A Study of ELX-02 in Patients With Alport Syndrome

Inclusion Criteria: * A confirmed diagnosis of X-linked or autosomal recessive Alport Syndrome with a documented nonsense mutation of Col4A5 in a male or nonsense mutation of Col4A3 or Col4A4 (male or female) * The nonsense mutation should be UAG or UGA * eGFR\>60 ml/min/1.73 m2 (based on CKD-EPI for ages ≥18 and Schwartz formula for participants \<18) * Urinary protein based on two spot urine collections \[urine protein/creatinine ratio (UPCR) ≥ 500 mg/g\] * Stable regimen of ACEi/ARB for at least 4 weeks before screening (unless there is a contraindication) Exclusion Criteria: * History of any organ transplantation * Mutation consistent with autosomal dominant Alport Syndrome * Liver disease characterized by cirrhosis or portal hypertension. Participants with alanine aminotransferase (ALT), aspartate aminotransferase (AST), and/or a total bilirubin 3.0 times the upper limit of normal (ULN) will be excluded * History of congestive heart failure diagnosed clinically or with documented left ventricular ejection fraction (LVEF) ≤ 40% * History of dialysis