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    Clinical Trial Results:
    Phase 3, Randomized, Double-Blind, Placebo-Controlled Trial, with Cross-Over, of Posoleucel (ALVR105) for the Treatment of Adenovirus Infection in Pediatric and Adult Participants Receiving Standard of Care Following Allogeneic Hematopoietic Cell Transplantation

    Summary
    EudraCT number
    		 2021-003450-22
    Trial protocol
    		 SE   IT   ES  
    Global end of trial date
    31 Jan 2024

    Results information
    Results version number
    		 v1(current)
    This version publication date
    01 May 2024
    First version publication date
    01 May 2024
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    P-105-303
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT05179057
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    AlloVir, Inc.
    Sponsor organisation address
    1100 Winter Street, Waltham, United States, MA 02451
    Public contact
    Clinical Trials Information Line, AlloVir, Inc., +1 617-433-2605, ClinicalTrials@allovir.com
    Scientific contact
    Clinical Trials Information Line, AlloVir, Inc., +1 617-433-2605, ClinicalTrials@allovir.com
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    No
    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
    Final
    Date of interim/final analysis
    31 Jan 2024
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    31 Jan 2024
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    The main objectives of the study were: - To compare the percent of participants who have clearance of adenovirus viremia at Day 29 in participants receiving posoleucel and standard of care (SoC) to that in participants receiving placebo and SoC. - To determine the safety and tolerability of posoleucel by analyzing the incidence and severity of treatment-emergent adverse events (TEAEs), including individual adverse events of special interest (AESIs).
    Protection of trial subjects
    This study was performed in compliance with the principles of Good Clinical Practice, including the archiving of essential documents.
    Background therapy
    		 Participants in both treatment arms continued to receive SoC per their treating physician.
    Evidence for comparator
    		 -
    Actual start date of recruitment
    26 Apr 2022
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    Yes
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    Canada: 6
    Country: Number of subjects enrolled
    United Kingdom: 21
    Country: Number of subjects enrolled
    United States: 17
    Country: Number of subjects enrolled
    Italy: 9
    Country: Number of subjects enrolled
    Spain: 3
    Country: Number of subjects enrolled
    Sweden: 1
    Worldwide total number of subjects
    57
    EEA total number of subjects
    13
    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)
    6
    Children (2-11 years)
    28
    Adolescents (12-17 years)
    13
    Adults (18-64 years)
    8
    From 65 to 84 years
    2
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 Participants were enrolled at 47 study centers in the United States, Canada, Italy, Spain, Sweden, and the United Kingdom, and participated from April 2022 to January 2024.

    Pre-assignment
    Screening details
    		 Participants with adenovirus infection receiving standard of care following allogeneic hematopoietic stem cell transplant (allo-HCT) were randomized in a 1:1 ratio to receive either posoleucel or placebo. Randomization was stratified by level of viremia (≥10,000 copies/mL or <10,000 copies/mL adenovirus DNA) and age (≥12 years or <12 years).

    Period 1
    Period 1 title
    Overall Study (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Randomised - controlled
    Blinding used
    		 Double blind
    Roles blinded
    		 Investigator, Subject

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Posoleucel, Then Placebo
    Arm description
    		 Participants were randomized to receive 2 sequential infusions of posoleucel, separated by 14 ± 3 days. The Primary Study Period included a 4-week efficacy evaluation followed by a 20-week safety follow-up. Eligible participants who experienced progression to active target organ disease or progression of existing target organ disease could cross-over to placebo treatment between Day 29 and Week 10. In the Cross-Over Period, participants received 2 sequential infusions of placebo, separated by 14 ± 3 days. The Cross-over Period included a 4-week efficacy evaluation followed by a 20-week safety follow-up.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Posoleucel
    Investigational medicinal product code
    ALVR105
    Other name
    PSL, ALVR-105, Viralym-M
    Pharmaceutical forms
    Dispersion for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Administered as 2-4 milliliter infusion, visually identical to placebo.

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Dispersion for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Administered as 2-4 milliliter infusion, visually identical to Posoleucel.

    Arm title
    		 Placebo, Then Posoleucel
    Arm description
    		 Participants were randomized to receive 2 sequential infusions of placebo, separated by 14 ± 3 days. The Primary Study Period included a 4-week efficacy evaluation followed by a 20-week safety follow-up. Eligible participants who experienced progression to active target organ disease or progression of existing target organ disease could cross-over to posoleucel treatment between Day 29 and Week 10. In the Cross- Over Period, participants received 2 sequential infusions of placebo, separated by 14 ± 3 days. The Cross-over Period included a 4-week efficacy evaluation followed by a 20-week safety follow-up.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Posoleucel
    Investigational medicinal product code
    ALVR105
    Other name
    PSL, ALVR-105, Viralym-M
    Pharmaceutical forms
    Dispersion for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Administered as 2-4 milliliter infusion, visually identical to placebo.

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Dispersion for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Administered as 2-4 milliliter infusion, visually identical to Posoleucel.

    Number of subjects in period 1
    		Posoleucel, Then Placebo Placebo, Then Posoleucel
    Started
    30
    27
    Received Primary Study Treatment
    28
    23
    Met Crossover Eligibility Criteria
    4 [1]
    5 [2]
    Received Cross-over Treatment
    4 [3]
    5 [4]
    Completed
    10
    13
    Not completed
    20
    14
         Study terminated
    13
    7
         Discontinuation or withdrawal
    2
    2
         Adverse event, non-fatal
    3
    2
         Never received study treatment
    1
    3
         Noncompliance with protocol
    1
    -
    Notes
    [1] - The number of subjects at this milestone seems inconsistent with the number of subjects in the arm. It is expected that the number of subjects will be greater than, or equal to the number that completed, minus those who left.
    Justification: The milestone represents participants who met the cross-over eligibility criteria.
    [2] - The number of subjects at this milestone seems inconsistent with the number of subjects in the arm. It is expected that the number of subjects will be greater than, or equal to the number that completed, minus those who left.
    Justification: The milestone represents participants who received any cross-over treatment.
    [3] - The number of subjects at this milestone seems inconsistent with the number of subjects in the arm. It is expected that the number of subjects will be greater than, or equal to the number that completed, minus those who left.
    Justification: The milestone represents participants who received any cross-over treatment.
    [4] - The number of subjects at this milestone seems inconsistent with the number of subjects in the arm. It is expected that the number of subjects will be greater than, or equal to the number that completed, minus those who left.
    Justification: The milestone represents participants who met the cross-over eligibility criteria.

