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RECRUITING
NCT07441876
PHASE2/PHASE3

Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia

Sponsor: BioMarin Pharmaceutical

View on ClinicalTrials.gov

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

2026-07-06

Healthy Volunteers

No

Conditions

Interventions

DRUG

BMN 333

Administration: Weekly subcutaneous injection

DRUG

Vosoritide Injection [Voxzogo]

Administration: Daily subcutaneous injection

Locations (18)

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

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