NCT01697319

Efficacy and Safety Study of BMN 110 for Morquio A Syndrome Patients Who Have Limited Ambulation

A Phase 2, Open-label, Multinational Study to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome) Who Have Limited Ambulation

• Status: Terminated
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 16 (actual)
• Sponsor: BioMarin Pharmaceutical · Industry
• Sex: All
• Age: 5 Years
• Healthy volunteers: Not accepted

Summary

The primary objective of this study is to evaluate the effect of 2.0 mg/kg/week BMN 110 in a patient population that has limited ambulation, in a period of up to 144 weeks.

Detailed description

Effect is defined by the following key domains: * Upper extremity function and dexterity * Mobility

Conditions

• Mucopolysaccharidosis IVA
• Morquio A Syndrome
• MPS IVA

Interventions

Type	Name	Description
DRUG	BMN 110	Drug will be delivered through a 4 hour (approximate) IV infusion at a dosage amount of 2.0 mg/kg/week for up to 144 weeks of treatment.

Timeline

• Start date: 2012-08-01
• Primary completion: 2014-10-01
• Completion: 2014-10-01
• First posted: 2012-10-02
• Last updated: 2016-01-12
• Results posted: 2016-01-12

Locations

7 sites across 3 countries: United States, Germany, United Kingdom