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