Jazz 15-007
Research type
Research Study
Full title
A Phase 3, Randomized, Adaptive Study Comparing the Efficacy and Safety of Defibrotide vs Best Supportive Care in the Prevention of Hepatic Veno-Occlusive Disease in Adult and Pediatric Patients Undergoing Hematopoietic Stem Cell Transplant.
IRAS ID
212197
Contact name
Antonio Pagliuca
Contact email
Sponsor organisation
Jazz Pharmaceuticals, Inc
Eudract number
2016-002004-10
Clinicaltrials.gov Identifier
62,118, IND Number
Duration of Study in the UK
4 years, 8 months, 31 days
Research summary
This is a clinical research study to test a new medicine to prevent liver veno-occlusive disease (VOD) in patients undergoing hematopoietic stem cell transplant (HSCT). This research study is investigating a medicine called Defibrotide. These patients will also be at high risk or very high risk for liver VOD following HSCT. Liver VOD can be a complication of a bone marrow and/or stem cell transplant, which can be life threatening.
VOD is a condition that is a possible side effect of the chemotherapy or conditioning regimen patients receive to help treat their cancer. VOD causes damage to blood vessels in the liver and surrounding liver cells, and this can cause pain, fluid retention and liver function that is abnormal. This can be life-threatening. Defibrotide may possibly prevent liver VOD in adult and paediatric patients, and this study is designed to investigate this. This research study is for patients over the age of 1 month.
Defibrotide is an approved medicine in Europe, Israel, South Korea, and the United States with the brand name Defitelio® for the treatment of VOD. Information from other research studies suggests that Defibrotide may also help to prevent VOD before it even starts. Defibrotide has not yet been approved for the prevention of VOD in patients undergoing HSCT, so its use in preventing VOD is new and experimental.
A patient who wishes to participate in the research study will be participating for approximately 6 months from the date of their HSCT. 400-600 patients will take part in this study in approximately 100 study centres in countries throughout the world, including the United States. This research study is sponsored by Jazz Pharmaceuticals.REC name
East Midlands - Derby Research Ethics Committee
REC reference
16/EM/0381
Date of REC Opinion
5 Jan 2017
REC opinion
Further Information Favourable Opinion