NCT03197766

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

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

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 121 (actual)
• Sponsor: BioMarin Pharmaceutical · Industry
• Sex: All
• Age: 5 Years – 18 Years
• Healthy volunteers: Not accepted

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.

Detailed 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.

Conditions

• Achondroplasia

Interventions

Type	Name	Description
DRUG	BMN 111	Subcutaneous injection of 15 μg/kg of BMN 111 daily
DRUG	Placebo	Subcutaneous injection of 15 μg/kg of placebo daily

Timeline

• Start date: 2016-12-12
• Primary completion: 2019-10-30
• Completion: 2019-10-30
• First posted: 2017-06-23
• Last updated: 2022-03-02
• Results posted: 2022-03-02

Locations

24 sites across 7 countries: United States, Australia, Germany, Japan, Spain, Turkey (Türkiye), United Kingdom