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    Clinical Trial Results:
    A Multicenter, Randomized, Phase III Registration Trial of Transplantation of NiCord®, Ex Vivo Expanded, Umbilical Cord Blood-derived, Stem and Progenitor Cells, versus Unmanipulated Umbilical Cord Blood for Patients with Hematological Malignancies

    Summary
    EudraCT number
    		 2016-000704-28
    Trial protocol
    		 ES   NL   GB   IT   PT   FR  
    Global end of trial date
    07 Feb 2025

    Results information
    Results version number
    		 v2(current)
    This version publication date
    03 May 2025
    First version publication date
    30 Apr 2022
    Other versions
    		 v1
    Version creation reason
    • New data added to full data set
    Trial status update
    Summary report(s)
    		 Omidubicel Phase 3 Case Study Report 23JAN2022

    Trial information

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    Trial identification
    Sponsor protocol code
    GCP#05.01.020
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT02730299
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Gamida Cell Ltd.
    Sponsor organisation address
    5 Nahum Hatzfedi Street, Jerusalem, Israel, 91240
    Public contact
    Clinical Department, Gamida Cell Ltd, 972 26595666, stuart@gamida-cell.com
    Scientific contact
    Clinical Department, Gamida Cell Ltd, 023730649 26595666, stuart@gamida-cell.com
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    Yes
    EMA paediatric investigation plan number(s)
    		 EMEA-001913-PIP02-18
    Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Results analysis stage
    Analysis stage
    Interim
    Date of interim/final analysis
    29 Apr 2021
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    07 Feb 2025
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    The overall study objective 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
    Protection of trial subjects
    Study records that identify subjects are kept confidential as required by national and state law. National and State Privacy Regulations provide safeguards for privacy, security, and authorized access. Except when required by law, subjects are not identified by name, address, telephone number, or any other direct personal identifier in study records disclosed outside of hospitals / institutions. Subjects are assigned a unique study number. The information linking subjects’ name to the study number is stored in a database on a secure server. No identifiable subject information will be given to researchers, nor will it be published or presented at scientific meetings.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    20 Dec 2016
    Long term follow-up planned
    Yes
    Long term follow-up rationale
    		 Safety, Efficacy
    Long term follow-up duration
    		 4 Years
    Independent data monitoring committee (IDMC) involvement?
    Yes
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    Netherlands: 6
    Country: Number of subjects enrolled
    Spain: 15
    Country: Number of subjects enrolled
    United Kingdom: 2
    Country: Number of subjects enrolled
    United States: 87
    Country: Number of subjects enrolled
    Singapore: 9
    Country: Number of subjects enrolled
    Brazil: 4
    Country: Number of subjects enrolled
    Israel: 2
    Worldwide total number of subjects
    125
    EEA total number of subjects
    21
    Number of subjects enrolled per age group
    In utero
    0
    Preterm newborn - gestational age < 37 wk
    0
    Newborns (0-27 days)
    0
    Infants and toddlers (28 days-23 months)
    0
    Children (2-11 years)
    0
    Adolescents (12-17 years)
    14
    Adults (18-64 years)
    111
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 125 patients were randomized at 33 centers in 7 countries, 87 (70%) patients were enrolled in United States, 15 (12%) from Spain, 9 (7%) from Singapore, and six (5%) from the Netherlands. All other countries contributed <5% of patients each. Patient characteristics was well balanced for age, disease, disease risk and specific site supportive care.

    Pre-assignment
    Screening details
    		 Subjects aged 12-65 years with a diagnosis of hematological malignancy and are candidates for unrelated cord blood (CB) transplantation with qualifying human leukocyte antigen (HLA)-matched unmanipulated CBUs with sufficient pre-cryopreserved total nucleated cell count and dose, and CD34+ cell dose, were included in the study.

    Period 1
    Period 1 title
    Arms > Overall Trial (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Randomised - controlled
    Blinding used
    		 Not blinded
    Blinding implementation details
    Sponsor maintained blinding of patient treatment assignment (including aggregate patient information) to minimize bias. Data Management Committee received unblinded reports and made recommendations to CRO for distribution to sponsor and clinical sites as requested. Selected CRO staff, all site research personnel were unblinded so as to evaluate safety and efficacy outcomes, and patients also knew their treatment assignments.

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Omidubicel
    Arm description
    		 Omidubicel (NiCord®) is a cryopreserved stem/progenitor cell based product comprised of: 1. ex vivo expanded, umbilical cord blood-derived hematopoietic CD34+ progenitor cells (omidubicel cultured fraction (CF)) 2. the non-cultured cell fraction of the same Cord Blood Unit (CBU) (omidubicel Non-cultured Fraction (NF)) consisting of mature myeloid and lymphoid cells. Both fractions, i.e. omidubicel CF and NiCord® NF, will be kept frozen until they are thawed and infused on the day of transplantation.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Omidubicel
    Investigational medicinal product code
    Other name
    NiCord
    Pharmaceutical forms
    Dispersion for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Both fractions, omidubicel Cultured Fraction (CF) and omidubicel Non-cultured Fraction (NF), will be kept frozen until they are thawed and infused on the day of transplantation. The final volume of omidubicel CF after thawing and reconstitution with a prepared Infusion Solution (IS) is 100 ml. The final volume of omidubicel NF after thawing and reconstitution with IS is 50 ml. omidubicel CF is infused first, followed immediately (up to 1 hour) by omidubicel NF and infused via the patient's central venous catheter. The infusion is given by gravity without additional support. Infusion of omidubicel CF and omidubicel NF target rate of 5 cc/kg/hr with a maximal rate of 10 cc/kg/hr.

