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    Clinical Trial Results:
    A SINGLE ARM, OPEN-LABEL, MULTI-CENTRE, PHASE I/II STUDY EVALUATING THE SAFETY AND CLINICAL ACTIVITY OF AUTO4, A CAR T CELL TREATMENT TARGETING TRBC1, IN PATIENTS WITH RELAPSED OR REFRACTORY TRBC1 POSITIVE SELECTED T CELL NON-HODGKIN LYMPHOMA

    Summary
    EudraCT number
    		 2017-001965-26
    Trial protocol
    		 GB   ES  
    Global end of trial date
    12 Dec 2024

    Results information
    Results version number
    		 v1(current)
    This version publication date
    20 Dec 2025
    First version publication date
    20 Dec 2025
    Other versions

    Trial information

    		 Close Top of page
    Trial identification
    Sponsor protocol code
    AUTO4-TL1
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT03590574
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Autolus Limited
    Sponsor organisation address
    191 Wood Lane, London, United Kingdom,
    Public contact
    Clinical Project manager, Autolus Ltd, 44 02038296230, clinicaltrials@autolus.com
    Scientific contact
    Clinical Project manager, Autolus Ltd, 44 02038296230, clinicaltrials@autolus.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
    03 Mar 2025
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    12 Dec 2024
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    Phase I: To assess the safety and tolerability of AUTO4 administration. To identify the recommended phase II dose (RP2D) and maximum tolerated dose (MTD), if an MTD exists. Phase II: To assess the safety and clinical activity of AUTO4 when administered at the RP2D
    Protection of trial subjects
    Standard drugs and palliative radiotherapy required by the participant could be administered alongside the trial protocol. Participants could receive bridging therapy, between leukapheresis and admission for pre-conditioning therapy, prior to AUTO4 infusion.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    13 Sep 2018
    Long term follow-up planned
    Yes
    Long term follow-up rationale
    		 Safety
    Long term follow-up duration
    		 15 Years
    Independent data monitoring committee (IDMC) involvement?
    Yes
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    Spain: 5
    Country: Number of subjects enrolled
    United Kingdom: 15
    Worldwide total number of subjects
    20
    EEA total number of subjects
    5
    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)
    0
    Adults (18-64 years)
    16
    From 65 to 84 years
    4
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 First participant enrolled: 22-Sep-2018 Last participant enrolled: 10-Oct-2023

    Pre-assignment
    Screening details
    		 -

    Pre-assignment period milestones
    Number of subjects started
    		 125 [1]
    Number of subjects completed
    		 15

    Pre-assignment subject non-completion reasons
    Reason: Number of subjects
    		 No TRBC1 status: 7
    Reason: Number of subjects
    		 TRBC1 negative: 68
    Reason: Number of subjects
    		 TRBC1 not evaluable: 10
    Reason: Number of subjects
    		 TRBC1 positive but not enrolled: 20
    Reason: Number of subjects
    		 enrolled - died prior to infusion: 2
    Reason: Number of subjects
    		 enrolled - withdrew due to "other" before infusion: 3
    Notes
    [1] - The number of subjects reported to have started the pre-assignment period are not the same as the worldwide number enrolled in the trial. It is expected that these numbers will be the same.
    Justification: The pre-assignment period includes all screened subjects. Of these 20 enrolled (the worldwide number enrolled), but 5 withdrew prior to receiving AUTO4 infusion, such that 15 patients are analysed (baseline period).
    Period 1
    Period 1 title
    Dose Escalation (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Non-randomised - controlled
    Blinding used
    		 Not blinded

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Cohort 1 (25x10^6 cells)
    Arm description
    		 Adult patients with relapsed or refractory TRBC1 positive selected T cell non-Hodgkin lymphoma. Pre-conditioning with Flu & CY; Days -6, -5, -4 and -3. Cohort 1 dosed with 25x10^6 CAR T cells using the original manufacturing process.
    Arm type
    		 Experimental

    Investigational medicinal product name
    AUTO4 (RQR8/ANTI-TRBC1 CAR Positive T Cells)
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Solution for infusion
    Routes of administration
    Infusion
    Dosage and administration details
    Following pre-conditioning with chemotherapy (fludarabine and cyclophosphamide) over Days -6 to -3, patients were treated with a single infusion of RQR8/aTRBC1-CAR positive T cells on Day 0. Provided that all criteria were satisfied, patients could receive re-treatment (second cycle) of AUTO4.

    Arm title
    		 Cohort 2 (75x10^6 cells)
    Arm description
    		 Adult patients with relapsed or refractory TRBC1 positive selected T cell non-Hodgkin lymphoma. Pre-conditioning with Flu & CY; Days -6, -5, -4 and -3. Cohort 2 dosed with 75x10^6 CAR T cells using the original manufacturing process.
    Arm type
    		 Experimental

    Investigational medicinal product name
    AUTO4 (RQR8/ANTI-TRBC1 CAR Positive T Cells)
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Solution for infusion
    Routes of administration
    Infusion
    Dosage and administration details
    Following pre-conditioning with chemotherapy (fludarabine and cyclophosphamide) over Days -6 to -3, patients were treated with a single infusion of RQR8/aTRBC1-CAR positive T cells on Day 0. Provided that all criteria were satisfied, patients could receive re-treatment (second cycle) of AUTO4.

    Arm title
    		 Cohort 3 (225x10^6 cells)
    Arm description
    		 Adult patients with relapsed or refractory TRBC1 positive selected T cell non-Hodgkin lymphoma. Pre-conditioning with Flu & CY; Days -6, -5, -4 and -3. Cohort 3 dosed with 225x10^6 CAR T cells using the original manufacturing process.
    Arm type
    		 Experimental

    Investigational medicinal product name
    AUTO4 (RQR8/ANTI-TRBC1 CAR Positive T Cells)
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Solution for infusion
    Routes of administration
    Infusion
    Dosage and administration details
    Following pre-conditioning with chemotherapy (fludarabine and cyclophosphamide) over Days -6 to -3, patients were treated with a single infusion of RQR8/aTRBC1-CAR positive T cells on Day 0. Provided that all criteria were satisfied, patients could receive re-treatment (second cycle) of AUTO4.

