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NCT00125879 · ClinicalTrials.gov registry record · Phase 2
Extension Study of Patients With Infantile-Onset Pompe Disease Who Were Previously Enrolled in Protocol AGLU01602
A Phase 2 study, sponsored by Genzyme, a Sanofi Company.
- Completed
- Registry status
- Phase 2
- Development phase
- 16
- Enrollment target
NCT00125879: Completed Phase 2 study, sponsored by Genzyme, a Sanofi Company.
NCT00125879 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
NCT00125879, 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) 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 overall objective of this study is to evaluate the long-term safety and efficacy of Myozyme treatment in patients with infantile-onset Pompe disease.
Interventions
- BIOLOGICAL Myozyme
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 16 participants |
| Start Date | 2005-06 |
| Est. Completion | 2006-12 |
| Phase | Phase 2 |
What the finished NCT00125879 record still lists
NCT00125879 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.
NCT00125879 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT00125879 about?
NCT00125879 is a clinical study titled "Extension Study of Patients With Infantile-Onset Pompe Disease Who Were Previously Enrolled in Protocol AGLU01602". 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 NCT00125879?
This trial is currently completed. It is a Phase 2 study. The enrollment target is 16 participants. The study started on 2005-06. Estimated completion is 2006-12.
What interventions are being tested in trial NCT00125879?
The interventions under investigation include: Myozyme (BIOLOGICAL).
Who is sponsoring clinical trial NCT00125879?
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 NCT00125879'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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