Compassionate Use of Defibrotide for Patients With Veno-occlusive Disease of the Liver
1 other identifier
expanded_access
N/A
1 country
1
Brief Summary
Veno-occlusive disease (VOD) of the liver is a significant complication for some patients undergoing hematopoietic stem cell transplantation. This disease is thought to be a toxicity secondary to chemotherapy or radiation-induced damage to the epithelial cells of the blood vessels in the liver. VOD is categorized as mild, moderate or severe. Historically, there has been no method to treat the disease. Recently, however, there have been investigations into the use of a new agent called defibrotide. The primary purpose of this protocol is to provide defibrotide to patients with severe VOD. Because this drug has not been approved by the FDA, use of this medication under the auspices of this IND treatment plan is for compassionate use only.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
1 active site
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
Study Start
First participant enrolled
November 1, 2003
CompletedFirst Submitted
Initial submission to the registry
September 1, 2005
CompletedFirst Posted
Study publicly available on registry
September 2, 2005
CompletedApril 25, 2017
April 1, 2017
September 1, 2005
April 24, 2017
Conditions
Keywords
Interventions
Defibrotide to be administered approximately every 6 hours at an initial dose of 6.25 mg/kg/dose. Subsequent doses will be escalated with a plateau of 15 mg/kg/dose (or 60 mg/kg/day total). For patients with persistent VOD, the dose may be escalated by 10 mg/kg/day as needed to a maximum of 110 mg/kg/day total.
Eligibility Criteria
You may qualify if:
- Patients with clinical or pathologic diagnosis of veno-occlusive disease. The following criteria will be used:
- Hyperbilirubinemia (greater than or equal to 2mg/dl) and two of the following: (hepatomegaly, ascites, or weight gain \[greater than or equal to 5% of baseline\]); OR,
- Patient who do not fulfill criteria in #1 but who have pathologic or radiographic evidence of VOD.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (1)
St. Jude Children's Research Hospital
Memphis, Tennessee, 38105, United States
Related Links
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
Wing Leung, M.D.
St. Jude Children's Research Hospital
Study Design
- Study Type
- expanded access
- Sponsor Type
- OTHER
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
September 1, 2005
First Posted
September 2, 2005
Study Start
November 1, 2003
Last Updated
April 25, 2017
Record last verified: 2017-04