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

Emory University

Status

Conditions

Liver Diseases

Treatments

Drug: Defibrotide

Study type

Expanded Access

Funder types

Other

Identifiers

NCT00263978

0966-2005

Details and patient eligibility

About

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.

Full 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.

Sex

All

Ages

1 to 22 years old

Volunteers

No Healthy Volunteers

Inclusion and exclusion criteria

Inclusion Criteria: