Defibrotide for Patients With Hepatic Veno-occlusive Disease: A Treatment IND Study
- Registration Number
- NCT00628498
- Lead Sponsor
- Jazz Pharmaceuticals
- Brief Summary
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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 1206
Entry criteria include the following:
-
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.
-
Patient must also provide written informed consent.
- 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.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Defibrotide Defibrotide Defibrotide 25 mg/kg day given in 4 divided doses approximately every 6 hours
- Primary Outcome Measures
Name Time Method Survival by Day+100 Post Stem Cell Transplant or Chemotherapy Day +100 from HSCT or 100 days from start of chemotherapy
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (125)
The Children's Hospital of Philadelphia
🇺🇸Philadelphia, Pennsylvania, United States
University of Maryland - Greenebaum Cancer Center
🇺🇸Baltimore, Maryland, United States
Johns Hopkins University
🇺🇸Baltimore, Maryland, United States
Pediatric Hematology/Oncology MC4060 - University of Chicago
🇺🇸Chicago, Illinois, United States
Children's Hospital of Pittsburgh of UPMC
🇺🇸Pittsburgh, Pennsylvania, United States
West Penn Allegheny Health System
🇺🇸Pittsburgh, Pennsylvania, United States
Jackson Memorial- University of Miami Health System- Pediatrics
🇺🇸Miami, Florida, United States
Tufts Medical Center
🇺🇸Boston, Massachusetts, United States
Riley Hospital for Children
🇺🇸Indianapolis, Indiana, United States
Indiana Blood and Marrow Transplantation Clinical Lab LLC/St. Francis Hospital
🇺🇸Indianapolis, Indiana, United States
Massachusetts General Hospital
🇺🇸Boston, Massachusetts, United States
Boston Children's Hospital DFCI/CHB-Pediatrics
🇺🇸Boston, Massachusetts, United States
Beth Israel Deaconess Medical Center
🇺🇸Boston, Massachusetts, United States
Children's Hospitals and Clinics of Minnesota and Minneapolis
🇺🇸Minneapolis, Minnesota, United States
University of Minnesota Medical Center
🇺🇸Minneapolis, Minnesota, United States
Robert J Tomsich Pathology & Laboratory Medicine Institute
🇺🇸Cleveland, Ohio, United States
Cincinnati Children's Hospital Medical Center
🇺🇸Cincinnati, Ohio, United States
The Jewish Hospital
🇺🇸Cincinnati, Ohio, United States
Baylor College of Medicine
🇺🇸Houston, Texas, United States
Texas Children's Hospital
🇺🇸Houston, Texas, United States
The University of Texas - MD Anderson Cancer Center
🇺🇸Houston, Texas, United States
University of Texas MD Anderson Cancer Center
🇺🇸Houston, Texas, United States
Methodist Healthcare System of San Antonio
🇺🇸San Antonio, Texas, United States
Fred Hutchinson Cancer Research Center/Seattle Children Hospital
🇺🇸Seattle, Washington, United States
University of Mississippi Medical Center
🇺🇸Jackson, Mississippi, United States
UF Health Shands Hospital
🇺🇸Gainesville, Florida, United States
Loma Linda University
🇺🇸Loma Linda, California, United States
Children's Hospital of Los Angeles
🇺🇸Los Angeles, California, United States
Nemours-Al duPont Hospital for Children
🇺🇸Wilmington, Delaware, United States
University of Arkansas for Medical Services
🇺🇸Little Rock, Arkansas, United States
Children's Hospital of Orange County (CHOC)
🇺🇸Orange, California, United States
Long Beach Memorial Medical Center
🇺🇸Long Beach, California, United States
Georgetown University Hospital
🇺🇸Washington, District of Columbia, United States
The University of California, San Diego
🇺🇸La Jolla, California, United States
Emory University Hospital