    Arm title
    		 Cohort 4 (450x10^6 cells)
    Arm description
    		 Adult patients with relapsed or refractory TRBC1 positive selected T cell non-Hodgkin lymphoma. Pre-conditioning with Flu & CY; Days -6, -5, -4 and -3. Cohort 4 dosed with 450x10^6 CAR T cells using the original manufacturing process.
    Arm type
    		 Experimental

    Investigational medicinal product name
    AUTO4 (RQR8/ANTI-TRBC1 CAR Positive T Cells)
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Solution for infusion
    Routes of administration
    Infusion
    Dosage and administration details
    Following pre-conditioning with chemotherapy (fludarabine and cyclophosphamide) over Days -6 to -3, patients were treated with a single infusion of RQR8/aTRBC1-CAR positive T cells on Day 0. Provided that all criteria were satisfied, patients could receive re-treatment (second cycle) of AUTO4.

    Arm title
    		 Cohort 3B (225x10^6 cells)
    Arm description
    		 Adult patients with relapsed or refractory TRBC1 positive selected T cell non-Hodgkin lymphoma. Pre-conditioning with Flu & CY; Days -6, -5, -4 and -3. Cohort 3B dosed with 225x10^6 CAR T cells using the modified manufacturing process.
    Arm type
    		 Experimental

    Investigational medicinal product name
    AUTO4 (RQR8/ANTI-TRBC1 CAR Positive T Cells)
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Solution for infusion
    Routes of administration
    Infusion
    Dosage and administration details
    Following pre-conditioning with chemotherapy (fludarabine and cyclophosphamide) over Days -6 to -3, patients were treated with a single infusion of RQR8/aTRBC1-CAR positive T cells on Day 0. Provided that all criteria were satisfied, patients could receive re-treatment (second cycle) of AUTO4.

    Arm title
    		 Cohort 4B (450x10^6 cells)
    Arm description
    		 Adult patients with relapsed or refractory TRBC1 positive selected T cell non-Hodgkin lymphoma. Pre-conditioning with Flu & CY; Days -6, -5, -4 and -3. Cohort 4B dosed with 450x10^6 CAR T cells using the modified manufacturing process.
    Arm type
    		 Experimental

    Investigational medicinal product name
    AUTO4 (RQR8/ANTI-TRBC1 CAR Positive T Cells)
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Solution for infusion
    Routes of administration
    Infusion
    Dosage and administration details
    Following pre-conditioning with chemotherapy (fludarabine and cyclophosphamide) over Days -6 to -3, patients were treated with a single infusion of RQR8/aTRBC1-CAR positive T cells on Day 0. Provided that all criteria were satisfied, patients could receive re-treatment (second cycle) of AUTO4.

    Arm title
    		 Cohort 5B (900x10^6 cells)
    Arm description
    		 Adult patients with relapsed or refractory TRBC1 positive selected T cell non-Hodgkin lymphoma. Pre-conditioning with Flu & CY; Days -6, -5, -4 and -3. Cohort 5B dosed with 900x10^6 CAR T cells using the modified manufacturing process.
    Arm type
    		 Experimental

    Investigational medicinal product name
    AUTO4 (RQR8/ANTI-TRBC1 CAR Positive T Cells)
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Solution for infusion
    Routes of administration
    Infusion
    Dosage and administration details
    Following pre-conditioning with chemotherapy (fludarabine and cyclophosphamide) over Days -6 to -3, patients were treated with a single infusion of RQR8/aTRBC1-CAR positive T cells on Day 0. Provided that all criteria were satisfied, patients could receive re-treatment (second cycle) of AUTO4.

    Number of subjects in period 1 [2]
    		Cohort 1 (25x10^6 cells) Cohort 2 (75x10^6 cells) Cohort 3 (225x10^6 cells) Cohort 4 (450x10^6 cells) Cohort 3B (225x10^6 cells) Cohort 4B (450x10^6 cells) Cohort 5B (900x10^6 cells)
    Started
    3
    2
    1
    4
    1
    3
    1
    Completed
    1
    1
    0
    2
    0
    0
    0
    Not completed
    2
    1
    1
    2
    1
    3
    1
         Adverse event, serious fatal
    -
    -
    -
    1
    -
    -
    1
         Consent withdrawn by subject
    -
    -
    -
    1
    1
    2
    -
         Other
    2
    1
    1
    -
    -
    -
    -
         Death
    -
    -
    -
    -
    -
    1
    -
    Notes
    [2] - The number of subjects reported to be in the baseline period are not the same as the worldwide number enrolled in the trial. It is expected that these numbers will be the same.
    Justification: Five participants enrolled did not receive AUTO4 infusion (2 died, 3 withdrew due to 'other' reasons). These participants are not included in the analyses.

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Cohort 1 (25x10^6 cells)
    Reporting group description
    		 Adult patients with relapsed or refractory TRBC1 positive selected T cell non-Hodgkin lymphoma. Pre-conditioning with Flu & CY; Days -6, -5, -4 and -3. Cohort 1 dosed with 25x10^6 CAR T cells using the original manufacturing process.

    Reporting group title
    Cohort 2 (75x10^6 cells)
    Reporting group description
    		 Adult patients with relapsed or refractory TRBC1 positive selected T cell non-Hodgkin lymphoma. Pre-conditioning with Flu & CY; Days -6, -5, -4 and -3. Cohort 2 dosed with 75x10^6 CAR T cells using the original manufacturing process.

    Reporting group title
    Cohort 3 (225x10^6 cells)
    Reporting group description
    		 Adult patients with relapsed or refractory TRBC1 positive selected T cell non-Hodgkin lymphoma. Pre-conditioning with Flu & CY; Days -6, -5, -4 and -3. Cohort 3 dosed with 225x10^6 CAR T cells using the original manufacturing process.

    Reporting group title
    Cohort 4 (450x10^6 cells)
    Reporting group description
    		 Adult patients with relapsed or refractory TRBC1 positive selected T cell non-Hodgkin lymphoma. Pre-conditioning with Flu & CY; Days -6, -5, -4 and -3. Cohort 4 dosed with 450x10^6 CAR T cells using the original manufacturing process.

    Reporting group title
    Cohort 3B (225x10^6 cells)
    Reporting group description
    		 Adult patients with relapsed or refractory TRBC1 positive selected T cell non-Hodgkin lymphoma. Pre-conditioning with Flu & CY; Days -6, -5, -4 and -3. Cohort 3B dosed with 225x10^6 CAR T cells using the modified manufacturing process.

    Reporting group title
    Cohort 4B (450x10^6 cells)
    Reporting group description
    		 Adult patients with relapsed or refractory TRBC1 positive selected T cell non-Hodgkin lymphoma. Pre-conditioning with Flu & CY; Days -6, -5, -4 and -3. Cohort 4B dosed with 450x10^6 CAR T cells using the modified manufacturing process.

