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Defibrotide for Patients With Hepatic Veno-occlusive Disease: A Treatment IND Study (Treatment IND)

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
Jazz Pharmaceuticals
ClinicalTrials.gov Identifier:
NCT00628498
First received: February 25, 2008
Last updated: September 1, 2016
Last verified: September 2016
  Purpose
Single arm, open-label study to provide Defibrotide to patients diagnosed with VOD. Defibrotide is no longer available though the Emergency Use IND mechanism (also known as compassionate use, or single patient named use). This protocol is the only mechanism by which Defibrotide can be made available to patients in the U.S.

Condition Intervention Phase
Hepatic Veno-Occlusive Disease
Drug: Defibrotide
Phase 3

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Defibrotide for Patients With Hepatic Veno-occlusive Disease: A Treatment IND Study

Resource links provided by NLM:


Further study details as provided by Jazz Pharmaceuticals:

Primary Outcome Measures:
  • Survival at Day +100 or from HSCT or 100 days from start of chemotherapy [ Time Frame: Day +100 from HSCT or 100 days from start of chemotherapy ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Tolerability & Safety Data from Patients with VOD [ Time Frame: From time of Consent to 30 Days Post of Last Administration of Study Drug ] [ Designated as safety issue: Yes ]

Enrollment: 1214
Study Start Date: December 2007
Estimated Study Completion Date: December 2016
Primary Completion Date: July 2016 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Defibrotide
Defibrotide 25 mg/kg day given in 4 divided doses approximately every 6 hours
Drug: Defibrotide

Defibrotide is a single-stranded polydeoxyribonucleotide derived from porcine intestinal mucosa by controlled depolymerisation. Defibrotide has a complex mechanism of action with antithrombotic, anti-ischemic, anti-inflammatory, anti-adhesive and thrombolytic properties but no significant systemic anti-coagulant effects.

Defibrotide is dose intravenously as a 2-hour infusion every 6 hours at a dose of 25 mg/kg/day. Recommended duration of therapy is 21 days.

Drug: Defibrotide

  Eligibility

Ages Eligible for Study:   Child, Adult, Senior
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

Entry criteria include the following:

  1. Clinical diagnosis of VOD, made by Baltimore Criteria, Modified Seattle Criteria, or biopsy proven:

    1.1 Baltimore Criteria- Bilirubin ≥2 mg/dL and at least 2 of the following clinical findings:

    • Ascites (radiographic or physical exam)
    • Weight gain of ≥5% compared to the day of conditioning-- if this value is not available, the weight on the date of admission to the SCT unit may be used)
    • Hepatomegaly; increased over baseline.

    1.2 Modified Seattle Criteria: At least two of the following

    • Bilirubin ≥2 mg/dL
    • Ascites (radiographic or physical exam) and/or weight gain ≥5% above baseline weight (defined as weight on the first day of conditioning- if this value is not available, the weight on the date of admission to the SCT unit may be used)
    • hepatomegaly increased over baseline

    1.3 Patients that do not meet the Baltimore Criteria or Modified Seattle Criteria and have biopsy proven VOD are eligible.

  2. Patient must also provide written informed consent.

Exclusion Criteria:

  • Use of any medication which increases the risk of hemorrhage is disallowed. Use of heparin or other anticoagulants is disallowed within 12 hours unless being used for routine central venous line management, fibrinolytic instillation for central venous line occlusion, intermittent dialysis or ultrafiltration of CVVH.
  • Clinically significant uncontrolled acute bleeding, defined as hemorrhage requiring > 15 cc/kg of packed red blood cells (e.g., a pediatric patient weighing 20 kg and requiring > 300cc of packed red blood cells/24 hours, or an adult patient weighing 70 kg and requiring >3 units of packed red blood cells/24 hours) to replace blood loss, OR bleeding from a site which in the Investigator's opinion constitutes a potential life-threatening source (e.g. pulmonary hemorrhage or CNS bleeding), irrespective of amount of blood loss, at any point from the date of SCT through the date of severe VOD diagnosis.
  • Hemodynamic instability as defined by a requirement for multiple pressors, or inability to maintain mean arterial pressure (for children: to maintain mean arterial pressure within 1 standard deviation of age-adjusted levels) with single pressor support.
  • Woman who are pregnant.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00628498

