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

Completed · Phase 3

Conditions studied: Hepatic Veno-Occlusive Disease

In brief

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.

Key facts

Study ID

NCT00628498

Run by

Jazz Pharmaceuticals

People needed

1206

Starts

2007-12-01

Expected to finish

2016-09-01

Last updated by the study team

2017-11-30

Who can join

Age: any. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Entry criteria include the following:
• Clinical diagnosis of VOD, made by Baltimore Criteria, Modified Seattle Criteria, or biopsy proven:
• 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.
• 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
• 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.

You may not qualify if…

• 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.

Where it is running

• Phoenix Children's Hospital — Phoenix, Arizona, United States
• Mayo Clinic Cancer Center — Phoenix, Arizona, United States
• Arizona Oncology — Scottsdale, Arizona, United States
• University Medical Center — Tucson, Arizona, United States
• University of Arkansas for Medical Services — Little Rock, Arkansas, United States
• City of Hope Medical Center — Duarte, California, United States
• The University of California, San Diego — La Jolla, California, United States
• Loma Linda University — Loma Linda, California, United States
• Long Beach Memorial Medical Center — Long Beach, California, United States
• Children's Hospital of Los Angeles — Los Angeles, California, United States
• Children's Hospital & Research Center Oakland — Oakland, California, United States
• Children's Hospital of Orange County (CHOC) — Orange, California, United States
• Sutter Institute for Medical Research — Sacramento, California, United States
• University of California Davis Health System — Sacramento, California, United States
• Rady's Children's Hospital - San Diego — San Diego, California, United States
• University of California San Francisco Medical Center — San Francisco, California, United States
• Stanford University Medical Center — Stanford, California, United States
• Children's Hospital Colorado — Aurora, Colorado, United States
• US Oncology - Denver — Denver, Colorado, United States
• Yale - New Haven Hospital — New Haven, Connecticut, United States
• Nemours-Al duPont Hospital for Children — Wilmington, Delaware, United States
• Georgetown University Hospital — Washington D.C., District of Columbia, United States
• Children's National Medical Center — Washington D.C., District of Columbia, United States
• UF Health Shands Hospital — Gainesville, Florida, United States
• Children's Hospital of Alabama — Birmingham, Alabama, United States

Full record on ClinicalTrials.gov

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