    Arm title
    		 Unmanipulated Cord Blood Transplant
    Arm description
    		 Unmanipulated cord blood unit(s)
    Arm type
    		 No intervention

    Investigational medicinal product name
    No investigational medicinal product assigned in this arm
    Number of subjects in period 1
    		Omidubicel Unmanipulated Cord Blood Transplant
    Started
    62
    63
    Completed
    59
    58
    Not completed
    3
    5
         Physician decision
    3
    3
         Consent withdrawn by subject
    -
    2

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Omidubicel
    Reporting group description
    		 Omidubicel (NiCord®) is a cryopreserved stem/progenitor cell based product comprised of: 1. ex vivo expanded, umbilical cord blood-derived hematopoietic CD34+ progenitor cells (omidubicel cultured fraction (CF)) 2. the non-cultured cell fraction of the same Cord Blood Unit (CBU) (omidubicel Non-cultured Fraction (NF)) consisting of mature myeloid and lymphoid cells. Both fractions, i.e. omidubicel CF and NiCord® NF, will be kept frozen until they are thawed and infused on the day of transplantation.

    Reporting group title
    Unmanipulated Cord Blood Transplant
    Reporting group description
    		 Unmanipulated cord blood unit(s)

    Reporting group values
    		 Omidubicel Unmanipulated Cord Blood Transplant Total
    Number of subjects
    		 62 63 125
    Age categorical
    Subjects must have been 12-65 years of age at the time of randomization
    Units: Subjects
        In utero
    		 0
        Preterm newborn infants (gestational age < 37 wks)
    		 0
        Newborns (0-27 days)
    		 0
        Infants and toddlers (28 days-23 months)
    		 0
        Children (2-11 years)
    		 0
        Adolescents (12-17 years)
    		 0
        Adults (18-64 years)
    		 0
        From 65-84 years
    		 0
        85 years and over
    		 0
    Age continuous
    Subjects must have been 12-65 years of age at the time of randomization
    Units: years
        median (full range (min-max))
    		 40 (13 to 62) 43 (13 to 65) -
    Gender categorical
    Units: Subjects
        Female
    		 30 23 53
        Male
    		 32 40 72

    End points

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    End points reporting groups
    Reporting group title
    Omidubicel
    Reporting group description
    		 Omidubicel (NiCord®) is a cryopreserved stem/progenitor cell based product comprised of: 1. ex vivo expanded, umbilical cord blood-derived hematopoietic CD34+ progenitor cells (omidubicel cultured fraction (CF)) 2. the non-cultured cell fraction of the same Cord Blood Unit (CBU) (omidubicel Non-cultured Fraction (NF)) consisting of mature myeloid and lymphoid cells. Both fractions, i.e. omidubicel CF and NiCord® NF, will be kept frozen until they are thawed and infused on the day of transplantation.

    Reporting group title
    Unmanipulated Cord Blood Transplant
    Reporting group description
    		 Unmanipulated cord blood unit(s)

    Primary: Time to Neutrophil Engraftment

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    End point title
    		 Time to Neutrophil Engraftment
    End point description
    The time to engraftment of neutrophils >500/μl was defined as per CIBMTR standards, requiring donor chimerism for neutrophil engraftment.
    End point type
    Primary
    End point timeframe
    Up to 42 days post-transplantation
    End point values
    		 Omidubicel Unmanipulated Cord Blood Transplant
    Number of subjects analysed
    62 [1]
    63 [2]
    Units: Days
        days
    12
    22
    Notes
    [1] - The number of patients randomized to the NiCord Intent to Treat group was 62
    [2] - The number of patients randomized to the Unmanipulated CB Transplant Intent to Treat group was 63
    Statistical analysis title
    		 Time to neutrophil engraftment following transplan
    Statistical analysis description
    Time to neutrophil engraftment (NE) following transplantation, defined as achieving an ANC ≥ 0.5 ×109/L on 3 consecutive measurements on different days with subsequent donor chimerism (≤10% host cells by peripheral blood (PB) chimerism or BM chimerism if PB chimerism not available). Moreover, the day of NE was designated as the first of the three consecutive measurements and must have occurred on or before 42 days post-transplantation and prior to infusion of any additional stem cell product.
    Comparison groups
    Omidubicel v Unmanipulated Cord Blood Transplant
    Number of subjects included in analysis
    125
    Analysis specification
    Pre-specified
    Analysis type
    		 other [3]
    P-value
    		 ≤ 0.001
    Method
    		 Wilcoxon (Mann-Whitney)
    Parameter type
    		 Hazard ratio (HR)
    Confidence interval
    Notes
    [3] - The primary endpoint was time from transplant to neutrophil engraftment. The primary analysis for comparing time to engraftment between the two treatment groups was based on the Mann-Whitney test statistic. This test was shown to be equivalent to using a Gehan-Wilcoxon alternative in a time-to-event analysis with competing risks.

    Secondary: Incidence of Grade 2/3 bacterial or invasive fungal infections

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    End point title
    		 Incidence of Grade 2/3 bacterial or invasive fungal infections
    End point description
    Incidence of First Bacterial Infection Grades 2-3 or Invasive Fungal Infection by 100 Days following Transplantation for the Intent to Treat Population
    End point type
    Secondary
    End point timeframe
    By 100 days post-transplant
    End point values
    		 Omidubicel Unmanipulated Cord Blood Transplant
    Number of subjects analysed
    62 [4]
    63 [5]
    Units: Subjects
        Subjects
    24
    38
    Notes
    [4] - The number of patients randomized to the NiCord Intent to Treat group was 62
    [5] - The number of patients randomized to the Unmanipulated CB Transplant Intent to Treat group was 63
    No statistical analyses for this end point

    Secondary: Days alive and out of hospital

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    End point title
    		 Days alive and out of hospital
    End point description
    Days alive and out of hospital in the first 100 Days post-transplantation for the Intent to Treat Population
    End point type
    Secondary
    End point timeframe
    The first 100 Days post-transplantation
    End point values
    		 Omidubicel Unmanipulated Cord Blood Transplant
    Number of subjects analysed
    62 [6]
    63 [7]
    Units: Days
    median (full range (min-max))
        Days
    60.5 (0.1 to 89.0)
    48.0 (0.1 to 79.0)
    Notes
    [6] - The number of patients randomized to the NiCord Intent to Treat group was 62
    [7] - The number of patients randomized to the Unmanipulated CB Transplant Intent to Treat group was 63
    No statistical analyses for this end point

