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Interventional Study of Vosoritide for the Treatment of Children With Hypochondroplasia
Sponsor: BioMarin Pharmaceutical
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).
Official title: A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of Vosoritide in Children With Hypochondroplasia
Key Details
Gender
All
Age Range
3 Years - 17 Years
Study Type
INTERVENTIONAL
Enrollment
80
Start Date
2024-06-17
Completion Date
2026-08-01
Last Updated
2026-01-20
Healthy Volunteers
No
Conditions
Interventions
Vosoritide
Subcutaneous injection of recommended dose of vosoritide based on weight-band dosing once daily.
Placebo
Subcutaneous injection of recommended dose of placebo
Locations (23)
Nemours Alfred I. DuPont Hospital for Children
Wilmington, Delaware, United States
Children's National Medical Center
Washington D.C., District of Columbia, United States
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States
Medical College of Wisconsin
Milwaukee, Wisconsin, United States
Murdoch Children's Research Institute
Parkville, Victoria, Australia
University of Alberta Stollery Children's Hospital
Edmonton, Alberta, Canada
SickKids - The Hospital for Sick Children
Toronto, Ontario, Canada
Centre Hospitalier Universitaire Sainte-Justine
Montreal, Quebec, Canada
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