A Phase I/II Study to Assess the Safety and Tolerability of ST-920
Research type
Research Study
Full title
A Phase I/II, Multicenter, Open-Label, Single-Dose, Dose-Ranging Study to Assess the Safety and Tolerability of ST-920, an AAV2/6 Human Alpha Galactosidase A Gene Therapy in Subjects with Fabry Disease
IRAS ID
261195
Contact name
Derralynn Arlene Hughes
Contact email
Sponsor organisation
Sangamo Therapeutics, Inc.
Eudract number
2019-000667-24
Duration of Study in the UK
1 years, 11 months, 1 days
Research summary
Fabry disease sometimes referred to as Anderson-Fabry disease, is a rare inherited lysosomal disease. In Fabry disease, an enzyme (a-galactosidase) responsible for the breakdown of waste products in the cells is deficient or absent.
Currently, the current standard of care is enzyme replacement therapy (ERT) using recombinant α-Gal A, Fabrazyme® (agalsidase beta or equivalent), or pharmacological chaperone therapy, which is available only for patients whose mutations are amenable to it. However, the short half-life of the recombinant α-Gal A used in ERT (approximately 1 hour in plasma) necessitates a lifetime of infusions every other week, with associated risk of infusion-related reactions in a significant proportion of patients, some of which are severe.
ST-920 is an investigational medicinal recombinant adeno-associated viral vector, AAV2 serotype 6 (rAAV2/6), encoding human GLA cDNA that is being developed by Sangamo Therapeutics, Inc.
The targeted patient population of this study will be male subjects ≥18 years of age with classical Fabry disease. Subjects who satisfy all inclusion/exclusion criteria will be enrolled into one of the 3 treatment dose cohorts.
This is a Phase I/II, multicenter, open-label, single-dose, dose-ranging study with 3 dose cohorts. Up to 18 subjects who satisfy all inclusion/exclusion criteria are eligible to dose in this study.
This study will test to see if ST-920 investigational therapy can help to provide patients with Fabry disease stable therapeutic,liver-specific expression of α-Gal A which may improve on the current clinical outcomes of ERT therapy and ultimately replace ERT altogether.
REC name
London - West London & GTAC Research Ethics Committee
REC reference
19/LO/1397
Date of REC Opinion
12 Nov 2019
REC opinion
Further Information Favourable Opinion