    Reporting group title
    Cohort 5B (900x10^6 cells)
    Reporting group description
    		 Adult patients with relapsed or refractory TRBC1 positive selected T cell non-Hodgkin lymphoma. Pre-conditioning with Flu & CY; Days -6, -5, -4 and -3. Cohort 5B dosed with 900x10^6 CAR T cells using the modified manufacturing process.

    Reporting group values
    		 Cohort 1 (25x10^6 cells) Cohort 2 (75x10^6 cells) Cohort 3 (225x10^6 cells) Cohort 4 (450x10^6 cells) Cohort 3B (225x10^6 cells) Cohort 4B (450x10^6 cells) Cohort 5B (900x10^6 cells) Total
    Number of subjects
    		 3 2 1 4 1 3 1 15
    Age categorical
    Units: Subjects
        In utero
    		 0 0 0 0 0 0 0 0
        Preterm newborn infants (gestational age < 37 wks)
    		 0 0 0 0 0 0 0 0
        Newborns (0-27 days)
    		 0 0 0 0 0 0 0 0
        Infants and toddlers (28 days-23 months)
    		 0 0 0 0 0 0 0 0
        Children (2-11 years)
    		 0 0 0 0 0 0 0 0
        Adolescents (12-17 years)
    		 0 0 0 0 0 0 0 0
        Adults (18-64 years)
    		 3 2 1 4 0 3 0 13
        From 65-84 years
    		 0 0 0 0 1 0 1 2
        85 years and over
    		 0 0 0 0 0 0 0 0
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    		 50.7 ( 14.57 ) 44.0 ( 12.73 ) 47.0 ( 0 ) 56.5 ( 8.58 ) 72.0 ( 0 ) 58.7 ( 3.51 ) 70.0 ( 0 ) -
    Gender categorical
    Units: Subjects
        Female
    		 2 1 0 0 0 0 0 3
        Male
    		 1 1 1 4 1 3 1 12
    Stage of Lymphoma at screening
    Units: Subjects
        One
    		 0 0 0 0 0 0 0 0
        Two
    		 0 2 0 0 0 0 0 2
        Three
    		 1 0 1 3 0 1 0 6
        Four
    		 2 0 0 1 1 2 1 7
    ECOG Score
    Units: Subjects
        0 - Fully Active
    		 1 1 0 1 0 1 1 5
        1 - Restricted
    		 2 1 1 3 1 2 0 10
        2 - Ambulatory
    		 0 0 0 0 0 0 0 0
        3 - Limited Self-care
    		 0 0 0 0 0 0 0 0
        4 - Completely Disabled
    		 0 0 0 0 0 0 0 0
        5 - Death
    		 0 0 0 0 0 0 0 0

    End points

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    End points reporting groups
    Reporting group title
    Cohort 1 (25x10^6 cells)
    Reporting group description
    		 Adult patients with relapsed or refractory TRBC1 positive selected T cell non-Hodgkin lymphoma. Pre-conditioning with Flu & CY; Days -6, -5, -4 and -3. Cohort 1 dosed with 25x10^6 CAR T cells using the original manufacturing process.

    Reporting group title
    Cohort 2 (75x10^6 cells)
    Reporting group description
    		 Adult patients with relapsed or refractory TRBC1 positive selected T cell non-Hodgkin lymphoma. Pre-conditioning with Flu & CY; Days -6, -5, -4 and -3. Cohort 2 dosed with 75x10^6 CAR T cells using the original manufacturing process.

    Reporting group title
    Cohort 3 (225x10^6 cells)
    Reporting group description
    		 Adult patients with relapsed or refractory TRBC1 positive selected T cell non-Hodgkin lymphoma. Pre-conditioning with Flu & CY; Days -6, -5, -4 and -3. Cohort 3 dosed with 225x10^6 CAR T cells using the original manufacturing process.

    Reporting group title
    Cohort 4 (450x10^6 cells)
    Reporting group description
    		 Adult patients with relapsed or refractory TRBC1 positive selected T cell non-Hodgkin lymphoma. Pre-conditioning with Flu & CY; Days -6, -5, -4 and -3. Cohort 4 dosed with 450x10^6 CAR T cells using the original manufacturing process.

    Reporting group title
    Cohort 3B (225x10^6 cells)
    Reporting group description
    		 Adult patients with relapsed or refractory TRBC1 positive selected T cell non-Hodgkin lymphoma. Pre-conditioning with Flu & CY; Days -6, -5, -4 and -3. Cohort 3B dosed with 225x10^6 CAR T cells using the modified manufacturing process.

    Reporting group title
    Cohort 4B (450x10^6 cells)
    Reporting group description
    		 Adult patients with relapsed or refractory TRBC1 positive selected T cell non-Hodgkin lymphoma. Pre-conditioning with Flu & CY; Days -6, -5, -4 and -3. Cohort 4B dosed with 450x10^6 CAR T cells using the modified manufacturing process.

    Reporting group title
    Cohort 5B (900x10^6 cells)
    Reporting group description
    		 Adult patients with relapsed or refractory TRBC1 positive selected T cell non-Hodgkin lymphoma. Pre-conditioning with Flu & CY; Days -6, -5, -4 and -3. Cohort 5B dosed with 900x10^6 CAR T cells using the modified manufacturing process.

    Subject analysis set title
    Infused Set - Phase I
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    The Infused Set comprises all patients who have received at least one infusion of AUTO4 treatment.

    Primary: Incidence of Grade 3 to Grade 5 toxicity occurring within 60 days of AUTO4 infusion.

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    End point title
    		 Incidence of Grade 3 to Grade 5 toxicity occurring within 60 days of AUTO4 infusion. [1]
    End point description
    To assess the safety and tolerability of AUTO4 administration. The incidence of Grade 3-5 toxicities occurring within 60 days of AUTO4 infusion. The analysis was conducted on the safety set which included all 15 participants enrolled and treated in the Phase 1 dose escalation.
    End point type
    Primary
    End point timeframe
    Within 60 days of AUTO4 infusion.
    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 is a Phase 1 study and evaluation of toxicity is not associated with a statistical analyses
    End point values
    		 Cohort 1 (25x10^6 cells) Cohort 2 (75x10^6 cells) Cohort 3 (225x10^6 cells) Cohort 4 (450x10^6 cells) Cohort 3B (225x10^6 cells) Cohort 4B (450x10^6 cells) Cohort 5B (900x10^6 cells)
    Number of subjects analysed
    3
    2
    1
    4
    1
    3
    1
    Units: Participants
    3
    2
    1
    4
    1
    0
    1
    No statistical analyses for this end point

    Primary: Frequency of DLT of AUTO4 within 28 days of AUTO4 infusion.

