Conditions: Late-onset Pompe Disease
Allocation: NA | Intervention model: SINGLE_GROUP
Enrollment: 24
Healthy volunteers: no

Brief 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.

Detailed description:

Arms:
arm 1: BMN 701 IV Infusion 20mg/kg every 2 weeks for 24 weeks followed by an optional extension of 240 weeks (total duration of therapy 264 weeks)

Interventions: intervention 1: BMN 701
Intervention descriptions: intervention 1: 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).

Protocol document: