NCT02055157

A Phase 2 Study of BMN 111 to Evaluate Safety, Tolerability, and Efficacy in Children With Achondroplasia

A Phase 2, Open-label, Sequential Cohort Dose-escalation Study of BMN 111 in Children With Achondroplasia

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 35 (actual)
• Sponsor: BioMarin Pharmaceutical · Industry
• Sex: All
• Age: 5 Years – 14 Years
• Healthy volunteers: Not accepted

Summary

This is a Phase 2, open-label, sequential cohort dose-escalation study of BMN 111 in children with achondroplasia. The primary objective is to assess the safety and tolerability of daily BMN 111 administered to children with achondroplasia.

Conditions

• Achondroplasia

Interventions

Type	Name	Description
DRUG	BMN 111	BMN 111 will be administered daily for 24 months in an open-label sequential dose adjustment fashion.

Timeline

• Start date: 2014-01-13
• Primary completion: 2017-10-02
• Completion: 2017-10-02
• First posted: 2014-02-05
• Last updated: 2021-01-15
• Results posted: 2021-01-15

Locations

9 sites across 4 countries: United States, Australia, France, United Kingdom