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Alglucosidase Alfa Temporary Access Program

Information source: Genzyme
Information obtained from ClinicalTrials.gov on October 04, 2010
Link to the current ClinicalTrials.gov record.

Condition(s) targeted: Glycogen Storage Disease Type II (GSD-II); Pompe Disease (Late-onset); Acid Maltase Deficiency Disease; Glycogenosis 2

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

Phase: N/A

Status: Available

Sponsored by: Genzyme

Official(s) and/or principal investigator(s):
Medical Monitor, Study Director, Affiliation: Genzyme

Overall contact:
Medical Information, Phone: 800-745-4447, Email: medinfo@genzyme.com

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.

Clinical Details

Official title: Alglucosidase Alfa Temporary Access Program

Study design: N/A

Eligibility

Minimum age: 18 Years. Maximum age: N/A. Gender(s): Both.

Criteria:

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.

Locations and Contacts

Medical Information, Phone: 800-745-4447, Email: medinfo@genzyme.com

Sheffield, Alabama, United States

Anchorage, Alaska, United States

Phoenix, Arizona, United States

Prescott, Arizona, United States

Scottsdale, Arizona, United States

Tucson, Arizona, United States

Bakersfield, California, United States

Beverly Hills, California, United States

Loma Linda, California, United States

Los Angeles, California, United States

Sacramento, California, United States

San Diego, California, United States

Stanford, California, United States

Aurora, Colorado, United States

Washington, District of Columbia, United States

Bradenton, Florida, United States

Coral Springs, Florida, United States

Daytona Beach, Florida, United States

Gainsville, Florida, United States

St. Petersburg, Florida, United States

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Iowa City, Iowa, United States

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West Colombia, South Carolina, United States

Houston, Texas, United States

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Salt Lake City, Utah, United States

Charlottesville, Virginia, United States

Chesapeake, Virginia, United States

Newport News, Virginia, United States

Richmond, Virginia, United States

Winchester, Virginia, United States

Pullman, Washington, United States

Seattle, Washington, United States

Morgantown, West Virginia, United States

Green Bay, Wisconsin, United States

Additional Information


Last updated: December 11, 2009

Page last updated: October 04, 2010

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