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

Shanghai Jiao Tong University

Status and phase: Enrolling; Phase 2

Conditions: Alport Syndrome, X-Linked

Treatments: Drug: Benazepril hydrochloride 10 milligram (mg) Tab; Drug: Hydroxychloroquine Sulfate 100 milligram (mg) Tab

Study type: Interventional

Funder types: Other

Identifiers

NCT04937907

2019YLYM06 (Other Grant/Funding Number)

2021SPNR001

Details and patient eligibility

About

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.

Full description

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.

Patients in the Phase 2 cohort will be randomized 1:1 to either Hydroxychloroquine Cohort or Comparator Cohort.

All patients in the study will follow the same visit and assessment schedule. Following randomization on Day 1, patients will be scheduled to be assessed during treatment at Weeks 4, 12, and 24. Patients will not receive study drug during a 24-week withdrawal period between Weeks 25 and 48. Patients will also be scheduled to be assessed at an in person follow up visit at Week 36, and 48.

Enrollment: 50 estimated patients

Sex: All

Ages: 3 to 18 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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50 participants in 2 patient groups

Hydroxychloroquine Cohort

Experimental group

Description:

Patients in the cohort will receive Hydroxychloroquine(HCQ) throughout the study.Patients administered HCQ by oral at a dose of 6.5mg per kilogram twice a day for 6 months. During treatment with HCQ, patients also received enalapril(5-10mg qd).

Treatment:

Drug: Hydroxychloroquine Sulfate 100 milligram (mg) Tab

Drug: Benazepril hydrochloride 10 milligram (mg) Tab

Comparator Cohort

Sham Comparator group

Description:

During treatment with HCQ, Patients randomized to Comparator Cohort only received enalapril(5-10mg qd).

Treatment:

Drug: Benazepril hydrochloride 10 milligram (mg) Tab