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Posoleucel, Then Placebo
    Reporting group description
    		 Participants were randomized to receive 2 sequential infusions of posoleucel, separated by 14 ± 3 days. The Primary Study Period included a 4-week efficacy evaluation followed by a 20-week safety follow-up. Eligible participants who experienced progression to active target organ disease or progression of existing target organ disease could cross-over to placebo treatment between Day 29 and Week 10. In the Cross-Over Period, participants received 2 sequential infusions of placebo, separated by 14 ± 3 days. The Cross-over Period included a 4-week efficacy evaluation followed by a 20-week safety follow-up.

    Reporting group title
    Placebo, Then Posoleucel
    Reporting group description
    		 Participants were randomized to receive 2 sequential infusions of placebo, separated by 14 ± 3 days. The Primary Study Period included a 4-week efficacy evaluation followed by a 20-week safety follow-up. Eligible participants who experienced progression to active target organ disease or progression of existing target organ disease could cross-over to posoleucel treatment between Day 29 and Week 10. In the Cross- Over Period, participants received 2 sequential infusions of placebo, separated by 14 ± 3 days. The Cross-over Period included a 4-week efficacy evaluation followed by a 20-week safety follow-up.

    Reporting group values
    		 Posoleucel, Then Placebo Placebo, Then Posoleucel Total
    Number of subjects
    		 30 27 57
    Age categorical
    Units: Subjects
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    		 16.2 ( 17.10 ) 14.9 ( 15.80 ) -
    Gender categorical
    Units: Subjects
        Female
    		 9 11 20
        Male
    		 21 16 37
    Ethnicity
    Units: Subjects
        Hispanic or Latino
    		 5 2 7
        Not Hispanic or Latino
    		 22 23 45
        Unknown or Not Reported
    		 3 2 5
    Race
    Units: Subjects
        Asian
    		 3 2 5
        Black or African American
    		 3 1 4
        White
    		 19 20 39
        Not Reported
    		 3 1 4
        Other
    		 2 3 5

    End points

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    End points reporting groups
    Reporting group title
    Posoleucel, Then Placebo
    Reporting group description
    		 Participants were randomized to receive 2 sequential infusions of posoleucel, separated by 14 ± 3 days. The Primary Study Period included a 4-week efficacy evaluation followed by a 20-week safety follow-up. Eligible participants who experienced progression to active target organ disease or progression of existing target organ disease could cross-over to placebo treatment between Day 29 and Week 10. In the Cross-Over Period, participants received 2 sequential infusions of placebo, separated by 14 ± 3 days. The Cross-over Period included a 4-week efficacy evaluation followed by a 20-week safety follow-up.

    Reporting group title
    Placebo, Then Posoleucel
    Reporting group description
    		 Participants were randomized to receive 2 sequential infusions of placebo, separated by 14 ± 3 days. The Primary Study Period included a 4-week efficacy evaluation followed by a 20-week safety follow-up. Eligible participants who experienced progression to active target organ disease or progression of existing target organ disease could cross-over to posoleucel treatment between Day 29 and Week 10. In the Cross- Over Period, participants received 2 sequential infusions of placebo, separated by 14 ± 3 days. The Cross-over Period included a 4-week efficacy evaluation followed by a 20-week safety follow-up.

    Subject analysis set title
    Posoleucel (Primary Study Period)
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    Participants were randomized to receive 2 sequential infusions of posoleucel, separated by 14 ± 3 days, during the Primary Study Period.

    Subject analysis set title
    Placebo (Primary Study Period)
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    Participants were randomized to receive 2 sequential infusions of placebo, separated by 14 ± 3 days, during the Primary Study Period.

    Subject analysis set title
    Posoleucel (Cross-over Period)
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    Participants were randomized to receive 2 sequential infusions of placebo in the Primary Study Period and were eligible to cross-over between Day 29 and Week 10. Participants received posoleucel during the Crossover Period.

    Subject analysis set title
    Placebo (Cross-over Period)
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    Participants were randomized to receive 2 sequential infusions of posoleucel in the Primary Study Period and were eligible to cross-over between Day 29 and Week 10. Participants received placebo during the Cross-over Period.

    Primary: Number of Participants With Undetectable Adenovirus Infection

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    End point title
    		 Number of Participants With Undetectable Adenovirus Infection
    End point description
    Viral load of adenovirus was measured at the central laboratory using quantitative polymerase chain reaction (qPCR) from blood and stool samples at each study visit and on Day 29 from a nasopharyngeal swab. There was a 14-day window for participants who crossed over from posoleucel to placebo; and for participants who crossed over from placebo to posoleucel, the pre-dose cross-over Day 1 viral load was used. Participants missing the primary endpoint but having undetectable viremia before Day 29 and after Day 43 were imputed as successes. Undetectable adenovirus viremia was less than the lower limit of quantification (LLOQ). The modified intent-to-treat (mITT) population included all randomized participants who received at least one dose of posoleucel or placebo. Only participants in the mITT population who completed through Day 29 or discontinued early were included.
    End point type
    Primary
    End point timeframe
    Day 29 through Day 43 (Day 29 + 14 days; up to 43 days post-first infusion)
    End point values
    		 Posoleucel, Then Placebo Placebo, Then Posoleucel
    Number of subjects analysed
    25
    21
    Units: participants
    11
    9
    Statistical analysis title
    		 Posoleucel versus Placebo
    Statistical analysis description
    A logistic regression model included treatment, level of viremia at baseline (≥10,000 copies/mL or <10,000 copies/mL adenovirus DNA), age (≥12 years or <12 years), and absolute lymphocyte counts at baseline. The null hypothesis is that the true percentage for posoleucel plus SoC is less than or equal to the true percentage for placebo plus SoC, and the alternative hypothesis is that it is greater.
    Comparison groups
    Posoleucel, Then Placebo v Placebo, Then Posoleucel
    Number of subjects included in analysis
    46
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    Method
    Parameter type
    		 Odds ratio (OR)
    Point estimate
    0.98
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 0.26
         upper limit
    		 3.69

