BMN 701 Phase 3 in rhGAA Exposed Subjects With Late Onset Pompe Disease (INSPIRE Study)
Status:
Terminated
Trial end date:
2016-09-12
Target enrollment:
Participant gender:
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.