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Locations
United States, Alabama
Children's Hospital of Alabama
Birmingham, Alabama, United States, 35233
United States, Arizona
Phoenix Children's Hospital
Phoenix, Arizona, United States, 85016
Mayo Clinic Cancer Center
Phoenix, Arizona, United States, 85054
Arizona Oncology
Scottsdale, Arizona, United States, 85258
University Medical Center
Tucson, Arizona, United States, 85724
United States, Arkansas
University of Arkansas for Medical Services
Little Rock, Arkansas, United States, 72205
United States, California
City of Hope Medical Center
Duarte, California, United States, 91010
The University of California, San Diego
La Jolla, California, United States, 92037
Loma Linda University
Loma Linda, California, United States, 92354
Long Beach Memorial Medical Center
Long Beach, California, United States, 90806
Children's Hospital of Los Angeles
Los Angeles, California, United States, 90027
Children's Hospital & Research Center Oakland
Oakland, California, United States, 94609
Children's Hospital of Orange County (CHOC)
Orange, California, United States, 92868
Sutter Institute for Medical Research
Sacramento, California, United States, 95816
University of California Davis Health System
Sacramento, California, United States, 95817
Rady's Children's Hospital - San Diego
San Diego, California, United States, 92123
University of California San Francisco Medical Center
San Francisco, California, United States, 94143
Stanford University Medical Center
Stanford, California, United States, 94305
United States, Colorado
Children's Hospital Colorado
Aurora, Colorado, United States, 80045
US Oncology - Denver
Denver, Colorado, United States, 80218
United States, Connecticut
Yale - New Haven Hospital
New Haven, Connecticut, United States, 06520
United States, Delaware
Nemours-Al duPont Hospital for Children
Wilmington, Delaware, United States, 19803
United States, District of Columbia
Georgetown University Hospital
Washington, District of Columbia, United States, 20007
Children's National Medical Center
Washington, District of Columbia, United States, 20010
United States, Florida
UF Health Shands Hospital
Gainesville, Florida, United States, 32610
Jackson Memorial- University of Miami Health System- Pediatrics
Miami, Florida, United States, 33136
Nicklaus Children's Hospital (Miami Children's Hospital)
Miami, Florida, United States, 33155
Florida Hospital
Orlando, Florida, United States, 42803
All Children's Hospital Specialty Physicians
St Petersburg, Florida, United States, 33701
H. Lee Moffitt Cancer Center & Research Institute
Tampa, Florida, United States, 33612
United States, Georgia
Childrens Healthcare of Atlanta
Atlanta, Georgia, United States, 30322
Emory University Hospital
Atlanta, Georgia, United States, 30322
Northside Hospital
Atlanta, Georgia, United States, 30342
United States, Hawaii
Kapi'olani Medical Center for Women and Children
Honolulu, Hawaii, United States, 96826
United States, Illinois
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States, 60611
Northwest Memorial Hospital
Chicago, Illinois, United States, 60611
Rush University Medical Center
Chicago, Illinois, United States, 60612
Pediatric Hematology/Oncology MC4060 - University of Chicago
Chicago, Illinois, United States, 60637
Cardinal Bernardin Cancer-Loyola University Medical Center
Maywood, Illinois, United States, 60153
United States, Indiana
Riley Hospital for Children
Indianapolis, Indiana, United States, 46202
Indiana Blood and Marrow Transplantation Clinical Lab LLC/St. Francis Hospital
Indianapolis, Indiana, United States, 46237
United States, Iowa
University of Iowa Hospitals & Clinics
Iowa City, Iowa, United States, 52242
United States, Kansas
University of Kansas Cancer Center
Kansas City, Kansas, United States, 66160
United States, Kentucky
Pediatric Blood and Cancer Disorders Clinic
Louisville, Kentucky, United States, 40202
University of Louisville Research Foundation
Louisville, Kentucky, United States, 40202
United States, Louisiana
Tulane University Hospital & Clinic (Tuhc) - Tulane Cancer Center Comprehensive Clinic (Tcccc)
