Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
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 is a Phase 1 study that has completed, run by Genzyme, a Sanofi Company. The registered enrollment target is 20 participants.
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 |
Interested in This Trial?
Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT00053573
The ClinicalTrials.gov registry entry for NCT00053573 describes a study currently listed as completed, categorized as Phase 1. The registered enrollment target is 20 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.
NCT00053573 reports 0 study locations.
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
Explore more on PlainTrial
Read our methodology - how this data is sourced, computed, and verified.
Related
Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This page mirrors this trial's own ClinicalTrials.gov registry record, live from the dataset. See our editorial standards & corrections policy, the methodology behind these numbers, or report a data error.