High Dose or High Dose Frequency Study of Alglucosidase Alfa

Status Completed

Clinical Trial Summary

Pompe disease (also known as glycogen storage disease Type II) is caused by a deficiency of a critical enzyme in the body called acid alpha-glucosidase (GAA). Normally, GAA is used by the body's cells to break down glycogen (a stored form of sugar) within specialized structures called lysosomes. In patients with Pompe disease, an excessive amount of glycogen accumulates and is stored in various tissues, especially heart and skeletal muscle, which prevents their normal function. The objective of this exploratory study is to evaluate the safety and efficacy of alternative dosing regimens of alglucosidase alfa in patients with Pompe disease who have not demonstrated an optimal response to the standard dosing regimen of 20 mg/kg every other week after a minimum of 6 months treatment immediately prior to study entry.

Clinical Trial Description

n/a

Study Design

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

Glycogen Storage Disease
Glycogen Storage Disease Type II
Glycogen Storage Disease Type II (GSD-II)
Glycogenesis 2 Acid Maltase Deficiency
Pompe Disease

Clinical Trial Details

NCT number	NCT00483379
Study type	Interventional
Source	Sanofi
Contact
Status	Completed
Phase	Phase 4
Start date	May 2007
Completion date	July 2010

View Details

See also
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