European VOD Registry
- Conditions
- Hepatic Veno-Occlusive Disease
- Registration Number
- NCT03032016
- Lead Sponsor
- Jazz Pharmaceuticals
- 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.
- Detailed Description
Defitelio® was granted a Marketing Authorisation in Europe under exceptional circumstances on 18 October 2013. Defitelio® is indicated for the treatment of severe hepatic Veno-Occlusive Disease (sVOD) also known as sinusoidal obstruction syndrome (SOS) in haematopoietic stem-cell transplantation (HSCT) therapy.
It is indicated in adults and in adolescents, children and infants over 1 month of age.
As required by the risk management plan, Gentium 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 is a multi-centre, multinational and prospective observational (non-interventional) disease registry of patients with severe hepatic VOD following HSCT and treated with Defitelio®.
The registry will be conducted in European transplant centres that are members of the European Society for Blood and Marrow Transplantation (EBMT).
The main objective of the registry is to assess the incidence rate of specific SAEs (Serious Adverse Events) of interest (including fatalities) in patients with severe hepatic VOD post-HSCT treated with Defitelio®.
Secondary objectives are:
* To describe the population treated with Defibrotide (age, gender, patients with pre-existing liver or severe renal insufficiency; patient with intrinsic lung disease)
* To determine the incidence rate of multiorgan failure (MOF) and Graft versus host disease (GvHD) in adult and paediatric patients receiving Defibrotide.
* To determine survival by Day+100 post-HSCT, overall mortality and mortality due to VOD in patients treated with Defibrotide.
* To determine the rate of VOD and VOD with MOF resolution any time after treatment initiation in patients treated with Defibrotide.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 176
- 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 .
- There will not be any specific exclusion criteria; however contraindications, special warnings and precautions for use as detailed in the SPC will have to be considered by the treating physician.
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Incidence rate of specific SAE of interest over 12 months Assess the incidence rate of specific SAEs of interest (including fatalities) in patients with severe hepatic VOD post-HSCT treated with Defitelio®
- Secondary Outcome Measures
Name Time Method Survival over 12 months To determine survival by Day+100 post-HSCT, overall mortality and mortality due to VOD in patients treated with Defitelio®
GvHD incidence over 12 months Determine the incidence rate of Graft versus host disease (GvHD) in adult and paediatric patients receiving Defitelio®
Describe the population treated Enrolment To describe the population treated with Defitelio® (age, gender, patients with pre-existing liver or severe renal insufficiency; patient with intrinsic lung disease)
VOD/MOF Resolution over 12 months To determine the rate of VOD and VOD with MOF resolution any time after treatment initiation in patients treated with Defitelio®
Trial Locations
- Locations (53)
CHU d'Estaing
🇫🇷Clermont Ferrand, France
IHOP
🇫🇷Lyon, France
Hôpital d'enfants de la Timone
🇫🇷Mare aux Daims, France
Institut Paoli Calmette
🇫🇷Marseille, France
CHU Lapeyronie
🇫🇷Montpellier, France
Hôpital de l'ARCHET
🇫🇷Nice, France
Hôpital Saint Antoine
🇫🇷Paris, 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
Institute G. Gaslini
🇮🇹Genova, Italy
Ospedale Ca' Granda Ospedale Maggiore di Milano
🇮🇹Milano, Italy
Ospedale San Raffaele
🇮🇹Milan, Italy
Universita Cattolica S. Cuore
🇮🇹Roma, Italy
IRRCS Ospedale Pediatrico Bambino Gesù
🇮🇹Roma, 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
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
Inst. Portugues Oncologia o Lisboa
🇵🇹Lisboa, Portugal
CHU Bordeaux Groupe hospitalier Pellegrin Enfants
🇫🇷Bordeaux, France
CHRU Angers, Maladies du Sang
🇫🇷Angers, France
CHRU Limoges
🇫🇷Limoges, France
CHRU de Lille
🇫🇷Lille, France
Hôpital Jeanne de Flandre
🇫🇷Lille, France
CHU Nantes
🇫🇷Nantes, France
Institut Gustave Roussy (Pediatrics)
🇫🇷Villejuif, France
Azienda Ospedaliera Papa Giovanni XXIII
🇮🇹Bergamo, Italy
Institut Gustave Roussy
🇫🇷Villejuif, France
UO Oncoematologia Ospedale Pediatrico Mayer
🇮🇹Firenze, Italy
Ospedale di Careggi
🇮🇹Florence, Italy
Ospedale di Niguarda Ca' Granda
🇮🇹Milano, Italy
Ospedale Santa Maria de la Misericorda
🇮🇹Perugia, Italy
Ospedale San Carlo
🇮🇹Potenza, Italy
Clinica di oncoematologia pediatrica
🇮🇹Padova, Italy
La Sapienza University Hospital
🇮🇹Rome, Italy
Policlinico G.B. Rossi, Clinica di Oncoematologia Pediatrica
🇮🇹Verona, Italy
Fondazione IRCCS Policlinico San Matteo
🇮🇹Pavia, Italy
Royal Manchester Children's Hospital
🇬🇧Manchester, United Kingdom
Great North Children's Hospital
🇬🇧Newcastle Upon Tyne, United Kingdom
Imperial College
🇬🇧London, United Kingdom
John Radccliffe Children's Hospital
🇬🇧Oxford, United Kingdom
Nottingham City Hospital
🇬🇧Nottingham, United Kingdom
Plymouth Hospitals NHS Trust
🇬🇧Plymouth, United Kingdom
St George's Hospital
🇬🇧London, United Kingdom