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Compassionate Use of Defibrotide for Patients With Veno-occlusive Disease of the Liver

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00143546
Expanded Access Status : No longer available
First Posted : September 2, 2005
Last Update Posted : April 25, 2017
Information provided by (Responsible Party):
St. Jude Children's Research Hospital

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.

Condition or disease Intervention/treatment
Hepatic Veno-occlusive Disease Drug: Defibrotide

Detailed Description:

Secondary objectives of this protocol include the following:

  • To describe the toxicities of defibrotide in patients with VOD.
  • To describe the response rate of VOD in patients receiving defibrotide.

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Study Type : Expanded Access
Official Title: Compassionate Use of Defibrotide for Patients With Veno-Occlusive Disease
Study Start Date : November 2003

Intervention Details:
  • Drug: Defibrotide
    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.
    Other Names:
    • Veno-occlusive disease
    • VOD
    • Hepatic veno-occlusive disease.

Information from the National Library of Medicine

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

Patients with clinical or pathologic diagnosis of veno-occlusive disease. The following criteria will be used:

  1. 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,
  2. Patient who do not fulfill criteria in #1 but who have pathologic or radiographic evidence of VOD.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00143546

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United States, Tennessee
St. Jude Children's Research Hospital
Memphis, Tennessee, United States, 38105
Sponsors and Collaborators
St. Jude Children's Research Hospital
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Principal Investigator: Wing Leung, M.D. St. Jude Children's Research Hospital
Additional Information:
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Responsible Party: St. Jude Children's Research Hospital Identifier: NCT00143546    
Other Study ID Numbers: DETIDE
First Posted: September 2, 2005    Key Record Dates
Last Update Posted: April 25, 2017
Last Verified: April 2017
Keywords provided by St. Jude Children's Research Hospital:
Hepatic Veno-occlusive Disease
Additional relevant MeSH terms:
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Hepatic Veno-Occlusive Disease
Liver Diseases
Digestive System Diseases
Vascular Diseases
Cardiovascular Diseases
Fibrinolytic Agents
Fibrin Modulating Agents
Molecular Mechanisms of Pharmacological Action
Platelet Aggregation Inhibitors