A multi-centre, multinational, prospective observational registry to collect safety and outcome data in patients diagnosed with severe hepatic veno-occlusive disease (VOD) following haematopoietic stem cell transplantation (HSCT) and treated with Defitelio® (DF VOD-2013-03-REG)

Study identification

• EU PAS number:
  EUPAS5592
• Study ID:
  36318
• Official title and acronym:
  A multi-centre, multinational, prospective observational registry to collect safety and outcome data in patients diagnosed with severe hepatic veno-occlusive disease (VOD) following haematopoietic stem cell transplantation (HSCT) and treated with Defitelio® (DF VOD-2013-03-REG)
• DARWIN EU® study:
  No
• Study countries:
  France

  Italy

  Portugal

  United Kingdom
• Study description:
  A multi-centre, multinational, prospective observational registry to collect safety and outcome data in patients diagnosed with severe hepatic VOD following hematopoietic stem cell transplantation (HSCT) and treated with Defitelio®.The registry is performed in collaboration with the European Society for Blood and Marrow Transplantation (EBMT). Participating sites are transplant centres that are members of EBMT.Following obtaining an informed consent, clinical data from patients who are treated with Defitelio® for severe hepatic VOD will be collected. In addition all patients prescribed Defitelio® regardless of indication will also be registered, and information will be collected on the indication for which Defitelio® has been administered.Patient clinical data will be collected on the registry form at 100 days, 6 months and 12 months post-HSCT. The objective of this non-interventional study is to assess the incidence rate of specific SAEs of interest (including fatalities) in patients with sVOD treated with Defitelio®. All SAEs of interest will be recorded by the treating physician or by the site staff, whether or not these are considered to be related to the treatment received.
• Study status:
  Finalised

Research institutions and networks

Institutions

Jazz Pharmaceuticals

Multiple centres: 36 centres are involved in the study

Networks

EBMT

Study timelines

Date when funding contract was signed

Planned:10/01/2014

Actual:10/01/2014

Study start date

Planned:01/04/2015

Actual:24/04/2015

Data analysis start date

Planned:18/10/2019

Actual:18/10/2019

Date of final study report

Planned:03/07/2020

Actual:18/06/2020

Sources of funding

Pharmaceutical company and other private sector 

More details on funding

Jazz Pharmaceuticals

Regulatory

• Was the study required by a regulatory body?:
  Yes
• Is the study required by a Risk Management Plan (RMP)?:
  EU RMP category 3 (required)