Growth Hormone Deficiency (GHD)Phase 3NCT06948214
Phase 3 Study of LUM-201 in Children With Growth Hormone Deficiency
A Phase 3 study testing LUM-201 in people with Growth Hormone Deficiency (GHD), run by Lumos Pharma at 55 sites in 8 countries.
Trial 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… Read more about this study
- Who is it for?
- Ages 3 to 11 years
- Conditions the study is for
- Growth Hormone Deficiency (GHD)
- What is being tested?
- LUM-201
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 150 participants
- Where
- 55 sitesin 8 countries
- Study dates
- 20 May 2026to Jan 2028
- Phase
- Phase 3
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 3 to 11 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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
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- 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
Who cannot join
- 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
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- 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
Trial locations and contacts
55 sites in 8 countries. Contact the study team or a site near you to ask about taking part.
55 sites in 8 countries
United States31 sites, 31 recruiting
- Recruiting
Lumos Pharma Investigational Site
Amarillo, Texas, 79106
- Recruiting
Lumos Pharma Investigational Site
Birmingham, Alabama, 35233
- Recruiting
Lumos Pharma Investigational Site
Centennial, Colorado, 80112
- Recruiting
Lumos Pharma Investigational Site
Charlottesville, Virginia, 22908
- Recruiting
Lumos Pharma Investigational Site
Columbia, South Carolina, 29203
- Recruiting
Lumos Pharma Investigational Site
Columbus, Ohio, 43205
- Recruiting
Lumos Pharma Investigational Site
Corpus Christi, Texas, 78411
- Recruiting
Lumos Pharma Investigational Site
Fort Worth, Texas, 76104
- Recruiting
Lumos Pharma Investigational Site
Greenwood Village, Colorado, 80111
- Recruiting
Lumos Pharma Investigational Site
Hollywood, Florida, 33021
- Recruiting
Lumos Pharma Investigational Site
Indianapolis, Indiana, 46202
- Recruiting
Lumos Pharma Investigational Site
Iowa City, Iowa, 52242
- Recruiting
Lumos Pharma Investigational Site
Jacksonville, Florida, 32207
- Recruiting
Lumos Pharma Investigational Site
Kansas City, Missouri, 64108
- Recruiting
Lumos Pharma Investigational Site
Las Vegas, Nevada, 89113
- Recruiting
Lumos Pharma Investigational Site
Madera, California, 93636
- Recruiting
Lumos Pharma Investigational Site
Miami, Florida, 33155
- Recruiting
Lumos Pharma Investigational Site
Minneapolis, Minnesota, 55454
- Recruiting
Lumos Pharma Investigational Site
New Brunswick, New Jersey, 08901
- Recruiting
Lumos Pharma Investigational Site
New Orleans, Louisiana, 70118
- Recruiting
Lumos Pharma Investigational Site
Oklahoma City, Oklahoma, 73104
- Recruiting
Lumos Pharma Investigational Site
Orange, California, 92868
- Recruiting
Lumos Pharma Investigational Site
Orlando, Florida, 32806
- Recruiting
Lumos Pharma Investigational Site
Sacramento, California, 95821
- Recruiting
Lumos Pharma Investigational Site
San Antonio, Texas, 78207
- Recruiting
Lumos Pharma Investigational Site
San Diego, California, 92123
- Recruiting
Lumos Pharma Investigational Site
Savannah, Georgia, 31406
- Recruiting
Lumos Pharma Investigational Site
Seattle, Washington, 98105
- Recruiting
Lumos Pharma Investigational Site
Staten Island, New York, 10306
- Recruiting
Lumos Pharma Investigational Site
Tallahassee, Florida, 32308
- Recruiting
Lumos Pharma Investigational Site
Washington D.C., District of Columbia, 20010
Thailand6 sites, 6 recruiting
- Recruiting
Lumos Pharma Investigational Site
Bangkok, Bangkok, 10400
- Recruiting
Lumos Pharma Investigational Site
Bangkok Noi, Bangkok, 10700
- Recruiting
