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

Launched by SHANGHAI CHILDREN'S HOSPITAL · Jun 17, 2021

Keywords

ClinConnect Summary

This clinical trial, called CHXLAS, is investigating the use of Hydroxychloroquine, a medication typically used to treat other conditions, in patients with X-linked Alport syndrome. The goal is to see if this medication is safe and effective for managing the symptoms of this genetic kidney disease. The study will involve up to 50 patients aged 3 to 18 years who have been diagnosed with Alport syndrome through genetic testing or specialized imaging. To participate, patients must have stable kidney function and not have used certain other medications recently.

Participants in this trial can expect to undergo regular check-ups and tests to monitor their health and the effects of the treatment. It’s important for families to know that the trial is currently recruiting participants, and only those who meet specific criteria will be eligible. This means that if a child has been diagnosed with Alport syndrome but also has other kidney issues or has previously taken Hydroxychloroquine, they may not qualify. Overall, this study aims to gather valuable information that could help improve treatment options for young people with Alport syndrome.

Gender

ALL

Eligibility 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.