A Phase 2b Multicenter, Single Dose, Randomized, Double Blind, Placebo-Controlled, Parallel-Group Study Evaluating the Safety and Efficacy of Two Doses of Stannsoporfin in Combination With Phototherapy in Neonates
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 91
- 试验地点
- 18
- 主要终点
- Total Serum Bilirubin (mg/dL)
研究概览
简要总结
It is normal for red blood cells to die, even in newborn babies. The waste from that is called bilirubin. The liver clears bilirubin out of the body.
Some babies are born with illness that makes red blood cells die too fast, so the liver is not strong enough to keep up with it.
The yellowish color in eyes or skin means there is too much bilirubin in the body. It can be dangerous if a baby's bilirubin gets too high.
Special lights are put on jaundiced babies (called phototherapy) to help the liver get rid of bilirubin.
This study tests an experimental drug to see if it can help the liver even more, by safely cutting down the amount of bilirubin the body is making in the first place.
详细描述
Participants randomized (1:1:1) to treatment groups will be term or near-term infants with isoimmune hemolytic disease or glucose-6-phosphate dehydrogenase (G6PD) deficiency.
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 (Care Provider, Investigator)
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
- •Term and near term infants ≥35 and ≤ 43 weeks gestational age (GA), age 0-48 hours with antibody (ABO) or rhesus factor (Rh) incompatibility (anti C, c, D, E or e) who are Coombs positive, or age 0-72 hours with G6PD deficiency
- •Parental or guardian consent
- •Birth weight ≥ 2500 grams
- •At or above the age-specific threshold for initiating phototherapy (PT) per the American Academy of Pediatrics (AAP) guidelines based on measurement of total serum bilirubin (TSB)
- •Parents agree to observe light precautions for 10 days post treatment
排除标准
- •Elevated direct bilirubin ≥2 mg/dL, OR > 20% of the total serum bilirubin
- •Alanine aminotransferase (ALT) > 2 times the upper limit of normal (ULN) and/or aspartate aminotransferase (AST) > 3 times ULN
- •Abnormal renal function defined as creatinine and/or blood urea nitrogen >2 times the ULN
- •Any other clinically significant abnormalities on screening laboratory evaluation [including electrocardiogram (ECG)] that in the opinion of the investigator makes the patient unsuitable for the clinical trial
- •Apgar score ≤6 at age 5 minutes
- •An unexplained existing rash or skin erythema
- •Prior exposure to PT
- •Clinical suggestion of neonatal thyroid disease or current uncontrolled thyroid disease in the mother (maternal Hashimoto's disease is not exclusionary)
- •Cardio-respiratory distress, defined as a respiratory rate >60 breaths per minute at time of enrollment
- •Any abnormal auditory or ophthalmologic findings on screening physical exam
- •Treatment or need for treatment in the neonate with medications that are photoreactive or may prolong the QT interval (erythromycin ointment for eye prophylaxis is permitted), or family history of Long QT syndrome
- •Known porphyrias or risk factors for porphyrias, including family history
- •A maternal history of systemic lupus erythematosus
- •Maternal use of phenobarbital 30 days before, or after delivery, if breast-feeding
- •Maternal current drug or alcohol abuse, or maternal history of drug or alcohol abuse that, in the opinion of the Investigator, would not make the patient a suitable candidate for participation in the clinical trial
- •Significant congenital anomalies or infections
- •Risk of requiring surgery or exposure to operating room (OR) lights in the first 2 weeks of life
- •Persistent hypoglycemia (blood glucose <40 mg/dL)
- •Temperature instability defined as temperature consistently (3 consecutive times) <36 degrees centigrade (ºC) and/or >37.5 degrees centigrade (ºC) axillary
- •Use of intravenous immunoglobulin (IVIg) or albumin prior to study drug administration
- •Post-delivery treatment with medications that are known or suspected to displace bilirubin from albumin (e.g., ceftriaxone or sulfa-based antibiotics)
- •Use of photosensitizing drugs or agents
- •Unwillingness of parents/guardians to adhere to recommendations regarding light precautions
- •Exposure to any investigational medications or devices after delivery, or participation in another clinical trial while participating in this trial
- •Any other concurrent medical condition, which in the opinion of the Investigator, makes the patient unsuitable for the clinical trial
研究组 & 干预措施
Stannsoporfin 3.0 mg/kg
Participants receive stannsoporfin (3.0 mg/kg) and phototherapy
干预措施: Stannsoporfin (Drug)
Stannsoporfin 4.5 mg/kg
Participants receive stannsoporfin (4.5 mg/kg) and phototherapy
干预措施: Phototherapy (Procedure)
Placebo
Participants receive placebo and phototherapy
干预措施: Phototherapy (Procedure)
Placebo
Participants receive placebo and phototherapy
干预措施: Placebo (Drug)
Stannsoporfin 3.0 mg/kg
Participants receive stannsoporfin (3.0 mg/kg) and phototherapy
干预措施: Phototherapy (Procedure)
Stannsoporfin 4.5 mg/kg
Participants receive stannsoporfin (4.5 mg/kg) and phototherapy
干预措施: Stannsoporfin (Drug)
结局指标
主要结局
Total Serum Bilirubin (mg/dL)
时间窗: Baseline, 48 hours post-treatment
Total serum bilirubin (TSB) was measured at baseline (the measure that qualified the baby for inclusion) and at 48 hours after treatment. If a baby was discharged before 48 hours, the last measurement before discharge was used \[last observation carried forward (LOCF)\].
次要结局
- Number of Participants With Rebound Hyperbilirubinemia(within 54 hours)
- Time (Hour) at Which TSB First Crosses at or Below the Defined Age-specific Threshold for 54-hours Post-treatment (PT)(within 54 hours)
- Number of Participants With Phototherapy (PT) Failure(within 30 days after discharge)