Human Umbilical Cord Mesenchymal Stem Cells for Alport Syndrome: Α Prospective Randomized, Single-blind, Placebo-controlled, Single-center Clinical Trial
试验速览
- 阶段
- 2 期
- 状态
- 尚未招募
- 发起方
- 入组人数
- 40
- 主要终点
- Urine protein remission rate
研究概览
简要总结
The goal of this clinical trial is to evaluate the safety and efficacy of human umbilical cord mesenchymal stem cells (hUC-MSC) in the treatment of Alport syndrome (AS) in a randomized, single-blind, placebo-controlled trial, to provide a clinical basis for the development of stem cell products for the treatment of AS, and to further clarify the therapeutic effect of hUC-MSC in the treatment of 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
- Single (Participant)
入排标准
- 年龄范围
- 3 Years 至 12 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •3 years old ≤Age ≤ 12 years old;
- •Meet the diagnostic criteria of Alport syndrome;
- •Positive proteinuria or combined hematuria;
- •Chronic kidney disease (CKD) stage: I-III stage children, that is, glomerular filtration rate greater than 60 ml/min.1.73m2;
- •No history of infectious diseases within 1 week before treatment;
- •Negative infectious disease screening;
- •No allergic state and related clinical manifestations;
- •Signed informed consent (children or their families).
排除标准
- •Age <3 years or >12 years old;
- •Alport syndrome patients with only microscopic hematuria and normal glomerular filtration rate;
- •Patients with significantly reduced renal function, chronic kidney disease stage IV or V, Alport syndrome;
- •Patients with other renal diseases;
- •Have a history of severe allergic reactions or be allergic to 2 or more foods or drugs;
- •Known allergy to stem cells or stem cell-derived products or ingredients in stem cell preparations;
- •Have severe heart, liver, lung and other organ dysfunction or have tumors;
- •Those with developmental malformations of the urinary system;
- •Those with autoimmune diseases and regular use of immunosuppressants;
- •Those with serious infectious diseases that are not under control;
- •Those with a history of infectious diseases such as HBV, HCV, HIV, syphilis;
- •History of surgery or acute trauma or blood loss exceeding 200ml within 3 months;
- •Participated in other clinical studies within 3 months;
- •Have received any cell product or derivative product treatment within 12 months;
- •Other circumstances that the researcher deems inappropriate for inclusion.
研究组 & 干预措施
MSC Theatment
Conventional symptomatic supportive treatment and human umbilical cord mesenchymal stem cells treatment
干预措施: hUC-MSC (Biological)
Placebo Control
Conventional symptomatic supportive treatment and normal saline as a placebo control
干预措施: Placebo control drug (Drug)
结局指标
主要结局
Urine protein remission rate
时间窗: 2 weeks to 12 months after treatment
Follow-up was performed at 2 weeks, 4 weeks, 6 weeks, 8 weeks, 3 months, 4 months, 5 months, 6 months, 8 months, 10 months, and 12 months after the last hUC-MSC infusion. Urinalysis, 24-hour urine protein quantification, and urine protein/creatinine ratio were tested to evaluate the remission of urine protein after treatment.
次要结局
- Hematuria remission rate(2 weeks to 12 months after treatment)
- Renal function improvement rate(2 weeks to 12 months after treatment)