LUM-201 for Human Growth Hormone Deficiency

(OraGrowtH213 Trial)

No longer recruiting at 10 trial locations

Age: < 18

Sex: Any

Trial Phase: Phase 2

Sponsor: Lumos Pharma

Must not be taking: CYP3A4 medications

Disqualifiers: Medical condition, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 6

Duration: 12 months

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to explore how well LUM-201 helps children with idiopathic growth hormone deficiency (a lack of growth hormone with no known cause) grow. These children have already received daily growth hormone shots for a year in a previous study. The trial is for those who participated in the LUM-201-01 trial and completed 12 months of daily treatment. Participants will receive LUM-201, a new potential drug, to determine if it can improve growth in a more manageable way. As a Phase 2 trial, the research focuses on measuring the treatment's effectiveness in an initial, smaller group of participants.

Do I have to stop taking my current medications for the trial?

You might need to stop taking certain medications if they can cause short stature or affect growth factors, or if they interact with LUM-201. It's best to discuss your current medications with the trial team to see if any changes are needed.

Is there any evidence suggesting that LUM-201 is likely to be safe for humans?

Research has shown that LUM-201, a daily pill for treating growth hormone deficiency, appears safe. In one study, children taking LUM-201 showed higher growth hormone levels, which is promising. Importantly, no serious side effects were reported in the studies so far, suggesting that the treatment is well-tolerated. Although this treatment is still under investigation, these early results are encouraging for those considering joining a trial. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard treatments for human growth hormone deficiency, which often involve regular injections of synthetic growth hormone, LUM-201 is an oral medication that stimulates the body's own production of growth hormone. This could mean fewer injections and an easier, less invasive treatment regimen for patients. Researchers are excited about LUM-201 because it targets the pituitary gland to naturally boost growth hormone levels, potentially improving patient compliance and convenience while maintaining effectiveness.

What evidence suggests that LUM-201 might be an effective treatment for growth hormone deficiency?

Research has shown that LUM-201 helps children with growth hormone deficiency (GHD) grow by boosting their body's natural growth hormone production. In one study, children who took LUM-201 for six months grew taller at a faster rate than before starting the treatment. Another study found that LUM-201 increased the body's own growth hormone levels, leading to better overall growth. This treatment uses a much smaller amount of growth hormone compared to other methods, potentially making it safer and more convenient. ^{3 4 5 6 7}

Are You a Good Fit for This Trial?

This trial is for children with growth hormone deficiency who have finished a previous 12-month treatment in the LUM-201-01 study. They must have consent from a parent or caregiver, and if old enough, give their own assent to participate. Children cannot join if they take other medications affecting growth or pose extra risks as determined by the study's lead researcher.

Inclusion Criteria

Is eligible for study participation as confirmed by the principal investigator (PI)

You must have already finished 12 months of daily rhGH treatment as part of the LUM-201-01 PGHD trial.

Parent/caregiver must sign the informed consent, and the subject must sign the assent, as applicable.

Exclusion Criteria

Has a medical condition that, in the opinion of the PI and/or MM, adds unwarranted risk to use of LUM-201.

I am not on medication that could affect my growth or response to growth treatments.

I am on long-term medication that affects how my body processes certain drugs.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

1 visit

1 visit (in-person)

Treatment

Participants receive LUM-201 as a daily oral dose each morning for 12 months

12 months

5 visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• LUM-201

Trial Overview The trial is testing LUM-201 on children with idiopathic growth hormone deficiency to see how it affects their growth after previously being treated with daily rhGH. It's an international study following up on earlier research to measure continued response.

How Is the Trial Designed?

Group I: LUM-201 (3.2 mg/kg/day) Experimental Treatment 1 Intervention

Who Is Running the Clinical Trial?

Lumos Pharma

Lead Sponsor

Trials

5

Recruited

180+

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11455403/

5162 Oral LUM-201 Restores Pulsatile Growth Hormone ... These results indicate that by restoring endogenous GH secretion, LUM-201 facilitates growth utilizing a much lower amount of GH than that ...

2. clinicaltrials.gov clinicaltrials.gov/study/NCT04614337

Phase 2 Study of LUM-201 in Children With Growth ... This is a multi-national trial. The goals of the trial are to study LUM-201 as a possible treatment for Pediatric Growth Hormone Deficiency (PGHD)

3. investors.lumos-pharma.com investors.lumos-pharma.com/news-releases/news-release-details/updated-phase-2-oragrowth-data-presented-espe-2024-demonstrate

Updated Phase 2 OraGrowtH Data Presented at ESPE 2024 ... Results showed that at six months on LUM-201, a significant increase over baseline in Annualized Height Velocity (AHV) was observed for the ...

4. endocrine-abstracts.org endocrine-abstracts.org/ea/0110/ea0110p648

Phase 3 pivotal trial design of daily oral LUM-201's efficacy ... The primary objective of the OraGrowtH Phase 3 study is to evaluate the efficacy of daily oral LUM-201 (1. 6 mg/kg) in improving growth velocity over 12 months ...

5. checkrare.com checkrare.com/phase-2-trial-evaluating-lum-201-for-treatment-of-growth-hormone-deficiency/

Phase 2 Trial Evaluating LUM-201 for Treatment of Growth ... These results suggest ibutamoren restores GH secretion, endogenous GH pulsatile secretion, and serum IGF-1 concentrations levels to ...

6. lumos-pharma.com lumos-pharma.com/our-pipeline/lum-201/

LUM-201 LUM-201, also known as ibutamoren, is an investigational, once-daily, orally administered small molecule that promotes the secretion of Growth Hormone (GH) ...

7. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC9227666/

A GH Secretagogue Receptor Agonist (LUM-201) Elicits ... LUM-201 elicits greater GH responses than standard stimuli in subjects with higher peak GH in response to conventional testing.