Overview

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.