Defibrotide for the Treatment of Severe Hepatic Veno-Occlusive Disease in Hematopoetic Stem Cell … (NCT00358501) | Clinical Trial Compass
CompletedPhase 3
Defibrotide for the Treatment of Severe Hepatic Veno-Occlusive Disease in Hematopoetic Stem Cell Transplant Patients
United States, Canada134 participantsStarted 2006-07
Plain-language summary
The purpose of this study is to (1) demonstrate the efficacy and safety (toxicity) of 25 mg/kg/day of Defibrotide in patients with severe veno-occlusive disease (sVOD) and (2) evaluate serum and endothelial markers of veno-occlusive disease (VOD) through the analysis of blood samples.
Who can participate
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria:
* Clinical diagnosis of VOD, defined by jaundice (bilirubin \>/= 2 mg/dL) and at least 2 of the following clinical findings, by Day+21 post stem cell transplant: ascites; weight gain \>/= 5% above baseline weight; hepatomegaly.
* Severe VOD, defined as VOD with multi-organ failure, i.e., presence of one or both of the following, by Day+28 post stem cell transplant: renal or pulmonary dysfunction.
* Provide voluntary written informed consent.
Exclusion Criteria:
* Pre-existing (prior to SCT) cirrhosis
* An alternative diagnosis for weight gain, ascites and jaundice
* Graft-versus-host disease (GVHD) grade B or higher involving liver or gut or grade C or higher involving skin
* Prior solid organ transplant
* Dependent on dialysis prior to and/or at the time of SCT
* Dependent on oxygen supplementation prior to SCT
* Significant acute bleeding or hemodynamic instability
* Requirement for the use of any medications that increase risk of hemorrhage will be excluded from the treatment group
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Survival at Day+100 Following Hematopoietic Stem Cell Transplant
Timeframe: Day+100 post hematopoietic stem cell transplant
2
Complete Response by Day+100 Post Hematopoietic Stem Cell Transplant
Timeframe: Day+100 post hematopoietic stem cell transplant