NCT07523581招募中2 期
A Randomized, Double-Blind, Placebo-Controlled, Delayed-Start Study to Evaluate the Efficacy and Safety of Exaluren in Alport Syndrome Patients With Nonsense Mutations in COL4A3/4/5 Genes
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 24
- 试验地点
- 12
- 主要终点
- The change in the degree of podocyte foot process effacement
研究概览
简要总结
This is a randomized, double-Blind, placebo-controlled study to evaluate the efficacy and safety of exaluren in Alport Syndrome patients with nonsense mutations in COL4A3/4/5 genes.
Targeted 24 patients aged 12 and older will be enrolled in the trial.
The study will be comprised of the following periods for each participant:
- a Screening period of up to 6 weeks (42 days)
- a total Treatment Period of exaluren 0.75 mg/kg or placebo administered daily subcutaneously for 32 weeks: Part 1: patients are randomized to either exaluren or placebo for 16 weeks. Part 2: all patients across both randomized arms receive exaluren for 16 additional weeks.
- a safety/efficacy Follow-up Period of 4 weeks after the last treatment
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 12 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •A confirmed diagnosis of X-linked or autosomal recessive Alport Syndrome with a documented nonsense mutation of COL4A5 in a male or nonsense mutation of COL4A3 or COL4A4 (male or female)
- •eGFR>45 ml/min/1.73 m2
- •Urinary protein based on two spot urine collections [urine protein/creatinine ratio (UPCR) ≥ 500 mg/g]
- •Stable regimen of ACEi/ARB for at least 12 weeks before Day 1
排除标准
- •History of any organ transplantation
- •Liver disease characterized by cirrhosis or portal hypertension. Participants with alanine aminotransferase (ALT), aspartate aminotransferase (AST), and/or a total bilirubin 1.5 times the upper limit of normal (ULN) will be excluded
- •History of dialysis
- •Acute kidney injury within 4 weeks before screening
- •Active dizziness
研究组 & 干预措施
Placebo
Placebo Comparator
干预措施: Exaluren (Drug)
Exaluren
Experimental
干预措施: Exaluren (Drug)
结局指标
主要结局
The change in the degree of podocyte foot process effacement
时间窗: Baseline to Week 16
Measured in kidney biopsies by the change in the Filtration Slit Density (FSD)
The change in the degree of podocyte foot process effacement
时间窗: Baseline to Week 32
Measured in kidney biopsies by the change in the Filtration Slit Density (FSD)
次要结局
- The percentage change in Urine Protein Creatinine Ratio (UPCR)(Baseline to Week 16)
- The percentage change in Urine Protein Creatinine Ratio (UPCR)(Baseline to Week 32)
研究者
研究点 (12)
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