Study of Hydroxychloroquine in Patients With X-linked Alport Syndrome in China (CHXLAS) (NCT04937907) | Clinical Trial Compass
CompletedPhase 2
Study of Hydroxychloroquine in Patients With X-linked Alport Syndrome in China (CHXLAS)
China50 participantsStarted 2021-09-08
Plain-language 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.
Who can participate
Age range3 Years – 18 Years
SexALL
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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.