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NCT04455230 Clinical Trial

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

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

Clinical Trial Details — Status: Completed

Administrative data
NCT number NCT04455230
Other study ID # FLT190-02
Secondary ID
Status Completed
Phase Phase 1/Phase 2
First received
Last updated
Start date September 8, 2020
Est. completion date September 28, 2023

Study information
Verified date December 2023
Source Freeline Therapeutics
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial 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.

Recruitment information / eligibility
Status Completed
Enrollment 3
Est. completion date September 28, 2023
Est. primary completion date September 28, 2023
Accepts healthy volunteers No
Gender Male
Age group 18 Years and older
Eligibility 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: - N/A

Study Design

Related Conditions & MeSH terms

Intervention

Genetic:
• FLT190
FLT190 is a recombinant adeno-associated viral (AAV) vector (AAVS3) containing the human aGLA gene as a single stranded (ss) deoxyribonucleic acid (DNA).

Locations
Country Name City State
Germany Charité - Universitätsmedizin Berlin Berlin
United Kingdom Royal Free London London

Sponsors (1)
Lead Sponsor Collaborator
Freeline Therapeutics

Countries where clinical trial is conducted

Germany,  United Kingdom, 

Outcome
Type Measure Description Time frame Safety issue
Primary 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
See also
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