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Clinical trial

A Phase 1/2 Open-label Study of the Safety, Tolerability, Pharmacokinetics, Pharmacodynamic and Preliminary Efficacy of BMN 701 (GILT-tagged Recombinant Human GAA) in Patients With Late-onset Pompe Disease

NCT01230801 is registered in ClinicalTrials.gov with BioMarin Pharmaceutical as lead sponsor.

NCT NCT01230801
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Registry record

As filed in ClinicalTrials.gov.

Title
A Phase 1/2 Open-label Study of the Safety, Tolerability, Pharmacokinetics, Pharmacodynamic and Preliminary Efficacy of BMN 701 (GILT-tagged Recombinant Human GAA) in Patients With Late-onset Pompe Disease
Lead sponsor
BioMarin Pharmaceutical
Phase
Phase 1/2
Overall status
Completed
Start date
January 17, 2011
Completion date
March 6, 2013
Enrollment
22 (actual)
Sites
10

Conditions

As the registry lists them; a condition with its own page is linked.

pompe disease