Back to resultsA Long Term Follow-Up Study of Fabry Disease Subjects Treated With FLT190
Primary Purpose
Fabry Disease, Lysosomal Storage Diseases
Status
Active
Phase
Phase 1
Locations
International
Study Type
Interventional
Intervention
FLT190
Sponsored by
About this trial
This is an interventional other trial for Fabry Disease focused on measuring Gene Therapy
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:
- N/A
Sites / Locations
- Charité - Universitätsmedizin Berlin
- Royal Free London
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Participants who have received gene therapy vector (FLT190)
Arm Description
Outcomes
Primary Outcome Measures
Safety endpoint will be assessed by the reporting of adverse events (AEs) according to Common Terminology Criteria for Adverse Events (CTCAE) version 5.0.
Secondary Outcome Measures
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT04455230
Brief Title
A Long Term Follow-Up Study of Fabry Disease Subjects Treated With FLT190
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
Study Type
Interventional
2. Study Status
Record Verification Date
February 2023
Overall Recruitment Status
Active, not recruiting
Study Start Date
September 8, 2020 (Actual)
Primary Completion Date
December 2030 (Anticipated)
Study Completion Date
December 2030 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Freeline Therapeutics
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Fabry Disease, Lysosomal Storage Diseases
Keywords
Gene Therapy
7. Study Design
Primary Purpose
Other
Study Phase
Phase 1, Phase 2
Interventional Study Model
Single Group Assignment
Model Description
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
Masking
None (Open Label)
Allocation
N/A
Enrollment
50 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Participants who have received gene therapy vector (FLT190)
Arm Type
Experimental
Intervention Type
Genetic
Intervention Name(s)
FLT190
Intervention Description
FLT190 is a recombinant adeno-associated viral (AAV) vector (AAVS3) containing the human αGLA gene as a single stranded (ss) deoxyribonucleic acid (DNA).
Primary Outcome Measure Information:
Title
Safety endpoint will be assessed by the reporting of adverse events (AEs) according to Common Terminology Criteria for Adverse Events (CTCAE) version 5.0.
Time Frame
Entry to 5 years post dosing
10. Eligibility
Sex
Male
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
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:
N/A
Facility Information:
Facility Name
Charité - Universitätsmedizin Berlin
City
Berlin
Country
Germany
Facility Name
Royal Free London
City
London
Country
United Kingdom
12. IPD Sharing Statement
Plan to Share IPD
No
Learn more about this trial
A Long Term Follow-Up Study of Fabry Disease Subjects Treated With FLT190
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