Lumos Pharma Investigational Site
Hat Yai, Changwat Songkhla, 90110
- Recruiting
Lumos Pharma Investigational Site
Muang, Changwat Khon Kaen, 40002
- Recruiting
Lumos Pharma Investigational Site
Muang, Chiang Mai, 50200
- Recruiting
Lumos Pharma Investigational Site
Pathum Wan, Bangkok, 10330
United Kingdom6 sites, 6 recruiting
- Recruiting
Lumos Pharma Investigational Site
Liverpool, England, L12 2AP
- Recruiting
Lumos Pharma Investigational Site
London, England, E1 1BB
- Recruiting
Lumos Pharma Investigational Site
London, England, W1G9AB
- Recruiting
Lumos Pharma Investigational Site
London, England, WC1N 3JH
- Recruiting
Lumos Pharma Investigational Site
Manchester, England, M13 9WL
- Recruiting
Lumos Pharma Investigational Site
Sheffield, England, S10 2TH
India5 sites, 5 recruiting
- Recruiting
Lumos Pharma Investigational Site
Chandigarh, 160012
- Recruiting
Lumos Pharma Investigational Site
Jaipur, Rajasthan, 302004
- Recruiting
Lumos Pharma Investigational Site
Madurai, Tamil Nadu, 625020
- Recruiting
Lumos Pharma Investigational Site
Mumbai, Maharashtra, 400012
- Recruiting
Lumos Pharma Investigational Site
Nashik, Maharashtra, 422002
Australia3 sites, 3 recruiting
- Recruiting
Lumos Pharma Investigational Site
Clayton, Victoria, 3168
- Recruiting
Lumos Pharma Investigational Site
Parkville, Victoria, 3052
- Recruiting
Children's Health Queensland Hospital and Health Service
South Brisbane, Queensland, 4101
QCH-Endoresearch@health.qld.gov.au
New Zealand2 sites, 2 recruiting
- Recruiting
University of Auckland (Liggins Institute, Maurice and Nessie Paykel Clinical Research Unit)
Auckland, 1142
Paul Hofman, p.hofman@auckland.ac.nz
Paul Hofman
- Recruiting
Wellington Regional Hospital
Wellington, 6242
Esko Wiltshire, esko.wiltshire@otago.ac.nz
Esko Wiltshire
Argentina1 site, 1 recruiting
- Recruiting
Lumos Pharma Investigational Site
San Miguel de Tucumán, Tucumán Province, 4000
Chile1 site, 1 recruiting
- Recruiting
Lumos Pharma Investigational Site
Santiago, Santiago Metropolitan, 8320000
About this study
Official title: 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.
- Conditions
- Growth Hormone Deficiency (GHD)
- Being tested
- LUM-201, Matched Placebo (Capsules)
- Keywords
- GHD, Pediatric Growth Hormone Deficiency, LUM-201, Growth hormone secretagogue, Height, Catch-up growth, PEM, Oral, Predictive Enrichment Marker, ibutamoren mesylate, OraGrowtH Phase 3 Trial, LUM-201 PEM, GH secretagogue
- Medical subject headings
- Dwarfism, Pituitary
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
LUM-201Drug
1.6 mg/kg/day, administered orally once daily
Matched Placebo (Capsules)Other
Administered orally once daily
| Study group | Role | What they receive |
|---|---|---|
| LUM-201 | Experimental | Not described Receives: Drug: LUM-201 |
| Placebo | Placebo comparator | Not described Receives: Other: Matched Placebo (Capsules) |
What is being measured
The primary outcome is the main question the study is designed to answer.
AHV after 12 months on LUM-201 compared to placebo
Annualized height velocity (AHV) measured as standing height with stadiometer
Measured: Day 1 to Month 12
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Apr 2025
- Study start20 May 2026
- Primary completionDec 2027 (estimated)
- Study completionJan 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Growth Hormone Deficiency (GHD)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 150 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Lumos Pharma
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06948214
- Sponsor's protocol ID
- LUM-201-10
- Status last verified
- May 2026
- First submitted
- 21 Apr 2025
- Last updated
- 19 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- Yes
- Individual data sharing
- Undecided