    Secondary: Platelet Engraftment by 42 days following transplantation

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    End point title
    		 Platelet Engraftment by 42 days following transplantation
    End point description
    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
    End point type
    Secondary
    End point timeframe
    By 42 days post-transplantation
    End point values
    		 Omidubicel Unmanipulated Cord Blood Transplant
    Number of subjects analysed
    62 [8]
    63 [9]
    Units: Subject
        Subjects
    34
    22
    Notes
    [8] - The number of patients randomized to the NiCord Intent to Treat group was 62
    [9] - The number of patients randomized to the Unmanipulated CB Transplant Intent to Treat group was 63
    No statistical analyses for this end point

    Other pre-specified: Non-relapse mortality by 210 Days following randomization

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    End point title
    		 Non-relapse mortality by 210 Days following randomization
    End point description
    Non-relapse mortality by 100 Days following transplant for the Intent to Treat population
    End point type
    Other pre-specified
    End point timeframe
    100 Days following transplant
    End point values
    		 Omidubicel Unmanipulated Cord Blood Transplant
    Number of subjects analysed
    62
    63
    Units: Subjects
        Subjects
    6
    8
    No statistical analyses for this end point

    Other pre-specified: Duration of primary hospitalization

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    End point title
    		 Duration of primary hospitalization
    End point description
    Length of time of Duration of primary hospitalization in days for the Transplanted Population
    End point type
    Other pre-specified
    End point timeframe
    Length of time of Duration of primary hospitalization in days
    End point values
    		 Omidubicel Unmanipulated Cord Blood Transplant
    Number of subjects analysed
    62
    63
    Units: Days
        Days
    27
    35
    No statistical analyses for this end point

    Other pre-specified: Overall survival at 210 Days following randomization

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    End point title
    		 Overall survival at 210 Days following randomization
    End point description
    Overall survival at 210 Days following randomization for the Intent to Treat population
    End point type
    Other pre-specified
    End point timeframe
    At 210 Days following ramdomization
    End point values
    		 Omidubicel Unmanipulated Cord Blood Transplant
    Number of subjects analysed
    62
    63
    Units: Number of deaths
        Deaths
    10
    20
    No statistical analyses for this end point

    Other pre-specified: Disease-free survival at 15 Months following randomization

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    End point title
    		 Disease-free survival at 15 Months following randomization
    End point description
    Disease-free survival at 15 Months following randomization for the Intent to Treat Population
    End point type
    Other pre-specified
    End point timeframe
    15 Months following randomization
    End point values
    		 Omidubicel Unmanipulated Cord Blood Transplant
    Number of subjects analysed
    62
    63
    Units: Number of deaths
    23
    28
    No statistical analyses for this end point

    Other pre-specified: Acute GvHD Grade II-IV by 100 Days following transplantation

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    End point title
    		 Acute GvHD Grade II-IV by 100 Days following transplantation
    End point description
    Acute GvHD Grade II-IV by 100 Days following transplantation
    End point type
    Other pre-specified
    End point timeframe
    By 100 Days following transplantation
    End point values
    		 Omidubicel Unmanipulated Cord Blood Transplant
    Number of subjects analysed
    59 [10]
    58 [11]
    Units: Number of patients
        Patients
    32
    25
    Notes
    [10] - Number of patients transplanted with omidubicel
    [11] - Number of patients transplanted with unmanipulated cord blood transplant
    No statistical analyses for this end point

    Other pre-specified: Acute GvHD Grade III-IV by 100 Days following transplantation

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    End point title
    		 Acute GvHD Grade III-IV by 100 Days following transplantation
    End point description
    Acute GvHD Grade III-IV by 100 Days following transplantation for Transplanted Populations
    End point type
    Other pre-specified
    End point timeframe
    By 100 Days following transplantation
    End point values
    		 Omidubicel Unmanipulated Cord Blood Transplant
    Number of subjects analysed
    59 [12]
    58 [13]
    Units: Number of Patients
        Patients
    8
    12
    Notes
    [12] - Number of patients transplanted with omidubicel product
    [13] - Number of patients transplanted with unmanipulated cord blood unit
    No statistical analyses for this end point

    Other pre-specified: Chronic GvHD by 180 Days following transplantation

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    End point title
    		 Chronic GvHD by 180 Days following transplantation
    End point description
    Chronic GvHD (mild/moderate/severe) by 180 Days following transplantation for transplanted population
    End point type
    Other pre-specified
    End point timeframe
    By 180 Days following transplantation
    End point values
    		 Omidubicel Unmanipulated Cord Blood Transplant
    Number of subjects analysed
    59 [14]
    58 [15]
    Units: Number of Patients
        Patients
    5
    6
    Notes
    [14] - Number of patients transplanted with omidubicel product
    [15] - Number of patients transplanted with unmanipulated cord blood unit(s)
    No statistical analyses for this end point

    Other pre-specified: Chronic GVHD by 1 year following transplantation

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    End point title
    		 Chronic GVHD by 1 year following transplantation
    End point description
    Chronic GVHD by 1 year following transplantation for transplanted populations
    End point type
    Other pre-specified
    End point timeframe
    By 1 year following transplantation
    End point values
    		 Omidubicel Unmanipulated Cord Blood Transplant
    Number of subjects analysed
    59 [16]
    58 [17]
    Units: Number of Patients
        Patients
    18
    15
    Notes
    [16] - Number of patients transplanted with omidubicel product
    [17] - Number of patients transplanted with unmanipulated cord blood units
    No statistical analyses for this end point

    Other pre-specified: Secondary graft failure by one year following transplantation

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    End point title
    		 Secondary graft failure by one year following transplantation
    End point description
    Secondary graft failure by one year following transplantation for transplanted populations
    End point type
    Other pre-specified
    End point timeframe
    By one year following transplantation
    End point values
    		 Omidubicel Unmanipulated Cord Blood Transplant
    Number of subjects analysed
    59 [18]
    58 [19]
    Units: Number of patients
        Patients
    1
    0
    Notes
    [18] - Number of patients transplanted with omidubicel product
    [19] - Number of patients transplanted with unmanipulated cord blood units
    No statistical analyses for this end point

    Other pre-specified: Grade 3 viral infections by 180 Days following transplantation

