Trial Outcomes & Findings for Stem Cell Transplantation With NiCord® (Omidubicel) vs Standard UCB in Patients With Leukemia, Lymphoma, and MDS (NCT NCT02730299)

Last Updated: 2026-02-25

Results Overview

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.

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

125 participants

Primary outcome timeframe

post-transplant up to 42 days

Results posted on

2026-02-25

Participant Flow

125 patients were screened, met inclusion criteria and were randomized to treatment from 33 transplant centers worldwide. First patient was consented on 20th December 2016 and the last patient consented on 27th December 2019.

Participant milestones

Participant milestones
Measure	NiCord® (Omidubicel) NiCord® is a cryopreserved stem/progenitor cell based product comprised of: 1. ex vivo expanded, umbilical cord blood-derived hematopoietic CD34+ progenitor cells (NiCord® cultured fraction (CF)) 2. the non-cultured cell fraction of the same Cord Blood Unit (CBU) (NiCord® Non-cultured Fraction (NF)) consisting of mature myeloid and lymphoid cells. Both fractions, i.e. NiCord® CF and NiCord® NF, will be kept frozen until they are thawed and infused on the day of transplantation. NiCord® (omidubicel)	Unmanipulated CBU(s) Unmanipulated cord blood unit(s)
Overall Study STARTED	62	63
Overall Study COMPLETED	62	63
Overall Study NOT COMPLETED	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Stem Cell Transplantation With NiCord® (Omidubicel) vs Standard UCB in Patients With Leukemia, Lymphoma, and MDS

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	NiCord® (Omidubicel) n=62 Participants NiCord® is a cryopreserved stem/progenitor cell based product comprised of: 1. ex vivo expanded, umbilical cord blood-derived hematopoietic CD34+ progenitor cells (NiCord® cultured fraction (CF)) 2. the non-cultured cell fraction of the same Cord Blood Unit (CBU) (NiCord® Non-cultured Fraction (NF)) consisting of mature myeloid and lymphoid cells. Both fractions, i.e. NiCord® CF and NiCord® NF, will be kept frozen until they are thawed and infused on the day of transplantation. NiCord® (omidubicel)	Unmanipulated CBU(s) n=63 Participants Unmanipulated cord blood unit(s)	Total n=125 Participants Total of all reporting groups
Race (NIH/OMB) White	35 Participants n=24 Participants	37 Participants n=20 Participants	72 Participants n=40 Participants
Age, Categorical <=18 years	8 Participants n=24 Participants	6 Participants n=20 Participants	14 Participants n=40 Participants
Age, Categorical Between 18 and 65 years	54 Participants n=24 Participants	57 Participants n=20 Participants	111 Participants n=40 Participants
Age, Categorical >=65 years	0 Participants n=24 Participants	0 Participants n=20 Participants	0 Participants n=40 Participants
Sex: Female, Male Female	30 Participants n=24 Participants	23 Participants n=20 Participants	53 Participants n=40 Participants
Sex: Female, Male Male	32 Participants n=24 Participants	40 Participants n=20 Participants	72 Participants n=40 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	10 Participants n=24 Participants	6 Participants n=20 Participants	16 Participants n=40 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	46 Participants n=24 Participants	52 Participants n=20 Participants	98 Participants n=40 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	6 Participants n=24 Participants	5 Participants n=20 Participants	11 Participants n=40 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=24 Participants	0 Participants n=20 Participants	0 Participants n=40 Participants
Race (NIH/OMB) Asian	7 Participants n=24 Participants	10 Participants n=20 Participants	17 Participants n=40 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=24 Participants	1 Participants n=20 Participants	1 Participants n=40 Participants
Race (NIH/OMB) Black or African American	11 Participants n=24 Participants	9 Participants n=20 Participants	20 Participants n=40 Participants
Race (NIH/OMB) More than one race	3 Participants n=24 Participants	1 Participants n=20 Participants	4 Participants n=40 Participants
Race (NIH/OMB) Unknown or Not Reported	6 Participants n=24 Participants	5 Participants n=20 Participants	11 Participants n=40 Participants
Region of Enrollment Netherlands	3 participants n=24 Participants	3 participants n=20 Participants	6 participants n=40 Participants
Region of Enrollment Singapore	4 participants n=24 Participants	5 participants n=20 Participants	9 participants n=40 Participants
Region of Enrollment United States	42 participants n=24 Participants	45 participants n=20 Participants	87 participants n=40 Participants
Region of Enrollment Brazil	3 participants n=24 Participants	1 participants n=20 Participants	4 participants n=40 Participants
Region of Enrollment United Kingdom	1 participants n=24 Participants	1 participants n=20 Participants	2 participants n=40 Participants
Region of Enrollment Israel	1 participants n=24 Participants	1 participants n=20 Participants	2 participants n=40 Participants
Region of Enrollment Spain	8 participants n=24 Participants	7 participants n=20 Participants	15 participants n=40 Participants

PRIMARY outcome

Timeframe: post-transplant up to 42 days

Population: intent to treat

Outcome measures

Outcome measures
Measure	NiCord® (Omidubicel) n=62 Participants NiCord® (omidubicel) is a cryopreserved stem/progenitor cell based product comprised of: 1. ex vivo expanded, umbilical cord blood-derived hematopoietic CD34+ progenitor cells (cultured fraction (CF) 2. the non-cultured cell fraction of the same Cord Blood Unit (CBU) Non-cultured Fraction (NF) consisting of mature myeloid and lymphoid cells.	Unmanipulated Cord Blood Unit(s) n=63 Participants Cord blood unit: Cord blood unit 1-2 units
Time to Neutrophil Engraftment	12 days Interval 10.0 to 14.0	22 days Interval 19.0 to 25.0

SECONDARY outcome

Timeframe: 100 days post-transplant

Population: Intent to Treat Population

First Bacterial Infection Grades 2-3 or Invasive Fungal Infection by 100 Days following Transplantation for the Intent to Treat Population

Outcome measures

Outcome measures
Measure	NiCord® (Omidubicel) n=62 Participants NiCord® (omidubicel) is a cryopreserved stem/progenitor cell based product comprised of: 1. ex vivo expanded, umbilical cord blood-derived hematopoietic CD34+ progenitor cells (cultured fraction (CF) 2. the non-cultured cell fraction of the same Cord Blood Unit (CBU) Non-cultured Fraction (NF) consisting of mature myeloid and lymphoid cells.	Unmanipulated Cord Blood Unit(s) n=63 Participants Cord blood unit: Cord blood unit 1-2 units
First Grade 2/3 Bacterial or Invasive Fungal Infections by 100 Days Following Transplantation	24 Participants	38 Participants

SECONDARY outcome

Timeframe: 100 days post-transplantation

Population: Intent to Treat Population

Days alive and out of hospital in the first 100 Days post-transplantation for the Intent to Treat Population

Outcome measures

Outcome measures
Measure	NiCord® (Omidubicel) n=62 Participants NiCord® (omidubicel) is a cryopreserved stem/progenitor cell based product comprised of: 1. ex vivo expanded, umbilical cord blood-derived hematopoietic CD34+ progenitor cells (cultured fraction (CF) 2. the non-cultured cell fraction of the same Cord Blood Unit (CBU) Non-cultured Fraction (NF) consisting of mature myeloid and lymphoid cells.	Unmanipulated Cord Blood Unit(s) n=63 Participants Cord blood unit: Cord blood unit 1-2 units
Days Alive and Out of Hospital in the First 100 Days Post-transplantation	60.5 Days Interval 0.0 to 89.0	48 Days Interval 0.0 to 79.0

SECONDARY outcome

Timeframe: 42 days post-transplantation

Population: Intent to Treat Population

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

Outcome measures

Outcome measures
Measure	NiCord® (Omidubicel) n=62 Participants NiCord® (omidubicel) is a cryopreserved stem/progenitor cell based product comprised of: 1. ex vivo expanded, umbilical cord blood-derived hematopoietic CD34+ progenitor cells (cultured fraction (CF) 2. the non-cultured cell fraction of the same Cord Blood Unit (CBU) Non-cultured Fraction (NF) consisting of mature myeloid and lymphoid cells.	Unmanipulated Cord Blood Unit(s) n=63 Participants Cord blood unit: Cord blood unit 1-2 units
Number of Participants With Platelet Engraftment by 42 Days Post-transplantation	34 Participants	22 Participants

OTHER_PRE_SPECIFIED outcome

Timeframe: By 5 years post-transplant

Population: Patients who were transplanted, alive at Year 1 post-transplant (end of main study) and provided consent to participate in the optional long-term follow-up sub-study

Kaplan Meier probability of donor chimerism \<95%

Outcome measures

Outcome measures
Measure	NiCord® (Omidubicel) n=39 Participants NiCord® (omidubicel) is a cryopreserved stem/progenitor cell based product comprised of: 1. ex vivo expanded, umbilical cord blood-derived hematopoietic CD34+ progenitor cells (cultured fraction (CF) 2. the non-cultured cell fraction of the same Cord Blood Unit (CBU) Non-cultured Fraction (NF) consisting of mature myeloid and lymphoid cells.	Unmanipulated Cord Blood Unit(s) n=32 Participants Cord blood unit: Cord blood unit 1-2 units
LTF Chimerism	0.97 Kaplan Meier probability	0.91 Kaplan Meier probability

