NCT04046224

Who might be able to join this trial:

• You are 18 years of age or older
• You have been officially diagnosed with Fabry disease
• You have one or more of the following symptoms: cloudy patches on the surface of the eye (cornea verticillata), burning or tingling pain in the hands and feet (acroparesthesia), inability to sweat normally (anhidrosis), or small dark red skin spots (angiokeratoma)
• You have been fully vaccinated against COVID-19 (following CDC guidelines in the US, or local guidelines in other countries) at least one month before receiving the study treatment

If joining the kidney (renal) group, you also need:

• A kidney function test result (eGFR) between 40 and 90 mL/min/1.73 m² at the screening visit
• Evidence that your kidney function has been declining at a certain rate, based on at least 3 blood test results taken within the past 18 months (confirm with trial site)

If joining the heart (cardiac) group, you also need:

• Evidence of thickening of the heart muscle (left ventricular hypertrophy) shown on an ultrasound or MRI scan, with walls measuring 12mm or more and no other explanation for this, OR signs that your heart condition is getting worse as shown by specific heart scan measurements (confirm with trial site)

Who may not be able to join:

• You have antibodies in your blood that could block a specific part of the study treatment (neutralizing antibodies to AAV6)
• Your kidney function test result (eGFR) is below 40 mL/min/1.73 m²
• Your heart failure is classified as moderate-to-severe or worse (New York Heart Association Class III or higher)
• You currently have an active infection with Hepatitis A, B, or C, HIV, or tuberculosis (TB)
• You have a history of significant liver disease such as serious fat build-up in the liver, liver scarring, a specific type of liver inflammation (NASH), cirrhosis, or bile duct problems within the past 6 months (a condition called Gilbert's syndrome is allowed)
• You have raised levels of a specific protein in your blood called alpha fetoprotein (AFP)
• You have had a serious allergic reaction to enzyme replacement therapy (ERT) within the past 6 months
• You are currently using, or have used in the past 6 months, certain medicines that affect the immune system taken by mouth or injection (topical creams or inhaled treatments are allowed)
• You cannot take corticosteroid medications (a type of anti-inflammatory drug) for any reason
• You have a history of cancer, except for certain skin cancers or prostate cancer that was fully treated
• You have a recent history of alcohol or substance misuse
• You have taken part in another clinical drug or medical device trial in the past 3 months, or plan to do so during this study
• You have previously received any gene therapy treatment
• You have a known allergy to any ingredient in the study treatment (ST-920)
• The trial doctor believes there is any other reason you would not be suitable for the study, including risk of COVID-19 infection

If joining the kidney (renal) group, you also cannot join if:

• You have ever been on kidney dialysis or had a kidney transplant
• You had a sudden serious loss of kidney function in the 6 months before screening
• You recently started or changed the dose of certain blood pressure or kidney-protecting medicines (ACE inhibitors or ARBs) within 4 weeks before screening
• You have a certain level of protein in your urine (UPCR above 0.5 g/g) and are not taking ACE inhibitor or ARB medicines (confirm with trial site)

If joining the heart (cardiac) group, you also cannot join if:

• You have significant scarring of the heart muscle shown on a cardiac MRI scan
• You have any reason you cannot have a cardiac MRI scan, based on your local hospital's guidelines
• You recently started or changed the dose of certain blood pressure or heart-protecting medicines (ACE inhibitors or ARBs) within 4 weeks before screening
• Your heart failure is classified as the most severe level (New York Heart Association Class IV)

Important: Always verify eligibility with the trial site directly before applying.