Interventional Study of Vosoritide for the Treatment of Children With Hypochondroplasia
Condition(s) studied
Investigational drug(s) / intervention(s)
Vosoritide: Subcutaneous injection of recommended dose of vosoritide based on weight-band dosing once daily.
Placebo: Subcutaneous injection of recommended dose of placebo
Study summary
The intent and design of this Phase 3 study is to assess vosoritide as a therapeutic option for the treatment of children with hypochondroplasia (HCH).
Eligibility
Primary outcome measure(s)
- Change from baseline in annualized growth velocity (AGV) at Week 52 versus placebo — At week 52
Trial sites (23)
| Facility | City | Region | Status |
|---|---|---|---|
| Nemours Alfred I. DuPont Hospital for Children | Wilmington | Delaware | |
| Children's National Medical Center | Washington D.C. | District of Columbia | |
| Ann & Robert H. Lurie Children's Hospital of Chicago | Chicago | Illinois | |
| Medical College of Wisconsin | Milwaukee | Wisconsin | |
| Murdoch Children's Research Institute | Parkville | Victoria | |
| University of Alberta Stollery Children's Hospital | Edmonton | Alberta | |
| SickKids - The Hospital for Sick Children | Toronto | Ontario | |
| Centre Hospitalier Universitaire Sainte-Justine | Montreal | Quebec | |
| Hospices Civils de Lyon - Hopital Femme Mere Enfant | Lyon | France | |
| Hopital de la Timone | Marseille | France | |
| Hopital Necker-Enfants Malade | Paris | France | |
| CHU de Toulouse | Toulouse | France | |
| Uniklinik Koln | Cologne | Germany | |
| Univeristatskinderklinik Magdeburg | Magdeburg | Germany | |
| Instituto Giannina Gaslini | Genoa | Italy | |
| Fondazione Policlinico Universitario Agostino Gemelli IRCCS | Rome | Italy | |
| Ospedale Pediatrico Bambino Gesu | Rome | Italy | |
| Osaka University Hosptial | Osaka | Japan | |
| Tokushima University Hospital | Tokushima | Japan | |
| Institute of Science Tokyo Hospital | Tokyo | Japan | |
| Tottori University Hospital | Tottori | Japan | |
| Vithas Hospital San Jose | Vitoria-Gasteiz | Spain | |
| Great Ormond Street Hospital for Children NHS Foundation Trust | London | United Kingdom |
More BioMarin Pharmaceutical trials in the UK
Other trials for the same condition
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT06455059 on ClinicalTrials.gov ↗ ← All trials in the UK