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    End point title
    		 Grade 3 viral infections by 180 Days following transplantation
    End point description
    Grade 3 viral infections by 180 Days after transplantation for intent to treat populations
    End point type
    Other pre-specified
    End point timeframe
    By 180 Days following transplantation
    End point values
    		 Omidubicel Unmanipulated Cord Blood Transplant
    Number of subjects analysed
    62
    63
    Units: Number of Patients
        Patients
    4
    14
    No statistical analyses for this end point

    Other pre-specified: Grade 3 viral infections by 1 year following transplantation

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    End point title
    		 Grade 3 viral infections by 1 year following transplantation
    End point description
    Grade 3 viral infections by 1 year following transplantation for the Intent to Treat populations
    End point type
    Other pre-specified
    End point timeframe
    By 1 year after transplantation
    End point values
    		 Omidubicel Unmanipulated Cord Blood Transplant
    Number of subjects analysed
    62
    63
    Units: Number of patients
        patients
    5
    17
    No statistical analyses for this end point

    Other pre-specified: Relapse by 15 Months following randomization

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    End point title
    		 Relapse by 15 Months following randomization
    End point description
    Relapse by 15 Months following randomization for Intent to Treat populations
    End point type
    Other pre-specified
    End point timeframe
    By 15 Months following randomization
    End point values
    		 Omidubicel Unmanipulated Cord Blood Transplant
    Number of subjects analysed
    62
    63
    Units: number of patients
        Patients
    14
    10
    No statistical analyses for this end point

    Other pre-specified: Neutrophil engraftment by 16 Days following transplantation

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    End point title
    		 Neutrophil engraftment by 16 Days following transplantation
    End point description
    Neutrophil engraftment by 16 Days following transplantation for Intent to Treat Population
    End point type
    Other pre-specified
    End point timeframe
    BY 16 Days following transplantation
    End point values
    		 Omidubicel Unmanipulated Cord Blood Transplant
    Number of subjects analysed
    62
    63
    Units: percent
    number (not applicable)
        Percent
    68
    24
    No statistical analyses for this end point

    Other pre-specified: Neutrophil engraftment by 42 Days following transplantation

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    End point title
    		 Neutrophil engraftment by 42 Days following transplantation
    End point description
    Neutrophil engraftment by 42 Days following transplantation for Intent to Treat Population
    End point type
    Other pre-specified
    End point timeframe
    By 42 Days following transplantation
    End point values
    		 Omidubicel Unmanipulated Cord Blood Transplant
    Number of subjects analysed
    62
    63
    Units: percent
        number (not applicable)
    89
    84
    No statistical analyses for this end point

    Other pre-specified: Time to Platelet Engraftment by Day 180 following Transplant

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    End point title
    		 Time to Platelet Engraftment by Day 180 following Transplant
    End point description
    Time to Platelet Engraftment by Day 180 following Transplant among Engrafters (Platelet Engrafting Population)
    End point type
    Other pre-specified
    End point timeframe
    By Day 180 following transplantation
    End point values
    		 Omidubicel Unmanipulated Cord Blood Transplant
    Number of subjects analysed
    44 [20]
    42 [21]
    Units: day
        median (full range (min-max))
    34 (21 to 180)
    42 (21 to 120)
    Notes
    [20] - Number of patients with Platelet Engraftment by Day 180 for patients treated with omidubicel product
    [21] - Number of patients with Platelet Engraftment by Day 180 for patients treated with unmanipulated CB
    No statistical analyses for this end point

    Other pre-specified: Non-relapse mortality by 130 Days following randomization

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    End point title
    		 Non-relapse mortality by 130 Days following randomization
    End point description
    Non-relapse mortality by 130 Days following randomization for the Intent to Treat Populations
    End point type
    Other pre-specified
    End point timeframe
    By 130 Days following randomization
    End point values
    		 Omidubicel Unmanipulated Cord Blood Transplant
    Number of subjects analysed
    62
    63
    Units: Number of subjects with events
        Subjects
    4
    9
    No statistical analyses for this end point

    Other pre-specified: Non-relapse mortality by 15 months following randomization

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    End point title
    		 Non-relapse mortality by 15 months following randomization
    End point description
    Non-relapse mortality by 15 months following randomization for the Intent to Treat Populations
    End point type
    Other pre-specified
    End point timeframe
    By 15 months following randomization
    End point values
    		 Omidubicel Unmanipulated Cord Blood Transplant
    Number of subjects analysed
    62
    63
    Units: Number of subjects with events
        Subjects
    9
    18
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    1 year
    Adverse event reporting additional description
    Treatment Emergent Serious Adverse Event (SAEs) Reported in at least 3% in either group of the Safety Population Treatment Emergent Adverse Events (AEs) with Severity Grades 3-5 Reported in at least 3% in either group of the Safety Population
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    23
    Reporting groups
    Reporting group title
    Unmanipulated CBUs
    Reporting group description
    		 Unmanipulated cord blood unit(s)

    Reporting group title
    Omidubicel
    Reporting group description
    		 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.