OTHER_PRE_SPECIFIED outcome

Timeframe: By 5 years post-transplant

Population: Patients who were transplanted, alive at Year 1 post-transplant (end of main study) and provided consent to participate in the optional long-term follow-up sub-study

Kaplan Meier probability of overall survival

Outcome measures

Outcome measures
Measure	NiCord® (Omidubicel) n=39 Participants NiCord® (omidubicel) is a cryopreserved stem/progenitor cell based product comprised of: 1. ex vivo expanded, umbilical cord blood-derived hematopoietic CD34+ progenitor cells (cultured fraction (CF) 2. the non-cultured cell fraction of the same Cord Blood Unit (CBU) Non-cultured Fraction (NF) consisting of mature myeloid and lymphoid cells.	Unmanipulated Cord Blood Unit(s) n=32 Participants Cord blood unit: Cord blood unit 1-2 units
LTF Overall Survival	0.82 KM probability	0.86 KM probability

OTHER_PRE_SPECIFIED outcome

Timeframe: By 5 years post-transplant

Population: Patients who were transplanted, alive at Year 1 post-transplant (end of main study) and provided consent to participate in the optional long-term follow-up sub-study

Number of patients with relapse during the long-term follow-up period

Outcome measures

Outcome measures
Measure	NiCord® (Omidubicel) n=39 Participants NiCord® (omidubicel) is a cryopreserved stem/progenitor cell based product comprised of: 1. ex vivo expanded, umbilical cord blood-derived hematopoietic CD34+ progenitor cells (cultured fraction (CF) 2. the non-cultured cell fraction of the same Cord Blood Unit (CBU) Non-cultured Fraction (NF) consisting of mature myeloid and lymphoid cells.	Unmanipulated Cord Blood Unit(s) n=32 Participants Cord blood unit: Cord blood unit 1-2 units
LTF Disease Progression/Relapse	2 Patients with relapse	1 Patients with relapse

OTHER_PRE_SPECIFIED outcome

Timeframe: By 5 years post-transplant

Population: Patients who were transplanted, alive at Year 1 post-transplant (end of main study) and provided consent to participate in the optional long-term follow-up sub-study

Cumulative Incidence of Chronic GvHD

Outcome measures

Outcome measures
Measure	NiCord® (Omidubicel) n=39 Participants NiCord® (omidubicel) is a cryopreserved stem/progenitor cell based product comprised of: 1. ex vivo expanded, umbilical cord blood-derived hematopoietic CD34+ progenitor cells (cultured fraction (CF) 2. the non-cultured cell fraction of the same Cord Blood Unit (CBU) Non-cultured Fraction (NF) consisting of mature myeloid and lymphoid cells.	Unmanipulated Cord Blood Unit(s) n=32 Participants Cord blood unit: Cord blood unit 1-2 units
LTF Chronic GvHD	0.56 Proportion	0.44 Proportion

Adverse Events

Omidubicel

Serious events: 47 serious events

Other events: 52 other events

Deaths: 19 deaths

Unmanipulated CBU(s)

Serious events: 51 serious events

Other events: 56 other events

Deaths: 24 deaths

Serious adverse events

Other adverse events

Additional Information

Associate Director, Clinical Operations

Gamida Cell

Phone: +972-073-2064774

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee Sponsor can review/comment at least 30 days before publication submission. At Sponsor request, site will withhold publication another 60 days to allow for patent application or other measures Sponsor deems appropriate to establish \& preserve its proprietary rights. Results publication made as part of those obtained by all sites performing protocol. Site free to publish based on its study results after publication of multi-center results or one year after completion/termination of site study.
Publication restrictions are in place

