Defibrotide for the Treatment of Severe Hepatic Veno-Occlusive Disease in Hematopoetic Stem Cell Transplant Patients
NCT ID: NCT00358501
Last Updated: 2017-01-30
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE3
134 participants
INTERVENTIONAL
2006-07-31
2015-03-31
Brief Summary
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Detailed Description
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In this study, the term "severe VOD" is defined as those patients who meet the Baltimore diagnostic criteria for VOD (total bilirubin \>/= 2.0 mg/dL plus two of the following: ascites, \>/=5% weight gain and hepatomegaly), who also have multi-organ failure (i.e., pulmonary and/or renal dysfunction). This represents a group of patients in whom mortality at Day+100 has been estimated to be \>80%.
Comparisons: The primary parameter is Complete Response at 100 days following stem cell transplant, utilizing historical controls as a comparator. The historical control database will be generated through a retrospective medical chart review performed at participating centers; the survival outcome of patients who would otherwise have met eligibility criteria for this trial will be compared to the survival observed in patients prospectively treated with Defibrotide. Secondary parameters include survival rate at 100 days and 6 months post stem cell transplantation (SCT), and special studies of endothelial and serum markers for VOD. This study will assess safety of the dose and schedule in this setting.
Conditions
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Study Design
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NON_RANDOMIZED
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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Defibrotide
Defibrotide treatment
Defibrotide
Defibrotide 6.25 mg/kg i.v. administered four times a day via 2 hour continuous infusion. Minimum duration 21 days.
Historical Control
Historical control group
No interventions assigned to this group
Interventions
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Defibrotide
Defibrotide 6.25 mg/kg i.v. administered four times a day via 2 hour continuous infusion. Minimum duration 21 days.
Eligibility Criteria
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Inclusion Criteria
* Severe VOD, defined as VOD with multi-organ failure, i.e., presence of one or both of the following, by Day+28 post stem cell transplant: renal or pulmonary dysfunction.
* Provide voluntary written informed consent.
Exclusion Criteria
* An alternative diagnosis for weight gain, ascites and jaundice
* Graft-versus-host disease (GVHD) grade B or higher involving liver or gut or grade C or higher involving skin
* Prior solid organ transplant
* Dependent on dialysis prior to and/or at the time of SCT
* Dependent on oxygen supplementation prior to SCT
* Significant acute bleeding or hemodynamic instability
* Requirement for the use of any medications that increase risk of hemorrhage will be excluded from the treatment group
ALL
No
Sponsors
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FDA Office of Orphan Products Development
FED
Jazz Pharmaceuticals
INDUSTRY
Responsible Party
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Principal Investigators
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Paul Richardson, M.D.
Role: PRINCIPAL_INVESTIGATOR
Dana-Farber Cancer Institute
Locations
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Duarte, California, United States
Palo Alto, California, United States
Denver, Colorado, United States
Atlanta, Georgia, United States
Chicago, Illinois, United States
Maywood, Illinois, United States
Indianapolis, Indiana, United States
Baltimore, Maryland, United States
Boston, Massachusetts, United States
Ann Arbor, Michigan, United States
Minneapolis, Minnesota, United States
Rochester, Minnesota, United States
St Louis, Missouri, United States
Omaha, Nebraska, United States
Hackensack, New Jersey, United States
New York, New York, United States
Durham, North Carolina, United States
Columbus, Ohio, United States
Portland, Oregon, United States
Philadelphia, Pennsylvania, United States
Houston, Texas, United States
Seattle, Washington, United States
Vancouver, British Columbia, Canada
Toronto, Ontario, Canada
Montreal, Quebec, Canada
Countries
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References
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Richardson PG, Murakami C, Jin Z, Warren D, Momtaz P, Hoppensteadt D, Elias AD, Antin JH, Soiffer R, Spitzer T, Avigan D, Bearman SI, Martin PL, Kurtzberg J, Vredenburgh J, Chen AR, Arai S, Vogelsang G, McDonald GB, Guinan EC. Multi-institutional use of defibrotide in 88 patients after stem cell transplantation with severe veno-occlusive disease and multisystem organ failure: response without significant toxicity in a high-risk population and factors predictive of outcome. Blood. 2002 Dec 15;100(13):4337-43. doi: 10.1182/blood-2002-04-1216. Epub 2002 Aug 1.
Richardson, Soiffer, Antin, Voss, Jin, Kurtzberget al. Defibrotide (DF) for the Treatment of Severe Veno-Occlusive Disease (VOD) and Multi-System Organ Failure (MOF) Post SCT: Final Results of a Phase II, Multicenter, Randomized Study and Preliminary Analyses of Surrogate Markers and Ultrasound Findings. [abstract]. Blood. 2004;104 (11).
Richardson PG, Riches ML, Kernan NA, Brochstein JA, Mineishi S, Termuhlen AM, Arai S, Grupp SA, Guinan EC, Martin PL, Steinbach G, Krishnan A, Nemecek ER, Giralt S, Rodriguez T, Duerst R, Doyle J, Antin JH, Smith A, Lehmann L, Champlin R, Gillio A, Bajwa R, D'Agostino RB Sr, Massaro J, Warren D, Miloslavsky M, Hume RL, Iacobelli M, Nejadnik B, Hannah AL, Soiffer RJ. Phase 3 trial of defibrotide for the treatment of severe veno-occlusive disease and multi-organ failure. Blood. 2016 Mar 31;127(13):1656-65. doi: 10.1182/blood-2015-10-676924. Epub 2016 Jan 29.
Related Links
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Click here for more information about Defibrotide.
Other Identifiers
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2005-01
Identifier Type: -
Identifier Source: org_study_id
NCT00410917
Identifier Type: -
Identifier Source: nct_alias
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