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

Sponsored by:	Emory University
Information provided by:	Emory University
ClinicalTrials.gov Identifier:	NCT00263978

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

MedlinePlus related topics: Liver Diseases

Drug Information available for: Defibrotide

U.S. FDA Resources

Study Type:	Interventional
Study Design:	Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study
Official Title:	Defibrotide Compassionate Use Protocol for Patients With Life Threatening Veno-Occlusive Disease of the Liver

Further study details as provided by Emory University:

Primary Outcome Measures:

treatment of veno-occlusive disease

Secondary Outcome Measures:

safety of patients

Estimated Enrollment:	20
Study Start Date:	November 2005
Estimated Study Completion Date:	October 2010

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

Contacts

Contact: Jo Ann Sadowski, BS	404-785-3531	joann.sadowski@choa.org
Contact: Pamela Rowland, CRN	404-785-1645	pamela.rowland@choa.org

Locations

United States, Georgia
Children's Healthcare of Atlanta	Recruiting
Atlanta, Georgia, United States, 30322
Principal Investigator: John Horan, MD

Sponsors and Collaborators

Emory University

Investigators

Principal Investigator:

John Horan, MD

Children's Healthcare of Atlanta

More Information

Study ID Numbers:	966-2005
Study First Received:	December 9, 2005
Last Updated:	February 21, 2007
ClinicalTrials.gov Identifier:	NCT00263978
Health Authority:	United States: Institutional Review Board

Keywords provided by Emory University:

Venous occlusive disease
VOD

Study placed in the following topic categories:

Hepatic venoocclusive disease
Liver Diseases
Digestive System Diseases

Vascular Diseases
Defibrotide
Hepatic Veno-Occlusive Disease

Additional relevant MeSH terms:

Fibrin Modulating Agents
Molecular Mechanisms of Pharmacological Action
Therapeutic Uses
Hematologic Agents
Platelet Aggregation Inhibitors

Fibrinolytic Agents
Cardiovascular Diseases
Cardiovascular Agents
Pharmacologic Actions

ClinicalTrials.gov processed this record on January 16, 2009