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LUM-201 treatment for children with growth hormone deficiency

A Multicenter, 12-Month, Randomized, Double Blind, Placebo-Controlled Phase 3 Efficacy and Safety Study of Daily Oral LUM-201 in Naïve-to-Treatment, Prepubertal Children With Growth Hormone Deficiency (GHD)

Phase 3 Interventional Lumos Pharma · NCT06948214

This trial tests whether daily oral LUM-201 can help prepubertal, treatment‑naïve children with growth hormone deficiency grow taller.

Quick facts

PhasePhase 3
Study typeInterventional
Enrollment150 (estimated)
Ages3 Years to 11 Years
SexAll
SponsorLumos Pharma Industry-sponsored
Locations29 sites (Birmingham, Alabama and 28 other locations)
Trial IDNCT06948214 on ClinicalTrials.gov

What this trial studies

This Phase 3, randomized trial gives eligible prepubertal, treatment‑naïve children with documented growth hormone deficiency daily oral LUM-201 or a matched placebo. Enrollment uses a LUM-201 predictive enrichment marker (PEM) strategy to select children more likely to respond to the oral secretagogue. Key measurements include height velocity, IGF-1 levels, bone age, and safety monitoring over the treatment period. The trial aims both to measure growth benefit and to validate the PEM as a way to identify responders.

Who should consider this trial

Good fit: Ideal candidates are prepubertal, treatment‑naïve children aged ≥3 years and ≤10 years for girls or ≤11 years for boys with documented GH peak <10 ng/mL on two stimulation tests, height ≤ -2.0 SDS, delayed bone age, low IGF‑1 (SDS ≤ -1.0), and baseline low height velocity.

Not a fit: Children who are already receiving growth hormone therapy, are pubertal, have GH stimulation peaks ≥10 ng/mL, have normal IGF‑1 levels, or have other medical causes of short stature (e.g., Turner syndrome) are unlikely to benefit from this protocol.

Why it matters

Potential benefit: If successful, LUM-201 could offer an effective daily oral alternative to injections for some children with growth hormone deficiency, improving growth and treatment convenience.

How similar studies have performed: Early‑phase studies of LUM-201 and other GH secretagogues showed signals of increased IGF‑1 and growth in pediatric populations, but large Phase 3 confirmation is required.

Eligibility criteria

Show full inclusion / exclusion 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

Where this trial is running

Birmingham, Alabama and 28 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Growth Hormone DeficiencyGHDPediatric Growth Hormone DeficiencyLUM-201Growth hormone secretagogueHeightCatch-up growthPEM
Last reviewed 2026-06-10 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.