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    End point title
    		 Frequency of DLT of AUTO4 within 28 days of AUTO4 infusion. [2]
    End point description
    To identify the RP2D and MTD, if an MTD exists, of AUTO4 by monitoring the frequency of DLT of AUTO4 within 28 days of AUTO4 infusion. The analysis was conducted on the safety set which included all 15 participants enrolled and treated in the Phase 1 dose escalation.
    End point type
    Primary
    End point timeframe
    28 days of AUTO4 infusion
    Notes
    [2] - 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 is a Phase 1 study and reporting of DLTs is not associated with a statistical analyses
    End point values
    		 Cohort 1 (25x10^6 cells) Cohort 2 (75x10^6 cells) Cohort 3 (225x10^6 cells) Cohort 4 (450x10^6 cells) Cohort 3B (225x10^6 cells) Cohort 4B (450x10^6 cells) Cohort 5B (900x10^6 cells)
    Number of subjects analysed
    3
    2
    1
    4
    1
    3
    1
    Units: Participants
    0
    0
    0
    0
    0
    0
    1
    No statistical analyses for this end point

    Secondary: Frequency and severity of all adverse events and serious adverse events.

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    End point title
    		 Frequency and severity of all adverse events and serious adverse events.
    End point description
    All adverse events (AEs)/serious adverse events (SAEs) were recorded from admission for pre-conditioning chemotherapy (Day -6 relative to AUTO4). Due to the long period between consent and AUTO4 treatment, any AEs/SAEs related to bridging chemotherapy not associated with study procedures did not require reporting as study AEs/SAEs. Any significant events were added to the patient's medical history. All AEs/SAEs related to study procedures (leukapheresis, bone marrow assessments) were reported. The analysis was conducted on the safety set which included all 15 participants enrolled and treated in the Phase 1 dose escalation.
    End point type
    Secondary
    End point timeframe
    24 months post-treatment
    End point values
    		 Cohort 1 (25x10^6 cells) Cohort 2 (75x10^6 cells) Cohort 3 (225x10^6 cells) Cohort 4 (450x10^6 cells) Cohort 3B (225x10^6 cells) Cohort 4B (450x10^6 cells) Cohort 5B (900x10^6 cells)
    Number of subjects analysed
    3
    2
    1
    4
    1
    3
    1
    Units: Participants
        Patients with any AE
    3
    2
    1
    4
    1
    2
    1
        Patients with any AE of Grade 3 or higher
    3
    2
    1
    4
    1
    1
    1
        Patients with any SAE
    2
    1
    0
    2
    0
    2
    1
        Patients with any SAE of Grade 3 or higher
    2
    0
    0
    1
    0
    1
    1
    No statistical analyses for this end point

    Secondary: Incidence and severity of opportunistic infections following AUTO4 infusion.

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    End point title
    		 Incidence and severity of opportunistic infections following AUTO4 infusion.
    End point description
    Incidence and severity of opportunistic infections following AUTO4 infusion. The analysis was conducted on the safety set which included all 15 participants enrolled and treated in the Phase 1 dose escalation.
    End point type
    Secondary
    End point timeframe
    24 months post-treatment
    End point values
    		 Cohort 1 (25x10^6 cells) Cohort 2 (75x10^6 cells) Cohort 3 (225x10^6 cells) Cohort 4 (450x10^6 cells) Cohort 3B (225x10^6 cells) Cohort 4B (450x10^6 cells) Cohort 5B (900x10^6 cells)
    Number of subjects analysed
    3
    2
    1
    4
    1
    3
    1
    Units: Participants
        Any infection or infestation post-infusion
    3
    1
    1
    1
    1
    1
    1
        Any infection or infestation ≥Grade3 post-infusion
    1
    0
    0
    0
    0
    1
    1
    No statistical analyses for this end point

    Secondary: Complete response (CR) rate

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    End point title
    		 Complete response (CR) rate
    End point description
    Participants achieving objective response per Lugano criteria based on independent central radiology review. The Lugano classification of response by FDG PET-CT: 1. no uptake or no residual uptake (when used interim) 2. slight uptake, but below blood pool (mediastinum) 3. uptake above mediastinal, but below or equal to uptake in the liver 4. uptake slightly to moderately higher than liver 5. markedly increased uptake or any new lesion (on response evaluation) Non-progressive disease: • Complete metabolic response – score of 1, 2, or 3 in nodal or extranodal sites with or without a residual mass • Partial metabolic response – score of 4 or 5 with reduced uptake compared with baseline and residual mass(es) of any size • Stable disease or no metabolic response – score of 4 or 5 with no obvious change in FDG uptake Progressive disease score of 4 or 5 in any lesion with an increase in intensity of FDG uptake from baseline.
    End point type
    Secondary
    End point timeframe
    Up to end of study
    End point values
    		 Cohort 1 (25x10^6 cells) Cohort 2 (75x10^6 cells) Cohort 3 (225x10^6 cells) Cohort 4 (450x10^6 cells) Cohort 3B (225x10^6 cells) Cohort 4B (450x10^6 cells) Cohort 5B (900x10^6 cells) Infused Set - Phase I
    Number of subjects analysed
    3
    2
    1
    4
    1
    3
    1
    15
    Units: Participants
    1
    0
    1
    3
    0
    0
    0
    5
    No statistical analyses for this end point

    Secondary: Progression-free survival

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    End point title
    		 Progression-free survival
    End point description
    PFS was defined as the time from the first treatment of AUTO4 to documented disease progression/relapse or death due to any cause. If a patient did not have relapse or death due to any reason prior to data cut-off, PFS was censored at the date of the last adequate assessment by default. Patients who proceeded to SCT after AUTO4 infusion were censored at the time of SCT (including the conditioning regimen for SCT). Patients who received new non-protocol anti-cancer therapies other than SCT were censored as the date of last adequate assessment prior to new therapy. Patients who experienced event after missing two or more scheduled disease assessments were censored at the date of last adequate assessment prior to the event. The analysis was conducted on the infused set which included all 15 participants enrolled and treated in the Phase 1 dose escalation.
    End point type
    Secondary
    End point timeframe
    Up to 30 months
    End point values
    		 Infused Set - Phase I
    Number of subjects analysed
    15
    Units: months
        median (confidence interval 95%)
    2.89 (0.95 to 6.54)
    No statistical analyses for this end point

    Secondary: Overall survival

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    End point title
    		 Overall survival
    End point description
    Number of participants alive at end of study following treatment with AUTO4. No median overall survival was evaluable.
    End point type
    Secondary
    End point timeframe
    Up to 30 months
    End point values
    		 Infused Set - Phase I
    Number of subjects analysed
    15
    Units: Participants
    9
    No statistical analyses for this end point

    Secondary: Time to response (PR and CR).

