A Multicenter, Long-term, Follow-up Study to Investigate the Safety and Durability of Response Following Dosing of an Adeno-associated Viral Vector (FLT190) in Subjects With Fabry Disease

FLT190-02

Fabry disease is a rare, X-linked inborn error of glycosphingolipid metabolism caused by an abnormal gene encoding the α-galactosidase A (αGLA) enzyme. The αGLA enzyme is ubiquitously expressed throughout the body and is responsible for the breakdown of glycosphingolipids, deficiency of which results in the accumulation of specific glycosphingolipids that are associated with the pathophysiology of the disease. Current treatment for Fabry disease is limited to the symptomatic management of pain, conventional management of complications, and methods to increase the availability of functional αGLA. This clinical study aims to investigate the long-term safety and durability of αGLA in patients who have been dosed with a new gene therapy product (FLT190) in earlier clinical studies.

2020-09-08

2023-09-28

2023-09-28

Completed

Early phase I

3

Inclusion Criteria: * Subjects who have previously received FLT190 * Provision of full informed consent and able to comply with all requirements of the study including long-term follow-up for 60 months (5 years) post-treatment. Exclusion Criteria: * N/A

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2023-12-07