European VOD Registry

Hepatic Veno-Occlusive Disease Clinical Trial

Official title:

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®

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03032016
• Other study ID #: DF VOD-2013-03-REG
• Status: Completed
• Start date: April 24, 2015
• Est. completion date: July 2019

Study information

• Verified date: October 2019
• Source: Jazz Pharmaceuticals
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

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 information / eligibility

• Status: Completed
• Enrollment: 176
• Est. completion date: July 2019
• Est. primary completion date: July 2019
• Accepts healthy volunteers: No
• Gender: All
• Age group: 1 Month and older

Eligibility

Inclusion Criteria:

• 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 .

Exclusion Criteria:

• 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.

Related Conditions & MeSH terms

• Hepatic Veno-Occlusive Disease

Locations

Country	Name	City	State
France	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 Robert Debré	Paris
France	Hôpital Saint Antoine	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	Villejuif
France	Institut Gustave Roussy (Pediatrics)	Villejuif
Italy	Azienda Ospedaliera Papa Giovanni XXIII	Bergamo
Italy	UO Oncoematologia Ospedale Pediatrico Mayer	Firenze
Italy	Ospedale di Careggi	Florence
Italy	Institute G. Gaslini	Genova
Italy	Ospedale San Raffaele	Milan
Italy	Ospedale Ca' Granda Ospedale Maggiore di Milano	Milano
Italy	Ospedale di Niguarda Ca' Granda	Milano
Italy	Clinica di oncoematologia pediatrica	Padova
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
Portugal	Inst. Portugues Oncologia o Lisboa	Lisboa
United Kingdom	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

Sponsors (2)

Lead Sponsor	Collaborator
Jazz Pharmaceuticals	European Group for Blood and Marrow Transplantation

Countries where clinical trial is conducted

France,  Italy,  Portugal,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	Describe the population treated	To describe the population treated with Defitelio® (age, gender, patients with pre-existing liver or severe renal insufficiency; patient with intrinsic lung disease)	Enrolment
Secondary	GvHD incidence	Determine the incidence rate of Graft versus host disease (GvHD) in adult and paediatric patients receiving Defitelio®	over 12 months
Secondary	Survival	To determine survival by Day+100 post-HSCT, overall mortality and mortality due to VOD in patients treated with Defitelio®	over 12 months
Secondary	VOD/MOF Resolution	To determine the rate of VOD and VOD with MOF resolution any time after treatment initiation in patients treated with Defitelio®	over 12 months

External Links

•   Home page of the European VOD Registry on EBMT Website