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Sponsors and Collaborators: |
Fred Hutchinson Cancer Research Center National Cancer Institute (NCI) |
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Information provided by: | National Cancer Institute (NCI) |
ClinicalTrials.gov Identifier: | NCT00410917 |
RATIONALE: Defibrotide may help heal liver damage that occurs after a stem cell transplant.
PURPOSE: This phase III trial is studying the side effects and how well defibrotide works in treating hepatic veno-occlusive disease in patients who have undergone a stem cell transplant.
Condition | Intervention | Phase |
---|---|---|
Cancer |
Drug: defibrotide Other: laboratory biomarker analysis |
Phase III |
Study Type: | Interventional |
Study Design: | Supportive Care, Open Label |
Official Title: | Defibrotide for the Treatment of Severe Hepatic Veno-Occlusive Disease in Hematopoietic Stem Cell Transplant Patients: A Historically-Controlled, Multi-Center Phase 3 Study to Determine Safety & Efficacy |
Estimated Enrollment: | 80 |
Study Start Date: | June 2006 |
Estimated Primary Completion Date: | November 2008 (Final data collection date for primary outcome measure) |
OBJECTIVES:
OUTLINE: This is an open-label, historically-controlled, multicenter study. Patients are stratified according to ventilator and/or dialysis dependence (yes vs no), age (≤ 16 years vs > 16 years), prior allogeneic bone marrow transplantation (yes vs no), and prior stem cell transplantation (yes vs no).
Patients receive defibrotide IV over 2 hours 4 times daily for a minimum of 21 days. Treatment continues, in the absence of unacceptable toxicity, until the patient is discharged from the hospital. Defibrotide may be reinitiated for patients who are rehospitalized for recurrent veno-occlusive disease (VOD).
Patients undergo collection of blood samples at baseline and periodically during study for biomarker correlative studies to evaluate potential serum and endothelial markers for VOD, including plasminogen activator inhibitor-1.
Patients are followed at 100 days and then again at 6 months after transplantation.
PROJECTED ACCRUAL: A total of 80 patients will be accrued for this study.
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
DISEASE CHARACTERISTICS:
Clinical diagnosis of hepatic veno-occlusive disease (VOD), as indicated by jaundice (i.e., bilirubin > 2 mg/dL) AND meets ≥ 2 of the following criteria by day 21 post hematopoietic stem cell transplantation (HSCT):
Hepatomegaly
Severe disease with multiorgan failure, as evidenced by any of the following by day 28 post HSCT:
Renal dysfunction, defined by any of the following:
Pulmonary dysfunction attributable to fluid overload or mechanical impingement from abdominal distention or hepatic enlargement not due to an infectious cause (e.g., documented pneumonia), as evidenced by any of the following:
PATIENT CHARACTERISTICS:
No documented diagnosis of grade B-D graft-versus-host disease (GVHD) involving the liver or gut or documented grade C or D GVHD involving skin of > 50% of body surface
No hemodynamic instability requiring multiple pressors
Able to maintain mean arterial pressure (MAP) with single-pressor support
Not oxygen-dependent during conditioning prior to HSCT
PRIOR CONCURRENT THERAPY:
Concurrent ursodiol allowed for prophylaxis if initiated at time of HSCT
United States, Washington | |
Fred Hutchinson Cancer Research Center | |
Seattle, Washington, United States, 98109-1024 |
Principal Investigator: | Gideon Steinbach, MD, PhD | Fred Hutchinson Cancer Research Center |
Responsible Party: | Gentium SpA ( Regulatory Affairs Associate ) |
Study ID Numbers: | CDR0000523392, FHCRC-2098.00, GENTIUM-2005-01 |
Study First Received: | December 11, 2006 |
Last Updated: | April 14, 2009 |
ClinicalTrials.gov Identifier: | NCT00410917 History of Changes |
Health Authority: | United States: Federal Government |
veno-occlusive disease adult acute myeloid leukemia with 11q23 (MLL) abnormalities adult acute myeloid leukemia with inv(16)(p13;q22) adult acute myeloid leukemia with t(15;17)(q22;q12) adult acute myeloid leukemia with t(16;16)(p13;q22) adult acute myeloid leukemia with t(8;21)(q22;q22) accelerated phase chronic myelogenous leukemia adult acute lymphoblastic leukemia in remission adult acute myeloid leukemia in remission atypical chronic myeloid leukemia blastic phase chronic myelogenous leukemia childhood acute lymphoblastic leukemia in remission childhood acute myeloid leukemia in remission childhood chronic myelogenous leukemia chronic eosinophilic leukemia |
chronic idiopathic myelofibrosis chronic myelomonocytic leukemia chronic neutrophilic leukemia chronic phase chronic myelogenous leukemia de novo myelodysplastic syndromes disseminated neuroblastoma extranodal marginal zone B-cell lymphoma of mucosa-associated lymphoid tissue juvenile myelomonocytic leukemia myelodysplastic/myeloproliferative disease, unclassifiable nodal marginal zone B-cell lymphoma noncontiguous stage II adult Burkitt lymphoma noncontiguous stage II adult diffuse large cell lymphoma noncontiguous stage II adult diffuse mixed cell lymphoma noncontiguous stage II adult diffuse small cleaved cell lymphoma noncontiguous stage II adult immunoblastic large cell lymphoma |
Chronic Myelomonocytic Leukemia Blast Crisis Liver Diseases Lymphoma, Mantle-Cell Mantle Cell Lymphoma Hepatic Veno-Occlusive Disease Follicular Lymphoma Mycoses Fibrin Modulating Agents Acute Myelocytic Leukemia Preleukemia Acute Myeloid Leukemia, Adult Leukemia, Lymphocytic, Chronic, B-Cell Wilms' Tumor Neoplasm Metastasis |
Hodgkin Disease Rhabdomyosarcoma Lymphoma, Large B-Cell, Diffuse Immunoproliferative Disorders Precursor Cell Lymphoblastic Leukemia-Lymphoma Testicular Cancer Leukemia, Myelomonocytic, Chronic Leukemia, Myeloid, Chronic, Atypical, BCR-ABL Negative Hairy Cell Leukemia Myeloproliferative Disorders Juvenile Myelomonocytic Leukemia Breast Neoplasms Leukemia, Myeloid Testicular Neoplasms Multiple Myeloma |
Liver Diseases Neoplasms by Histologic Type Immunoproliferative Disorders Molecular Mechanisms of Pharmacological Action Immune System Diseases Hematologic Agents Vascular Diseases Defibrotide Fibrinolytic Agents Hepatic Veno-Occlusive Disease Cardiovascular Agents Pharmacologic Actions |
Lymphatic Diseases Fibrin Modulating Agents Neoplasms Digestive System Diseases Therapeutic Uses Lymphoma, Large-Cell, Immunoblastic Platelet Aggregation Inhibitors Cardiovascular Diseases Lymphoma, Non-Hodgkin Lymphoproliferative Disorders Lymphoma |