4D-310 in Adults With Fabry Disease and Cardiac Involvement

Korábbi kezelés

• Presence of pre-existing antibodies to 4D-310 capsid or to AGA
• Undergone kidney transplantation or currently on hemodialysis or peritoneal dialysis
• Contraindication to systemic corticosteroid therapy or immunosuppressive therapy
• Chronic steroid use, defined as ≥ 3 months of oral corticosteroid use within the last 12 months.
• Currently receiving investigational drug, device or therapy or having ever received gene therapy

Más betegség

• HIV, active or chronic hepatitis B or C,
• Evidence of liver disease, severe pulmonary disease or diabetes with poor glycemic control
• History of stroke or transient ischemic attack within the last 12 months, or other significant thromboembolic disease history (e.g. pulmonary embolism)
• Moderately severe to severe cardiovascular disease or uncontrolled hypertension
• History of infusion related response to ERT or any adverse reaction leading to ERT discontinuation
• History of cancer within 2 years (exceptions include non-melanoma skin cancer, localized prostate cancer treated with curative intent)

Laborértékek

• eGFR <65 mL/min/1.73 m2
• Left ventricular ejection fraction of <45% on echocardiogram (ECHO)

Egyéb

• Pregnant or breast-feeding
  • Note: Other inclusion and exclusion criteria apply

• Male or female ≥ 18 to ≤65 years of age
• Pathogenic GLA mutation consistent with Fabry Disease
• Confirmed diagnosis of classic or late-onset Fabry disease with cardiac involvement
• Intolerant of ERT, unable or unwilling to receive ERT, or progressive disease despite ERT or migalastat
• Individuals receiving ERT, must be on a stable dose for at least 6 months
• Agree to use highly effective contraception