    Serious adverse events
    		 Unmanipulated CBUs Omidubicel
    Total subjects affected by serious adverse events
         subjects affected / exposed
    51 / 56 (91.07%)
    47 / 52 (90.38%)
         number of deaths (all causes)
    25
    17
         number of deaths resulting from adverse events
    25
    17
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Acute lymphocytic leukaemia recurrent
    Additional description: Treatment Emergent Serious Adverse Event Reported in at least 3% in either group of the Safety Population
         subjects affected / exposed
    0 / 56 (0.00%)
    3 / 52 (5.77%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 3
         deaths causally related to treatment / all
    0 / 0
    0 / 1
    Leukaemia recurrent
    Additional description: Treatment Emergent Serious Adverse Event Reported in at least 3% in either group of the Safety Population
         subjects affected / exposed
    6 / 56 (10.71%)
    4 / 52 (7.69%)
         occurrences causally related to treatment / all
    0 / 6
    0 / 4
         deaths causally related to treatment / all
    0 / 4
    0 / 1
    Injury, poisoning and procedural complications
    Femoral neck fracture
    Additional description: Treatment Emergent Serious Adverse Event Reported in at least 3% in either group of the Safety Population
         subjects affected / exposed
    0 / 56 (0.00%)
    2 / 52 (3.85%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Transplant failure
    Additional description: Treatment Emergent Serious Adverse Event Reported in at least 3% in either group of the Safety Population
         subjects affected / exposed
    5 / 56 (8.93%)
    3 / 52 (5.77%)
         occurrences causally related to treatment / all
    4 / 5
    2 / 3
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Nervous system disorders
    Subarachnoid haemorrhage
    Additional description: Treatment Emergent Serious Adverse Event Reported in at least 3% in either group of the Safety Population
         subjects affected / exposed
    0 / 56 (0.00%)
    2 / 52 (3.85%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Blood and lymphatic system disorders
    Febrile Neutropenia
    Additional description: Treatment Emergent Serious Adverse Event Reported in at least 3% in either group of the Safety Population
         subjects affected / exposed
    0 / 56 (0.00%)
    3 / 52 (5.77%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 3
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Thrombotic microangiopathy
    Additional description: Treatment Emergent Serious Adverse Event Reported in at least 3% in either group of the Safety Population
         subjects affected / exposed
    4 / 56 (7.14%)
    1 / 52 (1.92%)
         occurrences causally related to treatment / all
    0 / 4
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 1
    General disorders and administration site conditions
    Multiple organ dysfunction syndrome
    Additional description: Treatment Emergent Serious Adverse Event Reported in at least 3% in either group of the Safety Population
         subjects affected / exposed
    2 / 56 (3.57%)
    0 / 52 (0.00%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Pyrexia
    Additional description: Treatment Emergent Serious Adverse Event Reported in at least 3% in either group of the Safety Population
         subjects affected / exposed
    3 / 56 (5.36%)
    5 / 52 (9.62%)
         occurrences causally related to treatment / all
    0 / 3
    0 / 5
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Immune system disorders
    Acute graft versus host disease
    Additional description: Treatment Emergent Serious Adverse Event Reported in at least 3% in either group of the Safety Population
         subjects affected / exposed
    2 / 56 (3.57%)
    5 / 52 (9.62%)
         occurrences causally related to treatment / all
    1 / 2
    3 / 6
         deaths causally related to treatment / all
    0 / 0
    1 / 1
    Graft versus host disease
         subjects affected / exposed
    5 / 56 (8.93%)
    4 / 52 (7.69%)
         occurrences causally related to treatment / all
    4 / 6
    0 / 4
         deaths causally related to treatment / all
    2 / 2
    0 / 0
    Graft versus host disease in gastrointestinal tract
    Additional description: Treatment Emergent Serious Adverse Event Reported in at least 3% in either group of the Safety Population
         subjects affected / exposed
    6 / 56 (10.71%)
    5 / 52 (9.62%)
         occurrences causally related to treatment / all
    6 / 6
    5 / 5
         deaths causally related to treatment / all
    1 / 1
    2 / 2
    Graft versus host disease in skin
    Additional description: Treatment Emergent Serious Adverse Event Reported in at least 3% in either group of the Safety Population
         subjects affected / exposed
    0 / 56 (0.00%)
    2 / 52 (3.85%)
         occurrences causally related to treatment / all
    0 / 0
    2 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Gastrointestinal disorders
    Abdominal pain
    Additional description: Treatment Emergent Serious Adverse Event Reported in at least 3% in either group of the Safety Population
         subjects affected / exposed
    2 / 56 (3.57%)
    1 / 52 (1.92%)
         occurrences causally related to treatment / all
    0 / 2
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Diarrhoea
    Additional description: Treatment Emergent Serious Adverse Event Reported in at least 3% in either group of the Safety Population
         subjects affected / exposed
    3 / 56 (5.36%)
    1 / 52 (1.92%)
         occurrences causally related to treatment / all
    0 / 3
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Gastrointestinal haemorrhage
    Additional description: Treatment Emergent Serious Adverse Event Reported in at least 3% in either group of the Safety Population
         subjects affected / exposed
    2 / 56 (3.57%)
    0 / 52 (0.00%)
         occurrences causally related to treatment / all
    1 / 2
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Hepatobiliary disorders
    Veno-occlusive liver disease
    Additional description: Treatment Emergent Serious Adverse Event Reported in at least 3% in either group of the Safety Population
         subjects affected / exposed
    4 / 56 (7.14%)
    2 / 52 (3.85%)
         occurrences causally related to treatment / all
    0 / 4
    0 / 2
         deaths causally related to treatment / all
    0 / 1
    0 / 1
    Respiratory, thoracic and mediastinal disorders
    Respiratory failure
    Additional description: Treatment Emergent Serious Adverse Event Reported in at least 3% in either group of the Safety Population
         subjects affected / exposed
    5 / 56 (8.93%)
    2 / 52 (3.85%)
         occurrences causally related to treatment / all
    0 / 5
    0 / 2
         deaths causally related to treatment / all
    0 / 3
    0 / 0
    Acute respiratory distress syndrome
    Additional description: Treatment Emergent Serious Adverse Event Reported in at least 3% in either group of the Safety Population
         subjects affected / exposed
    2 / 56 (3.57%)
    0 / 52 (0.00%)
         occurrences causally related to treatment / all
    1 / 2
    0 / 0
         deaths causally related to treatment / all
    0 / 1
    0 / 0
    Acute respiratory failure
