Of the eight patients who have completed treatment in the study, two experienced grade IV acute graft-versus-host-disease, or aGVHD, a known potential side effect in transplantation. Both patients have recovered from the aGVHD, however, under the study protocol, the occurrence of two cases calls for a suspension of enrollment in the trial.
The company will review these cases and relevant data with the FDA and the institutional review boards for the clinical trial sites as part of obtaining their agreement to continue enrollment.
“Patient safety is our number one priority,” stated Marc Beer, president and chief executive officer of ViaCell. “Acute GVHD is a known side effect in transplantation. The protocol’s mandated stopping point for two cases of grade IV aGVHD is intended as a standard type of safeguard for phase I studies to allow us, along with the FDA and the sites, to assess the data. We are fully committed to working diligently with the FDA to find a path forward.”
Acute GVHD is a potential and common side effect of transplants associated with the use of bone marrow, peripheral blood or cord blood from a different person. In aGVHD, the transplanted immune cells recognize the tissue as not being from the recipient’s body.