🇺🇸Atlanta, Georgia, United States
City of Hope Medical Center
🇺🇸Duarte, California, United States
Childrens Healthcare of Atlanta
🇺🇸Atlanta, Georgia, United States
Rady's Children's Hospital - San Diego
🇺🇸San Diego, California, United States
Stanford University Medical Center
🇺🇸Stanford, California, United States
Ann & Robert H. Lurie Children's Hospital of Chicago
🇺🇸Chicago, Illinois, United States
Dana-Farber Cancer Institute
🇺🇸Boston, Massachusetts, United States
Cardinal Bernardin Cancer-Loyola University Medical Center
🇺🇸Maywood, Illinois, United States
Nicklaus Children's Hospital (Miami Children's Hospital)
🇺🇸Miami, Florida, United States
All Children's Hospital Specialty Physicians
🇺🇸Saint Petersburg, Florida, United States
Helen DeVos Children's Hospital - Spectrum Health
🇺🇸Grand Rapids, Michigan, United States
Northwest Memorial Hospital
🇺🇸Chicago, Illinois, United States
Levine Children's Hospital
🇺🇸Charlotte, North Carolina, United States
Washington University/St. Louis Children's Hospital
🇺🇸Saint Louis, Missouri, United States
Memorial Sloan Kettering Cancer Center
🇺🇸New York, New York, United States
Morgan Stanley Children's Hospital - Columbia University Medical Center
🇺🇸New York, New York, United States
Hackensack University Medical Center - Joseph M. Sanzari Childrens Hospital
🇺🇸Hackensack, New Jersey, United States
University of Rochester Medical Center, Strong Memorial Hospital
🇺🇸Rochester, New York, United States
The Ohio State University, James Cancer Hospital
🇺🇸Columbus, Ohio, United States
Roswell Park Cancer Institute
🇺🇸Buffalo, New York, United States
Steven and Alexandra Cohen Children's Medical Center
🇺🇸New Hyde Park, New York, United States
New York University School of Medicine
🇺🇸New York, New York, United States
Case Western Reserve University (Cwru) - University Hospitals of Cleveland (Uhc)
🇺🇸Cleveland, Ohio, United States
Children's Hospital Medical
🇺🇸Akron, Ohio, United States
Avera Cancer Institute
🇺🇸Sioux Falls, South Dakota, United States
San Antonio Military Medical Center
🇺🇸Fort Sam Houston, Texas, United States
University of Wisconsin, American Family Children's Hospital, Medical School
🇺🇸Madison, Wisconsin, United States
New York Medical College
🇺🇸Valhalla, New York, United States
University of Iowa Hospitals & Clinics
🇺🇸Iowa City, Iowa, United States
Arizona Oncology
🇺🇸Scottsdale, Arizona, United States
University Medical Center
🇺🇸Tucson, Arizona, United States
Children's Hospital & Research Center Oakland
🇺🇸Oakland, California, United States
Children's National Medical Center
🇺🇸Washington, District of Columbia, United States
Northside Hospital
🇺🇸Atlanta, Georgia, United States
University of Kansas Cancer Center
🇺🇸Kansas City, Kansas, United States
Cardinal Glennon Children's Medical Center/St. Louis University Department of Pediatrics
🇺🇸Saint Louis, Missouri, United States
Rutgers Cancer Institute of New Jersey
🇺🇸New Brunswick, New Jersey, United States
Mount Sinai Medical Center
🇺🇸New York, New York, United States
Mount Sinai Hospital - BMT Program
🇺🇸New York, New York, United States
Weill Cornell Medical College - New York Presbyterian Hospital
🇺🇸New York, New York, United States
Geisinger Medical Center
🇺🇸Danville, Pennsylvania, United States
Greenville Health System Cancer Institute
🇺🇸Greenville, South Carolina, United States
Methodist University Hospital
🇺🇸Memphis, Tennessee, United States
St. Jude Children's Research Hospital - Department of Bone Marrow Transplantation and Cellular Therapy
🇺🇸Memphis, Tennessee, United States
Cook Children's Health Care Hospital
