Efficacy and Safety of Hydroxychloroquine in Patients With X-linked Alport Syndrome in China (CHXLAS)
This Phase 2 randomized controlled trial will study the safety, tolerability, and efficacy of Hydroxychloroquine in qualified patients with Alport syndrome. The trial will be open-label, randomized, controlled and will enroll up to 50 patients.
• Male or female;
• Age 3-18 years old;
• Diagnosis of Alport syndrome by genetic testing (documented mutation in a gene associated with Alport syndrome, including COL4A3, COL4A4, or COL4A5) or histologic assessment 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 weeks prior 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;