Clinical trial NCT00520143: Alglucosidase Alfa Temporary Access Program
Official title: Alglucosidase Alfa Temporary Access Program
Study type: EXPANDED_ACCESS
Overall status: APPROVED_FOR_MARKETING
Conditions: Glycogen Storage Disease Type II (GSD-II)
Pompe Disease (Late-Onset)
Acid Maltase Deficiency Disease
Glycogenosis 2
Keywords: GSD-II, Pompe Disease, Glycogen Storage Disease II
Lead sponsor: Sanofi
Sponsor class: INDUSTRY
First posted: 2007-08-23
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT

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.


Eligibility 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.

Primary outcomes:
Secondary outcomes:
Other outcomes:
Locations:
city: Sheffield
country: United States
state: Alabama
city: Anchorage
country: United States
state: Alaska
city: Phoenix
country: United States
state: Arizona
city: Prescott
country: United States
state: Arizona
city: Scottsdale
country: United States
state: Arizona
city: Tucson
country: United States
state: Arizona
city: Bakersfield
country: United States
state: California
city: Beverly Hills
country: United States
state: California
city: Loma Linda
country: United States
state: California
city: Los Angeles
country: United States
state: California
city: Sacramento
country: United States
state: California
city: San Diego
country: United States
state: California
city: Stanford
country: United States
state: California
city: Aurora
country: United States
state: Colorado
city: Washington
country: United States
state: District of Columbia
city: Bradenton
country: United States
state: Florida
city: Coral Springs
country: United States
state: Florida
city: Daytona Beach
country: United States
state: Florida
city: Gainsville
country: United States
state: Florida
city: St. Petersburg
country: United States
state: Florida
city: Decatur
country: United States
state: Georgia
city: Boise
country: United States
state: Idaho
city: Chicago
country: United States
state: Illinois
city: Evansville
country: United States
state: Indiana
city: Iowa City
country: United States
state: Iowa
city: Kansas City
country: United States
state: Kansas
city: Louisville
country: United States
state: Kentucky
city: New Orleans
country: United States
state: Louisiana
city: Shreveport
country: United States
state: Louisiana
city: Augusta
country: United States
state: Maine
city: Baltimore
country: United States
state: Maryland
city: Boston
country: United States
state: Massachusetts
city: Springfield
country: United States
state: Massachusetts
city: Detroit
country: United States
state: Michigan
city: Grand Rapids
country: United States
state: Michigan
city: Lake Orion
country: United States
state: Michigan
city: Rochester Hills
country: United States
state: Michigan
city: St. Joseph
country: United States
state: Michigan
city: Minneapolis
country: United States
state: Minnesota
city: Missoula
country: United States
state: Montana
city: Omaha
country: United States
state: Nebraska
city: Midland Park
country: United States
state: New Jersey
city: Albuquerque
country: United States
state: New Mexico
city: Albany
country: United States
state: New York
city: Corning
country: United States
state: New York
city: Manhasset
country: United States
state: New York
city: New York
country: United States
state: New York
city: Asheville
country: United States
state: North Carolina
city: Durham
country: United States
state: North Carolina
city: Jacksonville
country: United States
state: North Carolina
city: Cincinnati
country: United States
state: Ohio
city: Cleveland
country: United States
state: Ohio
city: Greenville
country: United States
state: Ohio
city: Toledo
country: United States
state: Ohio
city: Oklahoma City
country: United States
state: Oklahoma
city: Tulsa
country: United States
state: Oklahoma
city: Portland
country: United States
state: Oregon
city: Philadelphia
country: United States
state: Pennsylvania
city: Pittsburgh
country: United States
state: Pennsylvania
city: Greenville
country: United States
state: South Carolina
city: West Colombia
country: United States
state: South Carolina
city: Houston
country: United States
state: Texas
city: San Antonio
country: United States
state: Texas
city: Waco
country: United States
state: Texas
city: Salt Lake City
country: United States
state: Utah
city: Charlottesville
country: United States
state: Virginia
city: Chesapeake
country: United States
state: Virginia
city: Newport News
country: United States
state: Virginia
city: Richmond
country: United States
state: Virginia
city: Winchester
country: United States
state: Virginia
city: Pullman
country: United States
state: Washington
city: Seattle
country: United States
state: Washington
city: Morgantown
country: United States
state: West Virginia
city: Green Bay
country: United States
state: Wisconsin
Study officials:
affiliation: Genzyme, a Sanofi Company
name: Medical Monitor
role: STUDY_DIRECTOR
References: