Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia
Start Date
4/20/2026
Completion Date
9/1/2029
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.
Detailed Description
The main purpose of this study is to evaluate the effects of BMN 333 on growth compared with vosoritide in participants with achondroplasia who have not received any growth-promoting treatments. The study includes 2 parts: the Phase 2 part will select the optimal BMN 333 dose to be used in Phase 3 and determine study continuation into Phase 3; the Phase 3 part will compare the effects of the selected dose of BMN 333 with vosoritide. Study details for either Phase 2 or Phase 3 include the following: * Study duration: up to 61 weeks (from screening to Safety Follow-up visit) * Treatment duration: 52 weeks. Treatment frequency: BMN 333, once weekly; vosoritide, once daily
Eligibility Criteria
Age Range: 2 years to 17 years
Interventions
BMN 333
Vosoritide Injection [Voxzogo]
Conditions
Locations
UCSF Benioff Children's Hospital Oakland
Oakland, California 94609
United States
Nemours Children's Health
Wilmington, Delaware 19803
United States
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois 60611
United States
Johns Hopkins Medicine
Baltimore, Maryland 21205
United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio 45229
United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania 19104
United States
University of Texas Southwestern Medical Center
Dallas, Texas 75207
United States
Texas Children Hospital, Baylor College of Medicine
Houston, Texas 77030
United States
Consano Clinical Research, LLC
San Antonio, Texas 78231
United States
Murdoch Children's Research Institute
Parkville, Victoria 3052
Australia
Universite de Montreal - Centre Hospitalier Universitaire Sainte-Justine
Montreal, Quebec H3T 1C5
Canada
Irccs Ospedale Gaslini Di Genova
Genova, 16147
Italy
Osaka City General Hospital
Osaka, 534-0021
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, 200642
Romania
Seoul National University Hospital
Seoul,
South Korea
Pusan National University Yangsan Hospital
Yangsan, 50612
South Korea
University Hospitals Bristol NHS Foundation Trust - Bristol Royal Hospital for Children
Bristol,
United Kingdom