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A Clinical Trial to Evaluate Safety of Vosoritide in At-risk Infants With Achondroplasia
Sponsor: BioMarin Pharmaceutical
Summary
Study 111-209 is a Phase 2 randomized, open-label clinical trial of BMN 111 in infants and young children with a diagnosis of Achondroplasia at a heightened risk of requiring cervicomedullary decompression surgery
Official title: A Randomized, Controlled, Open-label Clinical Trial With an Open-label Extension to Investigate the Safety of Vosoritide in Infants and Young Children With Achondroplasia at Risk of Requiring Cervicomedullary Decompression Surgery
Key Details
Gender
All
Age Range
0 Months - 12 Months
Study Type
INTERVENTIONAL
Enrollment
20
Start Date
2020-10-10
Completion Date
2027-12
Last Updated
2026-03-13
Healthy Volunteers
No
Conditions
Interventions
vosoritide
Subcutaneous injection of recommended dose of BMN 111 based on weight-band dosing once daily.
Locations (3)
Murdoch Children's Research Institute
Parkville, Victoria, Australia
Guy's and St. Thomas NHS Foundation Trust Evelina Children's Hospital
London, United Kingdom
Sheffield Children's NHS Foundation Trust
Sheffield, United Kingdom