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NCT00074932 · ClinicalTrials.gov registry record · NA
Expanded Access Use of Myozyme (Alglucosidase Alfa) in Patients With Late-onset Pompe Disease
A NA study, sponsored by Genzyme, a Sanofi Company.
- Completed
- Registry status
- NA
- Development phase
- 9
- Enrollment target
NCT00074932 is a NA study that has completed, run by Genzyme, a Sanofi Company. The registered enrollment target is 9 participants.
The verdict
NCT00074932, a NA study, has completed, sponsored by Genzyme, a Sanofi Company.
- COMPLETED
- Registry status
- NA
- Development phase
- 9 participants
- Enrollment target
Study 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 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.
Interventions
- BIOLOGICAL Myozyme
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 9 participants |
| Start Date | 2004-11 |
| Est. Completion | 2006-12 |
| Phase | NA |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT00074932
The ClinicalTrials.gov registry entry for NCT00074932 describes a study currently listed as completed, categorized as NA. The registered enrollment target is 9 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Genzyme, a Sanofi Company, which has 196 total studies on file at ClinicalTrials.gov.
The record links to 0 conditions, and to 1 intervention - of which Myozyme is the first listed.
NCT00074932 reports 0 study locations.
Frequently Asked Questions
What is clinical trial NCT00074932 about?
NCT00074932 is a clinical study titled "Expanded Access Use of Myozyme (Alglucosidase Alfa) in Patients With Late-onset Pompe Disease". 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 ...
What is the current status of trial NCT00074932?
This trial is currently completed. It is a NA study. The enrollment target is 9 participants. The study started on 2004-11. Estimated completion is 2006-12.
What interventions are being tested in trial NCT00074932?
The interventions under investigation include: Myozyme (BIOLOGICAL).
Who is sponsoring clinical trial NCT00074932?
This trial is sponsored by Genzyme, a Sanofi Company, which has 196 total clinical trials registered on ClinicalTrials.gov.
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