Hepatic Veno-Occlusive DiseaseNCT03032016
European VOD Registry
An observational study of Hepatic Veno-Occlusive Disease, run by Jazz Pharmaceuticals at 53 sites in 4 countries.
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… Read more about this study
- Who is it for?
- Ages 1 month and older
- Conditions the study is for
- Hepatic Veno-Occlusive Disease
- How many people
- 176 participants
- Where
- 53 sitesin 4 countries
- Study dates
- 24 Apr 2015to Jul 2019
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 1 month and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
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.
Who may be able to join
- 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 .
Who cannot join
- 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.
Trial locations and contacts
53 sites in 4 countries.
53 sites in 4 countries
France22 sites
CHRU Angers, Maladies du Sang
Angers
CHU Bordeaux Groupe hospitalier Pellegrin Enfants
Bordeaux
CHU d'Estaing
Clermont-Ferrand
CHRU de Lille
Lille
Hôpital Jeanne de Flandre
Lille
CHRU Limoges
Limoges
IHOP
Lyon
Hôpital d'enfants de la Timone
Mare aux Daims
Institut Paoli Calmette
Marseille
CHU Lapeyronie
Montpellier
CHU Nantes
Nantes
Hôpital de l'ARCHET
Nice
Hôpital Saint Antoine
Paris, 75012
Hôpital Robert Debré
Paris
Hôpital Tenon
Paris
Institut Curie
Paris
Hôpital Haut Leveque
Pessac
Hôpital de la Miletrie
Poitiers
CHU Hautepierre
Strasbourg
IUTC Oncopole
Toulouse
Institut Gustave Roussy (Pediatrics)
Villejuif
Institut Gustave Roussy
Villejuif
Italy19 sites
Azienda Ospedaliera Papa Giovanni XXIII
Bergamo
Ospedale di Careggi
Florence, 50134
UO Oncoematologia Ospedale Pediatrico Mayer
Florence
Institute G. Gaslini
Genova
Ospedale San Raffaele
Milan
Ospedale Ca' Granda Ospedale Maggiore di Milano
Milan
Ospedale di Niguarda Ca' Granda
Milan
Clinica di oncoematologia pediatrica
Padova
Fondazione IRCCS Policlinico San Matteo
Pavia
Ospedale Santa Maria de la Misericorda
Perugia
Ospedale San Carlo
Potenza
IRRCS Ospedale Pediatrico Bambino Gesù
Roma
Universita Cattolica S. Cuore
Roma
La Sapienza University Hospital
Rome
AOU Citta della salute e delle Scienza
Torino
Ospedale Infantile Regina Margherita
Torino
Istituto per l'Infanzia IRCCS Burlo Garofolo
Trieste
Clinica Ematologica-Azienda Ospedaliero Universitaria
Udine
Policlinico G.B. Rossi, Clinica di Oncoematologia Pediatrica
Verona
United Kingdom11 sites
Birmingham Children's Hospital
Birmingham
Birmingham Heartlands Hospital
Birmingham
Bristol Royal Hospital for Children
Bristol
Great Ormond Street Hospital Children's Charity
London
Imperial College
London
St George's Hospital
London
Royal Manchester Children's Hospital
Manchester
Great North Children's Hospital
Newcastle upon Tyne
Nottingham City Hospital
Nottingham
John Radccliffe Children's Hospital
Oxford
Plymouth Hospitals NHS Trust
Plymouth
About this study
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®
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.
Read the 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.
- Conditions
- Hepatic Veno-Occlusive Disease
- Keywords
- VOD, Defibrotide, Defitelio, Registry, SOS
- Medical subject headings
- Hepatic Veno-Occlusive Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| sVOD patient treated with defibrotide | Patient diagnosed with severe hepatic VOD and treated with defibrotide |
What is being measured
The primary outcome is the main question the study is designed to answer.
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®
Measured: over 12 months
Secondary outcomes (4)
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)
Measured: Enrolment
GvHD incidence
Determine the incidence rate of Graft versus host disease (GvHD) in adult and paediatric patients receiving Defitelio®
Measured: over 12 months
Survival
To determine survival by Day+100 post-HSCT, overall mortality and mortality due to VOD in patients treated with Defitelio®
Measured: over 12 months
VOD/MOF Resolution
To determine the rate of VOD and VOD with MOF resolution any time after treatment initiation in patients treated with Defitelio®
Measured: over 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Jan 2017 (estimated)
- Study start24 Apr 2015
- Primary completionJul 2019
- Study completionJul 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Hepatic Veno-Occlusive DiseaseStudies of the same condition, open and under way.
- More trials run by Jazz PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 176 participants (actual)
Sponsor and investigators
- Lead sponsor
- Jazz Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- European Society for Blood and Marrow Transplantation
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03032016
- Sponsor's protocol ID
- DF VOD-2013-03-REG
- Status last verified
- Oct 2019
- First submitted
- 24 Jan 2017
- Last updated
- 16 Oct 2019
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
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.
PubMed 34059801
Study at a glance
From the registry record, last updated by the sponsor on 16 Oct 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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®
- ClinicalTrials.gov ID
- NCT03032016
- Acronym / sponsor ID
- DF VOD-2013-03-REG
- Conditions
- Hepatic Veno-Occlusive Disease
- Keywords
- VOD, Defibrotide, Defitelio, Registry, SOS
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 176 participants (actual)
- Lead sponsor
- Jazz PharmaceuticalsIndustry (pharma, biotech, devices)
- Collaborators
- European Society for Blood and Marrow Transplantation
- Study start
- 24 Apr 2015 (actual)
- Primary completion
- Jul 2019 (actual)
- Study completion
- Jul 2019 (actual)
- First posted
- 26 Jan 2017 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| sVOD patient treated with defibrotide | – | –Patient diagnosed with severe hepatic VOD and treated with defibrotide |
Who may qualify
- Ages
- 1 month and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- 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.
Key inclusion criteria (2 listed)
- 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 .
Key exclusion criteria (1 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
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®
4 secondary outcomes
Describe the population treated
Time frame: Enrolment
GvHD incidence
Time frame: over 12 months
Survival
Time frame: over 12 months
VOD/MOF Resolution
Time frame: over 12 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Oct 2019.
- Derived from this study: PubMed 34059801Mohty 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.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
53 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hepatic Veno-Occlusive Disease, run by Jazz Pharmaceuticals at 53 sites in 4 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 16 Oct 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 53 sites in France, Italy, United Kingdom, Portugal. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence rate of specific SAE of interest (time frame: over 12 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03032016, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Oct 2019.