Conditions: Hepatic Veno-Occlusive Disease
Keywords: VOD
Allocation: NA | Intervention model: SINGLE_GROUP
Enrollment: 1206
Healthy volunteers: no

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:

Arms:
arm 1: Defibrotide 25 mg/kg day given in 4 divided doses approximately every 6 hours

Interventions: intervention 1: Defibrotide intervention 2: Defibrotide
Intervention descriptions: intervention 1: 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 2: None

Protocol document: