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

NCT04455230 | Completed Phase 1 FDA Drug

• 1 other identifier: FLT190-02
• Study Type: interventional
• Target: 3
• Locations: 2 countries
• Sites: 2

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.

Conditions

Fabry Disease; Lysosomal Storage Diseases

Keywords

Gene Therapy

Outcome Measures

Primary Outcomes (1)

• 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

Study Arms (1)

Participants who have received gene therapy vector (FLT190)

EXPERIMENTAL

Genetic: FLT190

Interventions

FLT190 GENETIC

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

Participants who have received gene therapy vector (FLT190)

Eligibility Criteria

• Age: 18 Years+
• Sex: male
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• N/A

Sponsors & Collaborators

• Spur Therapeutics: lead

Study Sites (2)

Charité - Universitätsmedizin Berlin

Berlin, Germany

Location

Royal Free London

London, United Kingdom

Location

MeSH Terms

Conditions

Fabry Disease; Lysosomal Storage Diseases

Condition Hierarchy (Ancestors)

Sphingolipidoses; Lysosomal Storage Diseases, Nervous System; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Cerebral Small Vessel Diseases; Cerebrovascular Disorders; Vascular Diseases; Cardiovascular Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolism, Inborn Errors; Lipidoses; Lipid Metabolism, Inborn Errors; Metabolic Diseases; Nutritional and Metabolic Diseases; Lipid Metabolism Disorders

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: OTHER
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: 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

Study Record Dates

• First Submitted: June 29, 2020
• First Posted: July 2, 2020
• Study Start: September 8, 2020
• Primary Completion: September 28, 2023
• Study Completion: September 28, 2023
• Last Updated: December 7, 2023

Record last verified: 2023-12

Data Sharing

• IPD Sharing: Will not share

Locations

GB (1); DE (1)