European VOD Registry
A Multi-centre, Multinational, Prospective Observational Registry to Collect Safety and Outcome Data in Patients Diagnosed With Severe Hepatic Veno-occlusive Disease (VOD) Following Hematopoietic Stem Cell Transplantation (HSCT) and Treated With Defitelio®
1 other identifier
observational
176
4 countries
53
Brief Summary
Following the licencing of a new drug, Defitelio®, indicated for the treatment of severe Veno-Occlusive Disease of the liver (sVOD), a rare but serious complication of haematopoietic stem cell transplantation (HSCT), as a specific obligation (SOB), the manufacture and marketing Authorisation Holder (MHA) (Gentium, a Jazz Pharmaceuticals Company) was required by PRAC (Pharmacovigilance Risk Assessment Committee) to set up a disease registry to collect safety and outcome data, and to assess patterns of utilization of Defitelio® in the post-approval setting. This registry is a Post Authorization Safety Study (PASS), is being coordinated in collaboration with the European Society for Blood and Marrow Transplantation (EBMT). For this study, anonymised clinical data are being collected from patients who develop VOD and and treated with and patients who have been treated with Defitelio® for conditions other than sVOD. The study DOES NOT involve decisions about treatment, which are clinical decisions, but merely collection of data for patients who develop this complication, whether or not they receive treatment and for patients who are treated with Defitelio® for any other reasons.The study DOES NOT involve decisions about treatment, which are clinical decisions, but merely collection of data for patients who develop this complication, whether or not they receive treatment and for patients who are treated with Defitelio® for any other reasons.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for all trials
Started Apr 2015
Longer than P75 for all trials
53 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
Study Start
First participant enrolled
April 24, 2015
CompletedFirst Submitted
Initial submission to the registry
January 24, 2017
CompletedFirst Posted
Study publicly available on registry
January 26, 2017
CompletedPrimary Completion
Last participant's last visit for primary outcome
July 1, 2019
CompletedStudy Completion
Last participant's last visit for all outcomes
July 1, 2019
CompletedOctober 16, 2019
October 1, 2019
4.2 years
January 24, 2017
October 14, 2019
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Incidence rate of specific SAE of interest
Assess the incidence rate of specific SAEs of interest (including fatalities) in patients with severe hepatic VOD post-HSCT treated with Defitelio®
over 12 months
Secondary Outcomes (4)
Describe the population treated
Enrolment
GvHD incidence
over 12 months
Survival
over 12 months
VOD/MOF Resolution
over 12 months
Study Arms (1)
sVOD patient treated with defibrotide
Patient diagnosed with severe hepatic VOD and treated with defibrotide
Eligibility Criteria
The patients will be selected by the treating physicians working in hospitals being part of the European society for Bone Marrow Transplantation network.
You may qualify if:
- Patients undergoing hematopoietic stem cell transplantation and diagnosed with severe hepatic VOD, who agree to participate in the study (Main population).
- Patients treated with defibrotide for another condition than severe hepatic VOD (Secondary population) in the scope of hematopoietic stem cell transplantation .
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (53)
CHRU Angers, Maladies du Sang
Angers, France
CHU Bordeaux Groupe hospitalier Pellegrin Enfants
Bordeaux, France
CHU d'Estaing
Clermont-Ferrand, France
CHRU de Lille
Lille, France
Hôpital Jeanne de Flandre
Lille, France
CHRU Limoges
Limoges, France
IHOP
Lyon, France
Hôpital d'enfants de la Timone
Mare aux Daims, France
Institut Paoli Calmette
Marseille, France
CHU Lapeyronie
Montpellier, France
CHU Nantes
Nantes, France
Hôpital de l'ARCHET
Nice, France
Hôpital Saint Antoine
Paris, 75012, France
Hôpital Robert Debré
Paris, France
Hôpital Tenon
Paris, France
Institut Curie
Paris, France
Hôpital Haut Leveque
Pessac, France
Hôpital de la Miletrie
Poitiers, France
CHU Hautepierre
Strasbourg, France
IUTC Oncopole
Toulouse, France
Institut Gustave Roussy (Pediatrics)
Villejuif, France
Institut Gustave Roussy
Villejuif, France
Azienda Ospedaliera Papa Giovanni XXIII
Bergamo, Italy
Ospedale di Careggi
Florence, 50134, Italy
UO Oncoematologia Ospedale Pediatrico Mayer
Florence, Italy
Institute G. Gaslini
Genova, Italy
Ospedale San Raffaele
Milan, Italy
Ospedale Ca' Granda Ospedale Maggiore di Milano
Milan, Italy
Ospedale di Niguarda Ca' Granda
Milan, Italy
Clinica di oncoematologia pediatrica
Padua, Italy
Fondazione IRCCS Policlinico San Matteo
Pavia, Italy
Ospedale Santa Maria de la Misericorda
Perugia, Italy
Ospedale San Carlo
Potenza, Italy
IRRCS Ospedale Pediatrico Bambino Gesù
Roma, Italy
Universita Cattolica S. Cuore
Roma, Italy
La Sapienza University Hospital
Rome, Italy
AOU Citta della salute e delle Scienza
Torino, Italy
Ospedale Infantile Regina Margherita
Torino, Italy
Istituto per l'Infanzia IRCCS Burlo Garofolo
Trieste, Italy
Clinica Ematologica-Azienda Ospedaliero Universitaria
Udine, Italy
Policlinico G.B. Rossi, Clinica di Oncoematologia Pediatrica
Verona, Italy
Inst. Portugues Oncologia o Lisboa
Lisbon, Portugal
Birmingham Children's Hospital
Birmingham, United Kingdom
Birmingham Heartlands Hospital
Birmingham, United Kingdom
Bristol Royal Hospital for Children
Bristol, United Kingdom
Great Ormond Street Hospital Children's Charity
London, United Kingdom
Imperial College
London, United Kingdom
St George's Hospital
London, United Kingdom
Royal Manchester Children's Hospital
Manchester, United Kingdom
Great North Children's Hospital
Newcastle upon Tyne, United Kingdom
Nottingham City Hospital
Nottingham, United Kingdom
John Radccliffe Children's Hospital
Oxford, United Kingdom
Plymouth Hospitals NHS Trust
Plymouth, United Kingdom
Related Publications (1)
Mohty M, Battista ML, Blaise D, Calore E, Cesaro S, Maximova N, Perruccio K, Renard C, Wynn R, Zecca M, Labopin M, Hanvesakul R, Amber V, Ryan RJ, Lawson S, Ciceri F. A multicentre, multinational, prospective, observational registry study of defibrotide in patients diagnosed with veno-occlusive disease/sinusoidal obstruction syndrome after haematopoietic cell transplantation: an EBMT study. Bone Marrow Transplant. 2021 Oct;56(10):2454-2463. doi: 10.1038/s41409-021-01265-2. Epub 2021 May 31.
PMID: 34059801DERIVED
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Design
- Study Type
- observational
- Observational Model
- COHORT
- Time Perspective
- PROSPECTIVE
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
January 24, 2017
First Posted
January 26, 2017
Study Start
April 24, 2015
Primary Completion
July 1, 2019
Study Completion
July 1, 2019
Last Updated
October 16, 2019
Record last verified: 2019-10
Data Sharing
- IPD Sharing
- Will not share