Study of Lademirsen (SAR339375) in Patients With Alport Syndrome

Inclusion Criteria

• •Male or female.
• •Confirmed diagnosis of Alport syndrome
• •1. Clinical diagnosis (hematuria, family history, hearing loss, ocular change), AND
• •2. Genetic confirmation of Alport Syndrome in the participant or the family member, OR
• •3. Kidney biopsy showing glomerular basement membrane abnormalities (e.g., significant thinning, thickening, irregularity or lucencies) consistent with Alport Syndrome.
• •Age 18-55 years old.
• •eGFR \> 35 ml/min/1.73m\^2 and \<90 mL/min/1.73m\^2 (based on CKD-EPI) at screening.
• •Renal Function Criteria (participants must have met at least one of the following CRITERIA A, B or C):
• •* A) Decline in eGFR of \>=4 mL/min/1.73 m\^2/year (eGFR slope \<= -4) based on a linear regression slope analysis of \>=4 eGFR measurements within 3 years prior to the study and with a minimum of 2-year time span (the last, of the screening measurement, and first eGFR measurements should be separated by at least 2 years). eGFR was calculated by using either the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) creatinine equation.
• •* B) proteinuria (UPCR) \>2000 mg/g (UACR\>1000 mg/g).
• •* C) Age and sex adjusted eGFR (based on CKD-Epi; male 18-23 eGFR\<90 mL/min/1.73m\^2
• •ACE inhibitor and/or ARB, the dosing regimen should be stable for at least 30 days prior to screening.
• •Sexually active female participants of childbearing potential and sexually mature male participants must have agreed to practice true abstinence in line with their preferred and usual lifestyle or to use two acceptable effective methods of contraception for the entire duration of the study and for at least 6 weeks after last dose.
• •Negative drug screen for opiates, cocaine, heroin, phencyclidine, amphetamines (including ecstasy), barbiturates, benzodiazepines, and cannabinoids. At the Investigator's discretion, participants prescribed benzodiazepines, cannabinoids, or opiates with positive results on a drug screen were allowed.
• •Negative screening results for hepatitis B surface antigen (HBsAg), hepatitis C virus (HCV) antibody, and human immunodeficiency virus (HIV) antibody.
• •Normal biological tests.
• •Able to understand all study procedures in the informed consent form (ICF) and to comply with all aspects of the protocol.