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Alglucosidase Alfa Temporary Access Program

NCT00520143 · Status: APPROVED_FOR_MARKETING · Type: EXPANDED_ACCESS

Last updated 2014-02-06

No results posted yet for this study

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 expanded access study is to provide patients with Pompe disease in the United States (US), access to alglucosidase alfa produced from a scaled up manufacturing process for a limited time until production at this scale is approved for commercial use by the Food and Drug Administration.

Conditions

  • Glycogen Storage Disease Type II (GSD-II)
  • Pompe Disease (Late-Onset)
  • Acid Maltase Deficiency Disease
  • Glycogenosis 2

Interventions

BIOLOGICAL

alglucosidase alfa (recombinant human acid alpha-glucosidase [rhGAA])

IV infusion: 20mg/kg qow

Sponsors & Collaborators

  • Genzyme, a Sanofi Company

    lead INDUSTRY

Principal Investigators

  • Medical Monitor · Genzyme, a Sanofi Company

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT00520143 on ClinicalTrials.gov