Restriction type: OTHER

Measure	Omidubicel n=52 participants at risk omidubicel is a cryopreserved stem/progenitor cell based product comprised of: 1. ex vivo expanded, umbilical cord blood-derived hematopoietic CD34+ progenitor cells (cultured fraction (CF) 2. the non-cultured cell fraction of the same Cord Blood Unit (CBU) Non-cultured Fraction (NF) consisting of mature myeloid and lymphoid cells.	Unmanipulated CBU(s) n=56 participants at risk Unmanipulated cord blood unit(s)
Blood and lymphatic system disorders Febrile Neutropenia	5.8% 3/52 • Number of events 3 • Up to 5 Years post-transplant for All-cause mortality Up to 1 year post-transplant for all other safety events reported Safety population (N=108). Serious Adverse Events (SAEs) \& Adverse Events (AEs) relate to safety population (SP) (N=108).	0.00% 0/56 • Up to 5 Years post-transplant for All-cause mortality Up to 1 year post-transplant for all other safety events reported Safety population (N=108). Serious Adverse Events (SAEs) \& Adverse Events (AEs) relate to safety population (SP) (N=108).
Blood and lymphatic system disorders Thrombotic microangiopathy	1.9% 1/52 • Number of events 1 • Up to 5 Years post-transplant for All-cause mortality Up to 1 year post-transplant for all other safety events reported Safety population (N=108). Serious Adverse Events (SAEs) \& Adverse Events (AEs) relate to safety population (SP) (N=108).	7.1% 4/56 • Number of events 4 • Up to 5 Years post-transplant for All-cause mortality Up to 1 year post-transplant for all other safety events reported Safety population (N=108). Serious Adverse Events (SAEs) \& Adverse Events (AEs) relate to safety population (SP) (N=108).
General disorders Multiple organ dysfunction syndrome	0.00% 0/52 • Up to 5 Years post-transplant for All-cause mortality Up to 1 year post-transplant for all other safety events reported Safety population (N=108). Serious Adverse Events (SAEs) \& Adverse Events (AEs) relate to safety population (SP) (N=108).	3.6% 2/56 • Number of events 2 • Up to 5 Years post-transplant for All-cause mortality Up to 1 year post-transplant for all other safety events reported Safety population (N=108). Serious Adverse Events (SAEs) \& Adverse Events (AEs) relate to safety population (SP) (N=108).
General disorders Pyrexia	9.6% 5/52 • Number of events 5 • Up to 5 Years post-transplant for All-cause mortality Up to 1 year post-transplant for all other safety events reported Safety population (N=108). Serious Adverse Events (SAEs) \& Adverse Events (AEs) relate to safety population (SP) (N=108).	5.4% 3/56 • Number of events 3 • Up to 5 Years post-transplant for All-cause mortality Up to 1 year post-transplant for all other safety events reported Safety population (N=108). Serious Adverse Events (SAEs) \& Adverse Events (AEs) relate to safety population (SP) (N=108).
Hepatobiliary disorders Veno-occlusive liver disease	3.8% 2/52 • Number of events 2 • Up to 5 Years post-transplant for All-cause mortality Up to 1 year post-transplant for all other safety events reported Safety population (N=108). Serious Adverse Events (SAEs) \& Adverse Events (AEs) relate to safety population (SP) (N=108).	7.1% 4/56 • Number of events 4 • Up to 5 Years post-transplant for All-cause mortality Up to 1 year post-transplant for all other safety events reported Safety population (N=108). Serious Adverse Events (SAEs) \& Adverse Events (AEs) relate to safety population (SP) (N=108).
Immune system disorders Graft-versus-host disease	32.7% 17/52 • Number of events 18 • Up to 5 Years post-transplant for All-cause mortality Up to 1 year post-transplant for all other safety events reported Safety population (N=108). Serious Adverse Events (SAEs) \& Adverse Events (AEs) relate to safety population (SP) (N=108).	25.0% 14/56 • Number of events 15 • Up to 5 Years post-transplant for All-cause mortality Up to 1 year post-transplant for all other safety events reported Safety population (N=108). Serious Adverse Events (SAEs) \& Adverse Events (AEs) relate to safety population (SP) (N=108).
Injury, poisoning and procedural complications Femoral Neck Failure	3.8% 2/52 • Number of events 2 • Up to 5 Years post-transplant for All-cause mortality Up to 1 year post-transplant for all other safety events reported Safety population (N=108). Serious Adverse Events (SAEs) \& Adverse Events (AEs) relate to safety population (SP) (N=108).	0.00% 0/56 • Up to 5 Years post-transplant for All-cause mortality Up to 1 year post-transplant for all other safety events reported Safety population (N=108). Serious Adverse Events (SAEs) \& Adverse Events (AEs) relate to safety population (SP) (N=108).
