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

Expanded access is no longer available for this treatment.
Sponsor:
Information provided by (Responsible Party):
St. Jude Children's Research Hospital
ClinicalTrials.gov Identifier:
NCT00143546
First received: September 1, 2005
Last updated: November 23, 2011
Last verified: June 2011
  Purpose

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 Intervention Phase
Hepatic Veno-occlusive Disease
Drug: Defibrotide
Phase 2

Study Type: Expanded Access     What is Expanded Access?
Official Title: Compassionate Use of Defibrotide for Patients With Veno-Occlusive Disease

Resource links provided by NLM:


Further study details as provided by St. Jude Children's Research Hospital:

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:
    • Defibrotide
    • Veno-occlusive disease
    • VOD
    • Hepatic veno-occlusive disease.
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.
  Eligibility

Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00143546

Locations
United States, Tennessee
St. Jude Children's Research Hospital
Memphis, Tennessee, United States, 38105
Sponsors and Collaborators
St. Jude Children's Research Hospital
Investigators
Principal Investigator: Wing Leung, M.D. St. Jude Children's Research Hospital
  More Information

Additional Information:
No publications provided

Responsible Party: St. Jude Children's Research Hospital
ClinicalTrials.gov Identifier: NCT00143546     History of Changes
Other Study ID Numbers: DETIDE
Study First Received: September 1, 2005
Last Updated: November 23, 2011
Health Authority: United States: Food and Drug Administration

Keywords provided by St. Jude Children's Research Hospital:
Hepatic Veno-occlusive Disease

Additional relevant MeSH terms:
Budd-Chiari Syndrome
Hepatic Veno-Occlusive Disease
Cardiovascular Diseases
Digestive System Diseases
Embolism and Thrombosis
Liver Diseases
Thrombosis
Vascular Diseases
Venous Thrombosis
Defibrotide
Cardiovascular Agents
Fibrin Modulating Agents
Fibrinolytic Agents
Hematologic Agents
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions
Platelet Aggregation Inhibitors
Therapeutic Uses

ClinicalTrials.gov processed this record on October 21, 2014