Fabry Disease Clinical Trial
Official title:
A Phase 1/2, Single Dose, Dose Escalation Study of Intravenous AAV5-GLA (AMT-191) in Adult Males With Classic Fabry Disease
This is an open-label multi-center study to evaluate safety and biomarkers of efficacy of a single dose of intravenously-administered AMT-191. The study will also include exploratory functional efficacy assessments. The plan is to investigate 2 sequential dose cohorts in 3-6 Participants per cohort. Participants will be monitored for 24 hours following AMT-191 administration then follow-up study visits will continue for 24 months, during which safety, pharmacokinetics/pharmacodynamics, biomarkers, and efficacy assessments will be performed. Participants will continue receiving their regularly scheduled enzyme replacement therapy (ERT) until they meet the criteria for withdrawal.
Fabry disease (FD) is a lysosomal storage disorder resulting from the absent or deficient activity of the lysosomal enzyme, α-galactosidase A (GLA). Despite availability of an enzyme replacement therapy (ERT) to prevent the progression of the renal, cardiac, and cerebrovascular symptoms, ERT is not curative and has variable impact on the disease progression for patients. A recombinant serotype 5 based adeno-associated viral vector (AMT-191) for one-time intravenous (IV) administration will be investigated in this study. This recombinant AAV5-based vector contains a coding deoxyribonucleic acid (DNA) sequence for human α-galactosidase A. Delivery of AMT-191 to the systemic circulation is expected to result in a therapeutic effect by promoting the liver expression of the lysosomal enzyme GLA in plasma and leukocytes levels in patients with FD. ;
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