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NCT00051935 · ClinicalTrials.gov registry record · Phase 2
A Study of the Safety and Pharmacokinetics of rhGAA in Siblings With Glycogen Storage Disease Type II
A Phase 2 study, sponsored by Genzyme, a Sanofi Company.
- Completed
- Registry status
- Phase 2
- Development phase
- 2
- Enrollment target
NCT00051935 is a Phase 2 study that has completed, run by Genzyme, a Sanofi Company. The registered enrollment target is 2 participants.
The verdict
NCT00051935, a Phase 2 study, has completed, sponsored by Genzyme, a Sanofi Company.
- COMPLETED
- Registry status
- Phase 2
- Development phase
- 2 participants
- Enrollment target
Study Summary
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, pharmacokinetics, pharmacodynamics and efficacy of recombinant human acid alpha-glucosidase (rhGAA) as a potential enzyme replacement therapy for a pair of siblings with GSD-II. To be eligible for this study, a patient must have a confirmed diagnosis of GSD-II and have a sister or brother who also has a confirmed diagnosis of GSD-II.
Interventions
- DRUG Alglucosidase alfa
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 2 participants |
| Start Date | 2003-01 |
| Est. Completion | 2003-10 |
| Phase | Phase 2 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT00051935
The ClinicalTrials.gov registry entry for NCT00051935 describes a study currently listed as completed, categorized as Phase 2. The registered enrollment target is 2 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.
NCT00051935 reports 0 study locations.
Frequently Asked Questions
What is clinical trial NCT00051935 about?
NCT00051935 is a clinical study titled "A Study of the Safety and Pharmacokinetics of rhGAA in Siblings With 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 e...
What is the current status of trial NCT00051935?
This trial is currently completed. It is a Phase 2 study. The enrollment target is 2 participants. The study started on 2003-01. Estimated completion is 2003-10.
What interventions are being tested in trial NCT00051935?
The interventions under investigation include: Alglucosidase alfa (DRUG).
Who is sponsoring clinical trial NCT00051935?
This trial is sponsored by Genzyme, a Sanofi Company, which has 196 total clinical trials registered on ClinicalTrials.gov.
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