity AHV after 12 months of treatment between the two groups. During the study, children will undergo regular assessments of growth and hormone levels to monitor response to treatment. Researchers will measure height velocity over 12 months to evaluate the effect of LUM-201 compared to placebo. The trial includes safety monitoring and aims to complete participant involvement by January 2028.

CONDITIONS

Brief 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 GH response below 10 ng/mL from 2 prior stimulation tests within 12 months
Height at least 2 standard deviations below average for age and sex
Morning or random cortisol level of at least 7.0 mcg/dL
Age between 3 years and 10 years for girls, and up to 11 years for boys
Baseline height velocity below the 25th percentile for age and sex based on 6 months of growth data
Bone age delayed by at least 12 months compared to chronological age
Girls must have genetic testing ruling out Turner syndrome and negative SHOX test if available
Normal thyroid function or successful treatment of hypothyroidism for at least 3 months
Baseline IGF-1 standard deviation score less than or equal to -1.0

You will not qualify if you...

Any medical or genetic condition causing short stature or limiting response to treatment
Arm span to height ratio more than 2 standard deviations below average
Conditions adding risk to LUM-201 use as judged by Investigator or Medical Monitor
Use of medications causing short stature or limiting growth response
Recent inflammatory diseases requiring corticosteroids for over 2 weeks within last 3 months
Use of hormone replacement therapy except for thyroid deficiency
Clinically significant ECG abnormalities at screening
Past or present intracranial tumor confirmed by imaging
Intracranial hypertension confirmed by fundoscopy or other tests
Elevated liver enzymes or bilirubin above normal limits
Suspicion of absent pituitary function with GH ≤ 3.0 ng/mL on prior tests
Body weight 14.0 kg or less
BMI outside -2 to +2 standard deviations for age and sex
Birth weight below 3rd percentile for gestational age
Use of strong CYP3A/4 inhibitors or inducers
History of spinal, cranial, or total body irradiation
Diagnosis of attention deficit hyperactivity disorder (ADHD)

Research Team

Lumos Pharma

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Phase 3 Study of Daily Oral LUM-201 for Treating Growth Hormone Deficiency in Prepubertal Children Aged 3 to 11 Years

On this page

AI-Summary

What this Trial Is About

Brief Title

Who Can Participate

Eligibility Criteria

Research Team

Not the Right Trial for You?

On this page

Who Can Participate

Eligibility Criteria