    Primary: Number of Participants Who Experienced TEAEs

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    End point title
    		 Number of Participants Who Experienced TEAEs [1]
    End point description
    A TEAE was defined as an adverse event (AE) with a start date and time on or after the first dose of study treatment. A serious AE (SAE) was an AE that met at least one of the following serious criteria: fatal, life-threatening, required inpatient hospitalization or prolongation of existing hospitalization; resulted in persistent or significant disability/ incapacity; was a congenital anomaly/birth defect; or other important medical event. Treatment-emergent AESI included acute or chronic graft versus host disease, cytokine release syndrome, infusion-related reactions, and graft failure or rejection. Treatment-related refers to the assessment of a relationship between study treatment and the event by the investigator. The safety population included all participants who received any amount of posoleucel or placebo and had at least one post-treatment safety assessment.
    End point type
    Primary
    End point timeframe
    Up to 34 weeks
    Notes
    [1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: This was a pre-specified endpoint in which descriptive statistics were planned, with no hypothesis testing.
    End point values
    		 Posoleucel (Primary Study Period) Placebo (Primary Study Period) Posoleucel (Cross-over Period) Placebo (Cross-over Period)
    Number of subjects analysed
    28
    23
    5
    4
    Units: participants
        Any TEAE
    27
    23
    5
    3
        Any TEAE related to study treatment
    7
    9
    1
    1
        Any AESI
    6
    9
    1
    2
        Any SAE
    16
    16
    3
    1
        Any SAE related to study treatment
    1
    1
    1
    1
        TEAE leading to study treatment discontinuation
    2
    1
    0
    2
        Any TEAE leading to study discontinuation
    1
    2
    0
    1
        Any TEAE leading to death
    2
    1
    0
    1
    No statistical analyses for this end point

    Secondary: Number of Participants With Overall Disease Progression

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    End point title
    		 Number of Participants With Overall Disease Progression
    End point description
    Data not collected due to early termination after Data and Safety Monitoring Board (DSMB) futility analysis concluded the study was unlikely to meet its primary endpoint.
    End point type
    Secondary
    End point timeframe
    From Day 29 up to Week 10
    End point values
    		 Posoleucel, Then Placebo Placebo, Then Posoleucel
    Number of subjects analysed
    0 [2]
    0 [3]
    Units: participants
    Notes
    [2] - Data not collected due to early termination after DSMB futility analysis.
    [3] - Data not collected due to early termination after DSMB futility analysis.
    No statistical analyses for this end point

    Secondary: Area Under the Curve (AUC) Adenovirus Viral Load

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    End point title
    		 Area Under the Curve (AUC) Adenovirus Viral Load
    End point description
    Data not collected due to early termination after DSMB futility analysis concluded the study was unlikely to meet its primary endpoint.
    End point type
    Secondary
    End point timeframe
    Pre-dose and Day 29
    End point values
    		 Posoleucel, Then Placebo Placebo, Then Posoleucel
    Number of subjects analysed
    0 [4]
    0 [5]
    Units: copies/mL*h
        arithmetic mean (standard deviation)
    ( )
    ( )
    Notes
    [4] - Data not collected due to early termination after DSMB futility analysis.
    [5] - Data not collected due to early termination after DSMB futility analysis.
    No statistical analyses for this end point

    Secondary: Number of Participants Who Achieved Adenovirus Viremia <400 Copies/mL at Day 29

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    End point title
    		 Number of Participants Who Achieved Adenovirus Viremia <400 Copies/mL at Day 29
    End point description
    Data not collected due to early termination after DSMB futility analysis concluded the study was unlikely to meet its primary endpoint.
    End point type
    Secondary
    End point timeframe
    Day 29
    End point values
    		 Posoleucel, Then Placebo Placebo, Then Posoleucel
    Number of subjects analysed
    0 [6]
    0 [7]
    Units: participants
    Notes
    [6] - Data not collected due to early termination after DSMB futility analysis.
    [7] - Data not collected due to early termination after DSMB futility analysis.
    No statistical analyses for this end point

    Secondary: Time to Undetectable Adenovirus Viremia (Less Than LLOQ)

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    End point title
    		 Time to Undetectable Adenovirus Viremia (Less Than LLOQ)
    End point description
    Data not collected due to early termination after DSMB futility analysis concluded the study was unlikely to meet its primary endpoint.
    End point type
    Secondary
    End point timeframe
    Pre-dose to 34 weeks
    End point values
    		 Posoleucel, Then Placebo Placebo, Then Posoleucel
    Number of subjects analysed
    0 [8]
    0 [9]
    Units: hours
        median (full range (min-max))
    ( to )
    ( to )
    Notes
    [8] - Data not collected due to early termination after DSMB futility analysis.
    [9] - Data not collected due to early termination after DSMB futility analysis.
    No statistical analyses for this end point

    Secondary: Number of Participants With Adenovirus Disease Recurrence

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    End point title
    		 Number of Participants With Adenovirus Disease Recurrence
    End point description
    Data not collected due to early termination after DSMB futility analysis concluded the study was unlikely to meet its primary endpoint.
    End point type
    Secondary
    End point timeframe
    34 weeks
    End point values
    		 Posoleucel, Then Placebo Placebo, Then Posoleucel
    Number of subjects analysed
    0 [10]
    0 [11]
    Units: participants
    Notes
    [10] - Data not collected due to early termination after DSMB futility analysis.
    [11] - Data not collected due to early termination after DSMB futility analysis.
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Up to 34 weeks
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    25.1
    Reporting groups
    Reporting group title
    Posoleucel (Primary Study Period)
    Reporting group description
    		 Participants were randomized to receive 2 sequential infusions of posoleucel, separated by 14 ± 3 days, during the Primary Study Period.

