NCT00004467

Brief Summary

OBJECTIVES: I. Determine the safety and efficacy of a short course (72 hours) of intravenous acetylcysteine in patients with acute liver failure for whom no antidote or specific treatment is available.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
173

participants targeted

Target at P25-P50 for phase_3

Timeline
Completed

Started Jun 1998

Longer than P75 for phase_3

Geographic Reach
1 country

25 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

June 1, 1998

Completed
1.4 years until next milestone

First Submitted

Initial submission to the registry

October 18, 1999

Completed
1 day until next milestone

First Posted

Study publicly available on registry

October 19, 1999

Completed
7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

November 1, 2006

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

November 1, 2006

Completed
Last Updated

October 13, 2017

Status Verified

October 1, 2017

Enrollment Period

8.4 years

First QC Date

October 18, 1999

Last Update Submit

October 11, 2017

Conditions

Keywords

acute liver failuregastrointestinal disordersrare disease

Outcome Measures

Primary Outcomes (1)

  • Overall survival rate

    3 weeks

Secondary Outcomes (4)

  • Survival without liver transplantation (Spontaneous Survival

    3 weeks

  • Transplant rate

    3 weeks

  • Length of hospital stay

    3 weeks

  • Number of organ systems showing failure

    3 weeks

Study Arms (2)

Placebo

PLACEBO COMPARATOR
Drug: Placebo

N-acetylcysteine (NAC)

EXPERIMENTAL
Drug: N-acetylcysteine (NAC)

Interventions

Infusion of 5% dextrose with N-acetylcysteine with an initial loading dose of 150 mg/kg/h of NAC over 1 hour, followed by 12.5 mg/kg/h for 4 hours, then continuous infusions of 6.25 mg/kg/h for the remaining 67 hours.

Also known as: Acetadote
N-acetylcysteine (NAC)

Infusion of 5% dextrose

Placebo

Eligibility Criteria

Age18 Years - 70 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • This is a phase III blinded study, which will involve approximately 200 patients. For this purpose, acute liver failure will be defined as onset of any mental status alteration and coagulopathy (INR \> 1.5) within 26 weeks of onset of a hepatitic illness, with no evidence of underlying chronic liver disease. Eligible patients will be those admitted to study site hospital intensive care units with acute liver failure and who can be evaluated and started on treatment within the first 24 hours of hospitalization or those who evolve to altered mentation if already in the hospital. All subjects will be between 18 and 70 years. Patients transferred from referring hospitals to a study site may be considered for enrollment, provided that no other specific treatment protocol has begun, and that no liver support device (bioartificial liver (BAL), extracorporeal liver assist device (ELAD), transgenic pig perfusion) has been used or is contemplated. Use of fresh frozen plasma infusions will not disqualify patients from participation.

You may not qualify if:

  • Patients less than age 18 or over 70 years of age.
  • ALF patients where acetaminophen or mushroom poisoning is assessed or Suspected to be a significant contributing or sole cause of the illness. Both these diagnoses require specific antidote therapy, including NAC in the case of acetaminophen, rather than randomized or non-specific treatment.
  • Patients with a diagnosis of shock liver (ischemic hepatopathy), since the overall outcome for these patients in largely determined by the underlying etiology of the condition leading to shock. Heat stroke is not excluded.
  • Acute liver failure of pregnancy or the HELLP syndrome (pregnancy associated hemolysis and coagulopathy). The effect of NAC on the fetus or the mother has not been determined; in addition, pregnancy-related liver diseases usually mandate rapid delivery of the infant.
  • ALF thought secondary to intrahepatic malignancy. Patients with hepatic malignancy experiencing ALF have 100% mortality and are not transplant candidates.
  • Patients who exhibit signs of cerebral herniation at the time of enrollment.
  • Patients who demonstrate the presence of intractable arterial hypotension (arterial systolic blood pressure equal to or below 70 mmHg) present, or require inotropic drugs at the time of enrollment.
  • Severe sepsis (temperature \>39o C and/or significant bacteremia) present at the time of enrollment.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (25)

