Phase 3
Completed N=134
Defibrotide for the Treatment of Severe Hepatic Veno-Occlusive Disease in Hematopoetic Stem Cell Transplant Patients
Severe Hepatic Veno-Occlusive Disease
Source: ClinicalTrials.gov NCT00358501 ↗
Enrolled (actual)
134
Serious AEs
78.4%
Results posted
Jan 2017
Primary outcomePrimary: Survival at Day+100 Following Hematopoietic Stem Cell Transplant — 38.2; 25.0 percentage of participants
◆ Published Evidence
Highly cited
327citations · ~33 / year
Phase 3 trial of defibrotide for the treatment of severe veno-occlusive disease and multi-organ failure.
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.
Linked Publications
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Phase 3 trial of defibrotide for the treatment of severe veno-occlusive disease and multi-organ failure.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Survival at Day+100 Following Hematopoietic Stem Cell Transplant |
38.2; 25.0 | — |
| PRIMARY Complete Response by Day+100 Post Hematopoietic Stem Cell Transplant |
25.5; 12.5 | — |
| SECONDARY Survival at Day+180 Post Hematopoietic Stem Cell Transplantation |
32.4; 25.0 | — |
| SECONDARY Percentage of Participants With Treatment-Emergent Adverse Events |
97; 100; 64; 69; 64; 75 | — |
Eligibility Criteria
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
Data sourced from ClinicalTrials.gov (NCT00358501) and the linked publication. Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.