Clinical Trial: A Phase 2 Study of BMN 111 to Evaluate Safety, Tolerability, and Efficacy in Children With Achondroplasia
Study Status: Enrolling by invitation
Recruit Status: Enrolling by invitation
Study Type: Interventional
Official Title: A Phase 2, Open-label, Sequential Cohort Dose-escalation Study of BMN 111 in Children With Achondroplasia
Brief 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.
Detailed Summary:
Sponsor: BioMarin Pharmaceutical
Current Primary Outcome: Safety Measures [ Time Frame: 6 months and approximately 24 months ]
Original Primary Outcome: Safety Measures [ Time Frame: Approximately 6 months. Following completion of study (Month 6), a safety follow up visit will be conducted (Month 7). ]
Current Secondary Outcome: Efficacy measure [ Time Frame: 6 months and approximately 24 months ]
Original Secondary Outcome: Efficacy measure [ Time Frame: Approximately 6 months ]
Information By: BioMarin Pharmaceutical
Dates:
Date Received: April 18, 2013
Date Started: January 2014
Date Completion: January 2019
Last Updated: February 5, 2016
Last Verified: February 2016