NCT06948214|RecruitingPhase 3DMCFDA DrugFDA Device

Phase 3 Study of LUM-201 in 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)

Lumos Pharma ClinicalTrials.gov

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1 other identifier

LUM-201-10

Study Type

interventional

Target

150

Locations

4 countries

Sites

Timeline

RegisteredApr 2025

StartedApr 2026

CompletionJan 2028

Brief Summary

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.

Trial Health

On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment

150

participants targeted

Target at P25-P50 for phase_3

Timeline

20mo left

Started Apr 2026

Geographic Reach

4 countries

28 active sites

Status

recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

1.8 years study duration

Study Progress6%

Apr 2026Jan 2028

First Submitted

Initial submission to the registry

April 21, 2025

Completed

8 days until next milestone

First Posted

Study publicly available on registry

April 29, 2025

Completed

11 months until next milestone

Study Start

First participant enrolled

April 1, 2026

Completed

1.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 1, 2027

Expected

1 month until next milestone

Study Completion

Last participant's last visit for all outcomes

January 1, 2028

Last Updated

May 4, 2026

Status Verified

September 1, 2025

Enrollment Period

1.7 years

First QC Date

April 21, 2025

Last Update Submit

May 1, 2026

Conditions

Growth Hormone Deficiency (GHD)

Keywords

GHDPediatric Growth Hormone DeficiencyLUM-201Growth hormone secretagogueHeightCatch-up growthPEMOralPredictive Enrichment Markeributamoren mesylateOraGrowtH Phase 3 TrialLUM-201 PEMGH secretagogue

Outcome Measures

Primary Outcomes (1)

AHV after 12 months on LUM-201 compared to placebo
Annualized height velocity (AHV) measured as standing height with stadiometer
Day 1 to Month 12

Other Outcomes (1)

Percentage of subjects selected by PEM strategy who meet target growth
Day 1 to Month 12

Study Arms (2)

LUM-201

EXPERIMENTAL

Drug: LUM-201

Placebo

PLACEBO COMPARATOR

Other: Matched Placebo (Capsules)

Interventions

LUM-201DRUG

1.6 mg/kg/day, administered orally once daily

LUM-201

Matched Placebo (Capsules)OTHER

Administered orally once daily

Placebo

Eligibility Criteria

Age3 Years - 11 Years

Sexall

Healthy VolunteersNo

Age GroupsChild (0-17)

You may qualify if:

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

You may not qualify if:

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
+2 more criteria

Contact the study team to confirm eligibility.