    Additional description: Treatment Emergent Serious Adverse Event Reported in at least 3% in either group of the Safety Population
         subjects affected / exposed
    2 / 56 (3.57%)
    0 / 52 (0.00%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Renal and urinary disorders
    Acute kidney injury
    Additional description: Treatment Emergent Serious Adverse Event Reported in at least 3% in either group of the Safety Population
         subjects affected / exposed
    2 / 56 (3.57%)
    5 / 52 (9.62%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 5
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Cystitis haemorrhagic
    Additional description: Treatment Emergent Serious Adverse Event Reported in at least 3% in either group of the Safety Population
         subjects affected / exposed
    3 / 56 (5.36%)
    0 / 52 (0.00%)
         occurrences causally related to treatment / all
    0 / 3
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Infections and infestations
    Bacteraemia
    Additional description: Treatment Emergent Serious Adverse Event Reported in at least 3% in either group of the Safety Population
         subjects affected / exposed
    2 / 56 (3.57%)
    0 / 52 (0.00%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Cytomegalovirus infection reactivation
    Additional description: Treatment Emergent Serious Adverse Event Reported in at least 3% in either group of the Safety Population
         subjects affected / exposed
    1 / 56 (1.79%)
    2 / 52 (3.85%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Cytomegalovirus viraemia
    Additional description: Treatment Emergent Serious Adverse Event Reported in at least 3% in either group of the Safety Population
         subjects affected / exposed
    1 / 56 (1.79%)
    2 / 52 (3.85%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Herpes zoster
    Additional description: Treatment Emergent Serious Adverse Event Reported in at least 3% in either group of the Safety Population
         subjects affected / exposed
    0 / 56 (0.00%)
    2 / 52 (3.85%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Human herpesvirus 6 infection
    Additional description: Treatment Emergent Serious Adverse Event Reported in at least 3% in either group of the Safety Population
         subjects affected / exposed
    1 / 56 (1.79%)
    4 / 52 (7.69%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 4
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Pneumonia
    Additional description: Treatment Emergent Serious Adverse Event Reported in at least 3% in either group of the Safety Population
         subjects affected / exposed
    6 / 56 (10.71%)
    4 / 52 (7.69%)
         occurrences causally related to treatment / all
    0 / 8
    0 / 7
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Sepsis
    Additional description: Treatment Emergent Serious Adverse Event Reported in at least 3% in either group of the Safety Population
         subjects affected / exposed
    1 / 56 (1.79%)
    3 / 52 (5.77%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 3
         deaths causally related to treatment / all
    0 / 0
    0 / 2
    Septic shock
    Additional description: Treatment Emergent Serious Adverse Event Reported in at least 3% in either group of the Safety Population
         subjects affected / exposed
    8 / 56 (14.29%)
    1 / 52 (1.92%)
         occurrences causally related to treatment / all
    0 / 9
    0 / 1
         deaths causally related to treatment / all
    0 / 6
    0 / 0
    Staphylococcal bacteraemia
    Additional description: Treatment Emergent Serious Adverse Event Reported in at least 3% in either group of the Safety Population
         subjects affected / exposed
    0 / 56 (0.00%)
    2 / 52 (3.85%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Upper respiratory tract infection
    Additional description: Treatment Emergent Serious Adverse Event Reported in at least 3% in either group of the Safety Population
         subjects affected / exposed
    2 / 56 (3.57%)
    0 / 52 (0.00%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Viral infection
    Additional description: Treatment Emergent Serious Adverse Event Reported in at least 3% in either group of the Safety Population
         subjects affected / exposed
    0 / 56 (0.00%)
    2 / 52 (3.85%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 3%
    Non-serious adverse events
    		 Unmanipulated CBUs Omidubicel
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    56 / 56 (100.00%)
    52 / 52 (100.00%)
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Acute lymphocytic leukaemia recurrent
         subjects affected / exposed
    0 / 56 (0.00%)
    3 / 52 (5.77%)
         occurrences all number
    0
    3
    Leukaemia recurrent
         subjects affected / exposed
    5 / 56 (8.93%)
    4 / 52 (7.69%)
         occurrences all number
    5
    4
    Vascular disorders
    Haemorrhage
         subjects affected / exposed
    2 / 56 (3.57%)
    1 / 52 (1.92%)
         occurrences all number
    2
    1
    Hypertension
         subjects affected / exposed
    21 / 56 (37.50%)
    13 / 52 (25.00%)
         occurrences all number
    25
    16
    Hypotension
         subjects affected / exposed
    5 / 56 (8.93%)
    2 / 52 (3.85%)
         occurrences all number
    5
    2
    General disorders and administration site conditions
    Asthenia
    Additional description: Treatment-Emergent AEs with Severity Grades 3-5 Reported in at least 3% in either group of the Safety Population (SP)
         subjects affected / exposed
    11 / 56 (19.64%)
    2 / 52 (3.85%)
         occurrences all number
    13
    3
    Edema
    Additional description: Treatment-Emergent AEs with Severity Grades 3-5 Reported in at least 3% in either group of the Safety Population (SP)
         subjects affected / exposed
    4 / 56 (7.14%)
    1 / 52 (1.92%)
         occurrences all number
    4
    1
    Mucosal inflammation
    Additional description: Treatment-Emergent AEs with Severity Grades 3-5 Reported in at least 3% in either group of the Safety Population (SP)
         subjects affected / exposed
    19 / 56 (33.93%)
    16 / 52 (30.77%)
         occurrences all number
    19
    17
    Multiple organ dysfunction syndrome
    Additional description: Treatment-Emergent AEs with Severity Grades 3-5 Reported in at least 3% in either group of the Safety Population (SP)
         subjects affected / exposed
    2 / 56 (3.57%)
    0 / 52 (0.00%)
         occurrences all number
    2
    0
    Pain
    Additional description: Treatment-Emergent AEs with Severity Grades 3-5 Reported in at least 3% in either group of the Safety Population (SP)
         subjects affected / exposed
    10 / 56 (17.86%)
    17 / 52 (32.69%)
         occurrences all number
    12
    18
    Pyrexia
    Additional description: Treatment-Emergent AEs with Severity Grades 3-5 Reported in at least 3% in either group of the Safety Population (SP)
         subjects affected / exposed
    6 / 56 (10.71%)
    1 / 52 (1.92%)
         occurrences all number
    6
    1
    Immune system disorders
    Graft-versus-host disease in gastrointestinal tract
         subjects affected / exposed
    6 / 56 (10.71%)
    5 / 52 (9.62%)
         occurrences all number
    6
    5
    Graft-versus-host disease
         subjects affected / exposed
    5 / 56 (8.93%)
    3 / 52 (5.77%)
         occurrences all number
    6
    3
    Acute Graft-versus-host disease
         subjects affected / exposed
    1 / 56 (1.79%)
    4 / 52 (7.69%)
         occurrences all number
    1
    5
    Respiratory, thoracic and mediastinal disorders
    Acute respiratory distress syndrome
         subjects affected / exposed
    2 / 56 (3.57%)
    0 / 52 (0.00%)
         occurrences all number
    2
    0
    Acute respiratory failure
         subjects affected / exposed
    2 / 56 (3.57%)
    0 / 52 (0.00%)
         occurrences all number
    2
    0
    Dyspnoea
         subjects affected / exposed
    9 / 56 (16.07%)
    4 / 52 (7.69%)
         occurrences all number
    10
    5
    Epistaxis
         subjects affected / exposed
    4 / 56 (7.14%)
    3 / 52 (5.77%)
         occurrences all number
    4
    3
    Hypoxia
         subjects affected / exposed
    13 / 56 (23.21%)
    5 / 52 (9.62%)
         occurrences all number
    18
    5
    Respiratory failure
         subjects affected / exposed
    5 / 56 (8.93%)
    2 / 52 (3.85%)
         occurrences all number
    5
    2
    Psychiatric disorders
    Anxiety
         subjects affected / exposed
    3 / 56 (5.36%)
    1 / 52 (1.92%)
         occurrences all number
    5
    1
    Depression
         subjects affected / exposed
    2 / 56 (3.57%)
    0 / 52 (0.00%)
         occurrences all number
    2
    0
    Insomnia
         subjects affected / exposed
    2 / 56 (3.57%)
    1 / 52 (1.92%)
         occurrences all number
    2
    1
    Investigations
    Transaminases increased
         subjects affected / exposed
    1 / 56 (1.79%)
    4 / 52 (7.69%)
         occurrences all number
    1
    4
    weight decreased
         subjects affected / exposed
    0 / 56 (0.00%)
    3 / 52 (5.77%)
         occurrences all number
    0
    3
    Injury, poisoning and procedural complications
    Femoral neck fracture
         subjects affected / exposed
    0 / 56 (0.00%)
    2 / 52 (3.85%)
         occurrences all number
    0
    2
    Transplant failure
         subjects affected / exposed
    5 / 56 (8.93%)
    3 / 52 (5.77%)
         occurrences all number
    5
    3
    Nervous system disorders
    Syncope
         subjects affected / exposed
    2 / 56 (3.57%)
    3 / 52 (5.77%)
         occurrences all number
    2
    3
    Blood and lymphatic system disorders
    Febrile neutropenia
    Additional description: Treatment-Emergent AEs with Severity Grades 3-5 Reported in at least 3% in either group of the Safety Population (SP)
         subjects affected / exposed
    4 / 56 (7.14%)
    4 / 52 (7.69%)
         occurrences all number
    8
    4
    Thrombotic microangiopathy
    Additional description: Treatment-Emergent AEs with Severity Grades 3-5 Reported in at least 3% in either group of the Safety Population (SP)
         subjects affected / exposed
    3 / 56 (5.36%)
    1 / 52 (1.92%)
         occurrences all number
    3
    1
    Gastrointestinal disorders
    Diarrhoea
    Additional description: Treatment-Emergent AEs with Severity Grades 3-5 Reported in at least 3% in either group of the Safety Population (SP)
         subjects affected / exposed
    3 / 56 (5.36%)
    1 / 52 (1.92%)
         occurrences all number
    3
    1
    Dysphagia
    Additional description: Treatment-Emergent AEs with Severity Grades 3-5 Reported in at least 3% in either group of the Safety Population (SP)
         subjects affected / exposed
    7 / 56 (12.50%)
    6 / 52 (11.54%)
         occurrences all number
    8
    7
    Gastrointestinal haemorrhage
    Additional description: Treatment-Emergent AEs with Severity Grades 3-5 Reported in at least 3% in either group of the Safety Population (SP)
         subjects affected / exposed
    3 / 56 (5.36%)
    0 / 52 (0.00%)
         occurrences all number
    3
    0
    Gastrointestinal toxicity
    Additional description: Treatment-Emergent AEs with Severity Grades 3-5 Reported in at least 3% in either group of the Safety Population (SP)
         subjects affected / exposed
    19 / 56 (33.93%)
    10 / 52 (19.23%)
         occurrences all number
    22
    12
    Vomiting
    Additional description: Treatment-Emergent AEs with Severity Grades 3-5 Reported in at least 3% in either group of the Safety Population (SP)
         subjects affected / exposed
    6 / 56 (10.71%)
    1 / 52 (1.92%)
         occurrences all number
    6
    1
    Hepatobiliary disorders
    veno-occlusive liver disease
    Additional description: Treatment-Emergent AEs with Severity Grades 3-5 Reported in at least 3% in either group of the Safety Population (SP)
         subjects affected / exposed
    4 / 56 (7.14%)
    2 / 52 (3.85%)
         occurrences all number
    4
    2
    Renal and urinary disorders
    Acute kidney injury
         subjects affected / exposed
    3 / 56 (5.36%)
    4 / 52 (7.69%)
         occurrences all number
    3
    4
    Cystitis haemorrhagic
         subjects affected / exposed
    2 / 56 (3.57%)
    0 / 52 (0.00%)
         occurrences all number
    2
    0
    Musculoskeletal and connective tissue disorders
    Muscular weakness
         subjects affected / exposed
    2 / 56 (3.57%)
    1 / 52 (1.92%)
         occurrences all number
    2
    1
    Infections and infestations
    Cystitis
         subjects affected / exposed
    2 / 56 (3.57%)
    4 / 52 (7.69%)
         occurrences all number
    2
    4
    Cytomegalovirus viraemia
         subjects affected / exposed
    1 / 56 (1.79%)
    2 / 52 (3.85%)
         occurrences all number
    1
    2
    Herpes zoster
         subjects affected / exposed
    0 / 56 (0.00%)
    2 / 52 (3.85%)
         occurrences all number
    0
    2
    Human herpesvirus 6 infection
         subjects affected / exposed
    0 / 56 (0.00%)
    4 / 52 (7.69%)
         occurrences all number
    0
    4
    Pneumonia
         subjects affected / exposed
    5 / 56 (8.93%)
    4 / 52 (7.69%)
         occurrences all number
    6
    7
    Sepsis
         subjects affected / exposed
    1 / 56 (1.79%)
    3 / 52 (5.77%)
         occurrences all number
    1
    3
    Septic shock
         subjects affected / exposed
    8 / 56 (14.29%)
    1 / 52 (1.92%)
         occurrences all number
    9
    1
    Staphylococcal bacteraemia
         subjects affected / exposed
    0 / 56 (0.00%)
    2 / 52 (3.85%)
         occurrences all number
    0
    2
    Metabolism and nutrition disorders
    Dehydration
         subjects affected / exposed
    2 / 56 (3.57%)
    3 / 52 (5.77%)
         occurrences all number
    2
    3
    Hyperglycaemia
         subjects affected / exposed
    8 / 56 (14.29%)
    4 / 52 (7.69%)
         occurrences all number
    11
    5
    Hypoalbuminaemia
         subjects affected / exposed
    3 / 56 (5.36%)
    1 / 52 (1.92%)
         occurrences all number
    3
    1
    Hypocalcaemia
         subjects affected / exposed
    3 / 56 (5.36%)
    1 / 52 (1.92%)
         occurrences all number
    4
    1
    Hypokalaemia
         subjects affected / exposed
    5 / 56 (8.93%)
    6 / 52 (11.54%)
         occurrences all number
    5
    6
    Hypomagnesaemia
         subjects affected / exposed
    0 / 56 (0.00%)
    2 / 52 (3.85%)
         occurrences all number
    0
    2
    Hypophosphataemia
         subjects affected / exposed
    5 / 56 (8.93%)
    3 / 52 (5.77%)
         occurrences all number
    5
    3

