A Study of ELX-02 in Patients With Alport Syndrome

Inclusion Criteria:

• A confirmed diagnosis of X-linked or autosomal recessive Alport Syndrome with adocumented nonsense mutation of Col4A5 in a male or nonsense mutation of Col4A3 orCol4A4 (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 forparticipants <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 acontraindication)

Exclusion Criteria:

• History of any organ transplantation

• Mutation consistent with autosomal dominant Alport Syndrome

• Liver disease characterized by cirrhosis or portal hypertension. Participants withalanine aminotransferase (ALT), aspartate aminotransferase (AST), and/or a totalbilirubin 3.0 times the upper limit of normal (ULN) will be excluded

• History of congestive heart failure diagnosed clinically or with documented leftventricular ejection fraction (LVEF) ≤ 40%

• History of dialysis