Growth Hormone DeficiencyPhase 2OraGrowtH213NCT05250063
Phase 2 Study of LUM-201 in Children Who Have Previously Completed the LUM-201-01 Trial (OraGrowtH213)
A Phase 2 study testing LUM-201 in people with Growth Hormone Deficiency, run by Lumos Pharma at 10 sites in 2 countries.
Trial summary
This is a multi-national trial. The goals of the trial are to study the growth response to LUM-201 administration in children with idiopathic growth hormone deficiency (GHD) previously treated with daily rhGH for 12 months in the LUM-201-01 trial. Read more about this study
- Who is it for?
- Ages 3 to 13 years
- Conditions the study is for
- Growth Hormone Deficiency
- What is being tested?
- LUM-201
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- 10 sitesin 2 countries
- Study dates
- 18 Feb 2022to 22 Jan 2025
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 13 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Parent/caregiver must sign the informed consent, and the subject must sign the assent, as applicable.
- Must have previously completed 12 months of daily rhGH treatment as part of the LUM-201-01 PGHD trial.
- Is eligible for study participation as confirmed by the principal investigator (PI)
Who cannot join
- Has a medical condition that, in the opinion of the PI and/or MM, adds unwarranted risk to use of LUM-201.
- Uses any medication that, in the opinion of the PI and/or MM, can independently cause short stature or limit the response to exogenous growth factors.
- Has planned or is receiving current long-term treatment with medications known to act as substrates, inducers, or inhibitors of the cytochrome system CYP3A4 that metabolizes LUM-201
Trial locations and contacts
10 sites in 2 countries.
10 sites in 2 countries
United States7 sites
University of Virginia Health System
Charlottesville, Virginia, 22908
Nationwide Children's Hospital
Columbus, Ohio, 43205
Indiana University School of Medicine
Indianapolis, Indiana, 46202
The Children's Mercy Hospital
Kansas City, Missouri, 64111
M Health, Fairview Pediatric Specialty Clinics- Discovery Clinic
Minneapolis, Minnesota, 55454
Rady Children's Hospital
San Diego, California, 92123
Children's National Hospital
Washington D.C., District of Columbia, 20010
Poland3 sites
Klinika Endokrynologii i Chorob Metabolicznych, Instytut Centrum Zdrowia Matki Polki
Lodz
Sonomed - Centrum Medyczne
Szczecin
Klinika Endokrynologii i Diabetologii, Instytut "Pomnik Centrum Zdrowia Dziecka
Warsaw
About this study
Official title: A Multicenter, Open-Label, Phase 2 Study to Evaluate Growth and Safety of LUM-201 Following 12 Months of Daily rhGH Treatment in Children With Idiopathic Growth Hormone Deficiency Who Have Previously Completed the LUM-201-01 Trial
This is a multi-national trial. The goals of the trial are to study the growth response to LUM-201 administration in children with idiopathic growth hormone deficiency (GHD) previously treated with daily rhGH for 12 months in the LUM-201-01 trial.
Read the detailed description
This trial will have one screening/baseline visit with tests to assess if subjects are eligible to start study therapy. The screening/baseline visit can coincide with the Month 12 visit in the LUM-201-01 trial. Subjects will receive the same dose of LUM-201 as a daily oral dose each morning. LUM-201 will be adjusted for body weight increments at each clinic visit, as applicable.
The trial consists of 12 months of treatment. After screening, subjects will return to the clinic for 5 visits. During four of these clinic visits, safety laboratory samples will be collected.
- Conditions
- Growth Hormone Deficiency
- Being tested
- LUM-201
- Keywords
- LUM-201, PGHD, Height, Oral
- 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
Administered orally once daily
| Study group | Role | What they receive |
|---|---|---|
| LUM-201 (3.2 mg/kg/day) | Experimental | Not described Receives: Drug: LUM-201 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Annualized Height Velocity (AHV) Measured as Standing Height With Stadiometer
Mean change from day 1 to month 12, Annualized Height Velocity (AHV) Height is measured in triplicate, by a calibrated stadiometer and taking the mean value AVH equation is (h2-h1/t2-t1)*365.25; h1 = height measured at day 1 h2 = height measured at month 12 t1 = day 1 t2 = month 12
Measured: Day 1 to Month 12
Secondary outcomes (11)
Occurrence of Adverse Events in Children With Growth Hormone Deficiency (GHD)
Number of events
Measured: Day 1 to Month 12
Growth Hormone (GH) Values
Mean GH Values change from Day 1 to month 12 (Pre-Dose and 1 hour Post-Dose)
Measured: Day 1 to Month 12
Insulin-Like Growth Factor 1 (IGF-1) Values
Mean change from Day 1 to month 12 (IGF-1 Values Pre-Dose)
Measured: Day 1 to Month 12
Insulin-Like Growth Factor Binding Protein 3 (IGFBP-3) Values
Mean change from day 1 to month 12 (IGFBP-3 Values Pre-Dose)
Measured: Day 1 to Month 12
Height (HT) Standard Deviation Score (SDS)
Mean change from day 1 to month 12 in HT-SDS
Measured: Day 1 to Month 12
Change in Weight
Mean change in Weight from day 1 to month 12
Measured: Day 1 to Month 12
Change in Weight - Standard Deviation Score (SDS)
Mean change in Weight-SDS from day 1 to month 12
Measured: Day 1 to Month 12
Change in Body Mass Index (BMI)
Mean change from day 1 to month 12 in BMI
Measured: Day 1 to Month 12
Change in Body Mass Index (BMI) - Standard Deviation Score (SDS)
Mean change from Day 1 to month 12 in BMI-SDS
Measured: Day 1 to Month 12
Change in Bone Age
Mean change from day 1 to month 6 in bone age, measured by X-ray of left hand and wrist using Greulich \& Pyle atlas
Measured: Month 6
