NCT01924845

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

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
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 24 (actual)
• Sponsor: BioMarin Pharmaceutical · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Late-onset Pompe Disease

Interventions

Type	Name	Description
DRUG	BMN 701	BMN 701 20 mg/kg for intravenous administration over approximately 4 hours every 2 weeks over a 24-week Treatment Period (total of 13 doses), and every 2 weeks over a 240-week Extension Period (up to 120 additional doses).

Timeline

• Start date: 2014-04-01
• Primary completion: 2016-09-12
• Completion: 2016-09-12
• First posted: 2013-08-19
• Last updated: 2018-06-14
• Results posted: 2018-06-14

Locations

22 sites across 8 countries: United States, Belgium, France, Germany, Italy, Netherlands, Portugal, United Kingdom