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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 is a Phase 2 study that has completed, run by Genzyme, a Sanofi Company. The registered enrollment target is 16 participants.
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 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT00125879
The ClinicalTrials.gov registry entry for NCT00125879 describes a study currently listed as completed, categorized as Phase 2. The registered enrollment target is 16 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.
NCT00125879 reports 0 study locations.
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.
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