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NCT00976352 · ClinicalTrials.gov registry record · Phase 1
Safety Study of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase to Treat Pompe Disease
A Phase 1 study of Pompe Disease, sponsored by University of Florida.
- Completed
- Registry status
- Phase 1
- Development phase
- 9
- Enrollment target
- 1
- Study location
NCT00976352: Completed Phase 1 study of Pompe Disease, sponsored by University of Florida.
NCT00976352 is a Phase 1 study of Pompe Disease that has completed, run by University of Florida. The registered enrollment target is 9 participants, below the 232-participant average among 14 other Pompe Disease trials with a reported enrollment target (96% lower). The trial reports 1 study location across 1 state. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT00976352, a Phase 1 study of Pompe Disease, has completed, sponsored by University of Florida.
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 9 participants
- Enrollment target
- 1
- Study location
Study Summary
Pompe disease is an inherited condition of acid alpha-glucosidase (GAA) deficiency resulting in lysosomal accumulation of glycogen in all tissues. Glycogen accumulation leads to muscle dysfunction and profound muscle weakness. A wide spectrum of disease is characteristic and the most severe patients have cardiorespiratory failure, often fatal in the first two years of life. Researchers have developed a way to introduce the normal GAA gene into muscle cells with the expectation that the GAA protein will be produced at levels sufficient to reduce glycogen accumulation. This study will evaluate the safety of the experimental gene transfer procedure in individuals with GAA deficiency. The study will also determine what dose may be required to achieve improvement in measures of respiratory function.
Primary Outcome
Change in Adeno-associated virus (AAV) antibody level; Change in Alglucosidase alpha (GAA) Antibody level
Conditions Studied
Interventions
- DRUG rAAV1-CMV-GAA (study agent) Administration
- OTHER RMST
Study Locations (1)
Florida
- Shands at the University of Florida - Gainesville
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 9 participants |
| Start Date | 2010-09 |
| Est. Completion | 2015-12 |
| Phase | Phase 1 |
What the finished NCT00976352 record still lists
NCT00976352 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 9 participants, a relatively small participant target, below the 232-participant average among 14 other Pompe Disease trials with a reported enrollment target (96% lower).
The record links to 1 condition, with Pompe Disease appearing as the primary indexed condition, and to 2 interventions - of which rAAV1-CMV-GAA (study agent) Administration is the first listed.
NCT00976352 reports a single indexed study location in Florida.
Frequently Asked Questions
What is clinical trial NCT00976352 about?
NCT00976352 is a clinical study titled "Safety Study of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase to Treat Pompe Disease". Pompe disease is an inherited condition of acid alpha-glucosidase (GAA) deficiency resulting in lysosomal accumulation of glycogen in all tissues. Glycogen accumulation leads to muscle dysfunction and profound muscle weakness. A wide spectrum of disease is characteristic and the most severe patients...
What is the current status of trial NCT00976352?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 9 participants. The study started on 2010-09. Estimated completion is 2015-12.
What conditions does trial NCT00976352 study?
This clinical trial studies the following conditions: Pompe Disease.
What interventions are being tested in trial NCT00976352?
The interventions under investigation include: rAAV1-CMV-GAA (study agent) Administration (DRUG), RMST (OTHER).
Who is sponsoring clinical trial NCT00976352?
This trial is sponsored by University of Florida, which has 1,106 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT00976352 being conducted?
This trial has 1 study location across Florida. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
Similar trials for Pompe Disease
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT00976352's enrollment target sits among peer trials
9 12th of 14 higher than 3 of 14 other Pompe Disease trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Pompe Disease 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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