Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
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 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.
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.
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 |
Interested in This Trial?
Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT00976352
The ClinicalTrials.gov registry entry for NCT00976352 describes a study currently listed as completed, categorized as Phase 1. The registered enrollment target is 9 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 232-participant average among 14 other Pompe Disease trials with a reported enrollment target (96% lower). The listed sponsor is University of Florida, which has 1,106 total studies on file at ClinicalTrials.gov.
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 1 study location spanning 1 distinct geographic area - top geographies include 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.
-
A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)
ACTIVE NOT RECRUITING · Phase 1
-
AAV2/8-LSPhGAA (ACTUS-101) in Late-Onset Pompe Disease
ACTIVE NOT RECRUITING · Phase 1
-
Safety and Efficacy Evaluation of Repeat neoGAA Dosing in Late Onset Pompe Disease Patients.
COMPLETED · Phase 1
-
Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD)
RECRUITING · Phase 2
-
Cognitive and Neurological Pathologies in Pompe Disease
RECRUITING
-
Determination of CRIM Status and Longitudinal Follow-up of Individuals With Pompe Disease
RECRUITING
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.