Jazz JZP963-201
Research type
Research Study
Full title
A Phase 2, Prospective, Randomized, Open-label Study on the Efficacy of Defibrotide Added to Standard of Care Immunoprophylaxis for the Prevention of Acute Graft-versus-Host-Disease in Adult and Pediatric Patients After Allogeneic Hematopoietic Stem Cell Transplant.
IRAS ID
241341
Contact name
Christopher Parrish
Contact email
Sponsor organisation
Jazz Pharmaceuticals Inc
Eudract number
2017-003309-16
Duration of Study in the UK
1 years, 5 months, 11 days
Research summary
Allogeneic Haematopoietic Stem Cell Transplant (HSCT) is a procedure used to treat patients diagnosed with acute leukaemia or myelodysplastic syndrome (MDS). It involves the receipt of bone marrow or stem cells from a donor. A common complication of allogeneic stem cell transplant is acute Graft versus Host Disease (aGvHD). This is where the cells from the donor start an immune reaction in the patient’s body. It occurs in approximately 40–59% of patients who receive allogeneic HSCT, and it may be life threatening.
This clinical research study will test a new medicine, called defibrotide, to prevent aGvHD. Patients being considered for allogeneic HSCT who have been diagnosed with acute leukaemia or MDS may be eligible for this study.
Everyone who participates in the study will receive standard treatment to prevent aGvHD. Participants will be randomly assigned, with a 50/50 chance, to receive defibrotide as well as standard treatment.
This study will recruit 150 participants in hospitals across Europe and North America.
Participation is expected to last approximately 6 months. There is a screening period to check eligibility, the treatment period while the participant undergoes HSCT, and a follow-up period after hospital discharge.
Study procedures include a physical exam, height, weight, review of medical history and medications. In addition, a serum pregnancy test (for females of child bearing potential), and blood tests will be completed. Bone marrow aspirates and biopsies will also be completed. Questionnaires asking about current health status will be administered.
Defibrotide is administered intravenously (a needle inserted into the vein or via a central line) as 2-hour infusions every 6 hours for a recommended minimum of 21 days, and no longer than 30 days after HSCT.
This study is sponsored by Jazz Pharmaceuticals, Inc..REC name
Yorkshire & The Humber - Leeds West Research Ethics Committee
REC reference
18/YH/0055
Date of REC Opinion
11 May 2018
REC opinion
Further Information Favourable Opinion