Potential study benefits and risks
It is possible that taking part in this study may improve the outcome of your Fabry disease, however this is not known. ST-920 may improve your symptoms, but there is also a chance that your symptoms may be no better, or worse, than they would have been if you had not received ST-920.
Even if there is no benefit to you, the results will help to shape future scientific research that could result in better treatment success in the future for people with Fabry disease.
Medical advances depend on participation of people in clinical studies, and every participant in a study makes a positive contribution towards learning more about medicine and future medical treatments, particularly for rare diseases where fewer patients are available to help.
People who enter into clinical studies are contributing to the generation of data that may advance future treatments for the benefit of more patients.
The full potential benefits and risks of participating in the study will be described in detail with the study doctor during the next stage of your discussions about the STAAR Study.
All investigational medications carry a risk of side effects.
Unexpected and severe risks are possible in research studies like STAAR where genes have been modified, and have not previously been tested in humans.
ST-920 is a once-only infusion that cannot be reversed. It is currently not known if ST-920 will increase α-Gal A enzyme in the blood, or if there will be any side effects in humans, as this is the first time that it will be studied in people. Therefore, people in STAAR will be closely monitored for any side effects. In addition, participants may become ineligible to other gene therapies using a similar vehicule due to exposure to ST-920.
For more details on the study, visit clinicaltrials.gov