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

Plain-language 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, whose deficiency leads to the buildup of specific glycosphingolipids in the body. This follow-up study investigates the long-term safety and durability of αGLA activity in people who received the gene therapy product FLT190 in earlier clinical studies. The Phase 1/2 study enrolled only those previously dosed with FLT190 who gave full informed consent and could comply with 60 months (5 years) of post-treatment follow-up.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

PHASE1/PHASE2

Status

Completed

Sponsor

Spur Therapeutics

Intervention

FLT190

Start date

9/8/2020

Estimated completion

9/28/2023

Countries

Germany, United Kingdom

Related genes

Locations

• Berlin, Germany
• London, United Kingdom

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.

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