Hydroxychloroquine treatment for X-linked Alport Syndrome in children

Efficacy and Safety of Hydroxychloroquine in Patients With X-linked Alport Syndrome in China (CHXLAS)

This study is testing if Hydroxychloroquine can help children with X-linked Alport syndrome feel better and manage their condition.

Quick facts

• Phase: PHASE2
• Study type: Interventional
• Enrollment: 50 (estimated)
• Ages: 3 Years to 18 Years
• Sex: All
• Sponsor: Shanghai Children's Hospital (other)
• Locations: 1 site (Shanghai, Shanghai)
• Trial ID: NCT04937907 on ClinicalTrials.gov

What this trial studies

This Phase 2 randomized controlled trial investigates the safety, tolerability, and efficacy of Hydroxychloroquine in patients diagnosed with X-linked Alport syndrome. The study will enroll up to 50 patients aged 3 to 18 years, who will be randomly assigned to receive either Hydroxychloroquine or a comparator treatment. Participants will undergo assessments at multiple time points throughout the treatment period, including a follow-up after a 24-week withdrawal phase. The trial aims to provide insights into the potential benefits of Hydroxychloroquine for managing this genetic condition.

Who should consider this trial

Good fit: Ideal candidates are children aged 3-18 years with a confirmed diagnosis of X-linked Alport syndrome.

Not a fit: Patients with chronic kidney disease from causes other than Alport syndrome or those who have previously used hydroxychloroquine may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could improve kidney function and overall health outcomes for patients with X-linked Alport syndrome.

How similar studies have performed: While there is limited data on the use of Hydroxychloroquine specifically for Alport syndrome, similar studies have explored its efficacy in other conditions, indicating potential for success.

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.

Where this trial is running

Shanghai, Shanghai

• Shanghai Children's Hospital — Shanghai, Shanghai, China (RECRUITING)

Study contacts

• Study coordinator: Wen-yan Huang, PhD

Conditions: Alport Syndrome, X-Linked, Alport Syndrome, Hydroxychloroquine, Treatment