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.

This study has been completed.
Sponsor:
Collaborator:
Information provided by (Responsible Party):
University of Pittsburgh
ClinicalTrials.gov Identifier:
NCT00248625
First received: November 3, 2005
Last updated: May 1, 2012
Last verified: April 2012
  Purpose

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.


Condition Intervention Phase
Acute Liver Failure
Hepatic Encephalopathy
Drug: N-acetylcysteine
Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator)
Primary Purpose: Treatment
Official 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.

Resource links provided by NLM:


Further study details as provided by University of Pittsburgh:

Primary Outcome Measures:
  • overall survival rate (spontaneous survival without transplant plus survival following transplantation) at one year following entry into the study. [ Time Frame: One year following entry into the study ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • spontaneous recovery (survival without transplant), transplantation, length of hospital stay, number of organ systems failing, infectious complication, highest coma grade of hepatic ENC and the number of days until recovery or death. [ Time Frame: One year following entry into the study ] [ Designated as safety issue: Yes ]

Enrollment: 184
Study Start Date: January 2000
Study Completion Date: October 2010
Primary Completion Date: September 2009 (Final data collection date for primary outcome measure)
Intervention Details:
    Drug: N-acetylcysteine
    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.
Detailed Description:

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.

  Eligibility

Ages Eligible for Study:   up to 18 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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, ELAD, 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 APAP 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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00248625

  Show 20 Study Locations
Sponsors and Collaborators
University of Pittsburgh
Investigators
Principal Investigator: Robert H Squires, M.D. Children's Hospital of Pittsburgh, University of Pittsburgh
  More Information

Additional Information:
Publications:

Responsible Party: University of Pittsburgh
ClinicalTrials.gov Identifier: NCT00248625     History of Changes
Other Study ID Numbers: IRB #: 0608007, U01DK072146
Study First Received: November 3, 2005
Last Updated: May 1, 2012
Health Authority: United States: Food and Drug Administration

Keywords provided by University of Pittsburgh:
acute liver failure
hepatic encephalopathy
acetaminophen toxicity
N-acetylcysteine

Additional relevant MeSH terms:
Hepatic Encephalopathy
Liver Failure
Liver Failure, Acute
Hepatic Insufficiency
Liver Diseases
Digestive System Diseases
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Metabolic Diseases
Acetaminophen
Acetylcysteine
N-monoacetylcystine
Analgesics, Non-Narcotic
Analgesics
Sensory System Agents
Peripheral Nervous System Agents
Physiological Effects of Drugs
Pharmacologic Actions
Central Nervous System Agents
Therapeutic Uses
Antipyretics
Antiviral Agents
Anti-Infective Agents
Expectorants
Respiratory System Agents
Free Radical Scavengers
Antioxidants
Molecular Mechanisms of Pharmacological Action

ClinicalTrials.gov processed this record on September 30, 2014