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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: Completed Phase 4 study, sponsored by Genzyme, a Sanofi Company.
NCT00483379 is a Phase 4 study that has completed, run by Genzyme, a Sanofi Company. The registered enrollment target is 13 participants, below the 518-participant average among 9,235 other Phase 4 trials with a reported enrollment target (97% lower). According to ClinicalTrials.gov, the official US trial registry.
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.
Primary Outcome
Participants were enrolled based on clinical decline or sub-optimal clinical response in cardiac, respiratory and/or motor function parameters pre-study while on standard treatment. Each participant was evaluated at Week 52 for change from baseline in the criteria that declined; respiratory decline as measured by change in ventilator use is summarized in this outcome. Ventilator use might have improved (less use of ventilator support), had no change, or worsened (more use of ventilator support).
Interventions
- BIOLOGICAL alglucosidase alfa
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 13 participants |
| Start Date | 2007-05 |
| Est. Completion | 2010-07 |
| Phase | Phase 4 |
What the finished NCT00483379 record still lists
NCT00483379 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 13 participants, a relatively small participant target, below the 518-participant average among 9,235 other Phase 4 trials with a reported enrollment target (97% lower).
The record links to 0 conditions, and to 1 intervention - of which alglucosidase alfa is the first listed.
NCT00483379 does not publish any study locations in the registry export this page uses.
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.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT00483379's enrollment target sits among peer trials
13 1910th of 2000 higher than 88 of 2,000 other Phase 4 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 4 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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