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®
Trial Snapshot
- Phase
- Not Applicable
- Status
- Completed
- Sponsor
- Jazz Pharmaceuticals
- Enrollment
- 176
- Locations
- 53
- Primary Endpoint
- Incidence rate of specific SAE of interest
Study Overview
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).
Study Design
- Study Type
- Observational
- Observational Model
- Cohort
- Time Perspective
- Prospective
Eligibility Criteria
- Ages
- 1 Month to — (Child, Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
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.
Outcomes
Primary Outcomes
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 Outcomes
- Survival(over 12 months)
- GvHD incidence(over 12 months)
- Describe the population treated(Enrolment)
- VOD/MOF Resolution(over 12 months)