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Completed

NCT Number: NCT00248625

N-acetylcysteine in Non-Acetaminophen Pediatric Acute Liver Failure

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.

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Key information

Conditions

Acute Liver Failure Brain Diseases Brain Diseases, Metabolic Central Nervous System Diseases Digestive System Diseases Hepatic Encephalopathy Hepatic Insufficiency Liver Diseases Liver Failure Liver Failure, Acute Metabolic Diseases Nervous System Diseases Nutritional and Metabolic Diseases

Age range

Up to 18 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Hospital for Sick Children, Toronto, Ontario, Canada

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About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • 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.

Exclusion criteria

  • 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

Treatment and study plan

N-acetylcysteine

Drug

The study drug is administered as a continuous infusion at a dose of 150 mg/kg/day for up to 7 days following entry into the study. The infusion is discontinued at the time of death, liver transplant or discharge.

Other names: Mucomyst

Placebo

Drug

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 N-acetylcysteine (150 mg/kg/d) in 5% dextrose (D5W) and water or placebo consisting of an equal volume of D5W alone. Volumes were adjusted for small children. Study medications were infused over 24 hours for up to 7 consecutive days in a dedicated line without other medications. Treatment was stopped earlier than 7 days in the case of hospital discharge, liver transplantation, or death within 7 days of randomization.

Other names: dextrose in water

Primary outcomes

  1. Survival

    Time frame: One year following randomization

    Spontaneous survival without transplant plus survival following transplantation

Secondary outcomes

  1. Spontaneous Recovery

    Time frame: One year following randomization

    Survival without liver transplantation

  2. Cumulative Percent Incidence of Transplantation by 1 Year

    Time frame: Within 1 year of randomization

  3. Length of Hospital Stay

    Time frame: Randomization to hospital discharge

  4. Categorized Length of ICU Stay

    Time frame: Within 7 days of randomization

    The length of ICU stay was categorized as number of days in ICU within 7 days of randomization, unless participant either died or received an LTx within this time period. Special categories were created for these cases.

  5. Number of Organ Systems Failing

    Time frame: Within 7 days of randomization

  6. Highest Coma Grade of Hepatic Encephalopathy

    Time frame: Within 7 days of randomization

    West Haven Criteria for hepatic encephalopathy (Grade 0 - IV ) is used for participants > 3 year of age. Coma grade IV indicates a participant who is comatose , with no reflexes, is decerebrate and has abnormal EEG changes with very slow delta activity. For participants less than 3 years the Whittington Scale was used. The Whittington scale does not use EEG changes and has only 3 levels, early (grades I and II), Mid (III) with somnolence, stupor, combativeness and Late (IV) for participants who are comatose with absent reflexes and decerebrate or decorticate posturing.

  7. Infectious Complication

    Time frame: Within 7 days of randomization

Sponsors and collaborators

Lead sponsor

University of Pittsburgh

Other

Collaborators

  • National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

Registry information

Official study title

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.

Important dates

Study start
2000
Primary completion
2009
Study completion
2010
First posted
Nov 4, 2005
Registry last updated
Jul 28, 2016

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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