Phase 3 N=184 Randomized Triple-blind Treatment

N-acetylcysteine in Non-Acetaminophen Pediatric Acute Liver Failure

Acute Liver Failure · Hepatic Encephalopathy

Bottom Line

View on ClinicalTrials.gov: NCT00248625 ↗

Enrolled (actual)

184

Serious AEs

4.9%

Results posted

Jul 2016

Primary outcome: Primary: Survival — 68; 76 Participants — p=0.19

Study Design & Population

Study type: Interventional
Phase: Phase 3
Interventions: N-acetylcysteine (Drug); Placebo (Drug)
Age: Pediatric, Adult
Sex: All
Sponsor: University of Pittsburgh
Primary completion: Sep 2009

Outcome Measures

Outcome	Result	p-value
PRIMARY Survival	68; 76	0.19
SECONDARY Spontaneous Recovery	33; 49	0.03 sig
SECONDARY Cumulative Percent Incidence of Transplantation by 1 Year	45; 35	0.21
SECONDARY Length of Hospital Stay	103; 21	0.053
SECONDARY Categorized Length of ICU Stay	29; 34; 3; 4; 3; 4	0.29
SECONDARY Number of Organ Systems Failing	44; 46; 21; 23; 15; 12	0.74
SECONDARY Highest Coma Grade of Hepatic Encephalopathy	28; 27; 16; 21; 20; 19	0.54
SECONDARY Infectious Complication	20; 21	0.86

Summary

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.

Eligibility 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, 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

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT00248625). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.