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NCT00059280 · ClinicalTrials.gov registry record · Phase 2
A Study of the Safety and Efficacy of rhGAA in Patients With Infantile-onset Pompe Disease
A Phase 2 study, sponsored by Genzyme, a Sanofi Company.
- Completed
- Registry status
- Phase 2
- Development phase
- 16
- Enrollment target
NCT00059280: Completed Phase 2 study, sponsored by Genzyme, a Sanofi Company.
NCT00059280 is a Phase 2 study that has completed, run by Genzyme, a Sanofi Company. The registered enrollment target is 16 participants, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (88% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT00059280, a Phase 2 study, has completed, sponsored by Genzyme, a Sanofi Company.
- COMPLETED
- Registry status
- Phase 2
- Development phase
- 16 participants
- Enrollment target
Study Summary
Pompe disease (also known as glycogen storage disease type II, "GSD-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. This study is being conducted to evaluate the safety and effectiveness of recombinant human acid alpha-glucosidase (rhGAA) as a potential enzyme replacement therapy for Pompe disease. Patients diagnosed with infantile-onset Pompe disease who are less than or equal to 6 months old will be studied.
Interventions
- BIOLOGICAL Myozyme
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 16 participants |
| Start Date | 2003-04 |
| Est. Completion | 2005-09 |
| Phase | Phase 2 |
What the finished NCT00059280 record still lists
NCT00059280 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 16 participants, a relatively small participant target, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (88% lower).
The record links to 0 conditions, and to 1 intervention - of which Myozyme is the first listed.
NCT00059280 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT00059280 about?
NCT00059280 is a clinical study titled "A Study of the Safety and Efficacy of rhGAA in Patients With Infantile-onset Pompe Disease". Pompe disease (also known as glycogen storage disease type II, "GSD-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 lyso...
What is the current status of trial NCT00059280?
This trial is currently completed. It is a Phase 2 study. The enrollment target is 16 participants. The study started on 2003-04. Estimated completion is 2005-09.
What interventions are being tested in trial NCT00059280?
The interventions under investigation include: Myozyme (BIOLOGICAL).
Who is sponsoring clinical trial NCT00059280?
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 NCT00059280's enrollment target sits among peer trials
16 1908th of 2000 higher than 82 of 2,000 other Phase 2 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 2 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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