Expanded Access Use of Myozyme (Alglucosidase Alfa) in Patients With Late-onset Pompe Disease
Public ClinicalTrials.gov record NCT00074932. 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 protocol is to provide enzyme replacement therapy with alglucosidase alfa on an expanded access basis, to severely affected patients with late-onset Pompe disease for whom there is no alternative treatment and who do not meet the clinical characteristics described in the inclusion criteria for participation in other Genzyme Corporation-sponsored studies currently enrolling patients with late-onset Pompe disease.
Study identification
- NCT ID
- NCT00074932
- Recruitment status
- Completed
- Study type
- Interventional
- Phase
- Not applicable
- Enrollment
- 9 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- Not listed
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Nov 2004 (Type not available)
- Primary completion
- Aug 2006 (Actual)
- Completion
- Dec 2006 (Actual)
- Last update posted
- Feb 6, 2014
2004 – 2006
United States locations
- U.S. sites
- 8
- U.S. states
- 6
- U.S. cities
- 8
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Colorado Health Science Center | Aurora | Colorado | — | — |
| Galichia Heart Hospital | Wichita | Kansas | — | — |
| Genzyme Medical Information | Cambridge | Massachusetts | 02142 | — |
| Freeman Health Systems | Joplin | Missouri | — | — |
| The Women's and Children's Hospital of Buffalo | Buffalo | New York | — | — |
| North Shore University Hospital | Manhasset | New York | — | — |
| Macoumb/Oakland Adult Medicine | Rochester Hills | New York | — | — |
| Riverside Regional Medical Center | Newport News | Virginia | — | — |
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 NCT00074932, 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 24, 2026
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Open the official record
The complete public registry record, listed eligibility criteria, and available contact information for NCT00074932 live on ClinicalTrials.gov.