Diseases and Conditions Researched
Hepatic Veno-Occlusive Disease
What is the purpose of this trial?
Single arm, open-label study to provide Defibrotide to patients diagnosed with VOD. Defibrotide is no longer available though the Emergency Use IND mechanism (also known as compassionate use, or single patient named use). This protocol is the only mechanism by which Defibrotide can be made available to patients in the U.S.
Participation Guidelines
Age: Any
Gender: Both
Click here for
detailed participation information for this trial.
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Sponsor:
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Gentium SpA |
| Dates: |
February 25, 2008 |
| Last Updated: |
October 5, 2012 |
| Study HIC#: |
0908005547 |
| Clinicaltrials.gov ID: |
NCT00628498
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Condition
Hepatic Veno-Occlusive Disease
Trial Phase
Phase 3
Trial Purpose
Single arm, open-label study to provide Defibrotide to patients diagnosed with VOD. Defibrotide is no longer available though the Emergency Use IND mechanism (also known as compassionate use, or single patient named use). This protocol is the only mechanism by which Defibrotide can be made available to patients in the U.S.
Participation Guidelines
Age: Any
Gender: Both
Eligibility Criteria
Inclusion Criteria:
Entry criteria include the following:
1. Clinical diagnosis of VOD, made by Baltimore Criteria, Modified Seattle Criteria, or
biopsy proven:
1.1 Baltimore Criteria- Bilirubin =2 mg/dL and at least 2 of the following clinical
findings:
- Ascites (radiographic or physical exam)
- Weight gain of =5% compared to the day of conditioning-- if this value is not
available, the weight on the date of admission to the SCT unit may be used)
- Hepatomegaly; increased over baseline.
1.2 Modified Seattle Criteria: At least two of the following
- Bilirubin =2 mg/dL
- Ascites (radiographic or physical exam) and/or weight gain =5% above baseline
weight (defined as weight on the first day of conditioning- if this value is not
available, the weight on the date of admission to the SCT unit may be used)
- hepatomegaly increased over baseline
1.3 Patients that do not meet the Baltimore Criteria or Modified Seattle Criteria
and have biopsy proven VOD are eligible.
2. Patient must also provide written informed consent.
Exclusion Criteria:
- Use of any medication which increases the risk of hemorrhage is disallowed. Use of
heparin or other anticoagulants is disallowed within 12 hours unless being used for
routine central venous line management, fibrinolytic instillation for central venous
line occlusion, intermittent dialysis or ultrafiltration of CVVH.
- Clinically significant uncontrolled acute bleeding, defined as hemorrhage requiring >
15 cc/kg of packed red blood cells (e.g., a pediatric patient weighing 20 kg and
requiring > 300cc of packed red blood cells/24 hours, or an adult patient weighing 70
kg and requiring >3 units of packed red blood cells/24 hours) to replace blood loss,
OR bleeding from a site which in the Investigator's opinion constitutes a potential
life-threatening source (e.g. pulmonary hemorrhage or CNS bleeding), irrespective of
amount of blood loss, at any point from the date of SCT through the date of severe
VOD diagnosis.
- Hemodynamic instability as defined by a requirement for multiple pressors, or
inability to maintain mean arterial pressure (for children: to maintain mean arterial
pressure within 1 standard deviation of age-adjusted levels) with single pressor
support.
- Woman who are pregnant.
Publication
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Richardson PG, Murakami C, Jin Z, Warren D, Momtaz P, Hoppensteadt D, Elias AD, Antin JH, Soiffer R, Spitzer T, Avigan D, Bearman SI, Martin PL, Kurtzberg J, Vredenburgh J, Chen AR, Arai S, Vogelsang G, McDonald GB, Guinan EC. Multi-institutional use of defibrotide in 88 patients after stem cell transplantation with severe veno-occlusive disease and multisystem organ failure: response without significant toxicity in a high-risk population and factors predictive of outcome. Blood. 2002 Dec 15;100(13):4337-43. Epub 2002 Aug 1.
|
Sponsor:
|
Gentium SpA |
| Dates: |
February 25, 2008 |
| Last Updated: |
October 5, 2012 |
| Study HIC#: |
0908005547 |
| Clinicaltrials.gov ID: |
NCT00628498
|