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Defibrotide for Patients With Hepatic Veno-occlusive Disease: A Treatment IND Study (Under CFR 312.34)

Phase 3
Open (Enrolling)
Hepatic Veno-Occlusive Disease

Thank you

Trial Information

Defibrotide for Patients With Hepatic Veno-occlusive Disease: A Treatment IND Study (Under CFR 312.34)

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

- 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

- Woman who are pregnant.

Type of Study:


Study Design:

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Complete response of VOD

Outcome Time Frame:

D+100 from SCT or 100 days from start of chemotherapy

Safety Issue:


Principal Investigator

Carin Heringa, M.D.

Investigator Role:

Study Director

Investigator Affiliation:

Gentium, S.p.A.


United States: Food and Drug Administration

Study ID:




Start Date:

December 2007

Completion Date:

Related Keywords:

  • Hepatic Veno-Occlusive Disease
  • VOD
  • Hepatic Veno-Occlusive Disease
  • Budd-Chiari Syndrome



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