European VOD Registry

October 14, 2019 updated by: Jazz Pharmaceuticals

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®

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.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

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 Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Observational

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
176

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • France
      • Angers, France
        • CHRU Angers, Maladies du Sang
      • Bordeaux, France
        • CHU Bordeaux Groupe hospitalier Pellegrin Enfants
      • Clermont Ferrand, France
        • CHU d'Estaing
      • Lille, France
        • CHRU de Lille
      • Lille, France
        • Hôpital Jeanne de Flandre
      • Limoges, France
        • CHRU Limoges
      • Lyon, France
        • IHOP
      • Mare aux Daims, France
        • Hôpital d'Enfants de la Timone
      • Marseille, France
        • Institut Paoli Calmette
      • Montpellier, France
        • CHU Lapeyronie
      • Nantes, France
        • CHU Nantes
      • Nice, France
        • Hopital de l'archet
      • Paris, France, 75012
        • Hôpital Saint Antoine
      • Paris, France
        • Hopital Robert Debre
      • Paris, France
        • Institut Curie
      • Paris, France
        • Hopital Tenon
      • Pessac, France
        • Hôpital Haut Levêque
      • Poitiers, France
        • Hôpital de la Milétrie
      • Strasbourg, France
        • CHU Hautepierre
      • Toulouse, France
        • IUTC Oncopole
      • Villejuif, France
        • Institut Gustave Roussy
      • Villejuif, France
        • Institut Gustave Roussy (Pediatrics)
  • Italy
      • Bergamo, Italy
        • Azienda Ospedaliera Papa Giovanni XXIII
      • Firenze, Italy
        • UO Oncoematologia Ospedale Pediatrico Mayer
      • Florence, Italy, 50134
        • Ospedale di Careggi
      • Genova, Italy
        • Institute G. Gaslini
      • Milan, Italy
        • Ospedale San Raffaele
      • Milano, Italy
        • Ospedale Ca' Granda Ospedale Maggiore di Milano
      • Milano, Italy
        • Ospedale di Niguarda Ca' Granda
      • Padova, Italy
        • Clinica di Oncoematologia Pediatrica
      • Pavia, Italy
        • Fondazione IRCCS Policlinico San Matteo
      • Perugia, Italy
        • Ospedale Santa Maria de la Misericorda
      • Potenza, Italy
        • Ospedale San Carlo
      • Roma, Italy
        • IRRCS Ospedale Pediatrico Bambino Gesù
      • Roma, Italy
        • Universita Cattolica S. Cuore
      • Rome, Italy
        • La Sapienza University Hospital
      • Torino, Italy
        • Ospedale Infantile Regina Margherita
      • Torino, Italy
        • AOU Citta della salute e delle Scienza
      • Trieste, Italy
        • Istituto per l'Infanzia IRCCS Burlo Garofolo
      • Udine, Italy
        • Clinica Ematologica-Azienda Ospedaliero Universitaria
      • Verona, Italy
        • Policlinico G.B. Rossi, Clinica di Oncoematologia Pediatrica
  • Portugal
      • Lisboa, Portugal
        • Inst. Portugues Oncologia o Lisboa
  • United Kingdom
      • Birmingham, United Kingdom
        • Birmingham Heartlands Hospital
      • Birmingham, United Kingdom
        • Birmingham Children's Hospital
      • Bristol, United Kingdom
        • Bristol Royal Hospital for Children
      • London, United Kingdom
        • St George's Hospital
      • London, United Kingdom
        • Imperial College
      • London, United Kingdom
        • Great Ormond Street Hospital Children's Charity
      • Manchester, United Kingdom
        • Royal Manchester Children's Hospital
      • Newcastle Upon Tyne, United Kingdom
        • Great North Children's Hospital
      • Nottingham, United Kingdom
        • Nottingham City Hospital
      • Oxford, United Kingdom
        • John Radccliffe Children's Hospital
      • Plymouth, United Kingdom
        • Plymouth Hospitals NHS Trust

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

1 month and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Sampling Method

Non-Probability Sample

Study Population

The patients will be selected by the treating physicians working in hospitals being part of the European society for Bone Marrow Transplantation network.

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Cohort
  • Time Perspectives: Prospective

Cohorts and Interventions

Group / Cohort

Group / Cohort

A group or subgroup of participants in an observational study that is assessed for biomedical or health outcomes.
sVOD patient treated with defibrotide
Patient diagnosed with severe hepatic VOD and treated with defibrotide

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Incidence rate of specific SAE of interest
Time Frame: 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®
over 12 months

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Describe the population treated
Time Frame: Enrolment
To describe the population treated with Defitelio® (age, gender, patients with pre-existing liver or severe renal insufficiency; patient with intrinsic lung disease)
Enrolment
GvHD incidence
Time Frame: over 12 months
Determine the incidence rate of Graft versus host disease (GvHD) in adult and paediatric patients receiving Defitelio®
over 12 months
Survival
Time Frame: over 12 months
To determine survival by Day+100 post-HSCT, overall mortality and mortality due to VOD in patients treated with Defitelio®
over 12 months
VOD/MOF Resolution
Time Frame: over 12 months
To determine the rate of VOD and VOD with MOF resolution any time after treatment initiation in patients treated with Defitelio®
over 12 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Jazz Pharmaceuticals

Collaborators

Collaborators

An organization other than the sponsor that provides support for a clinical study. This support may include activities related to funding, design, implementation, data analysis, or reporting.
European Society for Blood and Marrow Transplantation

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
April 24, 2015

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
July 1, 2019

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
July 1, 2019

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
January 24, 2017

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
January 24, 2017

First Posted (Estimate)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
January 26, 2017

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
October 16, 2019

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
October 14, 2019

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
October 1, 2019

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • DF VOD-2013-03-REG

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

No

Drug and device information, study documents

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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