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    05 Aug 2016
    Amendment I Addition of NRM at 210 Days post-randomization as a secondary endpoint; Addition of NRM assessment at 1-year following randomization; Addition of Day 28 timepoint for immune reconstitution. Updated disease inclusion criteria for AML and ALL TBI dose and days for conditioning regimen option A.2. Determination of using a single or double cord in the control arm by the investigator prior to randomization was clarified. General language clarifications/refinements were made.
    27 Oct 2016
    Amendment II Updated secondary endpoint assessment dates for NRM, OS and DFS, relapse, and relapse mortality from one year to 15 Months post-randomization. AE collection time changed from initiation of conditioning regimen to time of consent. Added clarification of omidubicel infusion guidelines, including minimal infusion time. BM assessment changed from Day 270 to Day 365 post-transplant. Updates made to GvHD prophylaxis monitoring levels and the final process quality control testing and release criteria.
    30 Jul 2017
    Amendment III MDS disease criterion and MDS baseline disease assessment criterion were updated. Adjustments were made to refine logistic processes of CBU review to simplify study enrollment and workflow.
    10 Dec 2017
    Amendment IV Expanded eligibility criteria to include ALL, AML, and MDS patients and to patients with lymphoma and other rare hematologic malignancies that are typical candidates for transplant. Patient age adjusted from 16-60 years to 12-65 years to allow for a wider age range. Lansky performance scale added due to the inclusion of pediatric patients. Further clarification to the CBU logistic and review processes were made, as well as simplification of backup CBU criteria. Endpoint label modifications made per FDA request to separate into secondary, tertiary, and exploratory endpoints. NRM was changed from a secondary to tertiary endpoint. Details of when chimerism testing must show donor cells to be considered neutrophil engraftment were added. Updates to GvHD and infection prophylaxis and reVVcommendations for toxoplasmosis prophylaxis were added. Language for post- transplant assessments was updated to clarify required days of assessments and distinguish between required versus requested or as performed per standard of care assessments.
    01 May 2018
    Amendment V Additional (optional) sub-studies for long-term follow-up and immune reconstitution testing were added. Eligibility criteria for ALL, CML, MDS, and lymphoma broadened to align more with standard criteria for transplant candidates, together with disease baseline assessments. Site selection of conditioning regimen of choice broadened so clinical sites could select a regimen according to primary diagnosis/age group instead of relying solely on one regimen for all patients. Permission for GvHD prophylaxis adjustments depending on patient diagnosis or age group. Regimen A.2 modified to allow for TBI to be given as either 1320 cGy or 1200 cy total per institutional practice. Calculation for adjusted body weight formula modified to be specific for the regimen selected. Calcineurin inhibitor tapering was adjusted to allow to begin at Day 100 instead of Day 150. Amendment V was recalled by Gamida Cell from submission to sites as of May 14, 2018, and CROs notified accordingly, following a meeting with FDA that required implementation of changes to the protocol.
    22 May 2018
    Amendment V.1 Provided to sites for submission included all changes made in Amd V Updates added regarding required disease assessment including flow cytometry, cytogenetics, molecular markers and/or BM morphology and other applicable assessments for all randomized patients at specified visits prior to visit on Day 365 post-transplant per FDA request.
    22 Jan 2019
    Amendment VI Addition of new disease criterion of CMMoL and MDS/CMMoL overlap to broaden the list of rare diseases allowed per study Wording adjustments to ALL and MDS criteria. Screening test results requirements adapted to accommodate a pediatric population. Regimen A.1 modified to allow for TBI to be given as either 1350 cGy or 1200 cy total as per institutional practice. Regimen B modified to allow for an alternate dose of busulfan to be added. FACT-BMT quality of life assessment modified for pediatric population. Lymphoma disease assessment was updated to allow for either CT or PET-CT scan of chest, abdomen, and pelvis at baseline and post-randomization. Chimerism assessment allowed to be performed as whole blood or myeloid fraction at the required study visit days. Release criteria tables for the CF, NF, and infusion solution were removed from the protocol as the Certificates of Analysis are provided to the site by Gamida Cell for each batch product to inform of all product specifications. Clarification of SUSAR reporting for omidubicel arm only and based on Regulatory Authorities requirements. All modifications in local amendments IV.1 and IV.2 were included in this Amendment VI.

    Interruptions (globally)
    Were there any global interruptions to the trial? No

    Limitations and caveats
    Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.
    None reported
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