Insulin-Like Growth Factor 1 (IGF-1) - Standard Deviation Score (SDS)
Mean Change from day 1 to month 12 for IGF-1 - SDS Pre-dose Values
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 posted22 Feb 2022
- Study start18 Feb 2022
- Primary completion22 Jan 2025
- Study completion22 Jan 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Growth Hormone DeficiencyStudies 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 15 participants (actual)
Sponsor and investigators
- Lead sponsor
- Lumos Pharma
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05250063
- Sponsor's protocol ID
- LUM-201-04
- Acronym
- OraGrowtH213
- Status last verified
- Apr 2026
- First submitted
- 10 Feb 2022
- Last updated
- 15 May 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 15 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter, Open-Label, Phase 2 Study to Evaluate Growth and Safety of LUM-201 Following 12 Months of Daily rhGH Treatment in Children With Idiopathic Growth Hormone Deficiency Who Have Previously Completed the LUM-201-01 Trial
- ClinicalTrials.gov ID
- NCT05250063
- Acronym / sponsor ID
- OraGrowtH213 / LUM-201-04
- Conditions
- Growth Hormone Deficiency
- Keywords
- LUM-201, PGHD, Height, Oral
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 15 participants (actual)
- Lead sponsor
- Lumos PharmaIndustry (pharma, biotech, devices)
- Study start
- 18 Feb 2022 (actual)
- Primary completion
- 22 Jan 2025 (actual)
- Study completion
- 22 Jan 2025 (actual)
- First posted
- 22 Feb 2022 (actual)
- Results
- Posted 13 Apr 2026
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a multi-national trial. The goals of the trial are to study the growth response to LUM-201 administration in children with idiopathic growth hormone deficiency (GHD) previously treated with daily rhGH for 12 months in the LUM-201-01 trial.
Detailed description
This trial will have one screening/baseline visit with tests to assess if subjects are eligible to start study therapy. The screening/baseline visit can coincide with the Month 12 visit in the LUM-201-01 trial. Subjects will receive the same dose of LUM-201 as a daily oral dose each morning. LUM-201 will be adjusted for body weight increments at each clinic visit, as applicable.
The trial consists of 12 months of treatment. After screening, subjects will return to the clinic for 5 visits. During four of these clinic visits, safety laboratory samples will be collected.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| LUM-201 (3.2 mg/kg/day) | Experimental | Drug: LUM-201 |
Who may qualify
- Ages
- 3 to 13 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (3 listed)
- Parent/caregiver must sign the informed consent, and the subject must sign the assent, as applicable.
- Must have previously completed 12 months of daily rhGH treatment as part of the LUM-201-01 PGHD trial.
- Is eligible for study participation as confirmed by the principal investigator (PI)
Key exclusion criteria (3 listed)
- Has a medical condition that, in the opinion of the PI and/or MM, adds unwarranted risk to use of LUM-201.
- Uses any medication that, in the opinion of the PI and/or MM, can independently cause short stature or limit the response to exogenous growth factors.
- Has planned or is receiving current long-term treatment with medications known to act as substrates, inducers, or inhibitors of the cytochrome system CYP3A4 that metabolizes LUM-201
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| LUM-201Administered orally once daily | Drug | LUM-201 (3.2 mg/kg/day) |
Primary outcome
Annualized Height Velocity (AHV) Measured as Standing Height With Stadiometer
Time frame: Day 1 to Month 12
Mean change from day 1 to month 12, Annualized Height Velocity (AHV) Height is measured in triplicate, by a calibrated stadiometer and taking the mean value AVH equation is (h2-h1/t2-t1)*365.25; h1 = height measured at day 1 h2 = height measured at month 12 t1 = day 1 t2 = month 12
11 secondary outcomes
Occurrence of Adverse Events in Children With Growth Hormone Deficiency (GHD)
Time frame: Day 1 to Month 12
Growth Hormone (GH) Values
Time frame: Day 1 to Month 12
Insulin-Like Growth Factor 1 (IGF-1) Values
Time frame: Day 1 to Month 12
Insulin-Like Growth Factor Binding Protein 3 (IGFBP-3) Values
Time frame: Day 1 to Month 12
Height (HT) Standard Deviation Score (SDS)
Time frame: Day 1 to Month 12
Change in Weight
Time frame: Day 1 to Month 12
Change in Weight - Standard Deviation Score (SDS)
Time frame: Day 1 to Month 12
Change in Body Mass Index (BMI)
Time frame: Day 1 to Month 12
Change in Body Mass Index (BMI) - Standard Deviation Score (SDS)
Time frame: Day 1 to Month 12
Change in Bone Age
Time frame: Month 6
Insulin-Like Growth Factor 1 (IGF-1) - Standard Deviation Score (SDS)
Time frame: Day 1 to Month 12
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 May 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: Undecided
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Enrolling by invitationObservational study
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
10 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing LUM-201 in people with Growth Hormone Deficiency, run by Lumos Pharma at 10 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 15 May 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 10 sites in United States, Poland. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 13 Apr 2026). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Annualized Height Velocity (AHV) Measured as Standing Height With Stadiometer (time frame: Day 1 to Month 12). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 15 May 2026.