A Multi-Center Group to Study Acute Liver Failure in Children
11. januar 2016 opdateret af: University of Pittsburgh
The PALF study group began with 20 sites and now continues with 12 sites (11 in the United States and 1 in Canada) in the new funding period.
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.
Studieoversigt
Status
Afsluttet
Betingelser
Detaljeret beskrivelse
The PALF study group will collect clinical, epidemiological and outcome data on children with ALF.
This information will be used to develop methods to predict whether a child will recover from the illness without the need for a liver transplant or other life-saving procedure.
We believe the methods to predict survival will vary with different patient age groups, but that diagnosis, multi-system organ failure, degree of encephalopathy and level of coagulopathy will be important regardless of patient age.
Biological samples, such as blood and liver tissue, will provide opportunities to identify subgroups of patients who have unique treatment requirements and outcomes.
In addition, we hope to identify unrecognized mechanisms of liver injury resulting in ALF in children.
Eligible study participants will be invited to participate in neurocognitive testing.
Since patients that develop acute liver failure experience varying levels of hepatic encephalopathy and cerebral edema, we suspect that there may be residual sub-clinical neurological injury that compromises long-term neurocognitive function.
Detailed neurocognitive testing has never been performed in a cohort of children that survive acute liver failure and this study seeks to close that information gap by defining the spectrum of neurocognitive outcomes in this population.
Undersøgelsestype
Observationel
Tilmelding (Faktiske)
158
Kontakter og lokationer
Dette afsnit indeholder kontaktoplysninger for dem, der udfører undersøgelsen, og oplysninger om, hvor denne undersøgelse udføres.
Studiesteder
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Ontario
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Toronto, Ontario, Canada, M5G 1X8
- Hospital for Sick Children
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Birmingham, Det Forenede Kongerige, B4 6NH
- Birmingham Children's Hospital
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London, Det Forenede Kongerige, SE59RS
- King's College Hospital (London, UK)
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California
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San Francisco, California, Forenede Stater, 94143
- University of California, San Francisco
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Colorado
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Aurora, Colorado, Forenede Stater, 80045
- Children's Hospital Colorado
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Georgia
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Atlanta, Georgia, Forenede Stater, 30322
- Emory University, Children's Healthcare of Atlanta
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Illinois
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Chicago, Illinois, Forenede Stater, 60611
- Ann & Robert H. Lurie Children's Hospital of Chicago
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Indiana
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Indianapolis, Indiana, Forenede Stater, 46202
- Riley Children's Hospital
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Maryland
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Baltimore, Maryland, Forenede Stater, 21287
- Johns Hopkins University
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Massachusetts
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Boston, Massachusetts, Forenede Stater, 02115
- Harvard University, Boston Children's Hospital
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Michigan
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Ann Arbor, Michigan, Forenede Stater, 48109
- University of Michigan
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Missouri
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St. Louis, Missouri, Forenede Stater, 63110
- St. Louis Children's Hospital
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New York
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New York, New York, Forenede Stater, 10029
- Mount Sinai Hospital
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New York, New York, Forenede Stater, 10032
- Columbia-Presbyterian
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Ohio
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Cincinnati, Ohio, Forenede Stater, 45229
- University of Cincinnati, Cincinnati Children's Hospital
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Pennsylvania
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Philadelphia, Pennsylvania, Forenede Stater, 19104
- Children's Hospital of Philadelphia
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Pittsburgh, Pennsylvania, Forenede Stater, 15224
- Children's Hospital of Pittsburgh of UPMC
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Texas
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Dallas, Texas, Forenede Stater, 75235
- Children's Medical Center of Dallas
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Houston, Texas, Forenede Stater, 77030
- Baylor College of Medicine
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Washington
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Seattle, Washington, Forenede Stater, 98105
- University of Washington
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Deltagelseskriterier
Forskere leder efter personer, der passer til en bestemt beskrivelse, kaldet berettigelseskriterier. Nogle eksempler på disse kriterier er en persons generelle helbredstilstand eller tidligere behandlinger.
Berettigelseskriterier
Aldre berettiget til at studere
Ikke ældre end 18 år (Barn, Voksen)
Tager imod sunde frivillige
Ingen
Køn, der er berettiget til at studere
Alle
Prøveudtagningsmetode
Sandsynlighedsprøve
Studiebefolkning
Children between birth and age 18 years who present with acute liver failure.
Beskrivelse
Inclusion Criteria:
- Written informed consent/assent
- Birth through 17 years of age
- Biochemical evidence of acute liver injury
Coagulopathy not corrected by vitamin K (or other intervention intended to correct coagulopathy)
- The presence of encephalopathy (ENC) is required if the INR is at least 1.5 and less than 2.0
- If INR is at least 2.0, the presence of ENC is not required
Exclusion Criteria:
- Known chronic underlying liver disease
- Multi-organ system failure following heart surgery or ECMO
- Solid organ or bone marrow transplantation
- Acute trauma
- Previously enrolled in the PALF Cohort Study
- Other severe illness, condition, or other reason in the opinion of the investigator that would make the patient unsuitable for the study
Studieplan
Dette afsnit indeholder detaljer om studieplanen, herunder hvordan undersøgelsen er designet, og hvad undersøgelsen måler.
Hvordan er undersøgelsen tilrettelagt?
Design detaljer
Samarbejdspartnere og efterforskere
Det er her, du vil finde personer og organisationer, der er involveret i denne undersøgelse.
Sponsor
Samarbejdspartnere
Efterforskere
- Ledende efterforsker: Robert H Squires, MD, Children's Hospital of Pittsburgh of UPMC, University of Pittsburgh
Publikationer og nyttige links
Den person, der er ansvarlig for at indtaste oplysninger om undersøgelsen, leverer frivilligt disse publikationer. Disse kan handle om alt relateret til undersøgelsen.
Generelle publikationer
- Narkewicz MR, Dell Olio D, Karpen SJ, Murray KF, Schwarz K, Yazigi N, Zhang S, Belle SH, Squires RH; Pediatric Acute Liver Failure Study Group. Pattern of diagnostic evaluation for the causes of pediatric acute liver failure: an opportunity for quality improvement. J Pediatr. 2009 Dec;155(6):801-806.e1. doi: 10.1016/j.jpeds.2009.06.005. Epub 2009 Jul 29.
- Rudnick DA, Dietzen DJ, Turmelle YP, Shepherd R, Zhang S, Belle SH, Squires R; Pediatric Acute Liver Failure Study Group. Serum alpha-NH-butyric acid may predict spontaneous survival in pediatric acute liver failure. Pediatr Transplant. 2009 Mar;13(2):223-30. doi: 10.1111/j.1399-3046.2008.00998.x. Epub 2008 Jul 17.
- James LP, Alonso EM, Hynan LS, Hinson JA, Davern TJ, Lee WM, Squires RH; Pediatric Acute Liver Failure Study Group. Detection of acetaminophen protein adducts in children with acute liver failure of indeterminate cause. Pediatrics. 2006 Sep;118(3):e676-81. doi: 10.1542/peds.2006-0069.
- Squires RH Jr, Shneider BL, Bucuvalas J, Alonso E, Sokol RJ, Narkewicz MR, Dhawan A, Rosenthal P, Rodriguez-Baez N, Murray KF, Horslen S, Martin MG, Lopez MJ, Soriano H, McGuire BM, Jonas MM, Yazigi N, Shepherd RW, Schwarz K, Lobritto S, Thomas DW, Lavine JE, Karpen S, Ng V, Kelly D, Simonds N, Hynan LS. Acute liver failure in children: the first 348 patients in the pediatric acute liver failure study group. J Pediatr. 2006 May;148(5):652-658. doi: 10.1016/j.jpeds.2005.12.051.
- Alonso EM. Acute liver failure in children: the role of defects in fatty acid oxidation. Hepatology. 2005 Apr;41(4):696-9. doi: 10.1002/hep.20680. No abstract available.
- Shneider BL, Rinaldo P, Emre S, Bucuvalas J, Squires R, Narkewicz M, Gondolesi G, Magid M, Morotti R, Hynan LS. Abnormal concentrations of esterified carnitine in bile: a feature of pediatric acute liver failure with poor prognosis. Hepatology. 2005 Apr;41(4):717-21. doi: 10.1002/hep.20631.
