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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


Phase 3
N/A
N/A
Not Enrolling
Both
Severe Hepatic Veno Occlusive Disease

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Trial Information

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


This is a historically-controlled, multicenter, open label Phase 3 study to determine the
safety and efficacy of 25 mg/kg/day of Defibrotide (DF) for the treatment of severe VOD in
hematopoietic SCT patients.

In this study, the term "severe VOD" is defined as those patients who meet the Baltimore
diagnostic criteria for VOD (total bilirubin >/= 2.0 mg/dL plus two of the following:
ascites, >/=5% weight gain and hepatomegaly), who also have MOF (i.e., pulmonary and/or
renal dysfunction). This represents a group of patients in whom mortality at D+100 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 eligiblity 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
SCT, and special studies of endothelial and serum markers for VOD. This study will assess
safety of the dose and schedule in this setting.


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

- 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

Type of Study:

Interventional

Study Design:

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Complete Response at D+100 post stem cell transplant

Outcome Time Frame:

Day + 100 post stem cell transplant

Safety Issue:

No

Principal Investigator

Paul Richardson, M.D.

Investigator Role:

Principal Investigator

Investigator Affiliation:

Dana-Farber Cancer Institute

Authority:

United States: Food and Drug Administration

Study ID:

2005-01

NCT ID:

NCT00358501

Start Date:

July 2006

Completion Date:

November 2008

Related Keywords:

  • Severe Hepatic Veno Occlusive Disease
  • Defibrotide
  • Severe veno occlusive disease
  • Multi organ failure
  • Stem cell transplant
  • Liver
  • Regimen related toxicity
  • Day 100 survival
  • Hepatic Veno-Occlusive Disease
  • Budd-Chiari Syndrome

Name

Location
Hinsdale, Illinois  60521
Alexandria, Minnesota  56308
Albany, Georgia  31701
Fountain Valley, California  92708
Columbia, Missouri  65203
Albany, New York  12208
Cleveland, Ohio  44195
Philadelphia, Pennsylvania  19104
Austin, Texas  78705
Seattle, Washington  98195
Flint, Michigan  48532
Omaha, Nebraska  68114
Hackensack, New Jersey  07601
Denver, Colorado  
Baltimore, Maryland  21287
Boston, Massachusetts  
Charlotte, North Carolina  
Eugene, Oregon  
Indianapolis, Indiana