A Randomized, Double-blind, Placebo-controlled, Parallel Group Phase 2a Study With an Extension Phase to Evaluate the Efficacy and Safety of BAY 3401016 in Participants Aged 18 to 45 With Alport Syndrome
试验速览
- 阶段
- 2 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 60
- 试验地点
- 168
- 主要终点
- Urinary albumin creatinine ratio (UACR) ratio to baseline averaged over 16, 20 and 24 weeks of treatment
研究概览
简要总结
Alport syndrome (AS) is a rare genetic condition that causes kidney disease, hearing loss, and eye abnormalities that occur due to changes in specific genes (COL4A3, COL4A4, and COL4A5). These genes help in producing an important protein called collagen. People with AS have a high risk of developing chronic kidney disease (CKD), a condition in which there is progressive loss in kidney function over time. The kidneys soon lose their ability to remove waste products from the body properly, resulting in end-stage kidney disease. A common sign of decreasing kidney function is the presence of excess protein in the urine that is not usually found with healthy kidneys. This condition is known as proteinuria. The study drug, BAY 3401016 (a monoclonal antibody), is a type of medicine that blocks a protein called Semaphorin 3A (Sema3A), which is thought to be involved in causing kidney damage in AS. By blocking the action of the Sema3A protein, BAY 3401016 may prevent proteinuria and slow down the loss in kidney function due to AS.
The main purpose of this study is to learn more about how well BAY 3401016 works in slowing down the loss in kidney function in adults with a rapidly progressing AS.
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
- Double (Participant, Investigator)
入排标准
- 年龄范围
- 18 Years 至 45 Years（Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participants must be 18 to 45 years of age inclusive
- •Participants with AS, either XLAS (male) or ARAS (male or female)
- •eGFR ≥ 45 mL/min/1.73m2
- •UACR ≥ 500mg/g
排除标准
- •Chronic kidney disease is different from AS
- •Clinically significant illness that could have influence on the safety of the participant and/or interfere with the study objectives
- •History or current existence of malignancy
- •Participants with history of severe allergies, multiple drug allergies or non-allergic drug reactions including allergies affecting the lower respiratory tract - allergic asthma, allergies requiring therapy with corticosteroids or urticaria
- •Participants with active skin disorders (e.g. atopic dermatitis, severe acne)
- •Systolic blood pressure above 140 mmHg
- •Diastolic blood pressure above 90 mmHg
研究组 & 干预措施
BAY 3401016
BAY 3401016 weekly for 24 weeks in addition to background therapy if eligible
干预措施: BAY 3401016 (Drug)
Placebo
Placebo weekly for 24 weeks in addition to background therapy if eligible
干预措施: Placebo (Other)
结局指标
主要结局
Urinary albumin creatinine ratio (UACR) ratio to baseline averaged over 16, 20 and 24 weeks of treatment
时间窗: From the start of study intervention, over 16, 20 and 24 weeks of treatment, until the last follow-up visit, 90 days ± 3 days after EoT
Progression of kidney disease, including Alport Syndrome (AS), to End-Stage Renal Disease (ESRD) takes years, making it challenging to establish drug efficacy in clinical trials without long follow-ups or large sample sizes. Using surrogate endpoints, such as early changes in albuminuria, can help address this issue. It is tested wether BAY 3401016 has an effect on albuminuria in AS patients. Research shows that albumin overload leads to renal function decline and podocyte injury.
次要结局
- Investigate the safety and tolerability of BAY 3401016 in participants with AS(From the start of study intervention until the last follow-up visit, 90 days ± 3 days after EoT)