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

  • STATUS
    Recruiting
  • End date
    Dec 5, 2026
  • participants needed
    50
  • sponsor
    Freeline Therapeutics
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Updated on 16 February 2024
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deficiency
gene therapy
glycosphingolipids
breakdown
fabry's disease

Summary

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.

Details
Condition Fabry's Disease, Lysosomal Storage Disease
Age 18years - 100years
Treatment FLT190
Clinical Study IdentifierNCT04455230
SponsorFreeline Therapeutics
Last Modified on16 February 2024

Eligibility

 Yes No Not Sure

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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