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    End point title
    		 Time to response (PR and CR).
    End point description
    To evaluate the overall clinical efficacy of AUTO4. The analysis was conducted on the infused set which included all 15 participants enrolled and treated in the Phase 1 dose escalation.
    End point type
    Secondary
    End point timeframe
    24 months post-treatment
    End point values
    		 Cohort 1 (25x10^6 cells) Cohort 2 (75x10^6 cells) Cohort 3 (225x10^6 cells) Cohort 4 (450x10^6 cells) Cohort 3B (225x10^6 cells) Cohort 4B (450x10^6 cells) Cohort 5B (900x10^6 cells)
    Number of subjects analysed
    3
    2
    1
    4
    1
    3
    1
    Units: Participants
        By Month 1
    1
    0
    1
    3
    0
    0
    1
        By Month 3
    0
    1
    0
    0
    0
    0
    0
        By Month 6
    0
    0
    0
    0
    0
    0
    0
        By Month 9
    0
    0
    0
    1
    0
    0
    0
        By Month 12
    0
    0
    0
    0
    0
    0
    0
        By Month 18
    0
    0
    0
    0
    0
    0
    0
        By Month 24
    0
    0
    0
    0
    0
    0
    0
    No statistical analyses for this end point

    Secondary: RQR8/aTRBC1-CAR positive T cells as determined by polymerase chain reaction and/or flow cytometry at a range of time points in the peripheral blood.

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    End point title
    		 RQR8/aTRBC1-CAR positive T cells as determined by polymerase chain reaction and/or flow cytometry at a range of time points in the peripheral blood.
    End point description
    To determine the expansion and persistence of AUTO4 following infusion. The analysis was conducted on the infused set which included all 15 participants enrolled and treated in the Phase 1 dose escalation.
    End point type
    Secondary
    End point timeframe
    At 24 months
    End point values
    		 Cohort 1 (25x10^6 cells) Cohort 2 (75x10^6 cells) Cohort 3 (225x10^6 cells) Cohort 4 (450x10^6 cells) Cohort 3B (225x10^6 cells) Cohort 4B (450x10^6 cells) Cohort 5B (900x10^6 cells)
    Number of subjects analysed
    0 [3]
    1 [4]
    0 [5]
    2 [6]
    0 [7]
    0 [8]
    0 [9]
    Units: copies/microgram DNA
        median (full range (min-max))
    ( to )
    0 (0 to 0)
    ( to )
    0 (0 to 0)
    ( to )
    ( to )
    ( to )
    Attachments
    		 Individual Concentration-time Profiles (PCR)
    Notes
    [3] - Participants with available data at Month 24
    [4] - Participants with available data at Month 24
    [5] - Participants with available data at Month 24
    [6] - Participants with available data at Month 24
    [7] - Participants with available data at Month 24
    [8] - Participants with available data at Month 24
    [9] - Participants with available data at Month 24
    No statistical analyses for this end point

    Secondary: Enumeration of circulating TRBC1 positive T cells assessed by flow cytometry at a range of time points in the peripheral blood.

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    End point title
    		 Enumeration of circulating TRBC1 positive T cells assessed by flow cytometry at a range of time points in the peripheral blood.
    End point description
    Duration of TRBC1 positive T cell aplasia. The analysis was conducted on the infused set which included all 15 participants enrolled and treated in the Phase 1 dose escalation.
    End point type
    Secondary
    End point timeframe
    At 24 months
    End point values
    		 Cohort 1 (25x10^6 cells) Cohort 2 (75x10^6 cells) Cohort 3 (225x10^6 cells) Cohort 4 (450x10^6 cells) Cohort 3B (225x10^6 cells) Cohort 4B (450x10^6 cells) Cohort 5B (900x10^6 cells)
    Number of subjects analysed
    0 [10]
    1 [11]
    0 [12]
    0 [13]
    0 [14]
    0 [15]
    0 [16]
    Units: cells/microlitre
        median (full range (min-max))
    ( to )
    61.0 (61.0 to 61.0)
    ( to )
    ( to )
    ( to )
    ( to )
    ( to )
    Attachments
    		 Individual Concentration-time Profiles (FC)
    Notes
    [10] - Data only analysed for participants with available data at the timepoint
    [11] - Data only analysed for participants with available data at the timepoint
    [12] - Data only analysed for participants with available data at the timepoint
    [13] - Data only analysed for participants with available data at the timepoint
    [14] - Data only analysed for participants with available data at the timepoint
    [15] - Data only analysed for participants with available data at the timepoint
    [16] - Data only analysed for participants with available data at the timepoint
    No statistical analyses for this end point

    Secondary: Duration of response

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    End point title
    		 Duration of response
    End point description
    DOR is defined as the time from the first observed CR or PR to documented disease progression or death due to any cause, for patients who are considered as responders.
    End point type
    Secondary
    End point timeframe
    Up to 27 months
    End point values
    		 Infused Set - Phase I
    Number of subjects analysed
    15
    Units: Months
        median (confidence interval 95%)
    4.27 (0.99 to 25.07)
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    From Day -6 up to 24 months. After Day 60, only the following were collected: SAEs and treatment-related non-serious AEs; AEs of special interest; AEs related to a study procedure.
    Adverse event reporting additional description
    Only AEs/SAEs related to study procedures were collected until admission for lymphodeletion chemotherapy. AEs related to intervening/bridging non-study related anti-cancer therapy administered prior to pre-conditioning or AEs associated with disease progression were not reported as AEs but were recorded as an update to the patients medical history.
    Assessment type
    		 Non-systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    25.0
    Reporting groups
    Reporting group title
    Cohort 1 (25x10^6 cells)
    Reporting group description
    		 Adult patients with relapsed or refractory TRBC1 positive selected T cell non-Hodgkin lymphoma. Pre-conditioning with Flu & CY; Days -6, -5, -4 and -3. Cohort 1 dosed with 25x10^6 CAR T cells using the original manufacturing process.

    Reporting group title
    Cohort 2 (75x10^6 cells)
    Reporting group description
    		 Adult patients with relapsed or refractory TRBC1 positive selected T cell non-Hodgkin lymphoma. Pre-conditioning with Flu & CY; Days -6, -5, -4 and -3. Cohort 2 dosed with 75x10^6 CAR T cells using the original manufacturing process.

    Reporting group title
    Cohort 3 (225x10^6 cells)
    Reporting group description
    		 Adult patients with relapsed or refractory TRBC1 positive selected T cell non-Hodgkin lymphoma. Pre-conditioning with Flu & CY; Days -6, -5, -4 and -3. Cohort 3 dosed with 225x10^6 CAR T cells using the original manufacturing process.

    Reporting group title
    Cohort 4 (450x10^6 cells)
    Reporting group description
    		 Adult patients with relapsed or refractory TRBC1 positive selected T cell non-Hodgkin lymphoma. Pre-conditioning with Flu & CY; Days -6, -5, -4 and -3. Cohort 4 dosed with 450x10^6 CAR T cells using the original manufacturing process.

    Reporting group title
    Cohort 3B (225x10^6 cells)
    Reporting group description
    		 Adult patients with relapsed or refractory TRBC1 positive selected T cell non-Hodgkin lymphoma. Pre-conditioning with Flu & CY; Days -6, -5, -4 and -3. Cohort 3B dosed with 225x10^6 CAR T cells using the modified manufacturing process.

    Reporting group title
    Cohort 4B (450x10^6 cells)
    Reporting group description
    		 Adult patients with relapsed or refractory TRBC1 positive selected T cell non-Hodgkin lymphoma. Pre-conditioning with Flu & CY; Days -6, -5, -4 and -3. Cohort 4B dosed with 450x10^6 CAR T cells using the modified manufacturing process.

    Reporting group title
    Cohort 5B (900x10^6 cells)
    Reporting group description
    		 Adult patients with relapsed or refractory TRBC1 positive selected T cell non-Hodgkin lymphoma. Pre-conditioning with Flu & CY; Days -6, -5, -4 and -3. Cohort 5B dosed with 900x10^6 CAR T cells using the modified manufacturing process.

    Reporting group title
    Total
    Reporting group description
    		 All patients infused.

