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Defibrotide For Patients With Hepatic Veno-Occlusive Disease
Supportive Care: Defibrotide For Patients With Hepatic Veno-Occlusive Disease (VOD): A Treatment IND Study (Under CFR 312.34)
||Jason Law, MD
Hepatic 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 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. This means that Defibrotide is not approved by the U.S. Food and Drug Administration (FDA) for use outside of a research study. The primary objective of this study is to provide defibrotide to patients with VOD. At present there is no established curative therapy for hepatic VOD. We want to better understand the effects (good and bad) of Defibrotide in patients with VOD.
- Diagnosis of Hepatic Veno-Occlusive Disease (VOD) according to certain criteria or liver biopsy
- No current bleeding problems
- Currently taking any medications that could increase the risk of bleeding
- Use of several drugs for hypotension to maintain stable blood pressure
Participants in this study will need to have some tests done to see if they are eligible to be in the research study. These tests are called screening tests. The research study has certain requirements that must be met. If the screening tests show that a participant can be in the research study, he/she will be able to start the study drug. If a person is able to participate, Defibrotide will be administered as an intravenous infusion in divided doses four times a day. Defibrotide will be given for at least three weeks (21 days) and will continue until the disease resolves or that person is ready to leave the hospital, as circumstances allow. During this study, participants will have physical exams, urine tests, and daily blood tests. Participants will be asked at discharge to return to the study center either 100 days after receiving their stem cell transplant or 100 days after the start of chemotherapy to have a physical examination and routine blood tests.