A Phase 2 Study of Vosoritide in Children With Idiopathic Short Stature

Recruiting now · Phase 2 · Has a placebo group

Conditions studied: Idiopathic Short Stature

In brief

The purpose of this study is to evaluate i) the effect of multiple doses of vosoritide and ii) the effect of the therapeutic dose of vosoritide compared to human growth hormone (hGH)(hGH; only in the United States), in children with idiopathic short stature (ISS).

Key facts

Study ID

NCT06382155

Run by

BioMarin Pharmaceutical

People needed

100

Starts

2024-10-21

Expected to finish

2036-12-01

Last updated by the study team

2026-05-22

Who can join

Age: 3 and older, up to 11. Sex: any. Healthy volunteers: not accepted.

Where it is running

• Chungnam National University Sejong Hospital (CNUSH) — Sejong, South Korea (enrolling)
• Rady Children's Hospital - San Diego — California City, California, United States (enrolling)
• Children's Hospital of Orange County Main Campus - Orange — California City, California, United States (enrolling)
• Children's Hospital Los Angeles — Los Angeles, California, United States (enrolling)
• Children's Hospital Oakland Research Institute — Oakland, California, United States (enrolling)
• Center Of Excellence in Diabetes and Endocrinology — Sacramento, California, United States (enrolling)
• Lundquist Institute for Biomedical Innovation (LA BioMed) — Torrance, California, United States (enrolling)
• The Children's National Research Institute — Washington D.C., District of Columbia, United States (enrolling)
• Nemours Children's Specialty Care Jacksonville — Jacksonville, Florida, United States (enrolling)
• Nicklaus Children's Hospital — Miami, Florida, United States (enrolling)
• Optimal Research Sites — Orange City, Florida, United States (enrolling)
• Nemours Children's Health System - Corporate Headquarters — Pensacola, Florida, United States (enrolling)
• Centricity Research — Columbus, Georgia, United States (enrolling)
• IRCCS Istituto Giannina Gaslini — Genova, Italy (enrolling)
• Seoul National University Bundang Hospital — Seongnam-si, Gyeonggi-do, South Korea (enrolling)
• Inha University Hospital — Incheon, South Korea (enrolling)
• Consano Clinical Research, LLC — California City, California, United States (enrolling)
• Rocky Mountain Clinical Research - Idaho Falls — Idaho Falls, Idaho, United States (enrolling)
• University of Iowa Stead Family Children's Hospital — Iowa City, Iowa, United States (enrolling)
• Kentucky Children's Hospital — Lexington, Kentucky, United States (enrolling)
• University of Louisville School of Medicine — Louisville, Kentucky, United States (enrolling)
• Velocity Clinical Research - MedPharmics - Lafayette — Lafayette, Louisiana, United States (enrolling)
• University of Michigan — Ann Arbor, Michigan, United States (enrolling)
• University of Minnesota Masonic Children's Hospital — Minneapolis, Minnesota, United States (enrolling)
• UBMD Pediatrics — Buffalo, New York, United States (enrolling)

Full record on ClinicalTrials.gov

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