    Serious adverse events
    		 Cohort 1 (25x10^6 cells) Cohort 2 (75x10^6 cells) Cohort 3 (225x10^6 cells) Cohort 4 (450x10^6 cells) Cohort 3B (225x10^6 cells) Cohort 4B (450x10^6 cells) Cohort 5B (900x10^6 cells) Total
    Total subjects affected by serious adverse events
         subjects affected / exposed
    2 / 3 (66.67%)
    1 / 2 (50.00%)
    0 / 1 (0.00%)
    2 / 4 (50.00%)
    0 / 1 (0.00%)
    2 / 3 (66.67%)
    1 / 1 (100.00%)
    8 / 15 (53.33%)
         number of deaths (all causes)
    1
    1
    0
    1
    0
    2
    1
    6
         number of deaths resulting from adverse events
    0
    0
    0
    1
    0
    0
    1
    2
    Investigations
    Alanine aminotransferase increased
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    0 / 1 (0.00%)
    1 / 3 (33.33%)
    0 / 1 (0.00%)
    1 / 15 (6.67%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    1 / 1
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Aspartate aminotransferase increased
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    0 / 1 (0.00%)
    1 / 3 (33.33%)
    0 / 1 (0.00%)
    1 / 15 (6.67%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    1 / 1
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Neutrophil count decreased
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    0 / 1 (0.00%)
    0 / 3 (0.00%)
    0 / 1 (0.00%)
    1 / 15 (6.67%)
         occurrences causally related to treatment / all
    11 / 15
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    11 / 15
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Platelet count decreased
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    0 / 1 (0.00%)
    0 / 3 (0.00%)
    0 / 1 (0.00%)
    1 / 15 (6.67%)
         occurrences causally related to treatment / all
    4 / 5
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    4 / 5
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Plasmocytoma
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
    1 / 4 (25.00%)
    0 / 1 (0.00%)
    0 / 3 (0.00%)
    0 / 1 (0.00%)
    1 / 15 (6.67%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    1 / 1
    0 / 0
    0 / 0
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Injury, poisoning and procedural complications
    Transfusion reaction
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    0 / 1 (0.00%)
    0 / 3 (0.00%)
    0 / 1 (0.00%)
    1 / 15 (6.67%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Cardiac disorders
    Atrial fibrillation
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    0 / 1 (0.00%)
    0 / 3 (0.00%)
    0 / 1 (0.00%)
    1 / 15 (6.67%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Nervous system disorders
    Encephalopathy
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    0 / 1 (0.00%)
    0 / 3 (0.00%)
    1 / 1 (100.00%)
    1 / 15 (6.67%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    1 / 1
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    1 / 1
    1 / 1
    Immune effector cell-associated neurotoxicity syndrome
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    0 / 1 (0.00%)
    0 / 3 (0.00%)
    1 / 1 (100.00%)
    1 / 15 (6.67%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    4 / 4
    4 / 4
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    1 / 1
    1 / 1
    Blood and lymphatic system disorders
    Anaemia
         subjects affected / exposed
    2 / 3 (66.67%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    0 / 1 (0.00%)
    0 / 3 (0.00%)
    0 / 1 (0.00%)
    2 / 15 (13.33%)
         occurrences causally related to treatment / all
    4 / 7
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    4 / 7
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Immune thrombocytopenia
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    0 / 1 (0.00%)
    0 / 3 (0.00%)
    0 / 1 (0.00%)
    1 / 15 (6.67%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Lymph node pain
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    0 / 1 (0.00%)
    1 / 3 (33.33%)
    0 / 1 (0.00%)
    1 / 15 (6.67%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    1 / 1
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Neutropenia
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    0 / 1 (0.00%)
    0 / 3 (0.00%)
    0 / 1 (0.00%)
    1 / 15 (6.67%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    General disorders and administration site conditions
    Pyrexia
         subjects affected / exposed
    1 / 3 (33.33%)
    1 / 2 (50.00%)
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    0 / 1 (0.00%)
    1 / 3 (33.33%)
    0 / 1 (0.00%)
    3 / 15 (20.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
    0 / 0
    0 / 0
    0 / 0
    1 / 2
    0 / 0
    1 / 4
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Fatigue
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    0 / 1 (0.00%)
    1 / 3 (33.33%)
    0 / 1 (0.00%)
    1 / 15 (6.67%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    1 / 1
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Immune system disorders
    Cytokine release syndrome
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
    2 / 4 (50.00%)
    0 / 1 (0.00%)
    1 / 3 (33.33%)
    0 / 1 (0.00%)
    3 / 15 (20.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    2 / 2
    0 / 0
    1 / 1
    0 / 0
    3 / 3
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Gastrointestinal disorders
    Ascites
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
    1 / 4 (25.00%)
    0 / 1 (0.00%)
    0 / 3 (0.00%)
    0 / 1 (0.00%)
    1 / 15 (6.67%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 1
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Gastric haemorrhage
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
    1 / 4 (25.00%)
    0 / 1 (0.00%)
    0 / 3 (0.00%)
    0 / 1 (0.00%)
    1 / 15 (6.67%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 1
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 1
    0 / 0
    0 / 0
    0 / 0
    0 / 1
    Skin and subcutaneous tissue disorders
    Rash
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    0 / 1 (0.00%)
    0 / 3 (0.00%)
    0 / 1 (0.00%)
    1 / 15 (6.67%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Infections and infestations
    Epstein-Barr virus infection reactivation
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    0 / 1 (0.00%)
    1 / 3 (33.33%)
    0 / 1 (0.00%)
    2 / 15 (13.33%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    1 / 1
    0 / 0
    1 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Infection
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    0 / 1 (0.00%)
    0 / 3 (0.00%)
    0 / 1 (0.00%)
    1 / 15 (6.67%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 0%
    Non-serious adverse events
    		 Cohort 1 (25x10^6 cells) Cohort 2 (75x10^6 cells) Cohort 3 (225x10^6 cells) Cohort 4 (450x10^6 cells) Cohort 3B (225x10^6 cells) Cohort 4B (450x10^6 cells) Cohort 5B (900x10^6 cells) Total
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    3 / 3 (100.00%)
    2 / 2 (100.00%)
    1 / 1 (100.00%)
    4 / 4 (100.00%)
    1 / 1 (100.00%)
    2 / 3 (66.67%)
    1 / 1 (100.00%)
    15 / 15 (100.00%)
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Skin papilloma
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
    1 / 4 (25.00%)
    0 / 1 (0.00%)
    0 / 3 (0.00%)
    0 / 1 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    0
    1
    Vascular disorders
    Hypotension
         subjects affected / exposed
    2 / 3 (66.67%)
    1 / 2 (50.00%)
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    1 / 1 (100.00%)
    0 / 3 (0.00%)
    0 / 1 (0.00%)
    4 / 15 (26.67%)
         occurrences all number
    2
    1
    0
    0
    2
    0
    0
    5
    Flushing
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
    1 / 4 (25.00%)
    0 / 1 (0.00%)
    0 / 3 (0.00%)
    0 / 1 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    0
    1
    Raynaud's phenomenon
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    0 / 1 (0.00%)
    0 / 3 (0.00%)
    0 / 1 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    0
    1
    General disorders and administration site conditions
    Pyrexia
         subjects affected / exposed
    2 / 3 (66.67%)
    1 / 2 (50.00%)
    1 / 1 (100.00%)
    1 / 4 (25.00%)
    1 / 1 (100.00%)
    1 / 3 (33.33%)
    0 / 1 (0.00%)
    7 / 15 (46.67%)
         occurrences all number
    3
    2
    1
    2
    1
    1
    0
    10
    Fatigue
         subjects affected / exposed
    2 / 3 (66.67%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
    1 / 4 (25.00%)
    0 / 1 (0.00%)
    2 / 3 (66.67%)
    0 / 1 (0.00%)
    5 / 15 (33.33%)
         occurrences all number
    2
    0
    0
    1
    0
    2
    0
    5
    Asthenia
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 2 (50.00%)
    0 / 1 (0.00%)
    1 / 4 (25.00%)
    0 / 1 (0.00%)
    0 / 3 (0.00%)
    0 / 1 (0.00%)
    2 / 15 (13.33%)
         occurrences all number
    0
    3
    0
    1
    0
    0
    0
    4
    Chest pain
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    0 / 1 (0.00%)
    0 / 3 (0.00%)
