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

NCT ID: NCT04455230

Last Updated: 2023-12-07

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1/PHASE2

Total Enrollment

3 participants

Study Classification

INTERVENTIONAL

Study Start Date

2020-09-08

Study Completion Date

2023-09-28

Brief Summary

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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

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Conditions

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Fabry Disease Lysosomal Storage Diseases

Keywords

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Gene Therapy

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

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
Primary Study Purpose

OTHER

Blinding Strategy

NONE

Study Groups

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Participants who have received gene therapy vector (FLT190)

Group Type EXPERIMENTAL

FLT190

Intervention Type GENETIC

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

Interventions

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FLT190

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

Intervention Type GENETIC

Eligibility Criteria

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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
Minimum Eligible Age

18 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No

Sponsors

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Spur Therapeutics

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Charité - Universitätsmedizin Berlin

Berlin, , Germany

Site Status

Royal Free London

London, , United Kingdom

Site Status

Countries

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Germany United Kingdom

Other Identifiers

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FLT190-02

Identifier Type: -

Identifier Source: org_study_id