Defibrotide for Patients with Hepatic Veno-Occlusive Disease (VOD): A Treatment IND Study (UNDER CFR 312.34)
Trial Purpose and Description
This is a multicenter, single arm, open-label study to provide Defibrotide to patients diagnosed with VOD. The primary objective of this Treatment IND is to provide Defibrotide under 21 CFR 312.34 (Treatment use of an investigational new drug). The Treatment IND will collect additional usage, tolerability and safety data from patients with VOD. Efficacy analysis will be done for patients who meet eligibility criteria for the previous Phase 3 study Protocol 2005-01 Defibrotide for the Treatment of Severe Hepatic Veno-Occlusive Disease in Hematopoietic Stem Cell Transplant Patients: A Historically Controlled, Multi-Center Phase 3 Study to Determine Safety and Efficacy”. Additional safety data collected from all patients who receive a dose of Defibrotide will be used to support the New Drug Application and the labeling for Defibrotide.
- 18 Years and older
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:
· 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.
· 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.
- Gentium S.P.A.
- February 2012
- Last Updated:
- Study HIC#:
Clinicaltrials.gov ID: Yale5273815