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A Prospective, Observational Study in Patients With Late-Onset Pompe Disease

Glycogen Storage Disease Type II Clinical Trial

Official title:
A Prospective, Observational Study in Patients With Late-Onset Pompe Disease

Clinical Trial Summary

Pompe disease (also known as glycogen storage disease type II, "GSD-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.

This study is being conducted to collect prospective, observational data on patients with late-onset Pompe disease. Approximately 60 subjects with late-onset Pompe disease will be enrolled.

Clinical Trial Description

n/a

Study Design

Observational Model: Cohort, Time Perspective: Prospective

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT00077662
Study type Observational
Source Sanofi
Contact
Status Completed
Phase N/A
Start date March 2004
Completion date January 2006
View Details
See also
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Completed NCT00051935 - A Study of the Safety and Pharmacokinetics of rhGAA in Siblings With Glycogen Storage Disease Type II Phase 2
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Completed NCT03687333 - Evaluate Efficacy and Safety in Chinese Patients With Infantile-Onset Pompe Disease With One Year Alglucosidase Alfa Treatment Phase 4