Safety and Exercise Study of Two Doses of BMN 110 for Morquio A Syndrome
Status:
Terminated
Trial end date:
2014-11-01
Target enrollment:
Participant gender:
Summary
The primary objective of this study was to evaluate the safety of a 2.0 mg/kg/week and a 4.0
mg/kg/week of BMN 110 in patients with Morquio A syndrome for up to 196 weeks. Secondary
objectives were to investigate the effect of the two doses on exercise capacity for up to 196
weeks. In addition, the pharmacokinetic (PK) parameters of both doses of BMN 110 was
assessed.