Phase 3
N=134
Defibrotide for the Treatment of Severe Hepatic Veno-Occlusive Disease in Hematopoetic Stem Cell Transplant Patients
Severe Hepatic Veno-Occlusive Disease
Bottom Line
View on ClinicalTrials.gov: NCT00358501 ↗Enrolled (actual)
134
Serious AEs
78.4%
Results posted
Jan 2017
Primary outcome: Primary: Survival at Day+100 Following Hematopoietic Stem Cell Transplant — 38.2; 25.0 percentage of participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Defibrotide (Drug)
- Age
- Pediatric, Adult, Older Adult
- Sex
- All
- Sponsor
- Jazz Pharmaceuticals
- Primary completion
- Sep 2008
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 | — |
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.
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). 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.