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NCT00053573 · ClinicalTrials.gov registry record · Phase 1
rhGAA in Patients With Infantile-onset Glycogen Storage Disease-II (Pompe Disease)
A Phase 1 study, sponsored by Genzyme, a Sanofi Company.
- Completed
- Registry status
- Phase 1
- Development phase
- 20
- Enrollment target
NCT00053573: Completed Phase 1 study, sponsored by Genzyme, a Sanofi Company.
NCT00053573 is a Phase 1 study that has completed, run by Genzyme, a Sanofi Company. The registered enrollment target is 20 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (67% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT00053573, a Phase 1 study, has completed, sponsored by Genzyme, a Sanofi Company.
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 20 participants
- Enrollment target
Study Summary
Glycogen Storage Disease Type II ("GSD-II"; also known as Pompe disease) 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 GSD-II, 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 GSD-II. Patients diagnosed with infantile-onset GSD-II who are greater than 6 months old, but less than or equal to 36 months old will be studied.
Interventions
- BIOLOGICAL Myozyme
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 20 participants |
| Start Date | 2003-02 |
| Est. Completion | 2006-11 |
| Phase | Phase 1 |
What the finished NCT00053573 record still lists
NCT00053573 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 20 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (67% lower).
The record links to 0 conditions, and to 1 intervention - of which Myozyme is the first listed.
NCT00053573 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT00053573 about?
NCT00053573 is a clinical study titled "rhGAA in Patients With Infantile-onset Glycogen Storage Disease-II (Pompe Disease)". Glycogen Storage Disease Type II ("GSD-II"; also known as Pompe disease) 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 NCT00053573?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 20 participants. The study started on 2003-02. Estimated completion is 2006-11.
What interventions are being tested in trial NCT00053573?
The interventions under investigation include: Myozyme (BIOLOGICAL).
Who is sponsoring clinical trial NCT00053573?
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 NCT00053573's enrollment target sits among peer trials
20 1629th of 2000 higher than 285 of 2,000 other Phase 1 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 1 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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