    Reporting group title
    Placebo (Primary Study Period)
    Reporting group description
    		 Participants were randomized to receive 2 sequential infusions of placebo, separated by 14 ± 3 days, during the Primary Study Period.

    Reporting group title
    Posoleucel (Cross-over Period)
    Reporting group description
    		 Participants were randomized to receive 2 sequential infusions of placebo in the Primary Study Period and were eligible to cross-over between Day 29 and Week 10. Participants received posoleucel during the Crossover Period.

    Reporting group title
    Placebo (Cross-over Period)
    Reporting group description
    		 Participants were randomized to receive 2 sequential infusions of posoleucel in the Primary Study Period and were eligible to cross-over between Day 29 and Week 10. Participants received placebo during the Cross-over Period.

    Serious adverse events
    		 Posoleucel (Primary Study Period) Placebo (Primary Study Period) Posoleucel (Cross-over Period) Placebo (Cross-over Period)
    Total subjects affected by serious adverse events
         subjects affected / exposed
    16 / 28 (57.14%)
    16 / 23 (69.57%)
    3 / 5 (60.00%)
    1 / 4 (25.00%)
         number of deaths (all causes)
    2
    1
    0
    1
         number of deaths resulting from adverse events
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Leukaemia recurrent
         subjects affected / exposed
    1 / 28 (3.57%)
    0 / 23 (0.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Vascular disorders
    Capillary leak syndrome
         subjects affected / exposed
    1 / 28 (3.57%)
    0 / 23 (0.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    General disorders and administration site conditions
    Asthenia
         subjects affected / exposed
    1 / 28 (3.57%)
    1 / 23 (4.35%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Disease progression
         subjects affected / exposed
    0 / 28 (0.00%)
    1 / 23 (4.35%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
    0 / 0
    Influenza like illness
         subjects affected / exposed
    1 / 28 (3.57%)
    0 / 23 (0.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Pyrexia
         subjects affected / exposed
    4 / 28 (14.29%)
    3 / 23 (13.04%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences causally related to treatment / all
    0 / 6
    1 / 4
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Immune system disorders
    Acute graft versus host disease
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 23 (0.00%)
    0 / 5 (0.00%)
    1 / 4 (25.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Acute graft versus host disease in intestine
         subjects affected / exposed
    1 / 28 (3.57%)
    0 / 23 (0.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Acute graft versus host disease in skin
         subjects affected / exposed
    0 / 28 (0.00%)
    1 / 23 (4.35%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Chronic graft versus host disease
         subjects affected / exposed
    0 / 28 (0.00%)
    1 / 23 (4.35%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Chronic graft versus host disease in intestine
         subjects affected / exposed
    1 / 28 (3.57%)
    1 / 23 (4.35%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences causally related to treatment / all
    0 / 3
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Chronic graft versus host disease in lung
         subjects affected / exposed
    0 / 28 (0.00%)
    1 / 23 (4.35%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Chronic graft versus host disease oral
         subjects affected / exposed
    1 / 28 (3.57%)
    0 / 23 (0.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Graft versus host disease
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 23 (0.00%)
    1 / 5 (20.00%)
    0 / 4 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Graft versus host disease in gastrointestinal tract
         subjects affected / exposed
    1 / 28 (3.57%)
    0 / 23 (0.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Haemophagocytic lymphohistiocytosis
         subjects affected / exposed
    0 / 28 (0.00%)
    1 / 23 (4.35%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    Deep vein thrombosis
         subjects affected / exposed
    1 / 28 (3.57%)
    0 / 23 (0.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Hypoxia
         subjects affected / exposed
    1 / 28 (3.57%)
    0 / 23 (0.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences causally related to treatment / all
    3 / 3
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Pharyngeal inflammation
         subjects affected / exposed
    0 / 28 (0.00%)
    1 / 23 (4.35%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Pleural effusion
         subjects affected / exposed
    1 / 28 (3.57%)
    0 / 23 (0.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Pneumomediastinum
         subjects affected / exposed
    0 / 28 (0.00%)
    1 / 23 (4.35%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Pneumothorax
         subjects affected / exposed
    0 / 28 (0.00%)
    1 / 23 (4.35%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Investigations
    Weight decreased
         subjects affected / exposed
    1 / 28 (3.57%)
    0 / 23 (0.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Injury, poisoning and procedural complications
    Lower limb fracture
         subjects affected / exposed
    0 / 28 (0.00%)
    1 / 23 (4.35%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Nervous system disorders
    Cerebral haemorrhage
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 23 (0.00%)
    0 / 5 (0.00%)
    1 / 4 (25.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 1
    Seizure
         subjects affected / exposed
    1 / 28 (3.57%)
    0 / 23 (0.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Blood and lymphatic system disorders
    Autoimmune haemolytic anaemia
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 23 (0.00%)
    1 / 5 (20.00%)
    0 / 4 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Haemolytic anaemia
         subjects affected / exposed
    1 / 28 (3.57%)
    0 / 23 (0.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Thrombotic microangiopathy
         subjects affected / exposed
    1 / 28 (3.57%)
    1 / 23 (4.35%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Immune-mediated cytopenia
         subjects affected / exposed
    0 / 28 (0.00%)
    1 / 23 (4.35%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Gastrointestinal disorders
    Ascites
         subjects affected / exposed
    0 / 28 (0.00%)
    1 / 23 (4.35%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Diarrhoea
         subjects affected / exposed
    0 / 28 (0.00%)
    1 / 23 (4.35%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Gastrointestinal haemorrhage
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 23 (0.00%)
    1 / 5 (20.00%)
    0 / 4 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Nausea
         subjects affected / exposed
    1 / 28 (3.57%)
    0 / 23 (0.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Pancreatitis
         subjects affected / exposed
    2 / 28 (7.14%)
    0 / 23 (0.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences causally related to treatment / all
