Pediatric Trial Investigating the Incidence & Outcome of Veno-Occlusive Disease With the Prophylactic Use of Defibrotide
VOD-DF
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
2 other identifiers
interventional
360
10 countries
30
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for phase_2
Started Dec 2005
Typical duration for phase_2
30 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
Study Start
First participant enrolled
December 1, 2005
CompletedFirst Submitted
Initial submission to the registry
January 4, 2006
CompletedFirst Posted
Study publicly available on registry
January 9, 2006
CompletedPrimary Completion
Last participant's last visit for primary outcome
January 1, 2009
CompletedStudy Completion
Last participant's last visit for all outcomes
July 1, 2009
CompletedJune 9, 2011
June 1, 2011
3.1 years
January 4, 2006
June 8, 2011
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
The primary objective is to evaluate if prophylactic DF has an impact on the incidence of VOD
Day + 30 post HSCT
Secondary Outcomes (1)
Occurrence of Multi-System Organ Failure and Survival (all causes of mortality)
day +100 post HSCT
Study Arms (2)
Prophylaxis Arm
EXPERIMENTALControl Arm
ACTIVE COMPARATORInterventions
Eligibility Criteria
You may qualify if:
- 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)
You may not qualify if:
- Pregnant patients
- Patients who are transplanted but do not fulfill any of the above mentioned criteria
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (30)
University Hospital
Graz, Austria
St Anna Kinderspital
Vienna, Austria
Inst. Gustave Roussy
Villejuif, France
University Hospital
Dresden, Germany
Klinik Kinder-Onkologie
Düsseldorf, Germany
Johann-Wolfgang Goethe Universität
Frankfurt, Germany
Universitätsspital Eppendorf
Hamburg, Germany
Medical School
Hanover, Germany
University Hospital
Heidelberg, Germany
Kinderklinik
Jena, Germany
Christian-Albrecht -University
Kiel, Germany
Kinderpoliklinik Uni
München, Germany
Poliklinik Kinderheilkunde
Münster, Germany
Universitätsspital
Tübingen, Germany
Our Lady's Hospital for Sick Children
Dublin, Ireland
Schneider Children's MC
Petah Tikva, Israel
Institute G. Gaslini
Genova, Italy
Ospedale S. Gerardo
Monza, Italy
Clinica di Oncoematologia Pediatrica
Padua, Italy
University Hospital
Utrecht, Netherlands
Sahlgrenska University Hospital
Gothenburg, Sweden
University Hospital
Huddinge, Sweden
University Hospital
Lund, Sweden
University Hospital
Uppsala, Sweden
University Children Hospital
Basel, Switzerland
Inselspital
Bern, Switzerland
Hopital Cantonal Universitaire
Geneva, Switzerland
University Hospital
Zurich, Switzerland
Great Ormond Street Hospital
London, United Kingdom
Children's Hospital
Manchester, United Kingdom
Related Publications (2)
Corbacioglu S, Grupp SA, Richardson PG, Duarte R, Pagliuca A, Ruutu T, Mahadeo K, Carreras E. Prevention of veno-occlusive disease/sinusoidal obstruction syndrome: a never-ending story and no easy answer. Bone Marrow Transplant. 2023 Aug;58(8):839-841. doi: 10.1038/s41409-023-02007-2. Epub 2023 May 25. No abstract available.
PMID: 37231094DERIVEDCorbacioglu S, Cesaro S, Faraci M, Valteau-Couanet D, Gruhn B, Rovelli A, Boelens JJ, Hewitt A, Schrum J, Schulz AS, Muller I, Stein J, Wynn R, Greil J, Sykora KW, Matthes-Martin S, Fuhrer M, O'Meara A, Toporski J, Sedlacek P, Schlegel PG, Ehlert K, Fasth A, Winiarski J, Arvidson J, Mauz-Korholz C, Ozsahin H, Schrauder A, Bader P, Massaro J, D'Agostino R, Hoyle M, Iacobelli M, Debatin KM, Peters C, Dini G. Defibrotide for prophylaxis of hepatic veno-occlusive disease in paediatric haemopoietic stem-cell transplantation: an open-label, phase 3, randomised controlled trial. Lancet. 2012 Apr 7;379(9823):1301-9. doi: 10.1016/S0140-6736(11)61938-7. Epub 2012 Feb 23.
PMID: 22364685DERIVED
Related Links
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
Selim Corbacioglu, MD
University of Ulm, Germany
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- RANDOMIZED
- Masking
- NONE
- Purpose
- PREVENTION
- Intervention Model
- PARALLEL
- Sponsor Type
- NETWORK
Study Record Dates
First Submitted
January 4, 2006
First Posted
January 9, 2006
Study Start
December 1, 2005
Primary Completion
January 1, 2009
Study Completion
July 1, 2009
Last Updated
June 9, 2011
Record last verified: 2011-06