NCT01242111

A Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)

A Multicenter, Multinational, Open-Label, Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)

• Status: Terminated
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 20 (actual)
• Sponsor: BioMarin Pharmaceutical · Industry
• Sex: All
• Age: —
• Healthy volunteers: Not accepted

Summary

This multicenter, open-label extension study is designed to assess long-term efficacy and safety of 2.0 milligrams (mg)/kilogram(kg)/week of BMN 110 in patients diagnosed with Mucopolysaccharidosis IVA (MPS IVA). Patients with MPS IVA, who enrolled in a prior BioMarin sponsored clinical study of BMN 110 (NCT00884949; Study Identification Number MOR-002), were eligible to enroll in this study (except patients who enrolled in NCT01275066; Study Identification Number MOR-004).

Conditions

• MPS IV A
• Mucopolysaccharidosis IVA
• Morquio A Syndrome

Interventions

Type	Name	Description
DRUG	BMN 110	Patients will receive an intravenous infusion of BMN110 at 2.0mg/kg/week, over a period of approximately 4 hours per infusion, for up to 240 weeks.

Timeline

• Start date: 2010-11-01
• Primary completion: 2014-07-01
• Completion: 2014-07-01
• First posted: 2010-11-16
• Last updated: 2015-09-30
• Results posted: 2015-09-30

Locations

4 sites across 1 country: United Kingdom