A Long Term Follow-Up Study of Fabry Disease Subjects Tre... | NCT04455230

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

NCT04455230

Sponsor

Spur Therapeutics

Status

Completed

Last Update Posted

Dec 7, 2023Actual

Enrollment

3Actual

Phase

Phase 1Phase 2

Conditions

Fabry Disease
Lysosomal Storage Diseases

Interventions

FLT190

Countries

Germany
United Kingdom

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Dec 2023

Overall Recruitment Status or Expanded Access Status

Completed

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Sep 8, 2020Actual

Primary Completion Date

Sep 28, 2023Actual

Completion Date

Sep 28, 2023Actual

First Submitted Date

Jun 29, 2020

First Submission Date that Met QC Criteria

Jun 29, 2020

First Posted Date

Jul 2, 2020Actual

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Dec 6, 2023

Last Update Posted Date

Dec 7, 2023Actual

Oversight Module

Has Data Monitoring Committee (DMC)

Is FDA Regulated Drug

Yes

Is FDA Regulated Device

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

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 provided

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Phase 1Phase 2

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

3Actual

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
Participants who have received gene therapy vector (FLT190)	Experimental		Genetic: FLT190

Interventions

Name	Type	Description	Arm Group Labels	Other Names
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)

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
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 Outcomes

Not provided

Other Outcomes

Not provided

Eligibility Module

Eligibility Criteria

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:

Accepts Healthy Volunteers

Sex

Male

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

18 Years

Maximum Age

Not provided

Standard Ages

AdultOlder Adult

Study Population

Not provided

Sampling Method

Not provided

Contacts/Locations Module

Central Contacts

Not provided

Overall Officials

Not provided

Locations

Facility	Status	City	State	ZIP	Country	Contacts
Charité - Universitätsmedizin Berlin		Berlin			Germany
Royal Free London

IPD Sharing Statement Module

Plan to Share IPD

Description

Not provided

Types

Not provided

Time Frame

Not provided

Access Criteria

Not provided

URL

Not provided

Release Date	Unrelease Date	Unrelease Date Unknown	Reset Date	MCP Release Number
Jun 4, 2026			Jun 29, 2026	5

Condition Browse Module

MeSH Terms

ID	Term
D000795	Fabry Disease
D016464	Lysosomal Storage Diseases

Ancestor Terms

ID	Term
D013106	Sphingolipidoses
D020140	Lysosomal Storage Diseases, Nervous System
D020739	Brain Diseases, Metabolic, Inborn
D001928	Brain Diseases, Metabolic

Browse Leaves

Not provided

Browse Branches

Not provided