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:
University of Pittsburgh
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
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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:
Detailed Description:
-
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.
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
Please refer to this study by its ClinicalTrials.gov identifier: NCT00248625
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Hide Study LocationsLocations
| United States, California | |
| University of California, San Francisco | |
| San Francisco, California, United States, 94143 | |
| United States, Colorado | |
| University of Colorado, Denver Children's Hospital | |
| Denver, Colorado, United States, 80218 | |
| United States, Georgia | |
| Emory University, Children's Healthcare of Atlanta | |
| Atlanta, Georgia, United States, 30322 | |
| United States, Illinois | |
| Children's Memorial Hospital | |
| Chicago, Illinois, United States, 60614 | |
| United States, Indiana | |
| Riley Children's Hospital | |
| Indianapolis, Indiana, United States, 46202 | |
| United States, Maryland | |
| Johns Hopkins University | |
| Baltimore, Maryland, United States, 21287 | |
| United States, Massachusetts | |
| Harvard University, Boston Children's Hospital | |
| Boston, Massachusetts, United States, 02115 | |
| United States, Michigan | |
| University of Michigan | |
| Ann Arbor, Michigan, United States, 48109 | |
| United States, Missouri | |
| St. Louis Children's Hospital | |
| St. Louis, Missouri, United States, 63110 | |
| United States, New York | |
| Mount Sinai Hospital | |
| New York, New York, United States, 10029 | |
| Columbia-Presbyterian | |
| New York, New York, United States, 10032 | |
| United States, Ohio | |
| University of Cincinnati, Cincinnati Children's Hospital | |
| Cincinnati, Ohio, United States, 45229 | |
| United States, Pennsylvania | |
| Children's Hospital of Philadelphia | |
| Philadelphia, Pennsylvania, United States, 19104 | |
| Children's Hospital of Pittsburgh of UPMC | |
| Pittsburgh, Pennsylvania, United States, 15224 | |
| United States, Texas | |
| Children's Medical Center of Dallas | |
| Dallas, Texas, United States, 75235 | |
| Baylor College of Medicine | |
| Houston, Texas, United States, 77030 | |
| United States, Washington | |
| University of Washington | |
| Seattle, Washington, United States, 98105 | |
| Canada, Ontario | |
| Hospital for Sick Children | |
| Toronto, Ontario, Canada, M5G 1X8 | |
| United Kingdom | |
| Birmingham Children's Hospital | |
| Birmingham, United Kingdom, B4 6NH | |
| King's College Hospital (London, UK) | |
| London, United Kingdom, SE59RS | |
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 Brain Damage, Chronic Delirium Encephalitis Neurotoxicity Syndromes 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 |
Confusion Neurobehavioral Manifestations Neurologic Manifestations Signs and Symptoms Delirium, Dementia, Amnestic, Cognitive Disorders Mental Disorders Central Nervous System Viral Diseases Virus Diseases Central Nervous System Infections Poisoning Substance-Related Disorders Acetaminophen Acetylcysteine N-monoacetylcystine Antipyretics |
ClinicalTrials.gov processed this record on May 16, 2013