Growth Hormone Deficiency (GHD)Phase 3NCT07129759
Phase 3 Long Term Safety Extension 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.
Trial summary
This is a Multi-national Trial. The Goal of the Trial is to Offer Subjects Who Complete 12 Months in the LUM-201-10 Phase 3 Trial up to an Additional 36 Months of Treatment of LUM-201 While Evaluating Safety and Tolerability of LUM-201. Read more about this study
- Who is it for?
- Ages 4 to 12 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?
- No, everyone receives an active treatment
- How many people
- 150 participants
- Where
- No sites listed yet
- Study dates
- Feb 2027to Feb 2030
- 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 4 to 12 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Parent/caregiver must be willing to provide written informed consent, and the subject must sign the assent, as applicable.
- Subject must have successfully completed 12 months of participation in the LUM-201 Phase 3 GHD trial, and be eligible for continuation of treatment, pending all other enrollment criteria are met.
- Subject who is sexually active must use an acceptable form of contraception.
- Subject must be eligible for the Day 1 visit as confirmed by the Investigator.
Who cannot join
- Subject has a medical or genetic condition that, in the opinion of the Investigator and/or MMs, adds unwarranted risk to use of LUM-201.
- Pregnancy.
- Subject has planned or is receiving current long-term treatment with medications known to prolong the QT interval or act as substrates, inducers, or inhibitors of the cytochrome system cytochrome P450 type 3A4 that metabolizes LUM-201 (see Appendix 6 for list of example medications). Subjects receiving shorter-term (two weeks or less) treatment with these medications should be evaluated on case-by-case basis by the Investigator in consultation with the MMs.
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
About this study
Official title: A Long-term Extension Study to Evaluate the Safety and Tolerability of Daily Oral LUM-201 in Children With Growth Hormone Deficiency (GHD)
This is a Multi-national Trial. The Goal of the Trial is to Offer Subjects Who Complete 12 Months in the LUM-201-10 Phase 3 Trial up to an Additional 36 Months of Treatment of LUM-201 While Evaluating Safety and Tolerability of LUM-201.
- Conditions
- Growth Hormone Deficiency (GHD)
- Being tested
- LUM-201
- 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
| Study group | Role | What they receive |
|---|---|---|
| LUM-201 | Experimental | Not described Receives: Drug: LUM-201 |
What is being measured
The primary outcome is the main question the study is designed to answer.
To evaluate the long-term safety and tolerability of LUM-201
- The number of subjects with at least 1 (serious) treatment-related adverse event (TEAE).
- The number of subjects with at least 1 suspected unexpected serious adverse reaction.
- The number of subjects with clinically significant abnormalities related to trial laboratory tests or electrocardiograms (ECGs).
- The number of subjects with at least 1 adverse event of special interest (AESI).
- Annual change from baseline in body weight standard deviation score (SDS).
- Annual change from baseline in body mass index (BMI) SDS.
Measured: Day 1 - Month 36
Secondary outcomes (2)
To evaluate the long-term effect of LUM-201 on growth.
- Annual height velocity.
- Annual change in height standard deviation score (SDS).
- Annual change in body mass index (BMI).
- Annual change in BMI SDS.
- Annual change in bone age (BA)/chronological age (CA) ratio.
Measured: Day 1 - Month 36
To evaluate the long-term effect of LUM-201 on pharmacodynamic (PD) markers.
- Annual change in insulin-like growth factor (IGF)-1 SDS.
- Annual change in insulin-like growth binding protein 3 (IGFBP-3) SDS.
Measured: Day 1 - Month 36
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Aug 2025
- Study startFeb 2027 (estimated)
- Primary completionFeb 2030 (estimated)
- Study completionFeb 2030 (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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- 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
- NCT07129759
- Sponsor's protocol ID
- LUM-201-11
- Status last verified
- Aug 2025
- First submitted
- 5 Aug 2025
- Last updated
- 8 Jul 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided