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A Long Term Follow-Up Study of Fabry Disease Subjects Treated With FLT190

A Long Term Follow-Up Study of Fabry Disease Subjects Treated With FLT190

Recruiting
18 years and older
Male
Phase N/A

Overview

Fabry disease is a rare, X-linked inborn error of glycosphingolipid metabolism caused by an abnormal gene encoding the -galactosidase A (GLA) enzyme. The GLA 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 GLA.

This clinical study aims to investigate the long-term safety and durability of GLA in patients who have been dosed with a new gene therapy product (FLT190) in earlier clinical studies.

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

Study details
    Fabry's Disease
    Lysosomal Storage Disease

NCT04455230

Freeline Therapeutics

16 February 2024

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