NCT01924845
Late-onset Pompe Disease
The below information is taken directly from public registry websites such as ClinicalTrials.gov, EuClinicalTrials.eu, ISRCTN.com, etc. and has not been edited.
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.
All
From 18 Years
No
BMN 701
Phase 3
Interventional
24
2014-04
2018-06-14
Phoenix, Arizona, United States
Orange, California, United States
Gainesville, Florida, United States
Kansas City, Kansas, United States
Saint Louis, Missouri, United States
Durham, North Carolina, United States
Columbus, Ohio, United States
Salt Lake City, Utah, United States
Edegem, , Belgium
Garches, , France
Marseille, , France
Mainz, , Germany
München, , Germany
Münster, , Germany
Messina, ME, Italy
Milano, , Italy
Rotterdam, , Netherlands
Porto, , Portugal
Birmingham, , United Kingdom
London, , United Kingdom
London, , United Kingdom
Salford, , United Kingdom
1. has received treatment with commercial rhGAA for ≥ 48 weeks (but no more than 20% of the study population can have received treatment for ≥ 6 years).
2. has received > 80% of all scheduled treatments in the prior 48 weeks and ≥ 4 out of the prior 6 scheduled treatments.
3. has received and completed the last two infusions without a drug-related adverse event resulting in dose interruption.
4. has received last treatment of commercial rhGAA ≥ 10 and ≤ 31 days prior to anticipated initiation of treatment with BMN 701.
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