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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
Completion Date
2029-09
Last Updated
2026-05-29
Healthy Volunteers
No
Conditions
Interventions
BMN 333
Administration: Weekly subcutaneous injection
Vosoritide Injection [Voxzogo]
Administration: Daily subcutaneous injection
Locations (10)
UCSF Benioff Children's Hospital Oakland
Oakland, California, United States
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States
Johns Hopkins Medicine Julie
Baltimore, Maryland, United States
Texas Children Hospital, Baylor College of Medicine, Houston TX
Houston, Texas, United States
Consano Clinical Research, LLC
San Antonio, Texas, United States
Murdoch Children's Research Institute
Parkville, Victoria, Australia
Irccs Ospedale Gaslini Di Genova
Genova, Italy
Craiova Emergency Clinical County
Craiova, Romania
Seoul National University Hospital
Seoul, South Korea
University Hospitals Bristol NHS Foundation Trust - Bristol Royal Hospital for Children
Bristol, United Kingdom