🇺🇸Fort Worth, Texas, United States
Children's Medical Center of Dallas
🇺🇸Dallas, Texas, United States
Children's Hospital of the King's Daughter
🇺🇸Norfolk, Virginia, United States
Stony Brook University Hospital
🇺🇸Stony Brook, New York, United States
Presbyterian Hospital
🇺🇸Albuquerque, New Mexico, United States
Rush University Medical Center
🇺🇸Chicago, Illinois, United States
Milton S. Hershey Medical Center
🇺🇸Hershey, Pennsylvania, United States
Nationwide Children's Hospital - Ohio State University College of Medicine (OSUCOM)
🇺🇸Columbus, Ohio, United States
University of Virginia Health System
🇺🇸Charlottesville, Virginia, United States
Duke University Medical Center
🇺🇸Durham, North Carolina, United States
Providence Sacred Heart Medical Center and Children's Hospital
🇺🇸Spokane, Washington, United States
Mary Babb Randolph Cancer Center - West Virginia University Hospital
🇺🇸Morgantown, West Virginia, United States
Sutter Institute for Medical Research
🇺🇸Sacramento, California, United States
Florida Hospital
🇺🇸Orlando, Florida, United States
Wayne State University/Children's Hospital of Michigan
🇺🇸Detroit, Michigan, United States
Wake Forest Baptist Health
🇺🇸Winston-Salem, North Carolina, United States
The University of Oklahoma Health Science Center
🇺🇸Oklahoma City, Oklahoma, United States
H. Lee Moffitt Cancer Center & Research Institute
🇺🇸Tampa, Florida, United States
Henry Ford Hospital
🇺🇸Detroit, Michigan, United States
Phoenix Children's Hospital
🇺🇸Phoenix, Arizona, United States
Mayo Clinic Cancer Center
🇺🇸Phoenix, Arizona, United States
University of California Davis Health System
🇺🇸Sacramento, California, United States
University of California San Francisco Medical Center
🇺🇸San Francisco, California, United States
US Oncology - Denver
🇺🇸Denver, Colorado, United States
Yale - New Haven Hospital
🇺🇸New Haven, Connecticut, United States
National Institutes of Health
🇺🇸Bethesda, Maryland, United States
University of Nebraska Medical Center
🇺🇸Omaha, Nebraska, United States
Children's Hospital of Alabama
🇺🇸Birmingham, Alabama, United States
Children's Hospital Colorado
🇺🇸Aurora, Colorado, United States
Pediatric Blood and Cancer Disorders Clinic
🇺🇸Louisville, Kentucky, United States
University of Louisville Research Foundation
🇺🇸Louisville, Kentucky, United States
University of Michigan Health System
🇺🇸Ann Arbor, Michigan, United States
Mayo Clinic
🇺🇸Rochester, Minnesota, United States
Doernbecher Children's Hospital - Oregon Health and Science University
🇺🇸Portland, Oregon, United States
The Children's Hospital at TriStar Centennial
🇺🇸Nashville, Tennessee, United States
Oregon Health and Science University
🇺🇸Portland, Oregon, United States
Medical College of Wisconsin/ Children's Hospitalof Wisconsin
🇺🇸Milwaukee, Wisconsin, United States
Vanderbilt Ingram Cancer Center
🇺🇸Nashville, Tennessee, United States
University of Utah
🇺🇸Salt Lake City, Utah, United States
Kapi'olani Medical Center for Women and Children
🇺🇸Honolulu, Hawaii, United States
Tulane University Hospital & Clinic (Tuhc) - Tulane Cancer Center Comprehensive Clinic (Tcccc)
🇺🇸New Orleans, Louisiana, United States
Children's Hospital
🇺🇸New Orleans, Louisiana, United States
Medical University of South Carolina
🇺🇸Charleston, South Carolina, United States
Children's Mercy Hospital and Clinics - Infectious Diseases
🇺🇸Kansas City, Missouri, United States
The University of North Carolina at Chapel Hill
🇺🇸Chapel Hill, North Carolina, United States
Virginia Commonwealth University
🇺🇸Richmond, Virginia, United States
Montefiore Medical Center
🇺🇸The Bronx, New York, United States