NCT04455230

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

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

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 3 (actual)
• Sponsor: Spur Therapeutics · Industry
• Sex: Male
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

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.

Conditions

• Fabry Disease
• Lysosomal Storage Diseases

Interventions

Type	Name	Description
GENETIC	FLT190	FLT190 is a recombinant adeno-associated viral (AAV) vector (AAVS3) containing the human αGLA gene as a single stranded (ss) deoxyribonucleic acid (DNA).

Timeline

• Start date: 2020-09-08
• Primary completion: 2023-09-28
• Completion: 2023-09-28
• First posted: 2020-07-02
• Last updated: 2023-12-07

Locations

2 sites across 2 countries: Germany, United Kingdom

Regulatory

• FDA-regulated drug study