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NCT00025896 · ClinicalTrials.gov registry record · Phase 2
Safety and Efficacy of Recombinant Human Acid Alpha-Glucosidase in the Treatment of Classical Infantile Pompe Disease
A Phase 2 study, sponsored by Genzyme, a Sanofi Company.
- Completed
- Registry status
- Phase 2
- Development phase
- 8
- Enrollment target
NCT00025896 is a Phase 2 study that has completed, run by Genzyme, a Sanofi Company. The registered enrollment target is 8 participants.
The verdict
NCT00025896, a Phase 2 study, has completed, sponsored by Genzyme, a Sanofi Company.
- COMPLETED
- Registry status
- Phase 2
- Development phase
- 8 participants
- Enrollment target
Study Summary
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 infants with severe cases of Pompe disease (called Classical Infantile Pompe disease), an excessive amount of glycogen accumulates and is stored in various tissues, especially heart, skeletal muscle, and liver, which prevents their normal function. This study being conducted to evaluate the safety and effectiveness of recombinant human acid alpha-glucosidase (rhGAA) as a potential enzyme replacement therapy for Pompe disease. Patients diagnosed with Classical Infantile Pompe disease who have a small, but inactive, amount of natural GAA enzyme present in their bodies (called Cross-Reacting Immunologic Material-Positive or "CRIM (+)" patients), will be studied.
Interventions
- DRUG recombinant human acid alpha-glucosidase (rhGAA)
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 8 participants |
| Start Date | 2001-05 |
| Est. Completion | 2002-09 |
| Phase | Phase 2 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT00025896
The ClinicalTrials.gov registry entry for NCT00025896 describes a study currently listed as completed, categorized as Phase 2. The registered enrollment target is 8 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 recombinant human acid alpha-glucosidase (rhGAA) is the first listed.
NCT00025896 reports 0 study locations.
Frequently Asked Questions
What is clinical trial NCT00025896 about?
NCT00025896 is a clinical study titled "Safety and Efficacy of Recombinant Human Acid Alpha-Glucosidase in the Treatment of Classical Infantile Pompe Disease". 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 infants with severe cases of Pompe disease (calle...
What is the current status of trial NCT00025896?
This trial is currently completed. It is a Phase 2 study. The enrollment target is 8 participants. The study started on 2001-05. Estimated completion is 2002-09.
What interventions are being tested in trial NCT00025896?
The interventions under investigation include: recombinant human acid alpha-glucosidase (rhGAA) (DRUG).
Who is sponsoring clinical trial NCT00025896?
This trial is sponsored by Genzyme, a Sanofi Company, which has 196 total clinical trials registered on ClinicalTrials.gov.
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