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Re-administration of Intramuscular AAV9 in Patients With Late-Onset Pompe Disease

Phase 1
Completed
Conditions
Pompe Disease
Interventions
Genetic: Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase
Other: saline
Drug: Rapamycin
Drug: Rituxan
Drug: Diphenhydramine
Drug: Acetaminophen
Drug: Lidocaine
Drug: LMX 4 Topical Cream
First Posted Date
2014-09-15
Last Posted Date
2022-04-05
Lead Sponsor
University of Florida
Target Recruit Count
2
Registration Number
NCT02240407
Locations
🇺🇸

Clinical and Translational Research Building (CTRB), University of Florida, Gainesville, Florida, United States

Safety Study of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase to Treat Pompe Disease

Phase 1
Completed
Conditions
Pompe Disease
Interventions
Drug: rAAV1-CMV-GAA (study agent) Administration
Other: RMST
First Posted Date
2009-09-14
Last Posted Date
2018-09-14
Lead Sponsor
University of Florida
Target Recruit Count
9
Registration Number
NCT00976352
Locations
🇺🇸

Shands at the University of Florida, Gainesville, Florida, United States

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