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

Freeline Therapeutics

Status and phase: Completed; Phase 2; Phase 1

Conditions: Fabry Disease; Lysosomal Storage Diseases

Treatments: Genetic: FLT190

Study type: Interventional

Funder types: Industry

Identifiers

NCT04455230

FLT190-02

Details and patient eligibility

About

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.

Enrollment: 3 patients

Sex: Male

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Other

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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3 participants in 1 patient group

Participants who have received gene therapy vector (FLT190)

Experimental group

Treatment:

Genetic: FLT190