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Expanded Access Use of Myozyme (Alglucosidase Alfa) in Patients With Infantile-onset Pompe Disease

NCT00074919 · Status: APPROVED_FOR_MARKETING · Type: EXPANDED_ACCESS

Last updated 2014-02-05

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 protocol is to provide enzyme replacement therapy with rhGAA on an expanded access basis, to severely affected patients with infantile-onset Pompe disease for whom there is no alternative treatment and who do not meet the clinical characteristics described in the inclusion criteria for participation in other Genzyme Corporation-sponsored study currently enrolling patients with infantile-onset Pompe disease.

Conditions

  • Glycogen Storage Disease Type II
  • Glycogenosis 2

Interventions

BIOLOGICAL

alglucosidase alfa

20 mg/kg qow

Sponsors & Collaborators

  • Genzyme, a Sanofi Company

    lead INDUSTRY

Principal Investigators

  • Medical Monitor · Genzyme, a Sanofi Company

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2003-12-31
Primary Completion
2007-02-28
Completion
2007-02-28

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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 NCT00074919 on ClinicalTrials.gov