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

St. Jude Children's Research Hospital

Status

Conditions: Hepatic Veno-occlusive Disease

Treatments: Drug: Defibrotide

Study type: Expanded Access

Funder types: Other

Identifiers

NCT00143546

DETIDE

Details and patient eligibility

About

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.

Full 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.

Sex: All

Volunteers: No Healthy Volunteers

Inclusion and exclusion 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.