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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 is a Phase 2 study that has completed, run by Genzyme, a Sanofi Company. The registered enrollment target is 16 participants.
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 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT00059280
The ClinicalTrials.gov registry entry for NCT00059280 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.
NCT00059280 reports 0 study locations.
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.
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