High-Dose Erythropoietin in Very Low Birth Weight Infants for the Potential Treatment of Prematurity-Related Cerebral Hemorrhagic-Ischemic Injury: A Phase II Safety/Tolerability Study
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 入组人数
- 22
- 试验地点
- 1
- 主要终点
- Neurodevelopmental evaluations at 18 to 22 months corrected age (cerebral palsy, Bayley Scores of Infant Development Mental Development Index (MDI), Psychomotor Development Index (PDI), bilateral hearing aid use, and visual impairment)
研究概览
简要总结
The highest risk for perinatal brain injury occurs among extremely premature infants who weigh less than 1250 grams at birth. Such perinatal brain injury is currently irreversible, associated with neurodevelopmental disability, and without adequate treatment modalities. Research in recent years suggest in both animal and human studies that erythropoietin (Epo) may have significant neuroprotective effects. Given the historical safe medical profile of Epo when used for anemia of prematurity but the likely need for a greater dosage regimen for activation of neuroprotective pathways against neonatal brain injury, we therefore propose this phase II study of high-dose Epo in very low birth weight infants for the prevention and/or attenuation of prematurity-related cerebral hemorrhagic-ischemic injury.
详细描述
Eligible extremely premature infants will be enrolled in this double-blind, placebo-controlled randomized trial from the neonatal intensive care unit at Morristown Memorial Hospital (Morristown, New Jersey). Subjects will be enrolled within the first 24 hours of life and randomly assigned to receive Epo or saline vehicle placebo.
Standard NICU care will be provided to all subjects. Serial exams, CBC-d, reticulocyte counts, serum Epo levels, serial HUS, and head MRI will be collected at established time points during the study period. At 18 to 22 months corrected age, subjects will undergo a neurodevelopmental evaluation assessing for cerebral palsy, Bayley Scores of Infant Development-II (BSID-II) Mental Development Index (MDI), BSID-II Psychomotor Development Index (PDI), bilateral hearing aid use, and visual impairment.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Prevention
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- — 至 24 Hours（Child）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •500 to 1250 grams at birth
- •Less than 32 weeks gestation at birth
- •Less than 24 hours of life at time of enrollment
排除标准
- •Congenital anomalies (chromosomal, CNS, cardiac, GI, pulmonary)
- •Seizures within first 24 hours of life
- •Severe neutropenia (ANC < 500 cells/microL) within first 24 hours of life
- •Polycythemia (Hct > 65%) within first 24 hours of life
- •Thrombocytopenia (platelets < 50K cells/microL) within first 24 hours of life
- •Hypertension (SBP > 100mmHg) without vasopressor support within first 24 hours of life
研究组 & 干预措施
EPO###
All subjects will be identified as a such by the study identifier "EPO###" where EPO designates enrollment in this study and ### is a numeric identifier (e.g. 103).
干预措施: Saline placebo (Drug)
EPO ###
All subjects will be identified as a such by the study identifier "EPO###" where EPO designates enrollment in this study and ### is a numeric identifier (e.g. 103).
干预措施: Erythropoietin (Drug)
结局指标
主要结局
Neurodevelopmental evaluations at 18 to 22 months corrected age (cerebral palsy, Bayley Scores of Infant Development Mental Development Index (MDI), Psychomotor Development Index (PDI), bilateral hearing aid use, and visual impairment)
时间窗: 18-22 months corrected age
次要结局
- Severe intraventricular hemorrhage(First ten days of life)
- Polycythemia, neutropenia, thrombocytopenia, hypertension, sepsis, hemorrhage, seizure(NICU hospitalization)