A Phase I Safety Trial of Allogeneic Mesenchymal Stem Cells for Systemic Lupus Erythematosus
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 6
- 试验地点
- 2
- 主要终点
- Frequency of Grade 3 or higher adverse events
研究概览
简要总结
The purpose of this study is to evaluate the safety of mesenchymal stromal cells (MSCs) obtained from umbilical cords for the treatment of adults with active systemic lupus erythematosus (SLE).
详细描述
This open label trial will evaluate the safety of allogeneic MSCs for the treatment of adults with moderate to severely active systemic lupus erythematosus (SLE). MSCs will be derived from healthy donor umbilical cord cells and 1 dose of MSCs will be tested. MUSC has a good manufacturing practice (GMP) quality Clean Cell Facility to ensure the quality and safety of the MSCs prior to infusing into study participants. The goal of this study is to determine the safety of MSC infusion in patients with SLE when added to standard of care for SLE.
The MSCs used in this trial are cells that are obtained from the umbilical cords of healthy donors having an elective Caesarean section and who have been screened to be sure that they are free of any infectious diseases. These investigational cells will be collected and processed so that they can be used as an infusion treatment. An infusion is when a drug (in this case the MSCs) is administered directly into the blood stream via a vein, usually located in the arm or hand. All participants will receive standard of care and their safety will be monitored throughout the study.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 65 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients between 18 and 65 years old, male or female, of any race
- •Definite SLE by meeting either SLICC or ACR Classification Criteria for SLE
- •Evidence of a positive ANA (≥1:80 titer) or positive dsDNA antibody test within 6 months of screening
- •Clinically mild to moderately active SLE determined by SLEDAI score ≥4 and ≤10 at screening, despite SOC therapy
- •If the patient has BILAG A or two BILAG Bs in the renal organ system, he/she must have completed at least 6 months of therapy with either mycophenolate mofetil or cyclophosphamide for the current episode of nephritis
- •Able and willing to give written informed consent
排除标准
- •Active CNS lupus affecting mental status
- •Active lupus nephritis requiring dialysis
- •Laboratory exclusions: eGFR <30, WBC <2.0/mm3, hemoglobin <8 g/dL, platelet count <30,000/mm3, liver enzymes AST or ALT >4 times upper limit normal; Positive testing for HIV, hepatitis B or hepatitis C
- •History of malignant neoplasm within the last 3 years, except for adequately treated cancers of the skin (basal or squamous cell) or carcinoma in situ of the uterine cervix
- •Pregnant or breast feeding; males or females not willing to use adequate contraception
- •History of renal transplantation
- •Herpes zoster within the past 90 days or any infection requiring hospitalization or intravenous antibiotics within the past 60 days
- •Clinically significant EKG or chest X-ray abnormalities
- •Any other medical condition, related or unrelated to SLE, that in the opinion of the investigator would render the patient inappropriate or too unstable to complete study protocol
- •Use of prednisone >0.5 mg/kg/day (or equivalent corticosteroid) within 1 month of Baseline visit
- •Change or addition to immunosuppressant regimen within 3 months of Baseline visit (except corticosteroids); Use of other experimental therapeutic agents within 3 months of Baseline visit
- •Having received belimumab within 3 months of Baseline, or having received rituximab or other B cell depleting biologic therapy within 6 months of Baseline.
- •Comorbidities requiring corticosteroid therapy
- •Current substance abuse or recent (within 60 days) history of substance abuse
研究组 & 干预措施
Drug: Low Dose Mesenchymal Stem Cells ( MSCs)
Participants will receive a single IV infusion of Mesenchymal Stem Cells (MSCs) 1 x 10^6 cells/kg in Plasma-Lyte A solution. All participants will receive the infusion at the Baseline (Day 0) visit. All participants will continue on their standard-of-care therapy during the trial.
干预措施: Low Dose Mesenchymal Stem Cells (MSCs) (Drug)
结局指标
主要结局
Frequency of Grade 3 or higher adverse events
时间窗: Week 24
The primary outcome measure is the frequency of Grade 3 or higher adverse events (AEs) experienced by participants at or prior to Week 24.
次要结局
- Frequency of All Adverse Events(Baseline to Week 52)
- Change in Disease Activity(Baseline to Week 24)
- Change in Patient Reported Outcomes - Life(Baseline to Week 24)
- Change in Patient Reported Outcomes - Pain(Baseline to Week 24)
- Change in Disease Biomarkers - Serum(Baseline to Week 24)
- Change in Patient Reported Outcomes - Fatigue(Baseline to Week 24)
- Change in Patient Reported Outcomes - Depression(Baseline to Week 24)
- Change in Disease Biomarkers - Cellular(Baseline to Week 24)