Sangamo Fabry disease gene therapy study (ST-920-201)

Are you eligible to participate in this study?

You may be eligible for this study if you meet the following criteria:

  • Conditions:
    Fabry Disease
  • Age: Between 18 - 99 Years
  • Gender: Male
  • Other Inclusion Criteria:
    1. Male subjects who are 18 years of age or older
    2. Subjects with documented diagnosis of classical Fabry disease as defined by plasma a-Gal A activity and one or more characteristics of classical Fabry disease
    3. Subjects with BMI 20-45 kg/m2
    4. Subjects who are on ERT or is ERT-nai¨ve
    5. Sexually mature subjects must agree to use a condom and refrain from sperm donation from the time of ST-920 administration until a minimum of 3 consecutive semen samples are negative for AAV2/6 after administration of ST-920 and a minimum of 90 days after ST-920 administration
    6. Subjects must be able to provide written informed consent

You may not be eligible for this study if the following are true:

    1. Subjects known to be unresponsive to ERT in the opinion of the Site Investigator and Medical Monitor y
    2. Subjects currently receiving migalastat (GalafoldTM)
    3. Subjects with positive neutralizing antibody response to AAV6
    4. Subjects who has intercurrent illness expected to impair evaluation of safety or efficacy during the observation period of the study
    5. Subjects with eGFR = 60 ml/min/1.73m2
    6. Subjects with New York Heart Association Class III or higher
    7. Subjects with active infection with HAV, HBV, HCV (negative HCV-DNA), or HIVas measured by qPCR; or active infection with TB
    8. Subjects who have history of liver disease such as steatosis, cholangitis, cirrhosis, biliary disease; except for Gilbert’s syndrome
    9. Subjects with contraindication to use of corticosteroids for immunosuppression (e.g., diabetes, osteoporosis, etc.)
    10. Subjects who has history of malignancy except for non-melanoma skin cancer
    11. Subjects who has history of alcohol or substance abuse
    12. Subjects who participated in prior investigational interventional drug or medical device study within the previous 3 months
    13. Subjects who had prior treatment with a gene therapy product
    14. Subjects known hypersensitivity to components of ST-920 formulation

If you are registered as a volunteer, please login to the dashboard to send referrals.

Contact the research team to learn more about this study.

By clicking "Contact Research Team", your contact information will be sent securely to the research staff associated with the study. You will also receive a copy of this email in your inbox, as well as other notifications to determine your participation status in the study.