University of Alabama at Birmingham

Birmingham, Alabama, 35294-0005, United States

Location

Mayo Clinic

Scottsdale, Arizona, 85259, United States

Location

University of California Los Angeles

Los Angeles, California, 90024, United States

Location

University of California Davis

Sacramento, California, 95817, United States

Location

University of California San Diego

San Diego, California, 92103-0707, United States

Location

University of California San Francisco

San Francisco, California, 94115, United States

Location

Mayo Clinic

Jacksonville, Florida, 32216, United States

Location

Northwestern University Medical School

Chicago, Illinois, 60611, United States

Location

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

Location

University of Michigan Health Systems

Ann Arbor, Michigan, 48109, United States

Location

Mayo Clinic

Rochester, Minnesota, 55905, United States

Location

Washington University School of Medicine

St Louis, Missouri, 63110, United States

Location

University of Nebraska Medical Center

Omaha, Nebraska, 68198-3330, United States

Location

Mount Sinai Medical Center, NY

New York, New York, 10029, United States

Location

New York Presbyterian Hospital

New York, New York, 10032-3784, United States

Location

Duke University Medical Center

Durham, North Carolina, 27715, United States

Location

Oregon Health Sciences University

Portland, Oregon, 97201-3098, United States

Location

University of Pennsylvania

Philadelphia, Pennsylvania, 19104, United States

Location

Albert Einstein Medical Center

Philadelphia, Pennsylvania, 19141, United States

Location

University of Pittsburgh Medical Center

Pittsburgh, Pennsylvania, 15213, United States

Location

Medical University of South Carolina

Charleston, South Carolina, 29425, United States

Location

University of Texas Southwestern Medical Center at Dallas

Dallas, Texas, 75235-8897, United States

Location

Baylor University Medical Center

Dallas, Texas, 75246, United States

Location

Virginia Commonwealth University

Richmond, Virginia, 23298-0341, United States

Location

University of Washington Medical Center

Seattle, Washington, 98195-6043, United States

Location

Related Publications (3)

  • Lee WM, Hynan LS, Rossaro L, Fontana RJ, Stravitz RT, Larson AM, Davern TJ 2nd, Murray NG, McCashland T, Reisch JS, Robuck PR; Acute Liver Failure Study Group. Intravenous N-acetylcysteine improves transplant-free survival in early stage non-acetaminophen acute liver failure. Gastroenterology. 2009 Sep;137(3):856-64, 864.e1. doi: 10.1053/j.gastro.2009.06.006. Epub 2009 Jun 12.

  • Stravitz RT, Fontana RJ, Karvellas C, Durkalski V, McGuire B, Rule JA, Tujios S, Lee WM; Acute Liver Failure Study Group. Future directions in acute liver failure. Hepatology. 2023 Oct 1;78(4):1266-1289. doi: 10.1097/HEP.0000000000000458. Epub 2023 May 16.

  • Singh S, Hynan LS, Rule JA, Lee WM. Changes in alpha-foetoprotein and Gc-globulin in relation to outcomes in non-acetaminophen acute liver failure. Liver Int. 2019 Dec;39(12):2368-2373. doi: 10.1111/liv.14216. Epub 2019 Sep 10.

Related Links

MeSH Terms

Conditions

Liver Failure, AcuteGastrointestinal DiseasesRare Diseases

Interventions

Acetylcysteine

Condition Hierarchy (Ancestors)

Liver FailureHepatic InsufficiencyLiver DiseasesDigestive System DiseasesDisease AttributesPathologic ProcessesPathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

CysteineAmino Acids, SulfurSulfur CompoundsOrganic ChemicalsAmino AcidsAmino Acids, Peptides, and Proteins

Study Officials

  • William M. Lee, MD

    University of Texas Southwestern Medical Center at Dallas, Dallas, TX

    STUDY CHAIR

Study Design

Study Type
interventional
Phase
phase 3
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
NIH
Responsible Party
SPONSOR
Expanded Access
Yes

Study Record Dates

First Submitted

October 18, 1999

First Posted

October 19, 1999

Study Start

June 1, 1998

Primary Completion

November 1, 2006

Study Completion

November 1, 2006

Last Updated

October 13, 2017

Record last verified: 2017-10

Locations