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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: Completed Phase 2 study, sponsored by Genzyme, a Sanofi Company.
NCT00025896 is a Phase 2 study that has completed, run by Genzyme, a Sanofi Company. The registered enrollment target is 8 participants, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (94% lower). According to ClinicalTrials.gov, the official US trial registry.
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 |
What the finished NCT00025896 record still lists
NCT00025896 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 8 participants, a relatively small participant target, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (94% lower).
The record links to 0 conditions, and to 1 intervention - of which recombinant human acid alpha-glucosidase (rhGAA) is the first listed.
NCT00025896 does not publish any study locations in the registry export this page uses.
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.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT00025896's enrollment target sits among peer trials
8 1990th of 2000 higher than 5 of 2,000 other Phase 2 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 2 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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