Efficacy and Safety of Hydroxychloroquine in Patients With X-linked Alport Syndrome in China (CHXLAS)
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 50
- 试验地点
- 1
- 主要终点
- Change in urinary erythrocyte count(/HP)
研究概览
简要总结
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.
详细描述
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 3 Years 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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;
排除标准
- •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.
研究组 & 干预措施
Hydroxychloroquine Cohort
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).
干预措施: Hydroxychloroquine Sulfate 100 milligram (mg) Tab (Drug)
Hydroxychloroquine Cohort
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).
干预措施: Benazepril hydrochloride 10 milligram (mg) Tab (Drug)
Comparator Cohort
During treatment with HCQ, Patients randomized to Comparator Cohort only received enalapril(5-10mg qd).
干预措施: Benazepril hydrochloride 10 milligram (mg) Tab (Drug)
结局指标
主要结局
Change in urinary erythrocyte count(/HP)
时间窗: Baseline to maximum 48 weeks
To assess the change in urinary erythrocyte count(/HP) from baseline to week 48 for patients receiving active drug, compared to patients in Comparator Cohort.
次要结局
- Change in eGFR from baseline(Baseline to maximum 48 weeks)
- Change in urinary albumin characterization(Baseline to maximum 48 weeks)
- Change in urinary erythrocyte count(urinary sediment analyzer)(Baseline to maximum 48 weeks)
- Number of participants with treatment-related adverse events(Baseline to maximum 48 weeks)
- Change in 24-hour urinary protein quantity(Baseline to maximum 48 weeks)
- Change in urinary albumin to creatinine ratio(Baseline to maximum 48 weeks)