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

Primary Purpose

Hepatic Veno-Occlusive Disease

Status
Completed
Phase
Phase 3
Locations
United States
Study Type
Interventional
Intervention
Defibrotide
Defibrotide
Sponsored by
Jazz Pharmaceuticals
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Hepatic Veno-Occlusive Disease focused on measuring VOD

Eligibility Criteria

undefined - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

  • Children's Hospital of Alabama
  • Phoenix Children's Hospital
  • Mayo Clinic Cancer Center
  • Arizona Oncology
  • University Medical Center
  • University of Arkansas for Medical Services
  • City of Hope Medical Center
  • The University of California, San Diego
  • Loma Linda University
  • Long Beach Memorial Medical Center
  • Children's Hospital of Los Angeles
  • Children's Hospital & Research Center Oakland
  • Children's Hospital of Orange County (CHOC)
  • Sutter Institute for Medical Research
  • University of California Davis Health System
  • Rady's Children's Hospital - San Diego
  • University of California San Francisco Medical Center
  • Stanford University Medical Center
  • Children's Hospital Colorado
  • US Oncology - Denver
  • Yale - New Haven Hospital
  • Nemours-Al duPont Hospital for Children
  • Georgetown University Hospital
  • Children's National Medical Center
  • UF Health Shands Hospital
  • Jackson Memorial- University of Miami Health System- Pediatrics
  • Nicklaus Children's Hospital (Miami Children's Hospital)
  • Florida Hospital
  • All Children's Hospital Specialty Physicians
  • H. Lee Moffitt Cancer Center & Research Institute
  • Childrens Healthcare of Atlanta
  • Emory University Hospital
  • Northside Hospital
  • Kapi'olani Medical Center for Women and Children
  • Ann & Robert H. Lurie Children's Hospital of Chicago
  • Northwest Memorial Hospital
  • Rush University Medical Center
  • Pediatric Hematology/Oncology MC4060 - University of Chicago
  • Cardinal Bernardin Cancer-Loyola University Medical Center
  • Riley Hospital for Children
  • Indiana Blood and Marrow Transplantation Clinical Lab LLC/St. Francis Hospital
  • University of Iowa Hospitals & Clinics
  • University of Kansas Cancer Center
  • Pediatric Blood and Cancer Disorders Clinic
  • University of Louisville Research Foundation
  • Tulane University Hospital & Clinic (Tuhc) - Tulane Cancer Center Comprehensive Clinic (Tcccc)
  • Children's Hospital
  • University of Maryland - Greenebaum Cancer Center
  • Johns Hopkins University
  • National Institutes of Health
  • Tufts Medical Center
  • Massachusetts General Hospital
  • Boston Children's Hospital DFCI/CHB-Pediatrics
  • Dana-Farber Cancer Institute
  • Beth Israel Deaconess Medical Center
  • University of Michigan Health System
  • Wayne State University/Children's Hospital of Michigan
  • Henry Ford Hospital
  • Helen DeVos Children's Hospital - Spectrum Health
  • Children's Hospitals and Clinics of Minnesota and Minneapolis
  • University of Minnesota Medical Center
  • Mayo Clinic
  • University of Mississippi Medical Center
  • Children's Mercy Hospital and Clinics - Infectious Diseases
  • Cardinal Glennon Children's Medical Center/St. Louis University Department of Pediatrics
  • Washington University/St. Louis Children's Hospital
  • University of Nebraska Medical Center
  • Hackensack University Medical Center - Joseph M. Sanzari Childrens Hospital
  • Rutgers Cancer Institute of New Jersey
  • Presbyterian Hospital
  • Roswell Park Cancer Institute
  • Steven and Alexandra Cohen Children's Medical Center
  • New York University School of Medicine
  • Memorial Sloan Kettering Cancer Center
  • Mount Sinai Hospital - BMT Program
  • Mount Sinai Medical Center
  • Morgan Stanley Children's Hospital - Columbia University Medical Center
  • Weill Cornell Medical College - New York Presbyterian Hospital
  • University of Rochester Medical Center, Strong Memorial Hospital
  • Stony Brook University Hospital
  • Montefiore Medical Center
  • New York Medical College
  • The University of North Carolina at Chapel Hill
  • Levine Children's Hospital
  • Duke University Medical Center
  • Wake Forest Baptist Health
  • Children's Hospital Medical
  • Cincinnati Children's Hospital Medical Center
  • The Jewish Hospital
  • Case Western Reserve University (Cwru) - University Hospitals of Cleveland (Uhc)
  • Robert J Tomsich Pathology & Laboratory Medicine Institute
  • Nationwide Children's Hospital - Ohio State University College of Medicine (OSUCOM)
  • The Ohio State University, James Cancer Hospital
  • The University of Oklahoma Health Science Center
  • Doernbecher Children's Hospital - Oregon Health and Science University
  • Oregon Health and Science University
  • Geisinger Medical Center
  • Milton S. Hershey Medical Center
  • The Children's Hospital of Philadelphia
  • Children's Hospital of Pittsburgh of UPMC
  • West Penn Allegheny Health System
  • Medical University of South Carolina
  • Greenville Health System Cancer Institute
  • Avera Cancer Institute
  • Methodist University Hospital
  • St. Jude Children's Research Hospital - Department of Bone Marrow Transplantation and Cellular Therapy
  • The Children's Hospital at TriStar Centennial
  • Vanderbilt Ingram Cancer Center
  • Children's Medical Center of Dallas
  • San Antonio Military Medical Center
  • Cook Children's Health Care Hospital
  • Baylor College of Medicine
  • Texas Children's Hospital
  • The University of Texas - MD Anderson Cancer Center
  • University of Texas MD Anderson Cancer Center
  • Methodist Healthcare System of San Antonio
  • University of Utah
  • University of Virginia Health System
  • Children's Hospital of the King's Daughter
  • Virginia Commonwealth University
  • Fred Hutchinson Cancer Research Center/Seattle Children Hospital
  • Providence Sacred Heart Medical Center and Children's Hospital
  • Mary Babb Randolph Cancer Center - West Virginia University Hospital
  • University of Wisconsin, American Family Children's Hospital, Medical School
  • Medical College of Wisconsin/ Children's Hospitalof Wisconsin

