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Pediatric Trial Investigating the Incidence & Outcome of Veno-Occlusive Disease With the Prophylactic Use of Defibrotide (VOD-DF)

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ClinicalTrials.gov Identifier: NCT00272948
Recruitment Status : Completed
First Posted : January 9, 2006
Last Update Posted : June 9, 2011
Sponsor:
Collaborators:
Jazz Pharmaceuticals
Deutsche Krebshilfe e.V., Bonn (Germany)
Information provided by:
European Society for Blood and Marrow Transplantation

Study Description
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Brief Summary:
The aim of this trial is to evaluate whether the prophylactic use of Defibrotide (DF) in pediatric patients (age less than 18 years) undergoing stem cell transplantation and who are at high risk of developing hepatic Veno-occlusive Disease (VOD) will have an impact on the incidence and severity of the disease. Patients will be randomly assigned to one of two treatment arms: Those allocated to the Prophylactic Arm will receive the study drug (Defibrotide) from the day of conditioning onwards. Patients allocated to the Control Arm will receive the study drug (Defibrotide) from the day that VOD is diagnosed.

Condition or disease Intervention/treatment Phase
Hepatic Veno-Occlusive Disease Drug: Defibrotide Phase 2 Phase 3

Detailed Description:

Comparison/control intervention and duration of the intervention:

Patients will be assigned randomly to either the Defibrotide (DF) prophylaxis arm or the control arm. Those allocated to the DF prophylaxis arm (DF 25 mg/kg/d iv in 4 doses) will begin treatment at day of conditioning and stop at day +30 after Stem Cell Transplantation (SCT) or upon discharge from inpatient care. There is no dose adjustment for a patient of the study arm who developed VOD, they continue with the 25mg/kg/d iv.

Patients allocated to the control arm receive no prophylactic measures and will start DF (25 mg/kg/d iv in 4 doses) beginning at day of diagnosis of Veno-occlusive Disease (VOD) according to modified Seattle criteria. Treatment will be stopped at complete resolution of symptoms. In both arms patients who developed VOD will continue DF until:

  • complete resolution of the ascites and
  • reversion of the hepatopedal flow (if present) and
  • normalization of the total and direct bilirubin
Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 360 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Prevention
Official Title: Prospective Randomized Study of the Incidence and Outcome of Veno-Occlusive Disease (VOD) With the Prophylactic Use of Defibrotide (DF) in Pediatric Stem Cell Transplantation
Study Start Date : December 2005
Actual Primary Completion Date : January 2009
Actual Study Completion Date : July 2009

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: Prophylaxis Arm Drug: Defibrotide
Defibrotide 25 mg/kg/d

Drug: Defibrotide
Defibrotide 25 mg/kg/d iv in 4 doses beginning at day of conditioning until day +30 or until discharge from inpatient care (with a minimum treatment of 14 days) if VOD does not occur.
Active Comparator: Control Arm Drug: Defibrotide
Defibrotide 25 mg/kg/d

Drug: Defibrotide
Defibrotide 25 mg/kg/d iv therapeutically when patients fulfil modified Seattle criteria


Outcome Measures
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Primary Outcome Measures :
  1. The primary objective is to evaluate if prophylactic DF has an impact on the incidence of VOD [ Time Frame: Day + 30 post HSCT ]

Secondary Outcome Measures :
  1. Occurrence of Multi-System Organ Failure and Survival (all causes of mortality) [ Time Frame: day +100 post HSCT ]

Eligibility Criteria
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Ages Eligible for Study:   up to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Age <18 years

  • myeloablative conditioning and autologous or allogeneic stem cell transplantation with at least one of the following risk factors for VOD:

    1. Pre-existing liver disease
    2. Second myeloablative HSCT
    3. History of treatment with gemtuzumab ozogamicin (MYLOTARGÒ, GO, CMA-676, Wyeth)
    4. Allogeneic HSCT for leukemia beyond the second relapse
    5. Osteopetrosis (OP)
    6. Conditioning with busulfan and melphalan
    7. Macrophage activating syndromes (MAS, like hemophagocytic lymphohistiocytosis, Griscelli, Chediak-Higashi
    8. Adrenoleukodystrophy (ALD)

Exclusion Criteria:

  • Pregnant patients
  • Patients who are transplanted but do not fulfill any of the above mentioned criteria
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00272948


Locations
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Sponsors and Collaborators
European Society for Blood and Marrow Transplantation
Jazz Pharmaceuticals
Deutsche Krebshilfe e.V., Bonn (Germany)
Investigators
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Principal Investigator: Selim Corbacioglu, MD University of Ulm, Germany
More Information
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Additional Information:

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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Responsible Party: Liz Clark, European Group for Blood and Marrow Transplantation
ClinicalTrials.gov Identifier: NCT00272948    
Other Study ID Numbers: EudraCT Number:2004-000592-33
EBMT-PD-200601
First Posted: January 9, 2006    Key Record Dates
Last Update Posted: June 9, 2011
Last Verified: June 2011
Keywords provided by European Society for Blood and Marrow Transplantation:
Venoocclusive disease
Pediatric
myeloablative conditioning
stem cell transplant
Additional relevant MeSH terms:
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Hepatic Veno-Occlusive Disease
Liver Diseases
Digestive System Diseases
Vascular Diseases
Cardiovascular Diseases
 Defibrotide
Fibrinolytic Agents
Fibrin Modulating Agents
Molecular Mechanisms of Pharmacological Action
Platelet Aggregation Inhibitors