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

June 5, 2026 updated by: Lumos Pharma

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)

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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

150

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • Australia
    • Victoria
      • Parkville, Victoria, Australia, 3052
        • Recruiting
        • Lumos Pharma Investigational Site
  • New Zealand
      • Auckland, New Zealand, 1142
        • Recruiting
        • Lumos Pharma Investigational Site
      • Wellington, New Zealand, 6242
        • Recruiting
        • Lumos Pharma Investigational Site
  • United Kingdom
    • England
      • Liverpool, England, United Kingdom, L12 2AP
        • Recruiting
        • Lumos Pharma Investigational Site
      • London, England, United Kingdom, W1G9AB
        • Recruiting
        • Lumos Pharma Investigational Site
      • London, England, United Kingdom, E1 1BB
        • Recruiting
        • Lumos Pharma Investigational Site
  • United States
    • Alabama
      • Birmingham, Alabama, United States, 35233
        • Recruiting
        • Lumos Pharma Investigational Site
    • California
      • Madera, California, United States, 93636
        • Recruiting
        • Lumos Pharma Investigational Site
      • Sacramento, California, United States, 95821
        • Recruiting
        • Lumos Pharma Investigational Site
      • San Diego, California, United States, 92123
        • Recruiting
        • Lumos Pharma Investigational Site
    • Colorado
      • Centennial, Colorado, United States, 80112
        • Recruiting
        • Lumos Pharma Investigational Site
      • Greenwood Village, Colorado, United States, 80111
        • Recruiting
        • Lumos Pharma Investigational Site
    • District of Columbia
      • Washington D.C., District of Columbia, United States, 20010
        • Recruiting
        • Lumos Pharma Investigational Site
    • Florida
      • Hollywood, Florida, United States, 33021
        • Recruiting
        • Lumos Pharma Investigational Site
      • Miami, Florida, United States, 33155
        • Recruiting
        • Lumos Pharma Investigational Site
      • Orlando, Florida, United States, 32806
        • Recruiting
        • Lumos Pharma Investigational Site
      • Tallahassee, Florida, United States, 32308
        • Recruiting
        • Lumos Pharma Investigational Site
    • Indiana
      • Indianapolis, Indiana, United States, 46202
        • Recruiting
        • Lumos Pharma Investigational Site
    • Iowa
      • Iowa City, Iowa, United States, 52242
        • Recruiting
        • Lumos Pharma Investigational Site
    • Louisiana
      • New Orleans, Louisiana, United States, 70118
        • Recruiting
        • Lumos Pharma Investigational Site
    • Minnesota
      • Minneapolis, Minnesota, United States, 55454
        • Recruiting
        • Lumos Pharma Investigational Site
    • Missouri
      • Kansas City, Missouri, United States, 64108
        • Recruiting
        • Lumos Pharma Investigational Site
    • Nevada
      • Las Vegas, Nevada, United States, 89113
        • Recruiting
        • Lumos Pharma Investigational Site
    • New Jersey
      • New Brunswick, New Jersey, United States, 08901
        • Recruiting
        • Lumos Pharma Investigational Site
    • New York
      • Staten Island, New York, United States, 10306
        • Recruiting
        • Lumos Pharma Investigational Site
    • Oklahoma
      • Oklahoma City, Oklahoma, United States, 73104
        • Recruiting
        • Lumos Pharma Investigational Site
    • South Carolina
      • Columbia, South Carolina, United States, 29203
        • Recruiting
        • Lumos Pharma Investigational Site
    • Texas
      • Amarillo, Texas, United States, 79106
        • Recruiting
        • Lumos Pharma Investigational Site
      • Corpus Christi, Texas, United States, 78411
        • Recruiting
        • Lumos Pharma Investigational Site
      • Fort Worth, Texas, United States, 76104
        • Recruiting
        • Lumos Pharma Investigational Site
    • Virginia
      • Charlottesville, Virginia, United States, 22908
        • Recruiting
        • Lumos Pharma Investigational Site
    • Washington
      • Seattle, Washington, United States, 98105
        • Recruiting
        • Lumos Pharma Investigational Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child

Accepts Healthy Volunteers

No

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Triple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Placebo Comparator: Placebo
Other: Matched Placebo (Capsules)
Administered orally once daily
Experimental: LUM-201
Drug: LUM-201
1.6 mg/kg/day, administered orally once daily

What is the study measuring?

Primary Outcome Measures

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

Other Outcome Measures

Outcome Measure
Measure Description
Time Frame
Percentage of subjects selected by PEM strategy who meet target growth
Time Frame: Day 1 to Month 12
Annualized height velocity (AHV) measured as standing height with stadiometer
Day 1 to Month 12

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Lumos Pharma

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

May 20, 2026

Primary Completion (Estimated)

December 1, 2027

Study Completion (Estimated)

January 1, 2028

Study Registration Dates

First Submitted

April 21, 2025

First Submitted That Met QC Criteria

April 21, 2025

First Posted (Actual)

April 29, 2025

Study Record Updates

Last Update Posted (Actual)

June 9, 2026

Last Update Submitted That Met QC Criteria

June 5, 2026

Last Verified

May 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • LUM-201-10

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

UNDECIDED

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

Yes

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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