    1 / 2
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Hepatobiliary disorders
    Acute hepatic failure
         subjects affected / exposed
    1 / 28 (3.57%)
    0 / 23 (0.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
    Hepatic failure
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 23 (0.00%)
    0 / 5 (0.00%)
    1 / 4 (25.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Renal and urinary disorders
    Chronic kidney disease
         subjects affected / exposed
    0 / 28 (0.00%)
    1 / 23 (4.35%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Haematuria
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 23 (0.00%)
    1 / 5 (20.00%)
    0 / 4 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Nephrolithiasis
         subjects affected / exposed
    1 / 28 (3.57%)
    0 / 23 (0.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Renal tubular disorder
         subjects affected / exposed
    1 / 28 (3.57%)
    0 / 23 (0.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Infections and infestations
    Bacteraemia
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 23 (0.00%)
    1 / 5 (20.00%)
    0 / 4 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 2
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    COVID-19
         subjects affected / exposed
    2 / 28 (7.14%)
    0 / 23 (0.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Candida infection
         subjects affected / exposed
    0 / 28 (0.00%)
    1 / 23 (4.35%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Clostridium difficile colitis
         subjects affected / exposed
    1 / 28 (3.57%)
    0 / 23 (0.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Clostridium difficile infection
         subjects affected / exposed
    0 / 28 (0.00%)
    2 / 23 (8.70%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 2
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Cryptosporidiosis infection
         subjects affected / exposed
    0 / 28 (0.00%)
    1 / 23 (4.35%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Cytomegalovirus colitis
         subjects affected / exposed
    0 / 28 (0.00%)
    1 / 23 (4.35%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Cytomegalovirus infection reactivation
         subjects affected / exposed
    0 / 28 (0.00%)
    1 / 23 (4.35%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Device related infection
         subjects affected / exposed
    0 / 28 (0.00%)
    1 / 23 (4.35%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Enterocolitis infectious
         subjects affected / exposed
    0 / 28 (0.00%)
    1 / 23 (4.35%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Escherichia pyelonephritis
         subjects affected / exposed
    0 / 28 (0.00%)
    1 / 23 (4.35%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 2
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Gastroenteritis
         subjects affected / exposed
    1 / 28 (3.57%)
    1 / 23 (4.35%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Gastroenteritis rotavirus
         subjects affected / exposed
    1 / 28 (3.57%)
    0 / 23 (0.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Human herpesvirus 6 infection
         subjects affected / exposed
    0 / 28 (0.00%)
    1 / 23 (4.35%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Localised infection
         subjects affected / exposed
    1 / 28 (3.57%)
    0 / 23 (0.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Pancreatitis viral
         subjects affected / exposed
    0 / 28 (0.00%)
    1 / 23 (4.35%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Parainfluenzae virus infection
         subjects affected / exposed
    1 / 28 (3.57%)
    0 / 23 (0.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Parotitis
         subjects affected / exposed
    0 / 28 (0.00%)
    1 / 23 (4.35%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Phlebitis infective
         subjects affected / exposed
    0 / 28 (0.00%)
    1 / 23 (4.35%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Pneumococcal bacteraemia
         subjects affected / exposed
    1 / 28 (3.57%)
    0 / 23 (0.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Pneumonia
         subjects affected / exposed
    2 / 28 (7.14%)
    1 / 23 (4.35%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Respiratory syncytial virus infection
         subjects affected / exposed
    0 / 28 (0.00%)
    1 / 23 (4.35%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Sepsis
         subjects affected / exposed
    0 / 28 (0.00%)
    1 / 23 (4.35%)
    1 / 5 (20.00%)
    0 / 4 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Sinusitis
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 23 (0.00%)
    1 / 5 (20.00%)
    0 / 4 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Metabolism and nutrition disorders
    Electrolyte imbalance
         subjects affected / exposed
    1 / 28 (3.57%)
    0 / 23 (0.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Hypokalaemia
         subjects affected / exposed
    0 / 28 (0.00%)
    1 / 23 (4.35%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Hypophosphataemia
         subjects affected / exposed
    1 / 28 (3.57%)
    0 / 23 (0.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Metabolic acidosis
         subjects affected / exposed
    1 / 28 (3.57%)
    0 / 23 (0.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Posoleucel (Primary Study Period) Placebo (Primary Study Period) Posoleucel (Cross-over Period) Placebo (Cross-over Period)
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    27 / 28 (96.43%)
    23 / 23 (100.00%)
    5 / 5 (100.00%)
    3 / 4 (75.00%)
    Vascular disorders
    Hypertension
         subjects affected / exposed
    4 / 28 (14.29%)
    1 / 23 (4.35%)
    0 / 5 (0.00%)
    1 / 4 (25.00%)
         occurrences all number
    4
    1
    0
    1
    General disorders and administration site conditions
    Asthenia
         subjects affected / exposed
    1 / 28 (3.57%)
    1 / 23 (4.35%)
    1 / 5 (20.00%)
    0 / 4 (0.00%)
         occurrences all number
    1
    1
    1
    0
    Gait disturbance
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 23 (0.00%)
    1 / 5 (20.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Pyrexia
         subjects affected / exposed
    10 / 28 (35.71%)
    8 / 23 (34.78%)
    1 / 5 (20.00%)
    0 / 4 (0.00%)
         occurrences all number
    10
    8
    1
    0
    Immune system disorders
    Acute graft versus host disease in intestine
         subjects affected / exposed
    1 / 28 (3.57%)
    3 / 23 (13.04%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    1
    3
    0
    0
    Acute graft versus host disease in skin
         subjects affected / exposed
    2 / 28 (7.14%)
    1 / 23 (4.35%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    2
    1
    0
    0
    Cytokine release syndrome
         subjects affected / exposed
    1 / 28 (3.57%)
    3 / 23 (13.04%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    1
    3
    0
    0
    Graft versus host disease
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 23 (0.00%)
    0 / 5 (0.00%)
    1 / 4 (25.00%)
         occurrences all number
    0
    0
    0
    1
    Graft versus host disease in liver
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 23 (0.00%)
