LUM-201 for Human Growth Hormone Deficiency
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial aims to test LUM-201, a new potential drug, as a treatment for Pediatric Growth Hormone Deficiency (PGHD), a condition where children produce insufficient growth hormone, affecting their growth. Researchers seek to determine if LUM-201 improves growth in these children and will also test a strategy to predict which children are likely to benefit from the treatment. Participants will be divided into groups receiving either LUM-201 or a placebo, which contains no active medication. Children who have never received growth hormone treatment and experience significantly slower growth for their age may be suitable candidates for this trial. As a Phase 3 trial, this study represents the final step before potential FDA approval, offering participants a chance to contribute to a treatment nearing widespread availability.
Do I need to stop my current medications to join the trial?
The trial does not specify if you need to stop all current medications, but you cannot take medications that might cause short stature or affect growth factor response. Also, you can't be on certain medications affecting liver enzymes or have used systemic corticosteroids recently.
Is there any evidence suggesting that LUM-201 is likely to be safe for humans?
Earlier studies have shown that LUM-201, also known as ibutamoren, is safe for children with Pediatric Growth Hormone Deficiency (PGHD). Research indicates that LUM-201 can trigger a stronger growth hormone response compared to standard treatments. So far, no major reports of serious side effects have emerged. This treatment is a once-a-day pill, making it easier and less invasive than injections. The current trial is in a later stage, indicating it has already passed initial safety checks. However, like any treatment, risks may exist, and discussing these with the trial team is important.12345
Why do researchers think this study treatment might be promising for growth hormone deficiency?
Unlike current treatments for human growth hormone deficiency, which typically involve daily injections of synthetic growth hormone, LUM-201 is an oral medication that stimulates the body's own production of growth hormone. This unique mechanism of action sets it apart from existing therapies by potentially reducing the need for frequent injections. Researchers are excited about LUM-201 because it promises a more convenient and less invasive option for patients, which could improve adherence to treatment and overall patient experience.
What evidence suggests that LUM-201 might be an effective treatment for Pediatric Growth Hormone Deficiency?
Research shows that LUM-201, a medicine that helps the body release growth hormone, may effectively treat children with Pediatric Growth Hormone Deficiency (PGHD). Studies have found that children taking LUM-201 grew taller more quickly, as indicated by their increased Annualized Height Velocity (AHV). Additionally, LUM-201 restored normal growth hormone patterns in these children, similar to those in healthy kids. These findings suggest that LUM-201 could help children with PGHD grow at a healthier rate. Participants in this trial will receive either LUM-201 or a placebo to further evaluate its effectiveness.34678
Are You a Good Fit for This Trial?
This trial is for prepubertal children who have not been treated before and are significantly shorter than average for their age due to growth hormone deficiency. They must be between 3-11 years old, with slow growth rates and delayed bone age. Girls need negative tests for Turner syndrome.Inclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive daily oral LUM-201 or placebo for 12 months
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- LUM-201
Find a Clinic Near You
Who Is Running the Clinical Trial?
Lumos Pharma
Lead Sponsor