Omidubicel is an investigational therapy for patients with high-risk hematologic malignancies.
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. Omidubicel is a stem/progenitor cell-based product composed of ex vivo expanded allogeneic cells from one entire unit of umbilical cord blood consisting of mature myeloid and lymphoid cells as follows: 1. Ex vivo expanded, umbilical cord blood-derived hematopoietic CD34+ progenitor cells (cultured fraction (CF)), containing a minimum of 8.0 × 10\^8 total viable cells of which a minimum of 8.7% is CD34+ cells and a minimum of 9.2 × 10\^7 CD34+ cells, and 2. the non-cultured cell fraction of the same Cord Blood Unit (CBU) (Non-cultured Fraction (NF)), containing a minimum of 4.0 × 10\^8 total viable cells with a minimum of 2.4 × 10\^7 CD3+ cells Omidubicel 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 overall study objectives are to provide access to omidubicel for transplantation in patients with hematological malignancies and to collect additional safety and efficacy data.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
36
hematopoietic stem cell transplant
UCLA
Los Angeles, California, United States
Stanford University Cancer Institute
Palo Alto, California, United States
Loyola University, Cardinal Bernardin Cancer Center
Maywood, Illinois, United States
University of Minnesota Masonic Cancer Center
Minneapolis, Minnesota, United States
Time From Transplant to Neutrophil Engraftment
Neutrophil engraftment was defined as achieving an absolute neutrophil count (ANC) greater than or equal to 0.5 x 10\^9/L on 3 consecutive measurements by Day 42 post-transplant inclusive. The first day of the three measurements was designated the day of neutrophil engraftment.
Time frame: by day 42 post-transplant inclusive
Cumulative Incidence of Neutrophil Engraftment
Death, second transplant, and relapse were competing risks at the time they occur if they occur prior to neutrophil engraftment, and no transplant was a competing risk at Day 0. If the patient failed to achieve neutrophil engraftment, they were considered to have a competing risk at Day 43.
Time frame: by day 42 post-transplant inclusive
Cumulative Incidence of Platelet Engraftment >20,000 Cells/uL
Time frame: By Day 42 and Day 180 post-transplant
Time to Platelet Engraftment >20,000 Cells/uL
Time to platelet engraftment \>20,000 cells/ul was defined as the number of days from transplant to the first day of a minimum of 3 consecutive measurements on different days in which the platelet count is 20,000 cells/ul or higher with no platelet transfusion within the previous 7 days (count day of engraftment as one of the preceding 7 days) was calculated. The first day of the three measurements was designated the day of platelet engraftment.
Time frame: By Day 730 post-transplant
Cumulative Incidence of Platelet Engraftment >50,000 Cells/uL
Time frame: By Day 42 and Day 180 post-transplant
Time to Platelet Engraftment >50,000 Cells/uL
Time to platelet engraftment \>50,000 cells/ul was defined as the number of days from transplant to the first day of a minimum of 3 consecutive measurements on different days in which the platelet count is 50,000 cells/ul or higher with no platelet transfusion within the previous 7 days (count day of engraftment as one of the preceding 7 days) was calculated. The first day of the three measurements was designated the day of platelet engraftment.
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Duke University Medical Center
Durham, North Carolina, United States
Oregon Health & Science University
Portland, Oregon, United States
Time frame: By Day 730 post-transplant
Non-relapse Mortality
Non-relapse mortality was defined as any death not preceded by relapse.
Time frame: By Day 180, Day 365 and Day 730 post-transplant
Overall Survival (OS)
OS probability was defined as the probability of participants remaining alive at specified time points following transplantation, estimated using Kaplan-Meier methods.
Time frame: By Day 180, Day 365 and Day 730 post-transplant
Disease Free Survival (DFS)
Disease-free survival was defined as the survival without disease relapse or death from any cause, whichever came first.
Time frame: By Day 365 and Day 730 post-transplant
Donor Chimerism
Patients considered to have donor chimerism when they had at least 95% donor chimerism
Time frame: By day 100 and Day 730 post-transplant
Secondary Graft Failure (SGF)
Time frame: By Day 730 post-transplant
Disease Relapse
Time frame: By Day 365 and Day 730 post-transplant
Cumulative Incidence of Acute GvHD Grade II-IV
Death, failure to achieve neutrophil engraftment, secondary graft failure, and relapse were considered competing events.
Time frame: By Day 100 post-transplant
Cumulative Incidence of aGvHD Grade III-IV
Death, failure to achieve neutrophil engraftment, secondary graft failure, and relapse were considered competing events.
Time frame: By Day 100 post-transplant
Cumulative Incidence of Chronic GvHD
Death, failure to achieve neutrophil engraftment, secondary graft failure, and relapse were considered competing events.
Time frame: By Day 180 and Day 730 post-transplant
Chronic GvHD-free Relapse-free Survival (cGRFS)
Chronic graft versus host disease-free, relapse-free survival (cGRFS) was defined as chronic GvHD, relapse, or death by any cause.
Time frame: By Day 365 and Day 730 post-transplant
GvHD-free Relapse-free Survival (GRFS)
Graft versus host disease-free, relapse-free survival (GRFS) was defined as acute GvHD Grade III-IV, chronic GvHD, relapse, or death by any cause
Time frame: By Day 365 and Day 730 post-transplant