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 ]

Safety evaluations by incidence of adverse events, serious adverse events, laboratory test results (urinalysis, chemistry, hematology), clinically significant changes in vital signs, physical examination, ECG and ECHO results, imaging, hip monitoring and anti-BMN 111 immunogenicity assessments.


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). ]

Safety evaluations by indicence of adverse events, serious adverse events, laboratory test results (urinaysis, chemistry, hematology), clinically significant changes in vital signs, physical examination, ECG and ECHO results, imaging, and anti-BMN 111 immunogenicity assessments.


Current Secondary Outcome: Efficacy measure [ Time Frame: 6 months and approximately 24 months ]

Efficacy will be assessed by change from baseline in height growth velocity (annualized to cm/yr), absolute growth, subject growth compared with ACH and non-ACH standardized pediatric growth curves, and change in body proportions. These will be assessed by anthropometric measurements and measurement ratios.


Original Secondary Outcome: Efficacy measure [ Time Frame: Approximately 6 months ]

Efficacy will be assessed by change from baseline in height growth velocity (annualized to cm/yr), absolute growth, subject growth compared with ACH and non-ACH standardized pediatric growth curves, and change in body proportions. These will be assessed by anthropometric measurements and measurement ratios.


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