New Orleans, Louisiana, United States, 70112
Children's Hospital
New Orleans, Louisiana, United States, 70118
United States, Maryland
University of Maryland - Greenebaum Cancer Center
Baltimore, Maryland, United States, 21201
Johns Hopkins University
Baltimore, Maryland, United States, 21287
National Institutes of Health
Bethesda, Maryland, United States, 20892
United States, Massachusetts
Tufts Medical Center
Boston, Massachusetts, United States, 02111
Massachusetts General Hospital
Boston, Massachusetts, United States, 02114
Boston Children's Hospital DFCI/CHB-Pediatrics
Boston, Massachusetts, United States, 02115
Dana-Farber Cancer Institute
Boston, Massachusetts, United States, 02115
Beth Israel Deaconess Medical Center
Boston, Massachusetts, United States, 02215
United States, Michigan
University of Michigan Health System
Ann Arbor, Michigan, United States, 48109
Henry Ford Hospital
Detriot, Michigan, United States, 48202
Wayne State University/Children's Hospital of Michigan
Detroit, Michigan, United States, 48201
Helen DeVos Children's Hospital - Spectrum Health
Grand Rapids, Michigan, United States, 49503
United States, Minnesota
Children's Hospitals and Clinics of Minnesota and Minneapolis
Minneapolis, Minnesota, United States, 55404
University of Minnesota Medical Center
Minneapolis, Minnesota, United States, 55455
Mayo Clinic
Rochester, Minnesota, United States, 55905
United States, Mississippi
University of Mississippi Medical Center
Jackson, Mississippi, United States, 39216
United States, Missouri
Children's Mercy Hospital and Clinics - Infectious Diseases
Kansas City, Missouri, United States, 64108
Cardinal Glennon Children's Medical Center/St. Louis University Department of Pediatrics
St. Louis, Missouri, United States, 63104
Washington University/St. Louis Children's Hospital
St. Louis, Missouri, United States, 63110
United States, Nebraska
University of Nebraska Medical Center
Omaha, Nebraska, United States, 68198
United States, New Jersey
Hackensack University Medical Center - Joseph M. Sanzari Childrens Hospital
Hackensack, New Jersey, United States, 07601
Rutgers Cancer Institute of New Jersey
New Brunswick, New Jersey, United States, 08903
United States, New Mexico
Presbyterian Hospital
Albequerque, New Mexico, United States, 87106
United States, New York
Montefiore Medical Center
Bronx, New York, United States, 10467
Roswell Park Cancer Institute
Buffalo, New York, United States, 14263
Steven and Alexandra Cohen Children's Medical Center
New Hyde Park, New York, United States, 11040
New York University School of Medicine
New York, New York, United States, 10016
Memorial Sloan Kettering Cancer Center
New York, New York, United States, 10021
Mount Sinai Hospital - BMT Program
New York, New York, United States, 10029
Mount Sinai Medical Center
New York, New York, United States, 10029
Morgan Stanley Children's Hospital - Columbia University Medical Center
New York, New York, United States, 10032
Weill Cornell Medical College - New York Presbyterian Hospital
New York, New York, United States, 10065
University of Rochester Medical Center, Strong Memorial Hospital
Rochester, New York, United States, 14642
Stony Brook University Hospital
Stony Brook, New York, United States, 11794
New York Medical College
Valhalla, New York, United States, 10595
United States, North Carolina
The University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, United States, 27599
Levine Children's Hospital
Charlotte, North Carolina, United States, 28203
Duke University Medical Center
Durham, North Carolina, United States, 27710
Wake Forest Baptist Health
Winston-Salem, North Carolina, United States, 27157
United States, Ohio
Children's Hospital Medical
Akron, Ohio, United States, 44308
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229
The Jewish Hospital
Cincinnati, Ohio, United States, 45236
Case Western Reserve University (Cwru) - University Hospitals of Cleveland (Uhc)
Cleveland, Ohio, United States, 44106
Robert J Tomsich Pathology & Laboratory Medicine Institute
