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A Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia

Achondroplasia Clinical Trial

Official title:
A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia

Clinical Trial 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.

Clinical Trial Description

This is a Phase 3 randomized, placebo-controlled, double-blind multicenter study with approximately 110 subjects, aged 5 to < 18 years old. Subjects with documented Achondroplasia confirmed by genetic testing will have been enrolled in Study 111-901 for at least a 6-month period immediately before entering into the 111-301 study. Eligible subjects will be randomly assigned to one of two treatment groups: placebo or BMN 111 at 15 μg/kg. The route of administration is subcutaneous injection and the frequency is daily. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT03197766
Study type Interventional
Source BioMarin Pharmaceutical
Contact
Status Completed
Phase Phase 3
Start date December 12, 2016
Completion date October 30, 2019
View Details
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