91´óÉñ

Study Location | Status

91´óÉñ Children’s Health, Pensacola | ·¡²Ô°ù´Ç±ô±ô¾±²Ô²µÌýÌý
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ParticipantÌýEligibility

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:Ìý Ìý Ìý Ìý Ìý Ìý Ìý Ìý Ìý Ìý Ìý Ìý Ìý Ìý Ìý Ìý Ìý Ìý Ìý Ìý Ìý Ìý Ìý Ìý ÌýÌý

• 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:ÌýA Phase 2, Randomized, Controlled, Multicenter Study of Vosoritide in Children With Idiopathic Short Stature

Study Type: InterventionalÌý(researchers intervene and assign treatments)

Study ID:Ìý2231167

Sponsor/Coordinating Site:ÌýBioMarin Pharmaceutical

Start Date:ÌýOctober 2024

Completion Date (Estimated):ÌýJune 2026

Enrolling Healthy Volunteers?ÌýNo
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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 injectionÌýof study drug under the skin.
• 1 screening visitÌýand approximatelyÌý1 observation visitÌý(if not previously enrolled).
• Up toÌý2 visitsÌýduring the observation period.
• Regular clinic visitsÌýuntil study completion.
• Blood draws: about 6 tablespoons (92 mL) in year 1, then about 3 tablespoons (40 mL) per year.
• DXA scansÌýto check bone density.
• Hand X-raysÌýto monitor growth over time.