A Phase 2b, Multicenter, Single-dose, Blinded, Randomized, Placebo-controlled, Dose-escalation, Safety and Efficacy Trial of Stannsoporfin in Neonates With Hyperbilirubinemia
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 发起方
- 入组人数
- 58
- 试验地点
- 23
- 主要终点
- Change in Adjusted Total Serum Bilirubin (TSB) From Baseline to 48 Hours After Treatment.
研究概览
简要总结
It is a normal process in the human body for red blood cells to die, which makes bilirubin.
Bilirubin is cleared away through the liver.
Some babies are born with livers that don't work well enough yet, or their red blood cells are dying too fast, so the baby looks yellow (jaundice).
This means there is too much bilirubin in the body. It can be dangerous if a baby's bilirubin gets too high.
Phototherapy is what they call the lights they shine on newborn babies to help the liver get rid of bilirubin.
This study tests an experimental drug to see if it can reduce how much bilirubin is being made in the first place.
详细描述
The purpose of this study is to determine if an experimental drug, stannsoporfin, is safe and effective in the treatment of hyperbilirubinemia in hemolyzing neonates.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Double (Participant, Investigator)
盲法说明
Clinical personnel at the site (including investigator, study coordinator and central cardiologist) were blinded to treatment group.
The research pharmacy and the health care provider responsible for giving the injection to patients knew what the treatment was (they were not blinded).
入排标准
- 年龄范围
- 1 Minute 至 48 Hours（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Babies may only participate if they meet all the following criteria:
- •Is a term or late preterm baby
- •Is at risk for protocol-defined hemolytic disease
- •Weighs at least 2500 g (5.5 lbs)
- •Has total serum bilirubin (TSB) a specified amount lower than the phototherapy threshold for the age
- •Has parents/guardians who are willing to follow light precautions and sign informed consent
排除标准
- •The following criteria will make a baby not eligible to participate:
- •Needs medications that may prolong the QT interval
- •Has family history or risk factors for Long QT Syndrome, Sudden Infant Death Syndrome, or Porphyrias
- •Has an Apgar score of 6 or below at age 5 minutes
- •Has abnormalities or infections (in mother or child) that per protocol or in the opinion of the investigator may compromise the safety and well-being of the baby or analysis of study results
研究组 & 干预措施
Cohort 1: Stannsoporfin 1.5 mg/kg
Participants receive a single dose of 1.5 mg/kg by intramuscular (IM) injection, along with PhotoTherapy (PT) if and when needed.
干预措施: Stannsoporfin (Drug)
Cohort 1: Stannsoporfin 1.5 mg/kg
Participants receive a single dose of 1.5 mg/kg by intramuscular (IM) injection, along with PhotoTherapy (PT) if and when needed.
干预措施: PhotoTherapy (as needed) (Other)
Cohort 2: Stannsoporfin 3.0 mg/kg
Participants receive a single dose of 3.0 mg/kg by intramuscular (IM) injection, along with PT if and when needed.
干预措施: Stannsoporfin (Drug)
Cohort 2: Stannsoporfin 3.0 mg/kg
Participants receive a single dose of 3.0 mg/kg by intramuscular (IM) injection, along with PT if and when needed.
干预措施: PhotoTherapy (as needed) (Other)
Cohort 3: Stannsoporfin 4.5 mg/kg
Participants receive a single dose of 4.5 mg/kg by intramuscular (IM) injection, along with PT if and when needed.
干预措施: Stannsoporfin (Drug)
Cohort 3: Stannsoporfin 4.5 mg/kg
Participants receive a single dose of 4.5 mg/kg by intramuscular (IM) injection, along with PT if and when needed.
干预措施: PhotoTherapy (as needed) (Other)
Cohort 4: Placebo
Participants receive a single dose of placebo (sterile saline solution) by IM injection, along with PT if and when needed.
干预措施: Placebo (Other)
Cohort 4: Placebo
Participants receive a single dose of placebo (sterile saline solution) by IM injection, along with PT if and when needed.
干预措施: PhotoTherapy (as needed) (Other)
结局指标
主要结局
Change in Adjusted Total Serum Bilirubin (TSB) From Baseline to 48 Hours After Treatment.
时间窗: Baseline, 48 hours
The adjusted TSB is a calculation of the percentage difference of the TSB level from the age-specific threshold for PT initiation per the American Academy of Pediatrics (AAP) Guidelines, ie, an indication of the distance below the PT threshold at the time \[(TSB - PT threshold/PT threshold) x 100%).
Change From Baseline in Total Serum Bilirubin (TSB) at 48 Hours (ITT Population
时间窗: Baseline, 48 hrs
Total bilirubin in blood serum was measured at baseline and at 48 hours after the shot. Change from baseline is calculated by subtracting the amount at baseline from the amount at 48 hours. Lower numbers are better.
次要结局
未报告次要终点