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NCT00483379 · ClinicalTrials.gov registry record · Phase 4
High Dose or High Dose Frequency Study of Alglucosidase Alfa
A Phase 4 study, sponsored by Genzyme, a Sanofi Company.
- Completed
- Registry status
- Phase 4
- Development phase
- 13
- Enrollment target
NCT00483379 is a Phase 4 study that has completed, run by Genzyme, a Sanofi Company. The registered enrollment target is 13 participants.
The verdict
NCT00483379, a Phase 4 study, has completed, sponsored by Genzyme, a Sanofi Company.
- COMPLETED
- Registry status
- Phase 4
- Development phase
- 13 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 exploratory study is to evaluate the safety and efficacy of alternative dosing regimens of alglucosidase alfa in patients with Pompe disease who have not demonstrated an optimal response to the standard dosing regimen of 20 mg/kg every other week after a minimum of 6 months treatment immediately prior to study entry.
Interventions
- BIOLOGICAL alglucosidase alfa
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 13 participants |
| Start Date | 2007-05 |
| Est. Completion | 2010-07 |
| Phase | Phase 4 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT00483379
The ClinicalTrials.gov registry entry for NCT00483379 describes a study currently listed as completed, categorized as Phase 4. The registered enrollment target is 13 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 alglucosidase alfa is the first listed.
NCT00483379 reports 0 study locations.
Frequently Asked Questions
What is clinical trial NCT00483379 about?
NCT00483379 is a clinical study titled "High Dose or High Dose Frequency Study of Alglucosidase Alfa". 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 NCT00483379?
This trial is currently completed. It is a Phase 4 study. The enrollment target is 13 participants. The study started on 2007-05. Estimated completion is 2010-07.
What interventions are being tested in trial NCT00483379?
The interventions under investigation include: alglucosidase alfa (BIOLOGICAL).
Who is sponsoring clinical trial NCT00483379?
This trial is sponsored by Genzyme, a Sanofi Company, which has 196 total clinical trials registered on ClinicalTrials.gov.
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