Long-Term Follow-up of Fabry Disease Subjects Treated with ST-920
Research type
Research Study
Full title
Long-Term Follow-up of Fabry Disease Subjects who were Treated with ST-920, an AAV2/6 Human Alpha Galactosidase A Gene Therapy
IRAS ID
1005663
Contact name
Priyal Dave
Contact email
Sponsor organisation
Sangamo Therapeutics, Inc.
Eudract number
2022-001411-86
Clinicaltrials.gov Identifier
Research summary
Fabry Disease is a rare genetic disease that is caused by mutations in the GLA gene, which encodes an enzyme (protein) called alpha-Gal A. Deficiency of alpha-Gal A causes a build-up of Gb3 and lyso-Gb3, leading to kidney and brain disease, and impairment of blood vessels around the brain, with a reduced life expectancy. ST-920 is a gene therapy product that has been designed to overcome the function of the broken gene by injecting a working copy. The gene was delivered into cells using a modified virus (adeno-associated virus (AAV)) in the parent study, ST-920-201.
ST-920-LT01 is a multi-centre, long-term follow-up (LTFU) study of subjects with Fabry Disease who received a gene therapy product, ST-920 in the parent trial.
Participants dosed with ST-920 in the parent study will be eligible for enrolment onto this long-term follow up once their written consent has been obtained. The primary objective of the study is to evaluate long term safety of ST-920 by evaluating occurrence of delayed Adverse Events (AEs). The follow up period will continue for up to 5 years from ST-920 infusion.REC name
North East - York Research Ethics Committee
REC reference
22/NE/0104
Date of REC Opinion
18 Aug 2022
REC opinion
Further Information Favourable Opinion