Clinical Trial: A Study to Evaluate Long-Term Safety, Tolerability, & Efficacy of BMN 111 in Children With Achondroplasia (ACH)
Study Status: Enrolling by invitation
Recruit Status: Enrolling by invitation
Study Type: Interventional
Official Title: A Phase 2, Open-Label, Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of BMN 111 in Children With Achondroplasia
Brief Summary: This is a Phase 2, Open-Label, Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of BMN 111 in Children with Achondroplasia. The primary objective is to evaluate the long-term safety and tolerability of daily SC injections of BMN 111 in children with ACH who have completed two years of treatment in the 111-202 study.
Detailed Summary:
Sponsor: BioMarin Pharmaceutical
Current Primary Outcome: Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability] [ Time Frame: 260 Weeks ]
- Number of study participants with treatment-emergent adverse events.
- Number of study participants with treatment-emergent serious adverse events
Original Primary Outcome: Same as current
Current Secondary Outcome:
- Growth Velocity [Efficacy] [ Time Frame: 260 Weeks ]Annualized growth velocity (cm/yr)
- Growth Parameters (Efficacy) [ Time Frame: 260 weeks ]Height standard score (Z-score)
- Body Proportions (Efficacy) [ Time Frame: 260 weeks ]Upper-to-lower body segment ratio
Original Secondary Outcome: Same as current
Information By: BioMarin Pharmaceutical
Dates:
Date Received: February 12, 2016
Date Started: January 2016
Date Completion: December 2022
Last Updated: March 25, 2016
Last Verified: March 2016