A Long Term Follow-Up Study of Fabry Disease Subjects Treated With FLT190
About the study
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.
Who can take part
You may be eligible to participate in the study if you meet the following criteria:
INCLUSION 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
Exclusion Criteria:
N/A
Study Locations
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How to Apply
Contact the study center to learn if this study is a good match for you.
Study’s details
Contition
Fabry Disease,Lysosomal Storage Diseases
Age (in years)
18+
Phase
Phase 1/Phase 2
Participants needed
50
Est. Completion Date
Dec 31, 2030
Treatment type
Interventional
Sponsor
Freeline Therapeutics
ClinicalTrials.gov identifier
NCT04455230
Study number
FLT190-02
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