Alglucosidase Alfa Temporary Access Program
Public ClinicalTrials.gov record NCT00520143. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
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.
Study identification
- NCT ID
- NCT00520143
- Recruitment status
- Approved For Marketing
- Study type
- Expanded access
- Phase
- Not listed
- Enrollment
- Not listed
Conditions and interventions
Conditions
Interventions
Biological
Eligibility (public fields only)
- Age range
- 18 Years and older
- Sex
- All
- Healthy volunteers
- Not listed
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Not listed
- Primary completion
- Not listed
- Completion
- Not listed
- Last update posted
- Feb 6, 2014
United States locations
- U.S. sites
- 74
- U.S. states
- 37
- U.S. cities
- 74
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Not listed | Sheffield | Alabama | — | — |
| Not listed | Anchorage | Alaska | — | — |
| Not listed | Phoenix | Arizona | — | — |
| Not listed | Prescott | Arizona | — | — |
| Not listed | Scottsdale | Arizona | — | — |
| Not listed | Tucson | Arizona | — | — |
| Not listed | Bakersfield | California | — | — |
| Not listed | Beverly Hills | California | — | — |
| Not listed | Loma Linda | California | — | — |
| Not listed | Los Angeles | California | — | — |
| Not listed | Sacramento | California | — | — |
| Not listed | San Diego | California | — | — |
| Not listed | Stanford | California | — | — |
| Not listed | Aurora | Colorado | — | — |
| Not listed | Washington D.C. | District of Columbia | — | — |
| Not listed | Bradenton | Florida | — | — |
| Not listed | Coral Springs | Florida | — | — |
| Not listed | Daytona Beach | Florida | — | — |
| Not listed | Gainsville | Florida | — | — |
| Not listed | St. Petersburg | Florida | — | — |
| Not listed | Decatur | Georgia | — | — |
| Not listed | Boise | Idaho | — | — |
| Not listed | Chicago | Illinois | — | — |
| Not listed | Evansville | Indiana | — | — |
| Not listed | Iowa City | Iowa | — | — |
| Not listed | Kansas City | Kansas | — | — |
| Not listed | Louisville | Kentucky | — | — |
| Not listed | New Orleans | Louisiana | — | — |
| Not listed | Shreveport | Louisiana | — | — |
| Not listed | Augusta | Maine | — | — |
| Not listed | Baltimore | Maryland | — | — |
| Not listed | Boston | Massachusetts | — | — |
| Not listed | Springfield | Massachusetts | — | — |
| Not listed | Detroit | Michigan | — | — |
| Not listed | Grand Rapids | Michigan | — | — |
| Not listed | Lake Orion | Michigan | — | — |
| Not listed | Rochester Hills | Michigan | — | — |
| Not listed | Saint Joseph | Michigan | — | — |
| Not listed | Minneapolis | Minnesota | — | — |
| Not listed | Missoula | Montana | — | — |
| Not listed | Omaha | Nebraska | — | — |
| Not listed | Midland Park | New Jersey | — | — |
| Not listed | Albuquerque | New Mexico | — | — |
| Not listed | Albany | New York | — | — |
| Not listed | Corning | New York | — | — |
| Not listed | Manhasset | New York | — | — |
| Not listed | New York | New York | — | — |
| Not listed | Asheville | North Carolina | — | — |
| Not listed | Durham | North Carolina | — | — |
| Not listed | Jacksonville | North Carolina | — | — |
| Not listed | Cincinnati | Ohio | — | — |
| Not listed | Cleveland | Ohio | — | — |
| Not listed | Greenville | Ohio | — | — |
| Not listed | Toledo | Ohio | — | — |
| Not listed | Oklahoma City | Oklahoma | — | — |
| Not listed | Tulsa | Oklahoma | — | — |
| Not listed | Portland | Oregon | — | — |
| Not listed | Philadelphia | Pennsylvania | — | — |
| Not listed | Pittsburgh | Pennsylvania | — | — |
| Not listed | Greenville | South Carolina | — | — |
| Not listed | West Colombia | South Carolina | — | — |
| Not listed | Houston | Texas | — | — |
| Not listed | San Antonio | Texas | — | — |
| Not listed | Waco | Texas | — | — |
| Not listed | Salt Lake City | Utah | — | — |
| Not listed | Charlottesville | Virginia | — | — |
| Not listed | Chesapeake | Virginia | — | — |
| Not listed | Newport News | Virginia | — | — |
| Not listed | Richmond | Virginia | — | — |
| Not listed | Winchester | Virginia | — | — |
| Not listed | Pullman | Washington | — | — |
| Not listed | Seattle | Washington | — | — |
| Not listed | Morgantown | West Virginia | — | — |
| Not listed | Green Bay | Wisconsin | — | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT00520143, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Feb 6, 2014 · Synced Oct 22, 2026
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Open the official record
The complete public registry record, listed eligibility criteria, and available contact information for NCT00520143 live on ClinicalTrials.gov.