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Trial · Interventional

RecruitingPhase 3Registry data

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

Overview

Status
Recruiting
Phase
Phase 3
Enrollment
150 participants (estimated)
Sponsor
Lumos Pharma · Industry
Design
Randomized · Triple masking
Countries
8 countries

United States, Argentina, Australia, Chile, India, New Zealand, Thailand, United Kingdom

Start
20 May 2026
Primary completion
Dec 2027 (estimated)
Completion
Jan 2028 (estimated)
First posted
29 Apr 2025
Last update posted
9 Oct 2026
Conditions
Growth Hormone Deficiency (GHD)

Timeline

1 trial · 1 ongoing

  • Completed
  • Ongoing
  • Not yet recruiting
  • Stopped
  • Estimated dates (est.)
  • Results posted
2026202720282029
  1. Ph 3

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

    NCT06948214

    20 May 2026 to est. Jan 2028

Registry summary

Text and fields below are the sponsor's entries at ClinicalTrials.gov, shown as posted.

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.

Interventions

  • LUM-201Drug
  • Matched Placebo (Capsules)Other

Arms2

  • Experimental

    LUM-201

  • Placebo comparator

    Placebo

Primary outcomes1

Outcomes the sponsor listed as primary.

  1. AHV after 12 months on LUM-201 compared to placebo

    Time frame · Day 1 to Month 12

    Registry description

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

Posted results

Values are shown as posted at ClinicalTrials.gov, with their units. PepBank has not converted, pooled or compared them.

No results are posted for this trial.

A trial that is still running, or that stopped early, may post results later.

Registry data comes from ClinicalTrials.gov. Study results come from studies, not from the registry. How PepBank works

Studies0

Studies that cite this trial.

No study cites this trial.

Eligibility

Who could join, as posted.

Sex
All
Age
from 3 Years up to 11 Years
Healthy volunteers
Not accepted
Inclusion and exclusion criteria, as posted
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

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