Dose-Ranging Study of ST-920, an AAV2/6 Human Alpha Galactosidase A Gene Therapy in Subjects With Fabry Disease (STAAR)

Exclusion Criteria

• •Neutralizing antibodies to AAV6
• •eGFR \< 40 ml/min/1.73m2
• •New York Heart Association Class III or higher
• •Active infection with hepatitis A, B or C, HIV or TB
• •History of liver disease such as clinically significant steatosis, fibrosis, non-alcoholic steatohepatitis (NASH) and cirrhosis, biliary disease within 6 months of informed consent; except for Gilbert's syndrome
• •Elevated circulating serum AFP
• •Recent or recurrent hypersensitivity response to ERT within within 6 months prior to consent
• •Current or history of systemic (IV or oral) immunomodulatory agents, or biologics or steroid use in the past 6 months prior to consent (topical treatment and inhaled allowed).
• •Contraindication to use of corticosteroids
• •History of malignancy except for non-melanoma skin cancer and localized prostate cancer treated with curative intent
• •Recent history of alcohol or substance abuse
• •Participation in investigational interventional drug or medical device study throughout the duration of this study and within previous 3 months prior to consent
• •Prior treatment with a gene therapy product
• •Known hypersensitivity to components of ST-920 formulation
• •Any other reason that, in the opinion of the Site Investigator or Medical Monitor, would render the subject unsuitable for participation in the study including but not limited to risk of COVID-19 infection
• •Renal cohort:
• •History of renal dialysis or transplantation
• •History of acute kidney insufficiency in the 6 months prior to screening
• •Angiotensin converting enzyme (ACE) inhibitor or angiotensin receptor blocker (ARB) therapy initiated within 4 weeks prior to screening or changed ACE inhibitor or ARB dose in the 4 weeks prior to screening
• •Urine protein to creatinine ratio (UPCR) \> 0.5 g/g who are not being treated with an ACE inhibitor or ARB
• •Cardiac cohort:
• •Significant cardiac fibrosis defined by late gadolinium enhancement on CMR
• •Any contraindications to CMR as per local hospital/institution guidelines
• •Angiotensin converting enzyme (ACE) inhibitor or angiotensin receptor blocker (ARB) therapy initiated within 4 weeks prior to screening or changed ACE inhibitor or ARB dose in the 4 weeks prior to screening
• •NYHA Class IV