Vosoritide for Idiopathic Short Stature in Children (Pensacola)
Studying Treatments for Idiopathic Short Stature
This research is being done to learn whether a study drug called vosoritide can help children with idiopathic short stature grow. The study will compare the effects, both benefits and potential risks, of vosoritide, human growth hormone, or placebo in children with this condition. Children who begin, or switch to, vosoritide may continue in that treatment group until they stop growing.
Study Location | Status
Nemours Children’s Health, Pensacola | Enrolling
Participant Eligibility
Clinical trials have inclusion and exclusion requirements that determine eligibility. Key requirements are listed below. Please note there may be additional qualifications.
Participants must be:
- Ages three to 10 years old (female).
- Ages three to 11 years old (male).
- Able to move and stand without help.
About the Study
Formal Study Name: A Phase 2, Randomized, Controlled, Multicenter Study of Vosoritide in Children With Idiopathic Short Stature
Study Type: Interventional (researchers intervene and assign treatments)
Study ID: 2231167
Sponsor/Coordinating Site: BioMarin Pharmaceutical
Start Date: October 2024
Completion Date (Estimated): June 2026
Enrolling Healthy Volunteers? No
What’s Involved
In this study, your child will receive either vosoritide, human growth hormone, or placebo. If your child receives placebo, they will switch to vosoritide after 6 months to reduce time without active treatment. Children on vosoritide may continue receiving it until they finish growing (about age 16 for girls and 18 for boys).
If your child was not in a related earlier study, they will complete a 6-month observation period before starting treatment.
Requirements include:
- Daily injection of study drug under the skin.
- 1 screening visit and approximately 1 observation visit (if not previously enrolled).
- Up to 2 visits during the observation period.
- Regular clinic visits until study completion.
- Blood draws: about 6 tablespoons (92 mL) in year 1, then about 3 tablespoons (40 mL) per year.
- DXA scans to check bone density.
- Hand X-rays to monitor growth over time.
How This Study Fits in
This study reflects our broader commitment at Nemours to improve care through research. Clinical trials like this one are shaped by the expertise of our medical teams and research programs. Learn how related research and clinical care support, and are strengthened by, studies like this one.
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CLINICAL CARE
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Learn what to expect from a clinical trial — how they work, why they matter, and what to consider when deciding if one is right for your child.
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