Finding Solutions to Thrive After Birth Asphyxia in Africa: An Open-label Dose-finding Clinical Trial (Phase Ib Study)
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 发起方
- 入组人数
- 18
- 试验地点
- 1
- 主要终点
- Maximum tolerable dose of sildenafil
研究概览
简要总结
Neonatal encephalopathy (NE) is the third leading cause of under 5-year mortality and contributes substantially to long-term neurological morbidity worldwide. In low-income countries (LICs), families often lack the resources to care for affected children. For those with disabilities, stigma is high, and social and emotional impacts are substantial. Improving our understanding of NE in LICs is crucial if intervention strategies are developed. Providing access to an affordable and easy-to-administer treatment after birth may improve survival, early brain development and later outcome, maximizing developmental potential.
The primary objective of this study is to investigate the feasibility, safety and tolerability of administering sildenafil as a neuroprotective/neurorestorative strategy to improve early brain development in a cohort of children with NE in Uganda.
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
- 0 Days 至 18 Months（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male and female neonates meeting the criteria for birth asphyxia
- •Gestational age ≥ 36 weeks and birth weight ≥ 1800 g;
- •Admitted to Kawempe Hospital or Nsambya Hospital within 48 hours of life;
- •Need for continued resuscitation after birth and/or 5-minute Apgar score ≤5;
- •Evidence of neonatal encephalopathy by an abnormal neurological exam (modified Sarnat score of 2-3 or abnormal aEEG).
排除标准
- •Absent heart rate at 10 minutes/imminent death
- •Neonates with major congenital malformations
- •Neonates with grade 3 AKI (serum creatinine rise ≥3x lowest previous creatinine or creatinine > 2.5 mg/dL = 221 mcmol/L or receipt of dialysis)
- •Neonates with intraventricular and/or intraparenchymal hemorrhage on cranial ultrasound (cUS) performed on day 1-2 of life
- •Mother living permanently outside 20km radius of Kawempe Hospital or Nsambya Hospital
- •Neonates whose parents are unwilling or unable to give informed written consent to enter the study
研究组 & 干预措施
Sildenafil
干预措施: Sildenafil (Drug)
结局指标
主要结局
Maximum tolerable dose of sildenafil
时间窗: within 30 days of drug administration
次要结局
- Sildenafil concentrations(within 30 days of drug administration)
- Incidence of adverse events (Safety and Tolerability)(within 30 days of drug administration)
研究者
Pia Wintermark
Associate Professor of Pediatrics
McGill University Health Centre/Research Institute of the McGill University Health Centre