NCT01609062

Safety and Exercise Study of Two Doses of BMN 110 for Morquio A Syndrome

A Randomized, Double-Blind, Pilot Study of the Safety and Physiological Effects of Two Doses of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)

• Status: Terminated
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 25 (actual)
• Sponsor: BioMarin Pharmaceutical · Industry
• Sex: All
• Age: 7 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Mucopolysaccharidosis IVA
• Morquio A Syndrome
• MPS IVA

Interventions

Type	Name	Description
DRUG	BMN 110	Weekly IV infusions of BMN 110 at 2.0 mg/kg/week over a period of approximately 4 hours per infusion for up to 192 weeks.
DRUG	BMN 110	Weekly IV infusions of BMN 110 at 4.0 mg/kg/week over a period of approximately 4 hours per infusion for 27 weeks, and will eventually transition to 2.0 mg/kg/week for up to an additional 166 weeks.

Timeline

• Start date: 2012-04-01
• Primary completion: 2014-11-01
• Completion: 2014-11-01
• First posted: 2012-05-31
• Last updated: 2016-02-01
• Results posted: 2016-02-01

Locations

11 sites across 4 countries: United States, Canada, Germany, United Kingdom