Safety of Autologous Human Umbilical Cord Blood Treatment for Perinatal
试验速览
- 阶段
- 1 期
- 状态
- 撤回
- 入组人数
- 10
- 试验地点
- 2
- 主要终点
- Composite Outcome: Renal Safety
研究概览
简要总结
Autologous human umbilical cord blood (hUCB) stored at Cord Blood Registry will be given to children who have suffered from a Perinatal Arterial Ischemic Stroke. The aim is to determine if hUCB infusion is safe, if late functional outcome is improved, if hUCB treatment improves physiologic response in the child's SSEP & EEG, and the effect of hUCB infusion in altering anatomic findings on MRI.
详细描述
Autologous human umbilical cord blood (hUCB) stored at Cord Blood Registry will be given to children who have suffered from a Perinatal Arterial Ischemic Stroke.
Subjects will come to Orlando for pretesting to include an MRI, SSEP, Urodynamics, blood work: CBC, CMP, Hepatic Function Panel, PT/PTT/INR, Chest Xray, EEG, Gross Motor Function Classification, Manual Ability Classification System, and a Speech and Language Evaluation.
After pretesting, the subjects will receive their autologous cord blood infusion intravenously. The subjects will then be monitored for 24 hours post infusion. After 24 hours, the subject will undergo repeat blood work and a chest x ray. Subjects will then be discharged home.
Subjects will follow up in Orlando at 6 months and 1 year post infusion. Follow up testing will repeat the exams performed at pretesting.
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
- 6 Weeks 至 6 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Between 6 weeks and 6 years of age on the day of study cord blood infusion.
- •MRI documented single arterial distribution infarction.
- •Initial injury occurring in the prenatal or perinatal period
- •Ability of child and caregiver to travel to Orlando, and stay for at least 4 days, and to return for all Follow-up visits (patient is responsible for cost of travel and lodging while in Orlando)
排除标准
- •Inability to obtain all pertinent medical records, including pertinent physician notes, laboratory findings, and radiographic images, related to the original injury, hospitalization and rehabilitation - must be sent to FHFC research team 14 days prior to scheduled study cord blood treatment. Need brain MRI with flair sequence <2 weeks old.
- •Recent radiographic evidence (imaging performed within past 2 weeks) of extensive stroke as evidenced by >100ml lesion
- •Multifocal infarctions on screening MRI.
- •Evidence of hypoxic-ischemic encephalopathy on screening MRI.
- •Uncorrected coagulopathy during the baseline period defined as INR > 1.4; PTT> 35 sec; PLT < 100,000
- •Known history of:
- •Recently diagnosed infection (within past 2 weeks) requiring treatment and/or medical intervention
- •Renal disease or altered renal function as defined by serum creatinine >1.5 mg/dL at admission
- •Hepatic disease or altered liver function as defined by SGPT > 150 U/L, and/or T. Bilirubin >1.3 mg/dL at enrollment
- •Immunosuppression as defined by WBC < 3 (10x3) at admission
- •Any evidence of active maternal infection during the pregnancy (Hepatitis A, Hepatitis B, Hepatitis C, HIV 1, HIV 2, Human T-lymphotropic Virus (HTLV) 1, HTLV 2
- •Pneumonia, or chronic lung disease requiring oxygen
- •Cord blood sample contamination
- •Participation in a concurrent intervention study
- •Desire for organ-donation in the event of death
- •Unwillingness or inability to stay for at least four days following cord blood infusion (should any problems arise following the infusion) and to return for 6 month, and 1 year follow-up visits
研究组 & 干预措施
Cord Blood Infusion
Autologous cord blood infusion
干预措施: Autologous Cord Blood Infusion (Biological)
结局指标
主要结局
Composite Outcome: Renal Safety
时间窗: 1 year
To minimize the effect of DSMO, our hUCB product will be washed according to Standard Operating Procedures prior to infusion. Though unlikely, it is possible that some quantity of DMSO will remain which could cause toxicity. Renal function/events corresponding to the CTCAE v3.0 Grade 3 will trigger the stopping rules
Composite Outcome: Neurological Safety
时间窗: 1 year
The patient's acute neurologic status will be monitored until discharged. Data recorded every 4 hours includes GCS, pupillary size/reactivity, motor/sensory evaluation of extremities, and seizure activity from infusion to discharge. Grade 3-5 CNS event as defined in the NCI CTCAE v3.0 occurring within 12 hours of cellular product infusion will trigger the stopping rules. Other changes temporally related to hUCB infusion (those events occurring within 12 hours of infusion) will be considered associated with the protocol and recorded as an adverse event.
Composite Outcome: Hemodynamic Safety
时间窗: 1 year
Three primary and two secondary hemodynamic indices will be monitored as indices of hemodynamic stability throughout the infusion and post-infusion periods. Heart rate, blood pressure, and oxygen saturation will be recorded every 5 minutes during the infusion, every 30 minutes for 2 hours after infusion and then hourly for 6 hours. A consistent, non-isolated 20% decrease in any of these indices will be prompt additional maneuvers to restore MAP. Two secondary hemodynamic indices will be monitored as indices of hemodynamic stability: capillary refill and heart rate. Prolongation of capillary refill by 2 seconds from baseline and/or \>20% change in heart rate during the procedure will prompt an evaluation as to the etiology of the change in hemodynamic status. An adverse event will be defined as a sustained (\> 10 minutes) \>20% decrease in MAP. Transient decreases in MAP that respond to fluid infusion or inotropes will not be considered adverse events.
Composite Outcome: Pulmonary Safety
时间窗: 1 year
A concern exists regarding the systemic infusion of leukocytes in a concentrated manner. Theoretically, activated monocytes could function to enhance PMN migration into the lung, as the lung is the primary "first pass" filter for intravenous infusion of any cellular product. PMN mediated organ injury typically occurs over a 6-24 hour time frame. Based on this, Chest radiographs will be performed and evaluated at Baseline and on Post-Infusion Day 1. Chest radiographs will be evaluated for systemic infusion of leukocytes in a concentrated manner. Additionally, blood-oxygen saturation will be monitored by finger oximeter. Moderate respiratory dysfunction within the first 48 hours post infusion will be considered an adverse event but will not warrant stopping the trial unless recommended by the DSMB. In the event of pulmonary dysfunction, standard supportive therapy will be given. Pulmonary symptoms/events corresponding to the CTCAE v3.0 Grade 3 will trigger the stopping rules.
次要结局
- EEG(1 year)
- Language(1 year)
- Speech(1 year)
- Fine and Gross Motor(1 year)
- Bladder: Urodynamics(1 year)
研究者
James Baumgartner, MD
Primary Investigator
AdventHealth