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Clinical trial

Open-Label, Pilot Study of the Safety, Pharmacokinetics and Pharmacodynamics of Recombinant Human Acid Alpha-Glucosidase (rhGAA) as Enzyme Replacement Therapy in Siblings With Glycogen Storage Disease Type II (GSD-II).

NCT00051935 is registered in ClinicalTrials.gov with Genzyme, a Sanofi Company as lead sponsor.

NCT NCT00051935
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Registry record

As filed in ClinicalTrials.gov.

Title
Open-Label, Pilot Study of the Safety, Pharmacokinetics and Pharmacodynamics of Recombinant Human Acid Alpha-Glucosidase (rhGAA) as Enzyme Replacement Therapy in Siblings With Glycogen Storage Disease Type II (GSD-II).
Lead sponsor
Genzyme, a Sanofi Company
Phase
Phase 2
Overall status
Completed
Start date
January 31, 2003
Completion date
October 31, 2003
Enrollment
2 (actual)
Sites
1

Conditions

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

acid maltase deficiency disease, glycogen storage disease type ii, glycogenosis 2, pompe disease