Evaluation of Re-administration of Recombinant Adeno-Associated Virus Acid Alpha- Glucosidase (rAAV9-DES-hGAA) in Patients with Late-Onset Pompe Disease (LOPD)
The purpose of this research study is to test the safety of administration and re-administration of the acid alpha-glucosidase (GAA) gene into muscle. This is the first time this gene transfer agent has ever been tested in human subjects.
If you decide to participate in this study, you will be randomly assigned, much like the flip of a coin, to receive either the gene therapy or placebo first to the right leg and four months later to the opposite leg.
- 18-50 years old
- Have a diagnosis of Pompe disease, as defined by protein assay AND/OR DNA sequence of the acid alpha-glucosidase gene, AND clinical symptoms of the disease
- Have residual ability to complete the 10-meter walk test
- Willing to discontinue aspirin, aspirin-containing products and other drugs that may alter platelet function, 7 days prior to dosing, resuming 24 hours after the dose has been administered
- Consistently taking enzyme replacement therapy (ERT) or remain off ERT from baseline until Day 520
- United States residents only.
For additional study information, please contact Samatha Norman:
- (352) 273-8218
Can be done from home
KeywordsPompe Disease, Neurology
Principal InvestigatorManuela Corti, PT, PhD
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Studies listed on this site have been approved by a UF Institutional Review Board (IRB), which works to ensure the welfare and rights of research participants as required by federal regulations. Study listings are provided by the UF Clinical and Translational Science Institute in collaboration with UF research teams and the UF IRBs.