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European VOD Registry

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ClinicalTrials.gov Identifier: NCT03032016
Recruitment Status : Completed
First Posted : January 26, 2017
Last Update Posted : October 16, 2019
Sponsor:
Collaborator:
European Group for Blood and Marrow Transplantation
Information provided by (Responsible Party):
Jazz Pharmaceuticals

Tracking Information
First Submitted Date January 24, 2017
First Posted Date January 26, 2017
Last Update Posted Date October 16, 2019
Actual Study Start Date April 24, 2015
Actual Primary Completion Date July 2019   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: February 12, 2019)
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®
Original Primary Outcome Measures
 (submitted: January 24, 2017)
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 treated with Defitelio® and to compare these rates with those occurring in a similar population not treated with Defitelio®.
Change History Complete list of historical versions of study NCT03032016 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures
 (submitted: February 12, 2019)
  • 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)
  • GvHD incidence [ Time Frame: over 12 months ]
    Determine the incidence rate of Graft versus host disease (GvHD) in adult and paediatric patients receiving Defitelio®
  • 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®
  • 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®
Original Secondary Outcome Measures
 (submitted: January 24, 2017)
  • Describe the population [ Time Frame: Enrolment ]
    To describe the population treated with Defitelio® and controls (age, gender, ethnicity, patients with pre-existing liver or severe renal insufficiency; patient with intrinsic lung disease)
  • GvHD incidence [ Time Frame: over 12 months ]
    Determine the incidence rate of Graft versus host disease (GvHD) in adult and paediatric patients receiving Defitelio® and in controls
  • 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® and in controls
  • VOD/MOF Resolution [ Time Frame: over 12 months ]
    To determine the rate of Veno Occlusive Disease/Multi Organ Failure resolution any time after treatment initiation in patients treated with Defitelio® and in controls.
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title European VOD Registry
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®
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.

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 Observational
Study Design Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration Not Provided
Biospecimen Not Provided
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.
Condition Hepatic Veno-Occlusive Disease
Intervention Not Provided
Study Groups/Cohorts sVOD patient treated with defibrotide
Patient diagnosed with severe hepatic VOD and treated with defibrotide
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Completed
Actual Enrollment
 (submitted: February 12, 2019)
176
Original Estimated Enrollment
 (submitted: January 24, 2017)
600
Actual Study Completion Date July 2019
Actual Primary Completion Date July 2019   (Final data collection date for primary outcome measure)
Eligibility 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.
Sex/Gender
Sexes Eligible for Study: All
Ages 1 Month and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers No
Contacts Contact information is only displayed when the study is recruiting subjects
Listed Location Countries France,   Italy,   Portugal,   United Kingdom
Removed Location Countries Ireland
 
Administrative Information
NCT Number NCT03032016
Other Study ID Numbers DF VOD-2013-03-REG
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement
Plan to Share IPD: No
Responsible Party Jazz Pharmaceuticals
Study Sponsor Jazz Pharmaceuticals
Collaborators European Group for Blood and Marrow Transplantation
Investigators Not Provided
PRS Account Jazz Pharmaceuticals
Verification Date October 2019