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ACTIVE NOT RECRUITING
NCT04554940
PHASE2

A Clinical Trial to Evaluate Safety of Vosoritide in At-risk Infants With Achondroplasia

Sponsor: BioMarin Pharmaceutical

View on ClinicalTrials.gov

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

BIOLOGICAL

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