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Alglucosidase Alfa Temporary Access Program

Information source: Sanofi
ClinicalTrials.gov processed this data on August 23, 2015
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: Approved for marketing

Sponsored by: Genzyme, a Sanofi Company

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


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


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


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

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

Decatur, Georgia, United States

Boise, Idaho, United States

Chicago, Illinois, United States

Evansville, Indiana, United States

Iowa City, Iowa, United States

Kansas City, Kansas, United States

Louisville, Kentucky, United States

New Orleans, Louisiana, United States

Shreveport, Louisiana, United States

Augusta, Maine, United States

Baltimore, Maryland, United States

Boston, Massachusetts, United States

Springfield, Massachusetts, United States

Detroit, Michigan, United States

Grand Rapids, Michigan, United States

Lake Orion, Michigan, United States

Rochester Hills, Michigan, United States

St. Joseph, Michigan, United States

Minneapolis, Minnesota, United States

Missoula, Montana, United States

Omaha, Nebraska, United States

Midland Park, New Jersey, United States

Albuquerque, New Mexico, United States

Albany, New York, United States

Corning, New York, United States

Manhasset, New York, United States

New York, New York, United States

Asheville, North Carolina, United States

Durham, North Carolina, United States

Jacksonville, North Carolina, United States

Cincinnati, Ohio, United States

Cleveland, Ohio, United States

Greenville, Ohio, United States

Toledo, Ohio, United States

Oklahoma City, Oklahoma, United States

Tulsa, Oklahoma, United States

Portland, Oregon, United States

Philadelphia, Pennsylvania, United States

Pittsburgh, Pennsylvania, United States

Greenville, South Carolina, United States

West Colombia, South Carolina, United States

Houston, Texas, United States

San Antonio, Texas, United States

Waco, Texas, United States

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: February 4, 2014

Page last updated: August 23, 2015

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