Defibrotide Compassionate Use for Patients With Life Threatening Veno-Occlusive Disease of the Liver

This study has been terminated.
(poor enrollment)
Sponsor:
Information provided by (Responsible Party):
John Horan, Emory University
ClinicalTrials.gov Identifier:
NCT00263978
First received: December 9, 2005
Last updated: November 26, 2013
Last verified: November 2013
  Purpose

Severe veno-occlusive disease (VOD) of the liver is a life threatening complication of blood and marrow transplantation. Treatment with currently available (Food and Drug Administration [FDA] approved) agents fails in most cases. Recently conducted clinical studies indicate that patients benefit from defibrotide, a non-FDA approved agent. This protocol has been developed not with a research intent, but rather to ensure that defibrotide is used by the blood and marrow transplant programs at Children's Healthcare of Atlanta and at Emory University in a safe, effective and ethical manner.


Condition Intervention
Liver Diseases
Drug: Defibrotide

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Defibrotide Compassionate Use Protocol for Patients With Life Threatening Veno-Occlusive Disease of the Liver

Resource links provided by NLM:


Further study details as provided by Emory University:

Primary Outcome Measures:
  • treatment of veno-occlusive disease [ Time Frame: enrollment ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • safety of patients [ Time Frame: enrollment ] [ Designated as safety issue: No ]

Enrollment: 2
Study Start Date: November 2005
Study Completion Date: June 2009
Primary Completion Date: June 2009 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
single arm
compassionate use study
Drug: Defibrotide

Detailed Description:

Severe veno-occlusive disease (VOD) of the liver is a life threatening complication of blood and marrow transplantation. Treatment with currently available (FDA approved) agents fails in most cases. Recently conducted clinical studies indicate that patients benefit from defibrotide, a non-FDA approved agent. This protocol has been developed not with a research intent, but rather to ensure that defibrotide is used by the blood and marrow transplant programs at Children's Healthcare of Atlanta and at Emory University in a safe, effective and ethical manner.

  Eligibility

Ages Eligible for Study:   1 Year to 22 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Veno-occlusive disease of the liver
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00263978

Locations
United States, Georgia
Children's Healthcare of Atlanta
Atlanta, Georgia, United States, 30322
Sponsors and Collaborators
Emory University
Investigators
Principal Investigator: John Horan, MD Children's Healthcare of Atlanta
  More Information

No publications provided

Responsible Party: John Horan, Principal Investigator, Emory University
ClinicalTrials.gov Identifier: NCT00263978     History of Changes
Other Study ID Numbers: 0966-2005
Study First Received: December 9, 2005
Last Updated: November 26, 2013
Health Authority: United States: Institutional Review Board

Keywords provided by Emory University:
Venous occlusive disease
VOD

Additional relevant MeSH terms:
Hepatic Veno-Occlusive Disease
Liver Diseases
Digestive System Diseases
Vascular Diseases
Cardiovascular Diseases
Defibrotide
Fibrinolytic Agents
Fibrin Modulating Agents
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions
Cardiovascular Agents
Therapeutic Uses
Hematologic Agents
Platelet Aggregation Inhibitors

ClinicalTrials.gov processed this record on April 17, 2014