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Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia
Sponsor: BioMarin Pharmaceutical
Summary
This is a multicenter, multinational, randomized, active-controlled, operationally seamless Phase 2/3 study of BMN 333 in treatment-naïve pediatric participants with achondroplasia (ACH). The study consists of a Phase 2 part and a Phase 3 part.
Official title: A Multicenter, Randomized, Operationally Seamless Phase 2/3 Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia
Key Details
Gender
All
Age Range
2 Years - 17 Years
Study Type
INTERVENTIONAL
Enrollment
160
Start Date
2026-04-20
Completion Date
2029-09
Last Updated
2026-08-21
Healthy Volunteers
No
Conditions
Interventions
BMN 333
Administration: Weekly subcutaneous injection
Vosoritide Injection [Voxzogo]
Administration: Daily subcutaneous injection
Locations (25)
UCSF Benioff Children's Hospital Oakland
Oakland, California, United States
Nemours Children's Health
Wilmington, Delaware, United States
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States
Johns Hopkins Medicine
Baltimore, Maryland, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
University of Texas Southwestern Medical Center
Dallas, Texas, United States
Texas Children Hospital, Baylor College of Medicine
Houston, Texas, United States
Consano Clinical Research, LLC
San Antonio, Texas, United States
Murdoch Children's Research Institute
Parkville, Victoria, Australia
Universite de Montreal - Centre Hospitalier Universitaire Sainte-Justine
Montreal, Quebec, Canada
Irccs Ospedale Gaslini Di Genova
Genova, Italy
Hokkaido University Hospital
Hokkaido, Japan
Osaka City General Hospital
Osaka, Japan
National Center for Child Health and Development
Tokyo, Japan
Uniwersytecki Szpital Kliniczny im. J. Mikulicza-Radeckiego we Wroclawiu Klinika Pediatrii i Chorob Infekcyjnych
Wroclaw, Poland
Institutul National de Endocrinologie C.I.Parhon
Bucharest, Romania
Craiova Emergency Clinical County
Craiova, Romania
Seoul National University Hospital
Seoul, South Korea
Pusan National University Yangsan Hospital
Yangsan, South Korea
University Hospitals Bristol NHS Foundation Trust - Bristol Royal Hospital for Children
Bristol, United Kingdom
Queen Elizabeth University Hospital
Glasgow, United Kingdom
Alder Hey Childrens NHS Foundation Trust
Liverpool, United Kingdom
Great Ormond Street Hospital
London, United Kingdom
University NHS Foundation Trust NIHR Wellcome Trust Clinical Research Facility
Manchester, United Kingdom