■免疫球蛋白A(IgA)肾病是一种表现为尿路症状的疾病,如肾小球性血尿和尿蛋白阳性,IgA主要沉积在肾小球系膜区域。皮质类固醇主要用于治疗;然而,感染是一种严重的不良事件,关于治疗效果的证据不足,因此,迫切需要新的治疗方法。间充质干细胞(MSCs)通过将白细胞的特性从炎症转化为抗炎并诱导器官组成细胞的增殖和分化,有助于炎症环境中炎症的改善和器官功能的恢复。分别。MSCs的这些特性导致其在各种炎症性疾病中的临床应用,但这项研究是世界上第一个用MSCs治疗难治性肾小球肾炎的临床试验。这项研究被注册并分配了编号,jRCT2043200002和NCT04342325。
■这将是第一阶段,开放标签,多中心,成人难治性IgA肾病患者对现有疗法耐药或难以治疗的剂量递增研究。ADR-001将在第一组中以1×108细胞的剂量静脉内给予3至6名患者一次,在第二组中以2周的间隔给6名患者两次。观察将持续到52周。主要终点是ADR-001开始后6周的不良事件评估。次要终点将是不良事件患者的相应百分比,临床缓解,部分缓解,缓解尿蛋白,血尿缓解,是时候缓解了,尿蛋白的变化,血尿,和估计的肾小球滤过率。
■IgA肾病患者服用ADR-001后,不良事件患者的百分比,无症状肺栓塞,临床缓解,部分缓解,尿蛋白缓解,血尿缓解,他们的时间来缓解,尿蛋白的变化,血尿,和肾小球滤过率将被确定。
■本研究将评估ADR-001的安全性和耐受性,并确认其在难治性IgA肾病成年患者中的疗效。
UNASSIGNED: Immunoglobulin A (IgA) nephropathy is a disease that presents with urinary symptoms such as glomerular hematuria and urinary protein positivity, with predominant deposition of IgA in the mesangial region of the glomerulus. Corticosteroids are mainly used for treatment; however, infection is a serious adverse event, and evidence regarding therapeutic efficacy is insufficient, thus new treatments are strongly desired. Mesenchymal stem cells (MSCs) contribute to the amelioration of inflammation and recovery of organ function in inflammatory environments by converting the character of leukocytes from inflammatory to anti-inflammatory and inducing the proliferation and differentiation of organ component cells, respectively. These properties of MSCs have led to their clinical application in various inflammatory diseases, but this
study is the first clinical
trial of MSCs for refractory glomerulonephritis in the world. This
study is registered and assigned the number, jRCT2043200002 and NCT04342325.
UNASSIGNED: This will be a phase 1, open-label, multiple-center, dose-escalation
study of adult patients with refractory IgA nephropathy resistant to or difficult to treat with existing therapies. ADR-001 will be administered intravenously to from three to six patients at a dose of 1 × 108 cells once in the first cohort and to six patients twice at 2-week intervals in the second cohort, and observation will continue until 52 weeks. The primary endpoint will be the evaluation of adverse events up to 6 weeks after the start of ADR-001 administration. Secondary endpoints will be the respective percentages of patients with adverse events, clinical remission, partial remission, remission of urine protein, remission of hematuria, time to remission, changes in urine protein, hematuria, and estimated glomerular filtration rate.
UNASSIGNED: Following the administration of ADR-001 to patients with IgA nephropathy, the respective percentages of patients with adverse events, asymptomatic pulmonary emboli, clinical remission, partial remission, urine protein remission, hematuria remission, their time to remission, changes in urine protein, hematuria, and glomerular filtration rate will be determined.
UNASSIGNED: This
study will evaluate the safety and tolerability of ADR-001 and confirm its therapeutic efficacy in adult patients with refractory IgA nephropathy.