Defibrotide for Patients With Hepatic Veno-occlusive Disease: A Treatment IND Study
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.
|Study Design:||Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||Defibrotide for Patients With Hepatic Veno-occlusive Disease: A Treatment IND Study (Under CFR 312.34)|
- Complete response of VOD [ Time Frame: D+100 from SCT or 100 days from start of chemotherapy ] [ Designated as safety issue: No ]
- Survival [ Time Frame: D+100 post SCT or 100 days from start of chemotherapy ] [ Designated as safety issue: No ]
- Number of Participants with Adverse Events [ Time Frame: From date study critera were met through 30 days from last Defibrotide dose ] [ Designated as safety issue: Yes ]
|Study Start Date:||December 2007|
|Estimated Primary Completion Date:||December 2013 (Final data collection date for primary outcome measure)|
Defibrotide 25 mg/kg day given in 4 divided doses approximately every 6 hours
Defibrotide is made up of a complex mixture of single stranded oligodeoxyribonucleotides having variable base sequences, chain lengths and molecular weights which form unfolded, folded or combined conformations. 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.
|Contact: Fahmina Rahman, MPH||1-312-706-6261||0265-002Gentium@iconplc.com|
|Contact: Alison Hannah, M.D.||firstname.lastname@example.org|
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|Study Director:||Carin Heringa, M.D.||Gentium, S.p.A.|
|Principal Investigator:||Paul Richardson, M.D.||Dana-Farber Cancer Institute|