Fabry disease is a rare, X-linked inborn error of glycosphingolipid metabolism caused by an abnormal gene encoding the α-galactosidase A (αGLA) enzyme, whose deficiency leads to the buildup of specific glycosphingolipids in the body. This follow-up study investigates the long-term safety and durability of αGLA activity in people who received the gene therapy product FLT190 in earlier clinical studies. The Phase 1/2 study enrolled only those previously dosed with FLT190 who gave full informed consent and could comply with 60 months (5 years) of post-treatment follow-up.
The summary above is a plain-language rendering of the official record. The original English title is shown for reference.
Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.
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