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

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 |
- The primary objective is to evaluate if prophylactic DF has an impact on the incidence of VOD [ Time Frame: Day + 30 post HSCT ]
- Occurrence of Multi-System Organ Failure and Survival (all causes of mortality) [ Time Frame: day +100 post HSCT ]

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Ages Eligible for Study: | up to 18 Years (Child, Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
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:
- Pre-existing liver disease
- Second myeloablative HSCT
- History of treatment with gemtuzumab ozogamicin (MYLOTARGÒ, GO, CMA-676, Wyeth)
- Allogeneic HSCT for leukemia beyond the second relapse
- Osteopetrosis (OP)
- Conditioning with busulfan and melphalan
- Macrophage activating syndromes (MAS, like hemophagocytic lymphohistiocytosis, Griscelli, Chediak-Higashi
- Adrenoleukodystrophy (ALD)
Exclusion Criteria:
- Pregnant patients
- Patients who are transplanted but do not fulfill any of the above mentioned criteria

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

Principal Investigator: | Selim Corbacioglu, MD | University of Ulm, Germany |
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
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 |
Venoocclusive disease Pediatric myeloablative conditioning stem cell transplant |
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 |