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Defibrotide for Hematopoietic Stem Cell Transplant Patients With Severe Hepatic Venocclusive Disease: A Phase I/II Study to Determine the Minimal Effective Dose


Phase 2
N/A
N/A
Open (Enrolling)
Both
Veno-Occlusive Disease

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Trial Information

Defibrotide for Hematopoietic Stem Cell Transplant Patients With Severe Hepatic Venocclusive Disease: A Phase I/II Study to Determine the Minimal Effective Dose


OBJECTIVES:

- Determine complete response rate in post-hematopoietic stem cell transplant patients
with severe veno-occlusive disease of the liver treated with defibrotide.

- Determine the minimal effective dose of this drug in these patients.

- Assess toxicity and adverse side effects of this drug in these patients.

OUTLINE: This is a randomized, multicenter study. All patients initially receive the same
dose of defibrotide IV over 2 hours every 6 hours on day 1. On day 2, patients are
randomized to 1 of 2 doses of defibrotide.

- Arm I: On days 2-14, patients receive a lower dose of defibrotide IV over 2 hours every
6 hours.

- Arm II: On days 2-14, patients receive a higher dose of defibrotide IV over 2 hours
every 6 hours.

In both arms, courses repeat every 14 days in the absence of disease progression or
unacceptable toxicity.

PROJECTED ACCRUAL: A total of 140 patients (70 per treatment arm) will be accrued for this
study.

Inclusion Criteria


DISEASE CHARACTERISTICS:

- Diagnosis of veno-occlusive disease (VOD) of the liver after hematopoietic stem cell
transplant as evidenced by 1 of the following:

- Jaundice (bilirubin at least 2 mg/dL) and at least 2 of the following: ascites,
weight gain over 5%, hepatomegaly, or right upper quadrant pain

- Jaundice and reversal of flow on doppler examination of portal vein with at
least 1 of the above mentioned criteria

- Diagnosed no more than 35 days prior to study entry

- Severity of VOD defined by Bearman model of 30% or more risk of severe disease and/or
evidence of multiorgan failure

- No concurrent grade B-D graft-versus-host disease (based on the International Bone
Marrow Transplant Registry Severity Index)

PATIENT CHARACTERISTICS:

Age:

- Not specified

Performance status:

- Not specified

Life expectancy:

- Not specified

Hematopoietic:

- Not specified

Hepatic:

- See Disease Characteristics

Renal:

- Not specified

Cardiovascular:

- Must be hemodynamically stable

PRIOR CONCURRENT THERAPY:

Biologic therapy:

- See Disease Characteristics

Chemotherapy:

- Not specified

Endocrine therapy:

- Not specified

Radiotherapy:

- Not specified

Surgery:

- Not specified

Other:

- No prior tissue plasminogen activator treatment

- No other concurrent experimental agent

Type of Study:

Interventional

Study Design:

Allocation: Randomized, Primary Purpose: Supportive Care

Outcome Measure:

Complete Response Rate as measured by a total bilirubin of < 2 mg/dL and resolution of multi-organ failure attributable to veno-occlusive disease (VOD)

Principal Investigator

Paul G.G. Richardson, MD

Investigator Role:

Study Chair

Investigator Affiliation:

Dana-Farber Cancer Institute

Authority:

United States: Federal Government

Study ID:

CDR0000067166

NCT ID:

NCT00003966

Start Date:

March 1999

Completion Date:

Related Keywords:

  • Veno-occlusive Disease
  • veno-occlusive disease

Name

Location

Fred Hutchinson Cancer Research CenterSeattle, Washington  98109
Memorial Sloan-Kettering Cancer CenterNew York, New York  10021
Duke Comprehensive Cancer CenterDurham, North Carolina  27710
Sidney Kimmel Comprehensive Cancer Center at Johns HopkinsBaltimore, Maryland  21231-2410
Dana-Farber/Harvard Cancer Center at Dana Farber Cancer InstituteBoston, Massachusetts  02115
City of Hope Comprehensive Cancer CenterDuarte, California  91010