A Safety Study of Intravenous Infusion of Bone Marrow Mesenchymal Stem Cell-derived Extracellular Vesicles (UNEX-42) in Preterm Neonates at High Risk for Bronchopulmonary Dysplasia
试验速览
- 阶段
- 1 期
- 状态
- 终止
- 发起方
- 入组人数
- 3
- 试验地点
- 6
- 主要终点
- Number of Subjects With Treatment-emergent Adverse Events During the Post-treatment Phase (Safety and Tolerability)
研究概览
简要总结
A multicenter, placebo-controlled, randomized, dose escalation, safety, and tolerability study of UNEX-42 in infants born at <27 weeks of gestational age (GA) at high risk for bronchopulmonary dysplasia (BPD).
详细描述
The study was discontinued by the Sponsor on 24 February 2021 due to a business decision, not related to reasons of safety or efficacy. Only data listings were created; no summary or inferential analyses were performed.
Subjects were assessed during Screening and Baseline (prior to randomization) for eligibility in the study. Subjects then received a single IV dose of UNEX-42 at 20 pmol phospholipid/kg body weight, or placebo. After randomization, subjects were monitored in the hospital through 40 Weeks postmentrual age (PMA) or the time of hospital discharge (whichever came first).
The following efficacy and safety assessments occurred during the course of the study:
Efficacy Assessments: incidence and severity of BPD, duration of hospitalization, duration of mechanical ventilation, duration of supplemental oxygen therapy, duration of postnatal steroids, tracheal aspirate inflammatory biomarkers, and Respiratory Severity Score.
Safety Assessments: physical examination, vital signs, adverse events, predefined complications of prematurity, clinical laboratory parameters, and chest x-ray.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 3 Days 至 14 Days（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Infant whose postnatal age was 3 to 14 days
- •Subjects met the following oxygen and birth weight criteria based on gestational age: 23 weeks to 24 weeks 6 days (any birth weight, any oxygen requirement) or 25 weeks to 26 weeks 6 days (fraction of inspired oxygen [FiO2] ≥35% [sustained for >2 hours] at any point during postnatal Days 1 to 14 AND birth weight ≤750 g)
- •Endotracheally intubated and receiving mechanical ventilation at the time of Screening and randomization.
- •Not expected to be extubated within the next 24 hours after randomization.
- •The subject had a parent/guardian who gave written informed consent.
排除标准
- •Had a congenital heart defect, except for PDA, atrial septal defect or a small/moderate, restrictive ventricular septal defect.
- •Had a serious malformation of the lung, such as pulmonary hypoplasia/aplasia, congenital diaphragmatic hernia, or any other congenital lung anomaly.
- •Was being treated with inhaled nitric oxide.
- •Had a known chromosomal abnormality (eg, Trisomy 18, Trisomy 13, or Trisomy 21) or a severe congenital malformation (eg, hydrocephalus and encephalocele, trachea-esophageal fistula, abdominal wall defects, and major renal anomalies).
- •Had a known severe congenital infectious disease (ie, herpes, toxoplasmosis rubella, syphilis, human immunodeficiency virus, cytomegalovirus, etc).
- •High clinical suspicion of active systemic infection, severe sepsis, or septic shock during Screening.
- •Underwent a surgical procedure (requiring admission to an operating room) within 72 hours before randomization or who was anticipated to have a surgical procedure (requiring admission to an operating room) within 72 hours before or following randomization.
- •Had a Grade 3 or 4 intracranial hemorrhage.
- •Had active pulmonary hemorrhage.
- •The subject was currently participating in any other interventional clinical study.
- •The subject was, in the opinion of the Investigator, so ill that death was inevitable, or was considered inappropriate for the study for any reason(s) other than those listed above.
研究组 & 干预措施
Placebo
Phosphate-buffered saline
干预措施: Phosphate-buffered saline (Biological)
200 pmol phospholipid/kg body weight
UNEX-42 administered at 200 pmol phospholipid/kg body weight
干预措施: UNEX-42 (Biological)
60 pmol phospholipid/kg body weight
UNEX-42 administered at 60 pmol phospholipid/kg body weight
干预措施: UNEX-42 (Biological)
20 pmol phospholipid/kg body weight
UNEX-42 administered at 20 pmol phospholipid/kg body weight
干预措施: UNEX-42 (Biological)
结局指标
主要结局
Number of Subjects With Treatment-emergent Adverse Events During the Post-treatment Phase (Safety and Tolerability)
时间窗: From Day 1 to 40 Weeks Post-menstrual Age or Hospital Discharge, whichever came first
The safety and tolerability of UNEX-42 in subjects with BPD was evaluated by the number of subjects with treatment-emergent adverse events, including death, computed by dose cohort and overall during the Post-treatment Phase.
次要结局
未报告次要终点