Jazz Pharmaceuticals has enrolled the first patient in a Phase II clinical trial to assess the efficacy and safety of defibrotide for treatment of an acute graft-versus-host-disease (aGvHD) in adult and pediatric patients after allogeneic hematopoietic stem cell transplant (HSCT).
GvHD is a life-threatening complication of HSCT and is considered to be the most frequent cause of morbidity and non-relapse mortality following allogeneic HSCT.
The prospective, randomised, open-label study will be conducted across around 60 medical centres in the US, Canada, and European Union.
As part of the study, defibrotide will be added to standard of care immunoprophylaxis for the prevention of aGvHD, and will be compared to the standard of care alone.
Around 150 adult and pediatric patients who have undergone allogeneic HSCT from an unrelated donor are expected to be enrolled in the study.
The trial’s primary endpoint is cumulative incidence of Grade B-D aGvHD by day +100 post-allogeneic HSCT.
Jazz Pharmaceuticals chief medical officer Karen Smith said: said: “Potential complications of hematopoietic stem cell transplant such as acute graft-versus-host disease can be life-threatening and even fatal.
“Despite the use of current immunoprophylaxis strategies, graft-versus-host disease remains a leading cause of non-relapse mortality after allogeneic stem cell transplant.”
The Phase II trial complements Jazz’s ongoing Phase III clinical trial of defibrotide to treat hepatic veno-occlusive disease (VOD) in high-risk adult and pediatric patients undergoing hematopoietic stem cell transplant.
VOD is a rare complication of HSCT that occurs in nearly 9%-14% of HSCT patients.