RecruitingPhase 2Phase 3NCT07441876

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

A Multicenter, Randomized, Operationally Seamless Phase 2/3 Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia

Sponsor

BioMarin Pharmaceutical

Enrollment

160 participants

Start Date

Apr 20, 2026

Study Type

INTERVENTIONAL

Conditions

Achondroplasia

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.

Eligibility

Min Age: 2 YearsMax Age: 17 Years

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying a drug called BMN 333 and a drug called Vosoritide Injection [Voxzogo] for people with achondroplasia. The study is currently recruiting participants at 19 locations. People eligible for this study include aged 2 Years to 17 Years.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

DRUGBMN 333

Administration: Weekly subcutaneous injection

DRUGVosoritide Injection [Voxzogo]

Administration: Daily subcutaneous injection

Locations(19)

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

Osaka City General Hospital

Osaka, 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

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NCT07441876

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