A Single Arm Single Centre Study To Investigate Safety And Efficacy Of Sildenafil In Near Term And Term Newborns With Persistent Pulmonary Hypertension Of The Newborn (PPHN)
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 发起方
- 入组人数
- 4
- 试验地点
- 1
- 主要终点
- Percentage of Participants Requiring Inhaled Nitric Oxide (iNO) or Extracorporeal Membrane Oxygenation (ECMO)
研究概览
简要总结
Sildenafil is efficacious in newborns with persistent pulmonary hypertension and its use will reduce the need for inhaled nitric oxide.
详细描述
Letter to investigator dated 18 June 2012 that study was to be terminated. Study terminated due to evolved and widespread use of standard of care, relevance of study questioned. No safety reasons or issues.
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
- 1 Hour 至 72 Hours（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •72 hours of age; and > or = to 34 weeks gestational age.
- •Persistent Pulmonary Hypertension of the Newborn or Hypoxic respiratory failure associated with:
- •Idiopathic PPHN or
- •Meconium aspiration syndrome or
- •Oxygenation Index (OI) >15 and <60 calculated
排除标准
- •Patients already receiving inhaled nitric oxide (iNO) on referral.
- •Prior or immediate need for full Cardio Pulmonary Resuscitation or Extracorporeal Membrane Oxygenation (ECMO).
- •Life threatening or lethal congenital anomaly.
- •Large left to right intracardiac or ductal shunting (diagnosed from echocardiogram on admission to GOSH).
- •Clinically significant active seizures as per clinical judgment.
- •Bleeding diathesis as per clinical judgment
研究组 & 干预措施
one
干预措施: sildanefil (Drug)
结局指标
主要结局
Percentage of Participants Requiring Inhaled Nitric Oxide (iNO) or Extracorporeal Membrane Oxygenation (ECMO)
时间窗: From start of infusion (baseline) up to Day 14
Percentage of participants who required standard therapy (iNO or ECMO) after failure of study treatment.
Number of Participants With Adverse Events (AEs) Based on Severity
时间窗: Baseline up to 28 days after last dose
AE:any untoward medical occurrence in participant who received study drug without regard to possibility of causal relationship. SAE:AE resulting in any of following outcomes or deemed significant for any other reason: death; initial/prolonged inpatient hospitalization; life-threatening experience; persistent/significant disability/incapacity; congenital anomaly. Severity criteria: "mild=does not interfere with participant's usual function; moderate=interferes to some extent with participant's usual function and severe=interferes significantly with participant's usual function".
Number of Participants With Abnormal Laboratory Data
时间窗: Screening, once daily for 3 days, every 48 hours thereafter till the end of infusion (up to Day 14)
Criteria for potentially clinically significant (PCS) laboratory values: hematocrit 29.2 percent (%); white blood cell (WBC) count 5.0\*10\^3, lymphocyte absolute 0.88\*10\^3, total neutrophils absolute 12.07\*10\^3, eosinophils absolute 0.50\*10\^3 per cubic millimeter (/mm\^3); calcium 6.8 milligram/deciliter (mg/dL); venous bicarbonate 47.0 milliequivalent/liter (meq/L).
次要结局
- Change From Baseline in Differential Saturation (Pre- And Post-ductal) at Hour 6 and 12(Baseline, Hour 6, 12)
- Change From Baseline in Ratio of Partial Pressure of Oxygen in Arterial Blood to the Fraction of Inspired Oxygen (P/F) at Hour 6 and 12(Baseline, Hour 6, 12)
- Maximum Observed Plasma Concentration (Cmax) of Sildenafil Metabolite (UK-103320)(Pre-dose, 5 and 30 minutes post-loading infusion, within 48 to 72, 96 to 120 hours during infusion, within 4 to 8, 18 to 24 and 44 to 48 hours post-maintenance infusion)
- Population Pharmacokinetics of Sildenafil(Pre-dose, 5 and 30 minutes post-loading infusion, within 48 to 72, 96 to 120 hours during infusion, within 4 to 8, 18 to 24 and 44 to 48 hours post-maintenance infusion)
- Time to Receipt of Standard Therapy (Inhaled Nitric Oxide [iNO] or Extracorporeal Membrane Oxygenation [ECMO])(Baseline up to 28 days after last dose)
- Change From Baseline in Oxygenation Index at Hour 6 and 12(Baseline, Hour 6, 12)
- Duration of Mechanical Ventilation(Baseline up to 28 days after last dose)