Injury, poisoning and procedural complications Transplant Failure	5.8% 3/52 • Number of events 3 • Up to 5 Years post-transplant for All-cause mortality Up to 1 year post-transplant for all other safety events reported Safety population (N=108). Serious Adverse Events (SAEs) \& Adverse Events (AEs) relate to safety population (SP) (N=108).	8.9% 5/56 • Number of events 5 • Up to 5 Years post-transplant for All-cause mortality Up to 1 year post-transplant for all other safety events reported Safety population (N=108). Serious Adverse Events (SAEs) \& Adverse Events (AEs) relate to safety population (SP) (N=108).
Nervous system disorders CNS events	7.7% 4/52 • Number of events 4 • Up to 5 Years post-transplant for All-cause mortality Up to 1 year post-transplant for all other safety events reported Safety population (N=108). Serious Adverse Events (SAEs) \& Adverse Events (AEs) relate to safety population (SP) (N=108).	3.6% 2/56 • Number of events 2 • Up to 5 Years post-transplant for All-cause mortality Up to 1 year post-transplant for all other safety events reported Safety population (N=108). Serious Adverse Events (SAEs) \& Adverse Events (AEs) relate to safety population (SP) (N=108).
Renal and urinary disorders Renal disorders	13.5% 7/52 • Number of events 7 • Up to 5 Years post-transplant for All-cause mortality Up to 1 year post-transplant for all other safety events reported Safety population (N=108). Serious Adverse Events (SAEs) \& Adverse Events (AEs) relate to safety population (SP) (N=108).	8.9% 5/56 • Number of events 5 • Up to 5 Years post-transplant for All-cause mortality Up to 1 year post-transplant for all other safety events reported Safety population (N=108). Serious Adverse Events (SAEs) \& Adverse Events (AEs) relate to safety population (SP) (N=108).
Respiratory, thoracic and mediastinal disorders Respiratory disorders	15.4% 8/52 • Number of events 8 • Up to 5 Years post-transplant for All-cause mortality Up to 1 year post-transplant for all other safety events reported Safety population (N=108). Serious Adverse Events (SAEs) \& Adverse Events (AEs) relate to safety population (SP) (N=108).	26.8% 15/56 • Number of events 15 • Up to 5 Years post-transplant for All-cause mortality Up to 1 year post-transplant for all other safety events reported Safety population (N=108). Serious Adverse Events (SAEs) \& Adverse Events (AEs) relate to safety population (SP) (N=108).
Gastrointestinal disorders GI Disorders	11.5% 6/52 • Number of events 6 • Up to 5 Years post-transplant for All-cause mortality Up to 1 year post-transplant for all other safety events reported Safety population (N=108). Serious Adverse Events (SAEs) \& Adverse Events (AEs) relate to safety population (SP) (N=108).	23.2% 13/56 • Number of events 13 • Up to 5 Years post-transplant for All-cause mortality Up to 1 year post-transplant for all other safety events reported Safety population (N=108). Serious Adverse Events (SAEs) \& Adverse Events (AEs) relate to safety population (SP) (N=108).
Infections and infestations Infections	50.0% 26/52 • Number of events 29 • Up to 5 Years post-transplant for All-cause mortality Up to 1 year post-transplant for all other safety events reported Safety population (N=108). Serious Adverse Events (SAEs) \& Adverse Events (AEs) relate to safety population (SP) (N=108).	50.0% 28/56 • Number of events 31 • Up to 5 Years post-transplant for All-cause mortality Up to 1 year post-transplant for all other safety events reported Safety population (N=108). Serious Adverse Events (SAEs) \& Adverse Events (AEs) relate to safety population (SP) (N=108).
Metabolism and nutrition disorders Metabolic disorders	5.8% 3/52 • Number of events 3 • Up to 5 Years post-transplant for All-cause mortality Up to 1 year post-transplant for all other safety events reported Safety population (N=108). Serious Adverse Events (SAEs) \& Adverse Events (AEs) relate to safety population (SP) (N=108).	5.4% 3/56 • Number of events 3 • Up to 5 Years post-transplant for All-cause mortality Up to 1 year post-transplant for all other safety events reported Safety population (N=108). Serious Adverse Events (SAEs) \& Adverse Events (AEs) relate to safety population (SP) (N=108).
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Hematologic malignancy	15.4% 8/52 • Number of events 8 • Up to 5 Years post-transplant for All-cause mortality Up to 1 year post-transplant for all other safety events reported Safety population (N=108). Serious Adverse Events (SAEs) \& Adverse Events (AEs) relate to safety population (SP) (N=108).	10.7% 6/56 • Number of events 6 • Up to 5 Years post-transplant for All-cause mortality Up to 1 year post-transplant for all other safety events reported Safety population (N=108). Serious Adverse Events (SAEs) \& Adverse Events (AEs) relate to safety population (SP) (N=108).