European VOD Registry

Sponsor

Jazz Pharmaceuticals (Industry)

Overall Status

Completed

CT.gov ID

NCT03032016

Collaborator

European Society for Blood and Marrow Transplantation (Other)

176

Enrollment

Locations

50.2

Actual Duration (Months)

3.3

Patients Per Site

0.1

Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Hepatic Veno-Occlusive Disease

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.

Study Design

Study Type:

Observational

Actual Enrollment :

176 participants

Observational Model:

Cohort

Time Perspective:

Prospective

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®

Actual Study Start Date :

Apr 24, 2015

Actual Primary Completion Date :

Jul 1, 2019

Actual Study Completion Date :

Jul 1, 2019

Arms and Interventions

Arm	Intervention/Treatment
sVOD patient treated with defibrotide Patient diagnosed with severe hepatic VOD and treated with defibrotide

Outcome Measures

Primary Outcome Measures

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

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)
GvHD incidence [over 12 months]
Determine the incidence rate of Graft versus host disease (GvHD) in adult and paediatric patients receiving Defitelio®
Survival [over 12 months]
To determine survival by Day+100 post-HSCT, overall mortality and mortality due to VOD in patients treated with Defitelio®
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®

Eligibility Criteria

Criteria

Ages Eligible for Study:

1 Month and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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.

Contacts and Locations

Locations

	Site	City	Country	Postal Code
1	CHRU Angers, Maladies du Sang	Angers	France
2	CHU Bordeaux Groupe hospitalier Pellegrin Enfants	Bordeaux	France
3	CHU d'Estaing	Clermont Ferrand	France
4	CHRU de Lille	Lille	France
5	Hôpital Jeanne de Flandre	Lille	France
6	CHRU Limoges	Limoges	France
7	IHOP	Lyon	France
8	Hôpital d'enfants de la Timone	Mare aux Daims	France
9	Institut Paoli Calmette	Marseille	France
10	CHU Lapeyronie	Montpellier	France
11	CHU Nantes	Nantes	France
12	Hôpital de l'ARCHET	Nice	France
13	Hôpital Saint Antoine	Paris	France	75012
14	Hôpital Robert Debré	Paris	France
15	Hôpital Tenon	Paris	France
16	Institut Curie	Paris	France
17	Hôpital Haut Leveque	Pessac	France
18	Hôpital de la Miletrie	Poitiers	France
19	CHU Hautepierre	Strasbourg	France
20	IUTC Oncopole	Toulouse	France
21	Institut Gustave Roussy (Pediatrics)	Villejuif	France
22	Institut Gustave Roussy	Villejuif	France
23	Azienda Ospedaliera Papa Giovanni XXIII	Bergamo	Italy
24	UO Oncoematologia Ospedale Pediatrico Mayer	Firenze	Italy
25	Ospedale di Careggi	Florence	Italy	50134
26	Institute G. Gaslini	Genova	Italy
27	Ospedale Ca' Granda Ospedale Maggiore di Milano	Milano	Italy
28	Ospedale di Niguarda Ca' Granda	Milano	Italy
29	Ospedale San Raffaele	Milan	Italy
30	Clinica di oncoematologia pediatrica	Padova	Italy
31	Fondazione IRCCS Policlinico San Matteo	Pavia	Italy
32	Ospedale Santa Maria de la Misericorda	Perugia	Italy
33	Ospedale San Carlo	Potenza	Italy
34	IRRCS Ospedale Pediatrico Bambino Gesù	Roma	Italy
35	Universita Cattolica S. Cuore	Roma	Italy
36	La Sapienza University Hospital	Rome	Italy
37	AOU Citta della salute e delle Scienza	Torino	Italy
38	Ospedale Infantile Regina Margherita	Torino	Italy
39	Istituto per l'Infanzia IRCCS Burlo Garofolo	Trieste	Italy
40	Clinica Ematologica-Azienda Ospedaliero Universitaria	Udine	Italy
41	Policlinico G.B. Rossi, Clinica di Oncoematologia Pediatrica	Verona	Italy
42	Inst. Portugues Oncologia o Lisboa	Lisboa	Portugal
43	Birmingham Children's Hospital	Birmingham	United Kingdom
44	Birmingham Heartlands Hospital	Birmingham	United Kingdom
45	Bristol Royal Hospital for Children	Bristol	United Kingdom
46	Great Ormond Street Hospital Children's Charity	London	United Kingdom
47	Imperial College	London	United Kingdom
48	St George's Hospital	London	United Kingdom
49	Royal Manchester Children's Hospital	Manchester	United Kingdom
50	Great North Children's Hospital	Newcastle Upon Tyne	United Kingdom
51	Nottingham City Hospital	Nottingham	United Kingdom
52	John Radccliffe Children's Hospital	Oxford	United Kingdom
53	Plymouth Hospitals NHS Trust	Plymouth	United Kingdom