This study is an open-label, controlled, multicenter, international, Phase III, randomized study of transplantation of NiCord® versus transplantation of one or two unmanipulated, unrelated cord blood units in patients with acute lymphoblastic leukemia or acute myeloid leukemia, myelodysplastic syndrome, chronic myeloid leukemia or lymphoma, all with required disease features rendering them eligible for allogeneic transplantation.
Successful blood and marrow transplantation (BMT) requires the infusion of a sufficient number of hematopoietic stem/progenitor cells (HSPCs), capable of both homing to the bone marrow and regenerating a full array of hematopoietic cell lineages with early and late repopulating ability in a timely fashion. A major drawback of Umbilical Cord Blood (UCB) is the low stem cell dose available for transplantation, compared to mobilized peripheral blood (PB) or bone marrow. This low stem cell dose can compromise the chances of engraftment and contributes to delayed kinetics of neutrophil and platelet recovery, as well as other transplant outcomes. The aim of ex vivo expansion of cord blood is to provide a graft with sufficient numbers of cells that have rapid and robust in vivo neutrophil and platelet producing potential to enable successful transplantation. NiCord® is a stem/progenitor cell-based product composed of ex vivo expanded allogeneic cells from one entire unit of UCB. NiCord® utilizes the small molecule nicotinamide (NAM), as an epigenetic approach to inhibit differentiation and to increase the migration, bone marrow (BM) homing and engraftment efficiency of Hematopoietic Progenitor Cells (HPC) expanded in ex vivo cultures. The chief aim of the study is to compare the safety and efficacy of NiCord® single ex-vivo expanded cord blood unit transplantation to unmanipulated cord blood unit transplantation in patients with hematological malignancies following conditioning therapy.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
125
Cord blood unit
UCLA
Los Angeles, California, United States
City of Hope
Los Angeles, California, United States
Stanford University Cancer Institute
Palo Alto, California, United States
UC San Diego Moores Cancer Center
San Diego, California, United States
Children's Hospital Colorado
Denver, Colorado, United States
Time to Neutrophil Engraftment
The time to engraftment of neutrophils \>500/μl was defined as per Center for International Blood and Marrow Transplant Research (CIBMTR) standards, requiring donor chimerism for neutrophil engraftment.
Time frame: post-transplant up to 42 days
First Grade 2/3 Bacterial or Invasive Fungal Infections by 100 Days Following Transplantation
First Bacterial Infection Grades 2-3 or Invasive Fungal Infection by 100 Days following Transplantation for the Intent to Treat Population
Time frame: 100 days post-transplant
Days Alive and Out of Hospital in the First 100 Days Post-transplantation
Days alive and out of hospital in the first 100 Days post-transplantation for the Intent to Treat Population
Time frame: 100 days post-transplantation
Number of Participants With Platelet Engraftment by 42 Days Post-transplantation
Platelet engraftment defined as the first day of a minimum of three consecutive measurements on different days such that the patient has achieved a platelet count \> 20 × 10\^9/L with no platelet transfusions during the preceding seven days (counting day of engraftment as one of the preceding seven days) for the Intent to Treat Population
Time frame: 42 days post-transplantation
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Northwestern University
Evanston, Illinois, United States
Loyola University, Cardinal Bernardin Cancer Center
Maywood, Illinois, United States
University of Kansas Cancer Center
Westwood, Kansas, United States
The University of Maryland Medicine Center
Baltimore, Maryland, United States
Boston Children's Hospital
Boston, Massachusetts, United States
...and 42 more locations