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

NCT04455230 · Status: COMPLETED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 3

Last updated 2023-12-07

No results posted yet for this study

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

Interventions

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).

Sponsors & Collaborators

  • Spur Therapeutics

    lead INDUSTRY

Study Design

Allocation
NA
Purpose
OTHER
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2020-09-08
Primary Completion
2023-09-28
Completion
2023-09-28
FDA Drug
Yes

Countries

  • Germany
  • United Kingdom

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04455230 on ClinicalTrials.gov