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

NCT02240407 · clinicaltrials.gov ↗

PHASE1

Phase

COMPLETED

Status

2

Enrollment

OTHER

Sponsor class

Conditions

Pompe Disease

Interventions

GENETIC: Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase
DRUG: Rapamycin
OTHER: saline
DRUG: Rituxan
DRUG: Diphenhydramine
DRUG: Acetaminophen
DRUG: Lidocaine
DRUG: LMX 4 Topical Cream

Sponsor

University of Florida

Collaborators

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