Dose-response and Efficacy of Umbilical Cord-derived Mesenchymal Stromal Cells in Renal Systemic Lupus Erythematosus
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 39
- 试验地点
- 2
- 主要终点
- Achievement of Global Renal Response (GR) at Study Endpoint
研究概览
简要总结
Phase II Clinical Trial to Assess the dose-response and Efficacy of Umbilical Cord-derived Mesenchymal Stromal Cells (MSCs) in Severe Renal Systemic Lupus Erythematosus (SLE).
详细描述
Phase IIa trial of escalating doses of intravenous (i.v.) MSCs in active SLE, followed by a Phase IIb, triple blind, controlled assessment of the selected MSC dosing versus Placebo, in SLE patients receiving Standard of Care Therapy for Severe Renal Disease,
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
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
盲法说明
Masking for patients, physicians providing patient care and outcome assessors.
入排标准
- 年龄范围
- 18 Years 至 75 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Fulfilling 1997 updated American College of Rheumatology (ACR) Criteria or 2012 SLICC Classification Criteria for SLE
- •Seropositive for antinuclear (≥1:80) and/or anti-DNA antibodies
- •Fulfilling following criteria for active renal disease:
- •Class III or IV proliferative disease (ISN/RPS) Renal Biopsy within 12 months plus...
- •Active Urinary Sediment (> 5 red blood cells/high-power field and/or >8 white blood cells/high-power field and/or cylindruria during the current flare).
- •UPC ratio ≥ 1
排除标准
- •Estimated GFR < 40ml/min/m2
- •Addition during prior 3 months of randomization of: Bolus methylprednisolone or new immunosuppressive drug or intravenous immunoglobulin (IVIG) or Plasmapheresis.
- •Addition during prior 6 months of randomization of Cyclophosphamide
- •Addition during prior 12 months of randomization of Biological anti-B cell therapy
- •Coexisting uncontrolled morbidity; Pregnancy or planned Pregnancy within next 12 months; uncontrolled infection or neoplastic disease. Pending unresolved surgical indication.
研究组 & 干预措施
MSC treatment
Intervention: a previously selected dose of MSCs (Phase IIa) will be administered by i.v. infusion at baseline and 6 months of follow-up (total 12 months), to patients with Severe Renal SLE subject also to Standard of Care treatment with Methylprednisolone and Cyclophosphamide followed by Mycophenolate.
干预措施: MSC treatment (Biological)
MSC treatment
Intervention: a previously selected dose of MSCs (Phase IIa) will be administered by i.v. infusion at baseline and 6 months of follow-up (total 12 months), to patients with Severe Renal SLE subject also to Standard of Care treatment with Methylprednisolone and Cyclophosphamide followed by Mycophenolate.
干预措施: Standard of Care (Drug)
Placebo
Intervention: A Placebo (infusion vehicle) will be administered by i.v. infusion at baseline and 6 months of follow-up (total 12 months), to patients with Severe Renal SLE subject also to Standard of Care treatment with Methylprednisolone and Cyclophosphamide followed by Mycophenolate.
干预措施: Standard of Care (Drug)
Placebo
Intervention: A Placebo (infusion vehicle) will be administered by i.v. infusion at baseline and 6 months of follow-up (total 12 months), to patients with Severe Renal SLE subject also to Standard of Care treatment with Methylprednisolone and Cyclophosphamide followed by Mycophenolate.
干预措施: Placebo (Drug)
结局指标
主要结局
Achievement of Global Renal Response (GR) at Study Endpoint
时间窗: 12 months
Proportion of Patients that achieve Complete (CR) or Partial (PR) Renal Response at Endpoint
次要结局
- Achievement of Complete Renal Response (CR) at Study Endpoint(12 months)
- Selena Sledai(12 months)
- Achievement of Partial Renal Response (PR) at Study Endpoint(12 months)
- Treatment Failure(24 weeks and 12 months)
- Response of SLE Responder Index (SRI).(12 months)
- BILAG score(12 months)
- Disease Flares(12 months)
- Biomarker Response(24 weeks and 12 months)
研究者
Fernando E. Figueroa MD
Program Director Translational Research in Cell Therapy
Universidad de los Andes, Chile