    0 / 5 (0.00%)
    1 / 4 (25.00%)
         occurrences all number
    0
    0
    0
    1
    Hypogammaglobulinaemia
         subjects affected / exposed
    3 / 28 (10.71%)
    0 / 23 (0.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    3
    0
    0
    0
    Reproductive system and breast disorders
    Pelvic pain
         subjects affected / exposed
    1 / 28 (3.57%)
    0 / 23 (0.00%)
    1 / 5 (20.00%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    1
    0
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    3 / 28 (10.71%)
    2 / 23 (8.70%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    3
    2
    0
    0
    Dyspnoea
         subjects affected / exposed
    3 / 28 (10.71%)
    0 / 23 (0.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    3
    0
    0
    0
    Epistaxis
         subjects affected / exposed
    2 / 28 (7.14%)
    1 / 23 (4.35%)
    1 / 5 (20.00%)
    0 / 4 (0.00%)
         occurrences all number
    2
    1
    1
    0
    Hypoxia
         subjects affected / exposed
    2 / 28 (7.14%)
    2 / 23 (8.70%)
    0 / 5 (0.00%)
    1 / 4 (25.00%)
         occurrences all number
    2
    2
    0
    1
    Pleural effusion
         subjects affected / exposed
    1 / 28 (3.57%)
    0 / 23 (0.00%)
    0 / 5 (0.00%)
    1 / 4 (25.00%)
         occurrences all number
    1
    0
    0
    1
    Tachypnoea
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 23 (0.00%)
    0 / 5 (0.00%)
    1 / 4 (25.00%)
         occurrences all number
    0
    0
    0
    1
    Investigations
    Activated partial thromboplastin time prolonged
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 23 (0.00%)
    0 / 5 (0.00%)
    1 / 4 (25.00%)
         occurrences all number
    0
    0
    0
    1
    Alanine aminotransferase increased
         subjects affected / exposed
    2 / 28 (7.14%)
    2 / 23 (8.70%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    2
    2
    0
    0
    Blood bicarbonate decreased
         subjects affected / exposed
    1 / 28 (3.57%)
    2 / 23 (8.70%)
    0 / 5 (0.00%)
    1 / 4 (25.00%)
         occurrences all number
    1
    2
    0
    1
    Blood bilirubin increased
         subjects affected / exposed
    2 / 28 (7.14%)
    1 / 23 (4.35%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    2
    1
    0
    0
    Blood creatinine increased
         subjects affected / exposed
    2 / 28 (7.14%)
    1 / 23 (4.35%)
    1 / 5 (20.00%)
    0 / 4 (0.00%)
         occurrences all number
    2
    1
    1
    0
    Enterobacter test positive
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 23 (0.00%)
    1 / 5 (20.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Gamma-glutamyltransferase increased
         subjects affected / exposed
    1 / 28 (3.57%)
    2 / 23 (8.70%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    1
    2
    0
    0
    Lipase increased
         subjects affected / exposed
    2 / 28 (7.14%)
    0 / 23 (0.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    2
    0
    0
    0
    Neutrophil count decreased
         subjects affected / exposed
    2 / 28 (7.14%)
    1 / 23 (4.35%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    2
    1
    0
    0
    Platelet count decreased
         subjects affected / exposed
    2 / 28 (7.14%)
    3 / 23 (13.04%)
    1 / 5 (20.00%)
    0 / 4 (0.00%)
         occurrences all number
    2
    3
    1
    0
    Weight decreased
         subjects affected / exposed
    2 / 28 (7.14%)
    2 / 23 (8.70%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    2
    2
    0
    0
    Injury, poisoning and procedural complications
    Traumatic haematoma
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 23 (0.00%)
    0 / 5 (0.00%)
    1 / 4 (25.00%)
         occurrences all number
    0
    0
    0
    1
    Congenital, familial and genetic disorders
    Phimosis
         subjects affected / exposed
    4 / 28 (14.29%)
    2 / 23 (8.70%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    4
    2
    0
    0
    Cardiac disorders
    Pericardial effusion
         subjects affected / exposed
    2 / 28 (7.14%)
    0 / 23 (0.00%)
    0 / 5 (0.00%)
    1 / 4 (25.00%)
         occurrences all number
    2
    0
    0
    1
    Sinus tachycardia
         subjects affected / exposed
    1 / 28 (3.57%)
    0 / 23 (0.00%)
    0 / 5 (0.00%)
    1 / 4 (25.00%)
         occurrences all number
    1
    0
    0
    1
    Nervous system disorders
    Somnolence
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 23 (0.00%)
    1 / 5 (20.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Blood and lymphatic system disorders
    Anaemia
         subjects affected / exposed
    6 / 28 (21.43%)
    5 / 23 (21.74%)
    2 / 5 (40.00%)
    1 / 4 (25.00%)
         occurrences all number
    6
    5
    2
    1
    Febrile neutropenia
         subjects affected / exposed
    2 / 28 (7.14%)
    1 / 23 (4.35%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    2
    1
    0
    0
    Thrombocytopenia
         subjects affected / exposed
    3 / 28 (10.71%)
    1 / 23 (4.35%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    3
    1
    0
    0
    Eye disorders
    Orbital haematoma
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 23 (0.00%)
    0 / 5 (0.00%)
    1 / 4 (25.00%)
         occurrences all number
    0
    0
    0
    1
    Vision blurred
         subjects affected / exposed
    0 / 28 (0.00%)
    2 / 23 (8.70%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    2
    0
    0
    Gastrointestinal disorders
    Abdominal distension
         subjects affected / exposed
    2 / 28 (7.14%)
    0 / 23 (0.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    2
    0
    0
    0
    Abdominal pain
         subjects affected / exposed
    6 / 28 (21.43%)
    2 / 23 (8.70%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    6
    2
    0
    0
    Ascites
         subjects affected / exposed
    2 / 28 (7.14%)
    0 / 23 (0.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    2
    0
    0
    0
    Constipation
         subjects affected / exposed
    3 / 28 (10.71%)
    2 / 23 (8.70%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    3
    2
    0
    0
    Diarrhoea
         subjects affected / exposed
    5 / 28 (17.86%)
    4 / 23 (17.39%)
    1 / 5 (20.00%)
    1 / 4 (25.00%)
         occurrences all number
    5
    4
    1
    1
    Gastritis
         subjects affected / exposed
    2 / 28 (7.14%)
    0 / 23 (0.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    2
    0
    0
    0
    Gastrointestinal haemorrhage
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 23 (0.00%)
    1 / 5 (20.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Nausea
         subjects affected / exposed
    6 / 28 (21.43%)
    1 / 23 (4.35%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    6
    1
    0
    0
    Stomatitis
         subjects affected / exposed
    0 / 28 (0.00%)
    3 / 23 (13.04%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    3
    0
    0
    Vomiting
         subjects affected / exposed
    8 / 28 (28.57%)
    4 / 23 (17.39%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    8
    4
    0
    0
    Hepatobiliary disorders
    Hypertransaminasaemia
         subjects affected / exposed
    2 / 28 (7.14%)
