A Multi-center Study of the Safety and Efficacy of N-acetylcysteine in the Treatment of Acute Liver Failure in Pediatric Patients Not Caused by Acetaminophen.
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 184
- 试验地点
- 20
- 主要终点
- Survival
研究概览
简要总结
We have completed patient enrollment in the the double blind, randomized, placebo-controlled trial of intravenous (IV) N-acetylcysteine (NAC) vs. placebo for the treatment of non-acetaminophen ALF. The purpose of this study is to examine the safety and efficacy of intravenous NAC in children with ALF for whom no antidote or other specific treatment is available. Inclusion in the NAC Study required enrollment in the Pediatric Acute Liver Failure (PALF) Study Registry.
详细描述
The Pediatric Acute Liver Failure (PALF) Study Group to identify, characterize, and develop management strategies for infants, children and adolescents who present with acute liver failure. The PALF study group includes 20 sites (17 in the United States, 2 in the United Kingdom, and 1 in Canada). The primary objective of the Pediatric Acute Liver Failure (PALF) study is to collect, maintain, analyze, and report clinical, epidemiological, and outcome data in children with ALF, including information derived from biospecimens.
Patients enrolled in the PALF study registry were able to enroll in the NAC study providing they met the additional required inclusion/exclusion criteria.
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
- Triple (Participant, Care Provider, Investigator)
入排标准
- 年龄范围
- — 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Meet entry criteria for and be enrolled in the Pediatric Acute Liver Failure prospective database.
- •Able to be evaluated and initiate treatment within the first 24 hours of hospitalization
- •Patients transferred from referring hospitals to the study site may be considered for enrollment, provided that no other treatment protocol has begun, and that no liver support device (BAL, extracorporeal liver assist device, transgenic pig perfusion) has been used or is contemplated.
- •Use of fresh frozen plasma infusions will not disqualify patients from participation.
排除标准
- •older than 18 years of age
- •pregnancy
- •ALF that is secondary to acute acetaminophen toxicity, mushroom poisoning, or a known malignancy.
- •Patients who exhibit signs of cerebral herniation, have intractable arterial hypotension, require inotropic drugs, or demonstrate signs of sepsis (temperature ≥ 39.5o C or bacteremia) at the time of enrollment
- •No exclusion will be made on the basis of race, ethnic group or gender.
- •Criteria for inclusion of females and minorities will be those established in the NIH guidelines
研究组 & 干预措施
N-acetylcysteine (NAC)
Eligible children were adaptively allocated by age (less than 2 years of age or at least 2 years old) and hepatic encephalopathy (grade 0-1 or 2-4) to receive N-acetylcysteine (150 mg/kg/d) in 5% dextrose (D5W) infused over 24 hours for up to 7 consecutive days
干预措施: N-acetylcysteine (Drug)
placebo
Eligible children were adaptively allocated within strata defined by age (less than 2 years of age or at least 2 years old) and hepatic encephalopathy (grade 0-1 or 2-4) to receive 5% dextrose (D5W) infused over 24 hours for up to 7 consecutive
干预措施: Placebo (Drug)
结局指标
主要结局
Survival
时间窗: One year following randomization
Spontaneous survival without transplant plus survival following transplantation
次要结局
- Cumulative Percent Incidence of Transplantation by 1 Year(Within 1 year of randomization)
- Spontaneous Recovery(One year following randomization)
- Length of Hospital Stay(Randomization to hospital discharge)
- Categorized Length of ICU Stay(Within 7 days of randomization)
- Number of Organ Systems Failing(Within 7 days of randomization)
- Highest Coma Grade of Hepatic Encephalopathy(Within 7 days of randomization)
- Infectious Complication(Within 7 days of randomization)
研究者
Robert Squires, Jr.
MD
University of Pittsburgh