A Long Term Follow-Up Study of Fabry Disease Subjects Treated With FLT190
- Conditions
- Lysosomal Storage DiseasesFabry Disease
- Interventions
- Genetic: FLT190
- Registration Number
- NCT04455230
- Lead Sponsor
- Freeline Therapeutics
- Brief 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- Male
- Target Recruitment
- 3
- 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.
- N/A
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Participants who have received gene therapy vector (FLT190) FLT190 -
- Primary Outcome Measures
Name Time Method Safety endpoint will be assessed by the reporting of adverse events (AEs) according to Common Terminology Criteria for Adverse Events (CTCAE) version 5.0. Entry to 5 years post dosing
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (2)
Royal Free London
🇬🇧London, United Kingdom
Charité - Universitätsmedizin Berlin
🇩🇪Berlin, Germany