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An Extension Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia
Sponsor: BioMarin Pharmaceutical
Summary
The intent and design of this Phase 3 study is to assess BMN 111 as a therapeutic option for the treatment of children with Achondroplasia
Official title: A Phase 3, Open-Label Long-Term Extension Study to Evaluate the Safety and Efficacy of BMN 111 in Children With Achondroplasia
Key Details
Gender
All
Age Range
6 Years - Any
Study Type
INTERVENTIONAL
Enrollment
119
Start Date
2017-12-12
Completion Date
2031-06
Last Updated
2026-03-13
Healthy Volunteers
No
Conditions
Interventions
BMN 111
Subcutaneous injection of recommended dose of BMN 111 based on weight-band dosing once daily.
Locations (24)
Children's Hospital & Research Center Oakland
Oakland, California, United States
Harbor - UCLA Medical Center
Torrance, California, United States
Alfred I. duPont Hospital for Children
Wilmington, Delaware, United States
Emory University
Atlanta, Georgia, United States
Ann and Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States
Johns Hopkins University
Baltimore, Maryland, United States
University of Missouri
Columbia, Missouri, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States
Baylor College of Medicine
Houston, Texas, United States
Seattle Children's Hospital
Seattle, Washington, United States
Medical College of Wisconsin, Children's Hospital
Milwaukee, Wisconsin, United States
The Children's Hospital at Westmead
Westmead, New South Wales, Australia
Murdoch Children's Research Institute
Parkville, Victoria, Australia
Otto-von-Gericke Universitaet, Universitaetskinderklinik
Magdeburg, Germany
Universitätsklinikum Münster
Münster, Germany
Osaka University Hospital
Osaka, Japan
Saitama Children's Medical Center
Saitama, Japan
Tokushima University Hospital
Tokushima, Japan
Institut Catala de Traumatologica I Medicina de l'Esport
Barcelona, Spain
Hospital Sant Joan de Deu
Barcelona, Spain
Hospital Universitario Virgen de la Victoria
Málaga, Spain
Acibadem University School of Medicine
Istanbul, Turkey (Türkiye)
Guy's and St. Thomas NHS Foundation Trust Evelina Children's Hospital
London, United Kingdom
Sheffield Children's NHS Foundation Trust
Sheffield, United Kingdom