Defibrotide Compassionate Use for Patients With Life Threatening Veno-Occlusive Disease of the Liver
- Conditions
- Liver Diseases
- Registration Number
- NCT00263978
- Lead Sponsor
- Emory University
- Brief Summary
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.
- 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.
Recruitment & Eligibility
- Status
- NO_LONGER_AVAILABLE
- Sex
- All
- Target Recruitment
- Not specified
- Veno-occlusive disease of the liver
Not provided
Study & Design
- Study Type
- EXPANDED_ACCESS
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (1)
Children's Healthcare of Atlanta
🇺🇸Atlanta, Georgia, United States