Defibrotide for Patients with Hepatic Veno-Occlusive Disease (VOD)

Trial Purpose and Description

Trial Purpose

Hepatic Veno-Occlusive Disease (VOD) can be a complication of bone marrow and/or stem cell transplant or high-dose chemotherapy, which may be life threatening. This condition is characterized by damage to the blood vessels in the liver and surrounding liver cells, leading to pain, fluid retention and abnormal liver function. This research study is evaluating an investigational drug called Defibrotide as a possible treatment for hepatic VOD.

Participation Guidelines

Eligibility Criteria

Inclusion Criteria:

Entry criteria include both of the following:

1.1 Clinical diagnosis of VOD, defined by jaundice (bilirubin >2 mg/dL) and at least 2 of the following clinical findings:

·         Ascites

·         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.

Patients that do not meet the criteria in Section 1.1 (i.e. have two of the major criteria but not three) and have biopsy proven VOD are eligible.

1.2 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.

Sponsor:

Gentium S.P.A.

Dates:

February 2012

Last Updated:

Study HIC#:

1202009694

Clinicaltrials.gov ID: Yale5273815