Phase I/II clinical study exploring the potential of ST-920 investigational gene therapy to treat Fabry disease
The STAAR Study is Recruiting Participants Now
Fabry disease is caused by shortage of an enzyme called alpha-galactosidase A (α-Gal A). This shortage happens when the GLA gene, which provides the body with instructions for making α-Gal A, is not working correctly.
The STAAR Phase I/II clinical study has been designed to investigate the safety and tolerability of an investigational gene therapy called isaralgagene civaparvovec, or ST-920, to treat Fabry disease. ST-920 aims to deliver a healthy copy of the GLA gene to the liver. It is hoped that the liver should then be able to produce the α-Gal A enzyme and secrete it via the blood stream to the rest of the body.
The STAAR Study is recruiting adults aged 18 and over who have been diagnosed with Fabry disease. Around twenty participants have already received the study medication, and participants continue to be enrolled at the highest dose. Preliminary clinical data were presented at the 19th Annual WORLDSymposium on February 24, 2023, by Dr. Robert J. Hopkin, an Associate Professor of clinical pediatrics at Cincinnati Children’s Hospital Medical Center in Ohio. The presentation is available on the Events & Presentations page of Sangamo’s website*.
Some highlights from the data presented at the conference:
- Sustained, elevated expression of alpha-galactosidase A (α-Gal A) activity observed in 13 participants for over two years for the longest treated patient as of cutoff date.
- One of the first participants to provide a kidney biopsy achieved 78% globotriaosylceramide (Gb3) substrate clearance at 6-months and 77% reduction in urine podocyte loss.
- Reported a clinically meaningful and statistically significant increase in mean general health scores, as measured by the SF-36 General Health survey.
- ST-920 is generally well tolerated with a favorable safety profile.
Twenty clinical sites are currently active in the United States, United Kingdom, Australia, Canada, Germany, Italy, and Taiwan.
Visit the STAAR Study website (www.staarclinicalstudy.com) where you can see if you qualify and find a study site near you. You can also discuss this further with the study team, who are more than happy to help.
Contact details: firstname.lastname@example.org
* This presentation is provided as general information only and not as a prediction of any potential participant’s outcome or results from the study.