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:
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Conditions: Fabry Disease
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Age: Between 18 - 99 Years
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Gender: Male
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Other Inclusion Criteria:
- Male subjects who are 18 years of age or older
- 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
- Subjects with BMI 20-45 kg/m2
- Subjects who are on ERT or is ERT-nai¨ve
- 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
- Subjects must be able to provide written informed consent
You may not be eligible for this study if the following are true:
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- Subjects known to be unresponsive to ERT in the opinion of the Site Investigator and Medical Monitor y
- Subjects currently receiving migalastat (GalafoldTM)
- Subjects with positive neutralizing antibody response to AAV6
- Subjects who has intercurrent illness expected to impair evaluation of safety or efficacy during the observation period of the study
- Subjects with eGFR = 60 ml/min/1.73m2
- Subjects with New York Heart Association Class III or higher
- Subjects with active infection with HAV, HBV, HCV (negative HCV-DNA), or HIVas measured by qPCR; or active infection with TB
- Subjects who have history of liver disease such as steatosis, cholangitis, cirrhosis, biliary disease; except for Gilbert’s syndrome
- Subjects with contraindication to use of corticosteroids for immunosuppression (e.g., diabetes, osteoporosis, etc.)
- Subjects who has history of malignancy except for non-melanoma skin cancer
- Subjects who has history of alcohol or substance abuse
- Subjects who participated in prior investigational interventional drug or medical device study within the previous 3 months
- Subjects who had prior treatment with a gene therapy product
- Subjects known hypersensitivity to components of ST-920 formulation
If you are registered as a volunteer, please log in to contact the study team/express interest in this study.