BMN 701 Phase 3 in rhGAA Exposed Subjects With Late Onset Pompe Disease (INSPIRE Study)

Late-onset Pompe Disease Clinical Trial

Official title: A Phase 3 Switchover Study of the Efficacy and Safety of BMN 701 (GILT-tagged Recombinant Human GAA) and Long-Term Study for Extended Treatment in rhGAA Exposed Subjects With Late-onset Pompe Disease

Status Terminated

Clinical Trial Summary

Study 701-301 is a single-arm, open-label, switchover study in patients with late-onset Pompe disease who have been receiving treatment with recombinant human acid alpha-glucosidase (rhGAA) for 48 weeks or longer. Ambulatory patients who have mild to moderate respiratory impairment will switch directly to receive BMN 701 20 mg/kg by IV infusion every other week. All participants will receive active drug. No dose of existing therapy will be missed - experimental drug is started immediately.

Clinical Trial Description

n/a

Related Conditions & MeSH terms

• Glycogen Storage Disease Type II
• Late-onset Pompe Disease

• NCT number: NCT01924845
• Study type: Interventional
• Source: BioMarin Pharmaceutical
• Status: Terminated
• Phase: Phase 3
• Start date: April 2014
• Completion date: September 12, 2016