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

• Status: Recruiting
• Trial end date: 2023-06-30
• Target enrollment: 0
• Participant gender: All

Summary

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.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Shanghai Children's Hospital
• Treatments: Benazepril; Hydroxychloroquine

Criteria

Inclusion Criteria:

• 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;

Exclusion Criteria:

• Causes of chronic kidney disease aside from Alport syndrome (including but not limited to 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 or medical condition (other than Alport syndrome) that could interfere with the subject's study compliance; confound the study results; impact subject safety; or significantly alter the absorption, distribution, metabolism, or excretion of drugs;

• Participation in other interventional clinical studies;

• Known hypersensitivity to any component of the study drug.