Intravenous Human Umbilical-Cord-Derived Mesenchymal Stem Cells For Premature Infants Ａt High Risk For Bronchopulmonary
试验速览
- 阶段
- 1 期
- 发起方
- 入组人数
- 20
- 试验地点
- 1
- 主要终点
- Number of participants with adverse reactions related to infusion after treatment
研究概览
简要总结
This study is an open-label, single-center, dose escalation study to evaluate of safety and efficacy of human umbilical cord -derived mesenchymal stem cells (hUC-MSCs) in premature infants at high risk for Bronchopulmonary Dysplasia（BPD）
详细描述
BPD is a chronic lung disease that occur in premature infants receiving prolonged oxygen pulmonary and ventilator therapy. It remains a main complication of extreme prematurity and currently lacks efficient treatment.The mortality rate of one year after birth is still high and the quality of life is not optimistic.
hUC-MSCs are widely used in clinic due to their low immunogenicity and convenient to get. Many animal study had shown that hUC-MSCs had therapeutic effects on a variety of animal models of lung disease.Furthermore,there are a large number of clinical trials of MSCs applied to various system diseases and the safety was verified.So, the main purpose of this study is to evaluate the safety and efficacy of hUC-MSCs in participants at high risk for BPD
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 4 Days 至 14 Days（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •An infant whose postnatal age is 3 to 14 days, inclusive (for treatment between 5 and 14 days after birth)
- •Gestational age is between 23 and 28 weeks (23 weeks ≤ gestational age (GA) < 28 weeks)
- •Birth weight is between 500g and 1000g, inclusive
- •Being intubated and receiving mechanical ventilation within 5-14 days after birth, with a fraction of inspired oxygen (FiO2) of 0.25 or greater at Screening
- •Written consent form signed by a legal representative or a parent.
排除标准
- •Although mechanical ventilation or oxygen is required in participants, there are no signs of dyspnea or BPD-related changes in lung imaging, such as central apnea or diaphragm paralysis.
- •The participants who have complex congenital heart disease.
- •The participants who have severe pulmonary hypertension(cardiac ultrasound confirmed) at the time of assessment.
- •The participants who have severe respiratory tract malformation: pierre-robin syndrome, tracheobronchomalacia, vascular ring syndrome, congenital tracheal stenosis, tracheo-esophageal fistula, pulmonary emphysema, pulmonary sequestration, congenital pulmonary dysplasia, congenital pulmonary cyst, congenital spasm, etc.
- •The participants who have severe chromosome anomalies :Edward syndrome, Patau syndrome, Down syndrome, etc) or severe congenital malformation (Hydrocephalus, Encephalocele, etc).
- •The participants who have severe congenital infection(Herpes, Toxoplasmosis, Rubella, Syphilis, AIDS, etc).
- •The participants who have severe sepsis or shock.
- •The participants who is going to have surgery 72 hours before/after this study drug administration.
- •The participants who have surfactant administration within 24 hours before this study drug administration.
- •The participants who have severe intracranial hemorrhage ≥ grade 3 or
- •The participants who have active pulmonary hemorrhage or active air leak syndrome at the time of assessment.
- •The participants who have the history of other clinical studies as a participant.
- •The participants who is considered inappropriate by the investigators.
研究组 & 干预措施
Transplantation of hUC-MSCs
Preterm infants at high risk for BPD will receive transplantation of hUC-MSCs.
干预措施: Transplantation of hUC-MSCs (Drug)
No transplantation of hUC-MSCs
Preterm infants at high risk for BPD will not receive transplantation of hUC-MSCs
干预措施: No transplantation of hUC-MSCs (Drug)
结局指标
主要结局
Number of participants with adverse reactions related to infusion after treatment
时间窗: 24 hours after administration
To evaluate the safety of hUC-MSCs for BPD.
次要结局
- Changes of respiratory rate in participants(3 days after administration)
- Changes of oxygen saturation in participants(3 days after administration)
- Changes of high-resolution chest CT in participants(within 2 years after administration)
- The incidence and severity of BPD defined by the National Institutes of Child Health and Human Development (NICHD) workshop.(at the corrected gestational age of 36 weeks)
- Changes of temperature in participants(3 days after administration)
- Changes of blood pressure in participants(3 days after administration)
研究者
Xia Yunqiu
Director
Children's Hospital of Chongqing Medical University