Lonapegsomatropin vs Somatropin for Dwarfism
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial aims to evaluate the effectiveness of a once-weekly treatment called lonapegsomatropin (a growth hormone therapy) compared to a daily treatment called somatropin (also a growth hormone therapy) for children with specific growth conditions. It focuses on conditions causing short stature and growth issues, such as Turner syndrome (TS), SHOX deficiency (SHOX-D), small for gestational age (SGA), or idiopathic short stature (ISS). Children who have not previously used growth hormone therapies and have documented growth issues may be suitable for this study. Participants will either receive weekly lonapegsomatropin for two years or start with daily somatropin for one year, followed by the weekly treatment for another year. The trial will occur in the U.S., France, Germany, Italy, Romania, Spain, and South Korea. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants a chance to contribute to the development of a potentially groundbreaking treatment.
Is there any evidence suggesting that this trial's treatments are likely to be safe?
Research has shown that lonapegsomatropin is safe and well-tolerated in children. Studies found that this once-a-week treatment did not raise any new safety concerns. Tested in children with growth hormone issues, it showed safety results similar to other treatments.
For somatropin, already approved by the FDA for growth hormone deficiency, evidence from its use confirms it is generally safe. It has been used for years to help children grow, with serious side effects being rare.
Both treatments have been studied in many children, and findings suggest they are safe options for managing growth conditions.12345Why are researchers excited about this trial's treatments?
Lonapegsomatropin is unique because it offers a new approach to treating dwarfism by potentially reducing the frequency of injections needed. Unlike standard treatments like somatropin, which require daily injections, lonapegsomatropin is designed for more extended release, potentially allowing for fewer injections over time. This could improve convenience and adherence for patients. Researchers are excited about this treatment because it maintains the benefits of growth hormone therapy while potentially offering a better quality of life for patients through easier administration.
What evidence suggests that this trial's treatments could be effective for dwarfism?
This trial will compare Lonapegsomatropin with Somatropin for treating dwarfism. Research has shown that Lonapegsomatropin, administered once a week, helps children with growth hormone deficiency (GHD) grow taller over time. Studies have found that it can significantly increase height in children without causing excessively rapid growth. It also helps reduce body fat, which can be a beneficial side effect. Children treated with Lonapegsomatropin have shown continued height improvements for up to six years. In contrast, Somatropin, administered daily, is already known to help children with similar conditions grow. These findings suggest that both treatments are effective for growth, but Lonapegsomatropin is more convenient due to fewer required doses. Participants in this trial will receive either Lonapegsomatropin or Somatropin to evaluate their effectiveness and convenience.12367
Who Is on the Research Team?
Medical Director, MD
Principal Investigator
Ascendis Pharma A/S
Are You a Good Fit for This Trial?
This trial is for children and adolescents aged 2 to less than 18 with specific growth disorders like Turner Syndrome, Dwarfism, or Idiopathic Short Stature. They must have normal growth hormone levels, be prepubertal, able to stand unassisted, and not previously treated with growth hormones. Girls need a confirmed XX chromosome count.Inclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive either lonapegsomatropin for 2 years or somatropin for 1 year followed by lonapegsomatropin for 1 year
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- Lonapegsomatropin
- Somatropin
Trial Overview
The study compares the effectiveness of weekly Lonapegsomatropin versus daily Somatropin injections in promoting growth. Participants are randomly assigned to receive either treatment for up to two years across several countries including the US and parts of Europe and Asia.
How Is the Trial Designed?
2
Treatment groups
Experimental Treatment
Active Control
Participants will receive Lonapegsomatropin by subcutaneous injection for 2 years (104 weeks)
Participants will receive somatropin by subcutaneous injection for 1 year (52 weeks) followed by lonapegsomatropin for 1 year (52 weeks)
Find a Clinic Near You
Who Is Running the Clinical Trial?
Ascendis Pharma A/S
Lead Sponsor
Citations
Safety and Efficacy of Lonapegsomatropin in Children With ...
Children treated with once-weekly lonapegsomatropin showed continued improvement of height SDS through the second year of therapy without excess advancement of ...
Efficacy and Safety of Once-Weekly Lonapegsomatropin in ...
At week 38, lonapegsomatropin significantly reduced trunk percent fat (–1.68% vs +0.37%; LS mean difference –2.04%, P<.001), increased total ...
Safety and effectiveness of lonapegsomatropin in children ...
The research showed that once-weekly lonapegsomatropin may be a good treatment option for children with GHD as it can help children reach their target height.
4.
karger.com
karger.com/hrp/article/doi/10.1159/000545064/923192/Children-with-Growth-Hormone-Deficiency-TreatedChildren with Growth Hormone Deficiency Treated ...
Children with growth hormone deficiency treated with Lonapegsomatropin demonstrated sustained height improvements for up to 6 years.
NCT04615273 | A Trial to Compare the Efficacy and Safety ...
A Trial to Compare the Efficacy and Safety of Once-weekly Lonapegsomatropin With Placebo and a Daily Somatropin Product in Adults With Growth Hormone Deficiency ...
A Safety, Tolerability and Efficacy Study of TransCon hGH ...
Approximately 150 children (males and females) with growth hormone deficiency (GHD) will be included. All study participants will receive TransCon hGH. This is ...
7.
endocrinologyadvisor.com
endocrinologyadvisor.com/news/lonapegsomatropin-promising-alternative-to-somatropin-in-growth-deficiency/Lonapegsomatropin Is Promising Alternative to Somatropin ...
Lonapegsomatropin demonstrated efficacy outcomes superior to daily somatropin in treatment of pediatric growth hormone deficiency.
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