Fabry Disease Clinical Trial
Official title:
A Multicenter, Long-term, Follow-up Study to Investigate the Safety and Durability of Response Following Dosing of an Adeno-associated Viral Vector (FLT190) in Subjects With Fabry Disease
Fabry disease is a rare, X-linked inborn error of glycosphingolipid metabolism caused by an abnormal gene encoding the α-galactosidase A (αGLA) enzyme. The αGLA 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 αGLA. This clinical study aims to investigate the long-term safety and durability of αGLA in patients who have been dosed with a new gene therapy product (FLT190) in earlier clinical studies.
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| Status | Clinical Trial | Phase | |
|---|---|---|---|
| Recruiting |
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N/A | |
| Completed |
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Phase 3 | |
| Completed |
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Phase 2 | |
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Phase 3 | |
| Withdrawn |
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Phase 4 | |
| Recruiting |
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Podocyturia - Predictor of Renal Dysfunction in Fabry Nephropathy
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N/A | |
| Completed |
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Sleepiness and Sleep-disordered Breathing in Fabry Disease. A Prospective Cohort Study.
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N/A | |
| Recruiting |
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N/A | |
| Completed |
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Phase 1 | |
| Completed |
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Phase 3 | |
| Completed |
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Dosing Study of Replagal in Patients With Fabry Disease
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Phase 2 | |
| Completed |
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Ophthalmic Findings During 10-year Enzyme Substitution of Danish Fabry Patients.
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Phase 4 | |
| Recruiting |
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Autoimmune and Inflammatory Response Biomarkers in Fabry Disease
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| Recruiting |
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| Active, not recruiting |
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N/A | |
| Terminated |
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Phase 2 | |
| Active, not recruiting |
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Open Label Extension Study of 1 mg/kg Pegunigalsidase Alfa Every 2 Weeks in Patients With Fabry Disease
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Phase 3 | |
| Recruiting |
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Assess Urine Biomarkers to Predict Nephropathy in Fabry Disease
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| Recruiting |
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