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Defibrotide

Arm Description

Defibrotide 25 mg/kg day given in 4 divided doses approximately every 6 hours

Outcomes

Primary Outcome Measures

Survival by Day+100 Post Stem Cell Transplant or Chemotherapy

Secondary Outcome Measures

Full Information

First Posted
February 25, 2008
Last Updated
November 2, 2017
Sponsor
Jazz Pharmaceuticals
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1. Study Identification

Unique Protocol Identification Number
NCT00628498
Brief Title
Defibrotide for Patients With Hepatic Veno-occlusive Disease: A Treatment IND Study
Acronym
Treatment IND
Official Title
Defibrotide for Patients With Hepatic Veno-occlusive Disease: A Treatment IND Study
Study Type
Interventional

2. Study Status

Record Verification Date
November 2017
Overall Recruitment Status
Completed
Study Start Date
December 2007 (undefined)
Primary Completion Date
July 2016 (Actual)
Study Completion Date
September 2016 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Jazz Pharmaceuticals

4. Oversight

Data Monitoring Committee
No

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Hepatic Veno-Occlusive Disease
Keywords
VOD

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
1206 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Defibrotide
Arm Type
Experimental
Arm Description
Defibrotide 25 mg/kg day given in 4 divided doses approximately every 6 hours
Intervention Type
Drug
Intervention Name(s)
Defibrotide
Intervention Description
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.
Intervention Type
Drug
Intervention Name(s)
Defibrotide
Primary Outcome Measure Information:
Title
Survival by Day+100 Post Stem Cell Transplant or Chemotherapy
Time Frame
Day +100 from HSCT or 100 days from start of chemotherapy

10. Eligibility

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

12. IPD Sharing Statement

Citations:
PubMed Identifier
12393437
Citation
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.
Results Reference
result
PubMed Identifier
32950693
Citation
Richardson PG, Smith AR, Kernan NA, Lehmann L, Ryan RJ, Grupp SA. Analysis of Time to Complete Response after Defibrotide Initiation in Patients with Hepatic Veno-Occlusive Disease/Sinusoidal Obstruction Syndrome after Hematopoietic Cell Transplantation. Transplant Cell Ther. 2021 Jan;27(1):88.e1-88.e6. doi: 10.1016/j.bbmt.2020.09.008. Epub 2020 Sep 17.
Results Reference
derived
PubMed Identifier
29767845
Citation
Kernan NA, Grupp S, Smith AR, Arai S, Triplett B, Antin JH, Lehmann L, Shore T, Ho VT, Bunin N, Iacobelli M, Liang W, Hume R, Tappe W, Soiffer R, Richardson P. Final results from a defibrotide treatment-IND study for patients with hepatic veno-occlusive disease/sinusoidal obstruction syndrome. Br J Haematol. 2018 Jun;181(6):816-827. doi: 10.1111/bjh.15267. Epub 2018 May 16.
Results Reference
derived

Learn more about this trial

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

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