A Study of Vosoritide Versus Placebo in Children With Hypochondroplasia Aged 0 to < 36 Months
Recruiting now · Phase 2 · Has a placebo group
Conditions studied: Hypochondroplasia
In brief
The purpose of this study is to evaluate the safety and efficacy of daily administration of vosoritide in participants with HCH aged 0 to \< 36 months over a 52-week period.
Key facts
- Study ID
- NCT07126262
- Run by
- BioMarin Pharmaceutical
- People needed
- 60
- Starts
- 2025-07-30
- Expected to finish
- 2028-06-30
- Last updated by the study team
- 2026-03-31
Who can join
Age: any, up to 3. Sex: any. Healthy volunteers: not accepted.
Where it is running
- University of Missouri — Columbia, Missouri, United States (enrolling)
- IRCCS Istituto Giannina Gaslini — Genova, Italy (enrolling)
- Kumamoto University Hospital — Kumamoto, Japan (enrolling)
- Osaka Women's and Children's Hospital — Osaka, Japan (enrolling)
- Institute of Science Tokyo Hospital — Tokyo, Japan (enrolling)
- Nihon University Itabashi Hospital — Tokyo, Japan (enrolling)
- Tottori University Hospital — Tottori, Japan (enrolling)
- Benioff Children's Hospital - Oakland — Oakland, California, United States (enrolling)
- Phoenix Children's Hospital - Thomas Campus (Main) — Phoenix, Arizona, United States (enrolling)
- Children's National Medical Center — Washington D.C., District of Columbia, United States (enrolling)
- Ann & Robert H. Lurie Children's Hospital of Chicago — Chicago, Illinois, United States (enrolling)
- Royal Children's Hospital Melbourne — Parkville, Victoria, Australia (enrolling)
- Hôpital Bicêtre — Le Kremlin-Bicêtre, Paris, France (enrolling)
- The Johns Hopkins University School of Medicine — Baltimore, Maryland, United States (enrolling)
- Uniklinik Köln — Cologne, North Rhine-Westphalia, Germany (enrolling)
- Universitätsklinikum des Saarlandes — Homburg, Saarland, Germany (enrolling)
- University of Minneasota Masonic Children's Hospital — Minneapolis, Minnesota, United States (enrolling)
- Great Ormond Street Hospital — London, England, United Kingdom
- Cedars-Sinai Medical Center — Los Angeles, California, United States
- Cook Children's Endocrinology — Fort Worth, Texas, United States
- Children's Wisconsin - Fox Valley Hospital — Neenah, Wisconsin, United States
- Children's Health Queensland Hospital and Health Service — South Brisbane, Queensland, Australia
- Hospices Civils de Lyon - Hôpital Femme Mère Enfant — Bron, Rhone-Alps, France
- Universitätskinderklinik Magdeburg — Magdeburg, Saxony-Anhalt, Germany
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS — Roma, Rome, Italy
Full record on ClinicalTrials.gov
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