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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: Completed NA study, sponsored by Genzyme, a Sanofi Company.
NCT00074932 is a NA study that has completed, run by Genzyme, a Sanofi Company. The registered enrollment target is 9 participants, below the 1,404-participant average among 58,714 other NA trials with a reported enrollment target (99% lower). According to ClinicalTrials.gov, the official US trial registry.
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 |
What the finished NCT00074932 record still lists
NCT00074932 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 9 participants, a relatively small participant target, below the 1,404-participant average among 58,714 other NA trials with a reported enrollment target (99% lower).
The record links to 0 conditions, and to 1 intervention - of which Myozyme is the first listed.
NCT00074932 does not publish any study locations in the registry export this page uses.
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.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT00074932's enrollment target sits among peer trials
9 1975th of 2000 higher than 25 of 2,000 other NA trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other NA trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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