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European VOD Registry

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ClinicalTrials.gov Identifier: NCT03032016
Recruitment Status : Active, not recruiting
First Posted : January 26, 2017
Last Update Posted : April 8, 2019
Sponsor:
Collaborator:
European Group for Blood and Marrow Transplantation
Information provided by (Responsible Party):
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.


Condition or disease
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.

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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 : April 24, 2015
Estimated Primary Completion Date : July 2019
Estimated Study Completion Date : July 2019

Resource links provided by the National Library of Medicine


Group/Cohort
sVOD patient treated with defibrotide
Patient diagnosed with severe hepatic VOD and treated with defibrotide



Primary Outcome Measures :
  1. 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®


Secondary Outcome Measures :
  1. 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)

  2. GvHD incidence [ Time Frame: over 12 months ]
    Determine the incidence rate of Graft versus host disease (GvHD) in adult and paediatric patients receiving Defitelio®

  3. 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®

  4. 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®



Information from the National Library of Medicine

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Ages Eligible for Study:   1 Month and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.
Criteria

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03032016


  Show 53 Study Locations
Sponsors and Collaborators
Jazz Pharmaceuticals
European Group for Blood and Marrow Transplantation

Additional Information:
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Responsible Party: Jazz Pharmaceuticals
ClinicalTrials.gov Identifier: NCT03032016     History of Changes
Other Study ID Numbers: DF VOD-2013-03-REG
First Posted: January 26, 2017    Key Record Dates
Last Update Posted: April 8, 2019
Last Verified: April 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Jazz Pharmaceuticals:
VOD
Defibrotide
Defitelio
Registry
SOS
Additional relevant MeSH terms:
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Hepatic Veno-Occlusive Disease
Liver Diseases
Digestive System Diseases
Vascular Diseases
Cardiovascular Diseases
Defibrotide
Fibrinolytic Agents
Fibrin Modulating Agents
Molecular Mechanisms of Pharmacological Action
Platelet Aggregation Inhibitors