Ignite Creation Date:
2024-05-05 @ 4:58 PM
Last Modification Date:
2024-10-26 @ 9:26 AM
Study NCT ID:
NCT00358501
Status:
COMPLETED
Last Update Posted:
2017-01-30
First Post:
2006-07-28
Brief Title:
Defibrotide for the Treatment of Severe Hepatic Veno-Occlusive Disease in Hematopoetic Stem Cell Transplant Patients
Sponsor:
Jazz Pharmaceuticals
Organization:
Jazz Pharmaceuticals
Study Overview
Official Title:
Defibrotide for the Treatment of Severe Hepatic Veno-Occlusive Disease in Hematopoetic Stem Cell Transplant Patients A Historically-Controlled Multi-Center Phase 3 Study to Determine Safety Efficacy
Status:
COMPLETED
Status Verified Date:
2016-12
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
The purpose of this study is to 1 demonstrate the efficacy and safety toxicity of 25 mgkgday 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
Detailed Description:
This is a historically-controlled multicenter open label Phase 3 study to determine the safety and efficacy of 25 mgkgday of Defibrotide DF for the treatment of severe VOD in hematopoietic stem cell transplantation HSCT patients
In this study the term severe VOD is defined as those patients who meet the Baltimore diagnostic criteria for VOD total bilirubin 20 mgdL plus two of the following ascites 5 weight gain and hepatomegaly who also have multi-organ failure ie pulmonary andor renal dysfunction This represents a group of patients in whom mortality at Day100 has been estimated to be 80
Comparisons The primary parameter is Complete Response at 100 days following stem cell transplant utilizing historical controls as a comparator The historical control database will be generated through a retrospective medical chart review performed at participating centers the survival outcome of patients who would otherwise have met eligibility criteria for this trial will be compared to the survival observed in patients prospectively treated with Defibrotide Secondary parameters include survival rate at 100 days and 6 months post stem cell transplantation SCT and special studies of endothelial and serum markers for VOD This study will assess safety of the dose and schedule in this setting
In this study the term severe VOD is defined as those patients who meet the Baltimore diagnostic criteria for VOD total bilirubin 20 mgdL plus two of the following ascites 5 weight gain and hepatomegaly who also have multi-organ failure ie pulmonary andor renal dysfunction This represents a group of patients in whom mortality at Day100 has been estimated to be 80
Comparisons The primary parameter is Complete Response at 100 days following stem cell transplant utilizing historical controls as a comparator The historical control database will be generated through a retrospective medical chart review performed at participating centers the survival outcome of patients who would otherwise have met eligibility criteria for this trial will be compared to the survival observed in patients prospectively treated with Defibrotide Secondary parameters include survival rate at 100 days and 6 months post stem cell transplantation SCT and special studies of endothelial and serum markers for VOD This study will assess safety of the dose and schedule in this setting
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None