Defibrotide for Hematopoietic Stem Cell Transplant Patients With Severe Hepatic Venocclusive Disease: A Phase I/II Study to Determine the Minimal Effective Dose
- Determine complete response rate in post-hematopoietic stem cell transplant patients
with severe veno-occlusive disease of the liver treated with defibrotide.
- Determine the minimal effective dose of this drug in these patients.
- Assess toxicity and adverse side effects of this drug in these patients.
OUTLINE: This is a randomized, multicenter study. All patients initially receive the same
dose of defibrotide IV over 2 hours every 6 hours on day 1. On day 2, patients are
randomized to 1 of 2 doses of defibrotide.
- Arm I: On days 2-14, patients receive a lower dose of defibrotide IV over 2 hours every
- Arm II: On days 2-14, patients receive a higher dose of defibrotide IV over 2 hours
every 6 hours.
In both arms, courses repeat every 14 days in the absence of disease progression or
PROJECTED ACCRUAL: A total of 140 patients (70 per treatment arm) will be accrued for this
Allocation: Randomized, Primary Purpose: Supportive Care
Complete Response Rate as measured by a total bilirubin of < 2 mg/dL and resolution of multi-organ failure attributable to veno-occlusive disease (VOD)
Paul G.G. Richardson, MD
Dana-Farber Cancer Institute
United States: Federal Government
|Fred Hutchinson Cancer Research Center||Seattle, Washington 98109|
|Memorial Sloan-Kettering Cancer Center||New York, New York 10021|
|Duke Comprehensive Cancer Center||Durham, North Carolina 27710|
|Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins||Baltimore, Maryland 21231-2410|
|Dana-Farber/Harvard Cancer Center at Dana Farber Cancer Institute||Boston, Massachusetts 02115|
|City of Hope Comprehensive Cancer Center||Duarte, California 91010|