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Alglucosidase Alfa Temporary Access Program (ATAP)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00520143
Expanded Access Status : Approved for marketing
First Posted : August 23, 2007
Last Update Posted : February 6, 2014
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )

Brief 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.

Condition or disease Intervention/treatment
Glycogen Storage Disease Type II (GSD-II) Pompe Disease (Late-Onset) Acid Maltase Deficiency Disease Glycogenosis 2 Biological: alglucosidase alfa (recombinant human acid alpha-glucosidase [rhGAA])

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Study Type : Expanded Access
  See clinical trials of the intervention/treatment in this expanded access record.
Official Title: Alglucosidase Alfa Temporary Access Program

Intervention Details:
  • Biological: alglucosidase alfa (recombinant human acid alpha-glucosidase [rhGAA])
    IV infusion: 20mg/kg qow
    Other Name: Alglucosidase alfa

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All

Inclusion Criteria:

  • The patient or patient's legal guardian must provide signed, informed consent prior to performing any study-related procedures.
  • The patient must reside in the US.
  • The patient must have a confirmed diagnosis of Pompe disease defined as documented acid alpha-glucosidase (GAA) enzyme deficiency from any tissue source and/or GAA gene mutations.
  • The patient must have/had documented clinical signs and symptoms of Pompe disease.
  • The patient must have/had prior treatment with alglucosidase alfa produced at commercial scale OR be naive to enzyme replacement therapy (ERT) for the treatment of Pompe disease and meet at least 1 of the following criteria: require a wheelchair OR require some respiratory assistance for any number of hours (including night time) through non-invasive ventilation.
  • The patient must be capable of complying with the required program schedule of assessments.

Exclusion Criteria:

  • Females who are pregnant or lactating
  • The patient has a clinical condition unrelated to Pompe disease that would interfere with program assessments.
  • The patient is currently enrolled in any clinical studies.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00520143

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Sponsors and Collaborators
Genzyme, a Sanofi Company
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Study Director: Medical Monitor Genzyme, a Sanofi Company
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Responsible Party: Genzyme, a Sanofi Company Identifier: NCT00520143    
Other Study ID Numbers: AGLU03907
First Posted: August 23, 2007    Key Record Dates
Last Update Posted: February 6, 2014
Last Verified: February 2014
Keywords provided by Sanofi ( Genzyme, a Sanofi Company ):
GSD-II, Pompe Disease, Glycogen Storage Disease II
Additional relevant MeSH terms:
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Glycogen Storage Disease Type II
Glycogen Storage Disease
Deficiency Diseases
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Carbohydrate Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases
Nutrition Disorders