Allogeneic Stem Cell Transplantation of NiCord®, Umbilical Cord Blood-derived Ex Vivo Expanded Stem and Progenitor Cells, in Adult Patients With Hematological Malignancies
Umbilical cord blood (UCB) is an alternative stem cell source for hematopoietic stem cell
transplantations (HSCT) and can be used for the treatment of various life-threatening
diseases, such as hematological malignancies or genetic blood disorders, in such cases where
a matched related stem cell donor is not available. However, the major drawback of using
this valuable stem cells source is the limited cell dose in a single cord blood unit (CBU),
which was shown to be associated with inadequate hematopoietic reconstitution and high risk
of transplant-related mortality. To improve outcomes and extend applicability of UCB
transplantation, one potential solution is ex vivo expansion of UCB-derived stem and
progenitor cells. NiCord® is a stem/progenitor cell based product composed of ex vivo
expanded allogeneic UCB cells. NiCord® is based on a novel technology for the ex vivo cell
expansion of cord blood derived hematopoietic progenitor cells. By increasing the number of
the short and long-term reconstitution progenitor cells transplanted, NiCord® has the
potential to enable the broader application of UCB transplantation, and improve the clinical
outcomes of UCB transplantation.
The study is designed as a multi center, single arm study, evaluating the safety and
efficacy of the transplantation of NiCord® to patients with hematological malignancies
following myeloablative therapy.
Total study duration is approximately 400 days from the signing of informed consent to the
last visit one year following transplantation
The overall study objective is to evaluate the safety and efficacy of NiCord®: single
ex-vivo expanded cord blood unit transplantation in patients with hematological malignancies
following myeloablative therapy as follows:
The main study objectives are to assess the cumulative incidence of patients with
NiCord®-derived neutrophil engraftment at 42 days following transplantation and to assess
the incidence of secondary graft failure at 180 days following transplantation of NiCord®
Ten evaluable patients recruited for the study should be 18-65 years of age, up to a maximum
of 15 treated patients.
Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Assess the cumulative incidence of patients with NiCord®-derived neutrophil engraftment at 42 days following transplantation.
Mitchell Horwitz, MD
United States: Food and Drug Administration
|Duke University Medical Center||Durham, North Carolina 27710|