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

  • End date
    Dec 25, 2026
  • participants needed
  • sponsor
    Freeline Therapeutics
Updated on 25 January 2021
gene therapy


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.

Condition Fabry's Disease, Fabry Disease, Lysosomal Storage Disease, Fabry Disease
Treatment FLT190
Clinical Study IdentifierNCT04455230
SponsorFreeline Therapeutics
Last Modified on25 January 2021


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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

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Additional screening procedures may be conducted by the study team before you can be confirmed eligible to participate.

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If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.

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Complete your scheduled study participation activities and then you are done. You may receive summary of study results if provided by the sponsor.

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