Beyond Achondroplasia

Growing together with Clara

Stages of implementation of BMN-111 in a short summary


BMN 111 Clinical Studies in Achondroplasia

(information by: BioMarin)


1-Double-Blind, Placebo-Controlled Phase 1 Study (Protocol 111-101)

Results of a two-part, double-blind, placebo-controlled Phase 1 study (Protocol 111-101) conducted in 48 healthy adult volunteers demonstrated that systemic exposure to BMN 111, at the doses evaluated, was similar to what has been observed to cause growth in healthy and disease model animals.1-3 BMN 111 was generally well-tolerated with observed mild, transient, self-limited hypotension that was asymptomatic in the majority of cases. All adverse events were of mild severity.

2-Observational Clinical Assessment Study (Protocol 111-901)

Currently, BioMarin is also conducting a study to collect consistent baseline growth measurements in children with achondroplasia (Protocol 111-901). Children who participate in Protocol 111-901 may also be considered for enrollment in the planned Phase 2 pediatric study (Protocol 111-202) scheduled to start by Q1 2014. Participation in Protocol 111-901 is a prerequisite for enrollment in the Phase 2 pediatric study.

3-A Phase 2, Open-label, Sequential Cohort Dose-Escalation Study (111-202)

This study is evaluating the safety and tolerability of daily subcutaneous injections of BMN 111 over a 6-month period in children (ages 5-14 years old) with achondroplasia.4 Secondary objectives include an assessment of the changes in annualized growth velocity, absolute growth parameters and body proportions, as well as other medically relevant and functional aspects of achondroplasia, including sleep apnea and joint range of motion. The first child was dosed with BMN 111 in January 2014


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