- Sundaram SS, Alonso EM, Narkewicz MR, Zhang S, Squires RH; Pediatric Acute Liver Failure Study Group. Characterization and outcomes of young infants with acute liver failure. J Pediatr. 2011 Nov;159(5):813-818.e1. doi: 10.1016/j.jpeds.2011.04.016. Epub 2011 May 31.
- Sundaram V, Shneider BL, Dhawan A, Ng VL, Im K, Belle S, Squires RH. King's College Hospital Criteria for non-acetaminophen induced acute liver failure in an international cohort of children. J Pediatr. 2013 Feb;162(2):319-23.e1. doi: 10.1016/j.jpeds.2012.07.002. Epub 2012 Aug 18.
- Leonis MA, Alonso EM, Im K, Belle SH, Squires RH; Pediatric Acute Liver Failure Study Group. Chronic acetaminophen exposure in pediatric acute liver failure. Pediatrics. 2013 Mar;131(3):e740-6. doi: 10.1542/peds.2011-3035. Epub 2013 Feb 25.
- Bucuvalas J, Filipovich L, Yazigi N, Narkewicz MR, Ng V, Belle SH, Zhang S, Squires RH. Immunophenotype predicts outcome in pediatric acute liver failure. J Pediatr Gastroenterol Nutr. 2013 Mar;56(3):311-5. doi: 10.1097/MPG.0b013e31827a78b2.
- Lu BR, Zhang S, Narkewicz MR, Belle SH, Squires RH, Sokol RJ; Pediatric Acute Liver Failure Study Group. Evaluation of the liver injury unit scoring system to predict survival in a multinational study of pediatric acute liver failure. J Pediatr. 2013 May;162(5):1010-6.e1-4. doi: 10.1016/j.jpeds.2012.11.021. Epub 2012 Dec 20.
- Feldman AG, Sokol RJ, Hardison RM, Alonso EM, Squires RH, Narkewicz MR; Pediatric Acute Liver Failure Study Group. Lactate and Lactate: Pyruvate Ratio in the Diagnosis and Outcomes of Pediatric Acute Liver Failure. J Pediatr. 2017 Mar;182:217-222.e3. doi: 10.1016/j.jpeds.2016.12.031. Epub 2017 Jan 12.
- Narkewicz MR, Horslen S, Belle SH, Rudnick DA, Ng VL, Rosenthal P, Romero R, Loomes KM, Zhang S, Hardison RM, Squires RH; Pediatric Acute Liver Failure Study Group. Prevalence and Significance of Autoantibodies in Children With Acute Liver Failure. J Pediatr Gastroenterol Nutr. 2017 Feb;64(2):210-217. doi: 10.1097/MPG.0000000000001363.
Datoer for undersøgelser
Disse datoer sporer fremskridtene for indsendelser af undersøgelsesrekord og resumeresultater til ClinicalTrials.gov. Studieregistreringer og rapporterede resultater gennemgås af National Library of Medicine (NLM) for at sikre, at de opfylder specifikke kvalitetskontrolstandarder, før de offentliggøres på den offentlige hjemmeside.
Studer store datoer
Studiestart
1. januar 2000
Primær færdiggørelse (Faktiske)
1. december 2015
Studieafslutning (Faktiske)
1. december 2015
Datoer for studieregistrering
Først indsendt
29. september 2009
Først indsendt, der opfyldte QC-kriterier
29. september 2009
Først opslået (Skøn)
30. september 2009
Opdateringer af undersøgelsesjournaler
Sidste opdatering sendt (Skøn)
12. januar 2016
Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier
11. januar 2016
Sidst verificeret
1. januar 2016
Mere information
Begreber relateret til denne undersøgelse
Yderligere relevante MeSH-vilkår
Andre undersøgelses-id-numre
- 1U01DK072146-01 (U.S. NIH-bevilling/kontrakt)
- U01DK072146 (U.S. NIH-bevilling/kontrakt)
- 2U01DK072146-06 (U.S. NIH-bevilling/kontrakt)
Disse oplysninger blev hentet direkte fra webstedet clinicaltrials.gov uden ændringer. Hvis du har nogen anmodninger om at ændre, fjerne eller opdatere dine undersøgelsesoplysninger, bedes du kontakte register@clinicaltrials.gov. Så snart en ændring er implementeret på clinicaltrials.gov, vil denne også blive opdateret automatisk på vores hjemmeside .