    2 / 23 (8.70%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    2
    2
    0
    0
    Skin and subcutaneous tissue disorders
    Dry skin
         subjects affected / exposed
    2 / 28 (7.14%)
    1 / 23 (4.35%)
    0 / 5 (0.00%)
    1 / 4 (25.00%)
         occurrences all number
    2
    1
    0
    1
    Rash
         subjects affected / exposed
    3 / 28 (10.71%)
    1 / 23 (4.35%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    3
    1
    0
    0
    Rash maculo-papular
         subjects affected / exposed
    3 / 28 (10.71%)
    1 / 23 (4.35%)
    1 / 5 (20.00%)
    0 / 4 (0.00%)
         occurrences all number
    3
    1
    1
    0
    Renal and urinary disorders
    Dysuria
         subjects affected / exposed
    2 / 28 (7.14%)
    1 / 23 (4.35%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    2
    1
    0
    0
    Glycosuria
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 23 (0.00%)
    1 / 5 (20.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Proteinuria
         subjects affected / exposed
    3 / 28 (10.71%)
    0 / 23 (0.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    3
    0
    0
    0
    Renal failure
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 23 (0.00%)
    0 / 5 (0.00%)
    1 / 4 (25.00%)
         occurrences all number
    0
    0
    0
    1
    Endocrine disorders
    Adrenal insufficiency
         subjects affected / exposed
    0 / 28 (0.00%)
    2 / 23 (8.70%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    2
    0
    0
    Musculoskeletal and connective tissue disorders
    Back pain
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 23 (0.00%)
    1 / 5 (20.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Pain in extremity
         subjects affected / exposed
    1 / 28 (3.57%)
    1 / 23 (4.35%)
    1 / 5 (20.00%)
    0 / 4 (0.00%)
         occurrences all number
    1
    1
    1
    0
    Infections and infestations
    Adenoviral hepatitis
         subjects affected / exposed
    2 / 28 (7.14%)
    0 / 23 (0.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    2
    0
    0
    0
    Adenovirus infection
         subjects affected / exposed
    3 / 28 (10.71%)
    0 / 23 (0.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    3
    0
    0
    0
    BK virus infection
         subjects affected / exposed
    3 / 28 (10.71%)
    1 / 23 (4.35%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    3
    1
    0
    0
    Bacteraemia
         subjects affected / exposed
    2 / 28 (7.14%)
    0 / 23 (0.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    2
    0
    0
    0
    COVID-19
         subjects affected / exposed
    3 / 28 (10.71%)
    1 / 23 (4.35%)
    0 / 5 (0.00%)
    1 / 4 (25.00%)
         occurrences all number
    3
    1
    0
    1
    Candida infection
         subjects affected / exposed
    2 / 28 (7.14%)
    1 / 23 (4.35%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    2
    1
    0
    0
    Cytomegalovirus infection reactivation
         subjects affected / exposed
    0 / 28 (0.00%)
    3 / 23 (13.04%)
    1 / 5 (20.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    3
    1
    0
    Cytomegalovirus viraemia
         subjects affected / exposed
    3 / 28 (10.71%)
    0 / 23 (0.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    3
    0
    0
    0
    Enterovirus infection
         subjects affected / exposed
    2 / 28 (7.14%)
    0 / 23 (0.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    2
    0
    0
    0
    Epstein-Barr viraemia
         subjects affected / exposed
    1 / 28 (3.57%)
    0 / 23 (0.00%)
    1 / 5 (20.00%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    1
    0
    Epstein-Barr virus infection reactivation
         subjects affected / exposed
    1 / 28 (3.57%)
    1 / 23 (4.35%)
    0 / 5 (0.00%)
    1 / 4 (25.00%)
         occurrences all number
    1
    1
    0
    1
    Gastroenteritis adenovirus
         subjects affected / exposed
    1 / 28 (3.57%)
    2 / 23 (8.70%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    1
    2
    0
    0
    Klebsiella bacteraemia
         subjects affected / exposed
    2 / 28 (7.14%)
    0 / 23 (0.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    2
    0
    0
    0
    Klebsiella infection
         subjects affected / exposed
    1 / 28 (3.57%)
    0 / 23 (0.00%)
    1 / 5 (20.00%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    1
    0
    Pneumonia
         subjects affected / exposed
    2 / 28 (7.14%)
    0 / 23 (0.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    2
    0
    0
    0
    Rhinovirus infection
         subjects affected / exposed
    2 / 28 (7.14%)
    3 / 23 (13.04%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    2
    3
    0
    0
    Sepsis
         subjects affected / exposed
    2 / 28 (7.14%)
    0 / 23 (0.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    2
    0
    0
    0
    Upper respiratory tract infection
         subjects affected / exposed
    3 / 28 (10.71%)
    0 / 23 (0.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    3
    0
    0
    0
    Viral rhinitis
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 23 (0.00%)
    1 / 5 (20.00%)
    0 / 4 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Metabolism and nutrition disorders
    Decreased appetite
         subjects affected / exposed
    1 / 28 (3.57%)
    2 / 23 (8.70%)
    1 / 5 (20.00%)
    0 / 4 (0.00%)
         occurrences all number
    1
    2
    1
    0
    Hyperammonaemia
         subjects affected / exposed
    0 / 28 (0.00%)
    0 / 23 (0.00%)
    0 / 5 (0.00%)
    1 / 4 (25.00%)
         occurrences all number
    0
    0
    0
    1
    Hypercalcaemia
         subjects affected / exposed
    1 / 28 (3.57%)
    0 / 23 (0.00%)
    1 / 5 (20.00%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    1
    0
    Hyperkalaemia
         subjects affected / exposed
    2 / 28 (7.14%)
    0 / 23 (0.00%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    2
    0
    0
    0
    Hypermagnesaemia
         subjects affected / exposed
    2 / 28 (7.14%)
    0 / 23 (0.00%)
    1 / 5 (20.00%)
    1 / 4 (25.00%)
         occurrences all number
    2
    0
    1
    1
    Hypervolaemia
         subjects affected / exposed
    2 / 28 (7.14%)
    1 / 23 (4.35%)
    1 / 5 (20.00%)
    0 / 4 (0.00%)
         occurrences all number
    2
    1
    1
    0
    Hypoalbuminaemia
         subjects affected / exposed
    2 / 28 (7.14%)
    2 / 23 (8.70%)
    1 / 5 (20.00%)
    0 / 4 (0.00%)
         occurrences all number
    2
    2
    1
    0
    Hypokalaemia
         subjects affected / exposed
    9 / 28 (32.14%)
    6 / 23 (26.09%)
    1 / 5 (20.00%)
    1 / 4 (25.00%)
         occurrences all number
    9
    6
    1
    1
    Hypomagnesaemia
         subjects affected / exposed
    5 / 28 (17.86%)
    3 / 23 (13.04%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    5
    3
    0
    0
    Hypophosphataemia
         subjects affected / exposed
    3 / 28 (10.71%)
    2 / 23 (8.70%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    3
    2
    0
    0
    Iron overload
         subjects affected / exposed
    2 / 28 (7.14%)
    1 / 23 (4.35%)
    0 / 5 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    2
    1
    0
    0

