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Phase 3 Study of LUM-201 for Growth Hormone Deficiency

This study is testing an investigational oral medication called LUM-201 for children with Growth Hormone Deficiency (GHD). GHD is a condition where the body doesn't make enough growth hormone, leading to slower growth. We are comparing LUM-201 to a placebo (an inactive capsule) to see if LUM-201 helps children grow more over 12 months. Children aged 3 to 11 years who have not received prior GHD treatment and meet specific height and hormone levels may be eligible. The study also aims to identify children most likely to respond to LUM-201. The current recruitment status is unclear.

Study design
This is a multi-national Phase 3 study planning to enroll 150 participants. Participants will receive either LUM-201 or a matched placebo daily.
What's involved
Not specified in the trial record.
Compensation
Not stated in the trial record.
Follow-up
The primary endpoint is measured after 12 months on LUM-201 or placebo.

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NCT06948214

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

Recruiting
PHASE3Ages 3–11InterventionalTreatment
Lumos Pharma
~150 participants
Updated 2026-08-21 on ClinicalTrials.gov
What's tested:LUM-201Matched Placebo (Capsules)

At a glance

Recruiting sites
47 of 47 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
AHV after 12 months on LUM-201 compared to placebo
Measured over Day 1 to Month 12

Conditions

Growth Hormone Deficiency (GHD)

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Where it's being run

47 sites across 30 states
Florida5
California4
Texas4
England4
Colorado2
Victoria2
New Zealand2
Bangkok2

Who to contact

Lumos Pharma
[email protected]
515-598-2921
Email the study team

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Do you actually qualify for this trial?

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Eligibility criteria

Inclusion

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

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

What this trial measures

  • AHV after 12 months on LUM-201 compared to placeboDay 1 to Month 12

    Annualized height velocity (AHV) measured as standing height with stadiometer