Cleveland, Ohio, United States, 44195
Nationwide Children's Hospital - Ohio State University College of Medicine (OSUCOM)
Columbus, Ohio, United States, 43205
The Ohio State University, James Cancer Hospital
Columbus, Ohio, United States, 43210
United States, Oklahoma
The University of Oklahoma Health Science Center
Oklahoma City, Oklahoma, United States, 73104
United States, Oregon
Doernbecher Children's Hospital - Oregon Health and Science University
Portland, Oregon, United States, 97239
Oregon Health and Science University
Portland, Oregon, United States, 97239
United States, Pennsylvania
Geisinger Medical Center
Danville, Pennsylvania, United States, 17822
Milton S. Hershey Medical Center
Hershey, Pennsylvania, United States, 17033
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, United States, 15219
West Penn Allegheny Health System
Pittsburgh, Pennsylvania, United States, 15224
United States, South Carolina
Medical University of South Carolina
Charleston, South Carolina, United States, 29425
Greenville Health System Cancer Institute
Greenville, South Carolina, United States, 29605
United States, South Dakota
Avera Cancer Institute
Sioux Falls, South Dakota, United States, 57105
United States, Tennessee
Methodist University Hospital
Memphis, Tennessee, United States, 38104
St. Jude Children's Research Hospital - Department of Bone Marrow Transplantation and Cellular Therapy
Memphis, Tennessee, United States, 38105
The Children's Hospital at TriStar Centennial
Nashville, Tennessee, United States, 37203
Vanderbilt Ingram Cancer Center
Nashville, Tennessee, United States, 37232
United States, Texas
Children's Medical Center of Dallas
Dallas, Texas, United States, 75235
San Antonio Military Medical Center
Fort Sam Houston, Texas, United States, 78234
Cook Children's Health Care Hospital
Fort Worth, Texas, United States, 76104
Baylor College of Medicine
Houston, Texas, United States, 77030
Texas Children's Hospital
Houston, Texas, United States, 77030
The University of Texas - MD Anderson Cancer Center
Houston, Texas, United States, 77030
University of Texas MD Anderson Cancer Center
Houston, Texas, United States, 77030
Methodist Healthcare System of San Antonio
San Antonio, Texas, United States, 78229
United States, Utah
University of Utah
Salt Lake City, Utah, United States, 84132
United States, Virginia
University of Virginia Health System
Charlottesville, Virginia, United States, 22908
Children's Hospital of the King's Daughter
Norfolk, Virginia, United States, 23507
Virginia Commonwealth University
Richmond, Virginia, United States, 23298
United States, Washington
Fred Hutchinson Cancer Research Center/Seattle Children Hospital
Seattle, Washington, United States, 98109
Providence Sacred Heart Medical Center and Children's Hospital
Spokane, Washington, United States, 99204
United States, West Virginia
Mary Babb Randolph Cancer Center - West Virginia University Hospital
Morgantown, West Virginia, United States, 26505
United States, Wisconsin
University of Wisconsin, American Family Children's Hospital, Medical School
Madison, Wisconsin, United States, 53792
Medical College of Wisconsin/ Children's Hospitalof Wisconsin
Milwaukee, Wisconsin, United States, 53226
Sponsors and Collaborators
Jazz Pharmaceuticals
Investigators
Study Chair: William Tappe, M.D. Jazz Pharmaceuticals
Principal Investigator: Paul Richardson, M.D. Dana-Farber Cancer Institute
  More Information

Publications:
Responsible Party: Jazz Pharmaceuticals
ClinicalTrials.gov Identifier: NCT00628498     History of Changes
Other Study ID Numbers: P2006-05 
Study First Received: February 25, 2008
Last Updated: September 1, 2016
Health Authority: United States: Food and Drug Administration

Keywords provided by Jazz Pharmaceuticals:
VOD

Additional relevant MeSH terms:
Hepatic Veno-Occlusive Disease
Liver Diseases
Digestive System Diseases
Vascular Diseases
Cardiovascular Diseases
Defibrotide
Fibrinolytic Agents
Fibrin Modulating Agents
Molecular Mechanisms of Pharmacological Action
Platelet Aggregation Inhibitors

ClinicalTrials.gov processed this record on September 23, 2016