Study of Hydroxychloroquine in Patients With X-linked Alport Syndrome in China (CHXLAS)

Inclusion Criteria:

• Male or female;
• Age 3-18 years old;
• Diagnosis of Alport syndrome by genetic testing (documented mutation in a geneassociated with Alport syndrome, including COL4A3, COL4A4, or COL4A5) or histologicassessment using electron microscopy;
• Screening eGFR ≥ 90 mL/min/1.73 m2;
• ACE inhibitor and/or ARB, the dosing regimen should be stable for at least 4 weeksprior to screening;
• No antirheumatic drugs such as hydroxychloroquine have been used;
• Willing and able to comply with scheduled visits, treatment plan, laboratory tests,and other study procedures;

Exclusion Criteria:

• Causes of chronic kidney disease aside from Alport syndrome (including but not limitedto other heritable disorders leading to chronic kidney disease, diabetic nephropathy,hypertensive nephropathy, lupus nephritis, IgA nephropathy);
• Prior exposure to hydroxychloroquine;
• Ongoing chronic hemodialysis or peritoneal dialysis therapy;
• Renal transplant recipient;
• Any clinically significant illness within 4 weeks before screening or surgical ormedical condition (other than Alport syndrome) that could interfere with the subject'sstudy compliance; confound the study results; impact subject safety; or significantlyalter the absorption, distribution, metabolism, or excretion of drugs;
• Participation in other interventional clinical studies;
• Known hypersensitivity to any component of the study drug.