A Long Term Follow-Up Study of Fabry Disease Subjects Treated With FLT190
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- STATUS
- Recruiting
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- End date
- Dec 5, 2026
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- participants needed
- 50
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- sponsor
- Freeline Therapeutics
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 Identifier | NCT04455230 |
| Sponsor | Freeline Therapeutics |
| Last Modified on | 19 February 2024 |
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