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    17 Dec 2021
    Major revisions include: - Labeled safety objectives and endpoints as primary. - Added a requirement for participants who receive a third dose for recurrence to undergo safety follow-up for 20 weeks after the third dose of study treatment. - Added a statement that participating sites will adhere to relevant safety standards. - Added a statement that participants will be approached for enrollment in a long-term registry study. - Provided additional guidance on permitted and prohibited medications and the recording of concomitant medications. - Updated text surrounding study treatment discontinuation. - Added the Intent-to-Treat Population. - Deleted the Modified Intent-to-Treat Population. - Updated the planned analysis of the primary efficacy endpoint to be analyzed based on logistic regression instead of Fisher’s exact test. - Provided justification for analysis of the primary efficacy endpoint at Day 29 and for durability of response assessments. - Revised schedule of assessments. - Provided details on interim analyses. - Added additional guidance on post-infusion monitoring. - Added a statement that all protocol amendments will be submitted to regulatory authorities and ethics committees/Institutional Review Boards as appropriate. - Added a statement that in some jurisdictions, legally effective informed consent must be obtained from all legal guardians for minor participants. - Provided guidance on estimating fraction of inspired oxygen.
    23 Aug 2022
    Major Revisions include: - Revised the order and timing of Study Objectives and Endpoints. - Revised Eligibility Criteria. - Added flexibility in blood specimen testing to allow blood specimen results from local laboratory results to be used for confirmation of eligibility and dosing if Adenovirus viral load results from the central laboratory are not available. - Expanded the screening window. - Added clarifying information regarding premature Crossover. - Added Data Safety Monitoring Board review and recommendation of preliminary safety data from 5 participants ≥1 and ≤6 years of age prior to enrolling participants under age 1. - Statistical Updates. - Added language regarding premedication with an antihistamine. - Streamlined study drug administration and dosing instructions. - Updated text to clarify that no subsequent infusion of blinded study treatment should occur in participants who develop new onset graft versus host disease (Grade >2) or worsening of graft versus host disease. - Added Clarifying information regarding Target Disease Endpoint Definitions.
    21 Sep 2023
    Major Revisions include: - Revised Eligibility Criteria. - Revised Concomitant Medications. - Analysis updates.
    30 Nov 2023
    Major Revisions include: - Addition of Interim analysis (sample size re-estimation and futility analysis). - Changes in Sample Size Determination. - Changes in Primary Analysis.

    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.
    The study was discontinued following a pre-planned DSMB futility analysis concluding that the study was unlikely to meet its primary endpoint, no safety concerns were identified.
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