A multicenter, randomized, double-blind, placebo -controlled, phase-II trial to assess safety, and efficacy of sovateltide in the treatment of hypoxic-ischemic encephalopathy in neonates
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 40
- 试验地点
- 5
- 主要终点
- Percentage of patients with death or disability (moderate/severe)
研究概览
简要总结
This is a multicenter, randomized, double-blind, placebo-controlled, phase-II study to assess the safety and efficacy of sovateltide for the treatment of perinatal asphyxia caused hypoxic-ischemic encephalopathy (HIE) in neonates.
This protocol is designed to develop a novel first-in-class treatment for hypoxic-ischemic encephalopathyin neonates.
For an individual patient, the duration of the study will be 24 months. At visit 1, a total of 40 patients with perinatal asphyxia/perinatal depression will be randomized 1:1 into 2 treatment groups after meeting the eligibility criteria. An Interactive Web Response System (IWRS) will be used to randomize the eligible patient to the treatment groups.
Group 1: Sovateltide + Supportive treatment for perinatal asphyxia
Group 2: Normal Saline (Equal volume) + Supportive treatment for perinatal asphyxia
Sovateltide or saline will be administered as an IV bolus dose over 1 minute. In the sovateltide group, 3 doses of sovateltide, at 0.3 μg/kg body weight will be administered as an (IV) bolus over 1 minute every 3 hours ± 1 hour on day 1, 3, and day 6 (total dose/day: 0.9 µg/kg body weight). In the saline group, 3 doses of an equal volume of normal saline will be administered as an IV bolus over 1 minute every 3 hours ± 1 hour on days 1, 3, and day 6 post-randomization. In both treatment groups, patients will be provided supportive treatment for perinatal asphyxia. Every effort will be made to have drug administration at the same time on days 1, 3 and, 6.
Each patient will be monitored closely throughout his/her hospitalization and will be followed for 24 months from randomization. Each subject will be assessed for efficacy and safety parameters over 24 months from randomization.
研究设计
- 研究类型
- Interventional
- 分配方式
- Computer generated randomization
- 盲法
- Participant and Investigator Blinded
入排标准
- 年龄范围
- 0.00 Day(s) 至 1.00 Day(s)（—）
- 性别
- All
入选标准
- •1.Either sex with ≥ 36 weeks of gestational age 2.Receiving supportive management for perinatal asphyxia 3.Perinatal depression, based on at least one of the following: (a)Apgar score of <5 at 10 minutes (b)Need for resuscitation (chest compressions or mechanical ventilation) at birth (c)pH <7.00 or base deficit ≥ 16 mmol/liter in the cord or arterial blood within 60 minutes of birth (d)Moderate/severe encephalopathy evident by at least 3 of 6 modified Sarnat criteria, present between 1 to 6 hours of birth.
- •4.Informed consent by one of the parents or a legal representative.
排除标准
- •1.Gestational age <36 weeks 2.Admitted to hospital12-hoursafter birth 3.A genetic or congenital condition that affects neuronal development 4.TORCH infection 5.Neonatal sepsis 6.Complex congenital heart disease 7.Severe dysmorphic feature 8.Microcephaly (head circumference < 2 SDs below mean for gestational age).
结局指标
主要结局
Percentage of patients with death or disability (moderate/severe)
时间窗: Day-1 to 24 Months
次要结局
- Change in the proportion of children with disabling cerebral palsy(24 months)
- Change in the proportion of patients with seizures Clinical or electrical seizuresat birth, at 6 hours, 12 hours, 24 hours, 48 hours, 72 hours, 7 days, 30 days, 6 months after initiation of treatment, and then at every 6 months interval(24 months)
- Cognitive, Language, Motor, Social-Emotional, and General Adaptive Scales Score as assessed by Bayley Scales of Infant and Toddler Development (BSID)TM measured(at 6 months after initiation of treatment and then at every 6 months interval)
- Changes in Bayley Scales of Infant and Toddler DevelopmentScores(24 months)
- The number of patients with brain injury (MRI or EEG evidence of brain injury).(14 days)
- Change in the proportion of patients with blindness or hearing impairment(24 months)
- Incidence of sovateltide related adverse events(24 months)
- The number of patients not receiving complete treatment due to intolerance to sovateltide(7 days)