    1 / 1 (100.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    1
    1
    Chills
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
    1 / 4 (25.00%)
    0 / 1 (0.00%)
    0 / 3 (0.00%)
    0 / 1 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    0
    1
    Gait disturbance
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    0 / 1 (0.00%)
    0 / 3 (0.00%)
    1 / 1 (100.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    1
    1
    Non-cardiac chest pain
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    0 / 1 (0.00%)
    1 / 3 (33.33%)
    0 / 1 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    0
    1
    Immune system disorders
    Cytokine release syndrome
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
    3 / 4 (75.00%)
    1 / 1 (100.00%)
    1 / 3 (33.33%)
    0 / 1 (0.00%)
    5 / 15 (33.33%)
         occurrences all number
    0
    0
    0
    3
    2
    1
    0
    6
    Reproductive system and breast disorders
    Vulval disorder
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    0 / 1 (0.00%)
    0 / 3 (0.00%)
    0 / 1 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    0
    1
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    1 / 3 (33.33%)
    1 / 2 (50.00%)
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    0 / 1 (0.00%)
    0 / 3 (0.00%)
    0 / 1 (0.00%)
    2 / 15 (13.33%)
         occurrences all number
    1
    1
    0
    0
    0
    0
    0
    2
    Dyspnoea
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 2 (50.00%)
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    0 / 1 (0.00%)
    1 / 3 (33.33%)
    0 / 1 (0.00%)
    2 / 15 (13.33%)
         occurrences all number
    0
    1
    0
    0
    0
    1
    0
    2
    Epistaxis
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    0 / 1 (0.00%)
    0 / 3 (0.00%)
    0 / 1 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    0
    1
    Nasal congestion
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    0 / 1 (0.00%)
    0 / 3 (0.00%)
    0 / 1 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    0
    1
    Oropharyngeal pain
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    0 / 1 (0.00%)
    1 / 3 (33.33%)
    0 / 1 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    0
    1
    Psychiatric disorders
    Insomnia
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    0 / 1 (0.00%)
    0 / 3 (0.00%)
    0 / 1 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    0
    1
    Investigations
    Neutrophil count decreased
         subjects affected / exposed
    1 / 3 (33.33%)
    1 / 2 (50.00%)
    0 / 1 (0.00%)
    3 / 4 (75.00%)
    1 / 1 (100.00%)
    0 / 3 (0.00%)
    0 / 1 (0.00%)
    6 / 15 (40.00%)
         occurrences all number
    6
    3
    0
    8
    7
    0
    0
    24
    Lymphocyte count decreased
         subjects affected / exposed
    1 / 3 (33.33%)
    1 / 2 (50.00%)
    1 / 1 (100.00%)
    1 / 4 (25.00%)
    1 / 1 (100.00%)
    0 / 3 (0.00%)
    0 / 1 (0.00%)
    5 / 15 (33.33%)
         occurrences all number
    9
    4
    3
    4
    2
    0
    0
    22
    Alanine aminotransferase increased
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 2 (0.00%)
    1 / 1 (100.00%)
    1 / 4 (25.00%)
    0 / 1 (0.00%)
    0 / 3 (0.00%)
    1 / 1 (100.00%)
    3 / 15 (20.00%)
         occurrences all number
    0
    0
    1
    1
    0
    0
    1
    3
    Aspartate aminotransferase increased
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 2 (50.00%)
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    0 / 1 (0.00%)
    0 / 3 (0.00%)
    1 / 1 (100.00%)
    2 / 15 (13.33%)
         occurrences all number
    0
    1
    0
    0
    0
    0
    1
    2
    Blood creatine phosphokinase increased
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
    1 / 4 (25.00%)
    0 / 1 (0.00%)
    0 / 3 (0.00%)
    1 / 1 (100.00%)
    2 / 15 (13.33%)
         occurrences all number
    0
    0
    0
    2
    0
    0
    1
    3
    Weight decreased
         subjects affected / exposed
    2 / 3 (66.67%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    0 / 1 (0.00%)
    0 / 3 (0.00%)
    0 / 1 (0.00%)
    2 / 15 (13.33%)
         occurrences all number
    2
    0
    0
    0
    0
    0
    0
    2
    Blood bilirubin increased
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 2 (0.00%)
    1 / 1 (100.00%)
    0 / 4 (0.00%)
    0 / 1 (0.00%)
    0 / 3 (0.00%)
    0 / 1 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    0
    1
    Blood thyroid stimulating hormone increased
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    0 / 1 (0.00%)
    1 / 3 (33.33%)
    0 / 1 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    0
    1
    Clostridium test positive
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    0 / 1 (0.00%)
    0 / 3 (0.00%)
    1 / 1 (100.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    1
    1
    Epstein-Barr virus test positive
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    0 / 1 (0.00%)
    0 / 3 (0.00%)
    1 / 1 (100.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    1
    1
    Platelet count decreased
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    0 / 1 (0.00%)
    0 / 3 (0.00%)
    0 / 1 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    2
    0
    0
    0
    0
    0
    0
    2
    Injury, poisoning and procedural complications
    Fall
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    0 / 1 (0.00%)
    0 / 3 (0.00%)
    0 / 1 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    0
    1
    Cardiac disorders
    Atrial fibrillation
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    0 / 1 (0.00%)
    0 / 3 (0.00%)
    0 / 1 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    2
    0
    0
    0
    0
    0
    0
    2
    Palpitations
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    0 / 1 (0.00%)
    0 / 3 (0.00%)
    0 / 1 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    0
    1
    Nervous system disorders
    Dizziness
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 2 (50.00%)
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    0 / 1 (0.00%)
    1 / 3 (33.33%)
    0 / 1 (0.00%)
    2 / 15 (13.33%)
         occurrences all number
    0
    1
    0
    0
    0
    1
    0
    2
    Headache
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 2 (50.00%)
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    1 / 1 (100.00%)
    0 / 3 (0.00%)
    0 / 1 (0.00%)
    2 / 15 (13.33%)
         occurrences all number
    0
    1
    0
    0
    1
    0
    0
    2
    Immune effector cell-associated neurotoxicity syndrome
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    0 / 1 (0.00%)
    0 / 3 (0.00%)
    1 / 1 (100.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    1
    1
    Blood and lymphatic system disorders
    Anaemia
         subjects affected / exposed
    3 / 3 (100.00%)
    1 / 2 (50.00%)
    0 / 1 (0.00%)
    2 / 4 (50.00%)
    1 / 1 (100.00%)
    1 / 3 (33.33%)
    0 / 1 (0.00%)
    8 / 15 (53.33%)
         occurrences all number
    13
    6
    0
    5
    2
    1
    0
    27
    Neutropenia
         subjects affected / exposed
    2 / 3 (66.67%)
    1 / 2 (50.00%)
    0 / 1 (0.00%)
    1 / 4 (25.00%)
    0 / 1 (0.00%)
    0 / 3 (0.00%)
    0 / 1 (0.00%)
    4 / 15 (26.67%)
         occurrences all number
    4
    10
    0
    4
    0
    0
    0
    18
    Thrombocytopenia
         subjects affected / exposed
    1 / 3 (33.33%)
    1 / 2 (50.00%)
    0 / 1 (0.00%)
    1 / 4 (25.00%)
    0 / 1 (0.00%)
    0 / 3 (0.00%)
    0 / 1 (0.00%)
    3 / 15 (20.00%)
         occurrences all number
    1
    1
    0
    3
    0
    0
    0
    5
    Lymphopenia
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 2 (50.00%)
    0 / 1 (0.00%)
    1 / 4 (25.00%)
    0 / 1 (0.00%)
    0 / 3 (0.00%)
    0 / 1 (0.00%)
    2 / 15 (13.33%)
         occurrences all number
    0
    8
    0
    3
    0
    0
    0
    11
    Febrile neutropenia
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    0 / 1 (0.00%)
    0 / 3 (0.00%)
    0 / 1 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    0
    1
    Immune thrombocytopenia
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    0 / 1 (0.00%)
    0 / 3 (0.00%)
    0 / 1 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    6
    0
    0
    0
    0
    0
    0
    6
    Eye disorders
    Dry eye
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 2 (50.00%)
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    0 / 1 (0.00%)
    0 / 3 (0.00%)
    0 / 1 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    1
    0
    0
    0
    0
    0
    1
    Gastrointestinal disorders
    Diarrhoea
         subjects affected / exposed
    1 / 3 (33.33%)
    1 / 2 (50.00%)
    1 / 1 (100.00%)
    1 / 4 (25.00%)
    1 / 1 (100.00%)
    0 / 3 (0.00%)
    0 / 1 (0.00%)
    5 / 15 (33.33%)
         occurrences all number
    2
    1
    2
    1
    2
    0
    0
    8
    Nausea
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
    1 / 4 (25.00%)
    1 / 1 (100.00%)
    1 / 3 (33.33%)
    0 / 1 (0.00%)
    3 / 15 (20.00%)
         occurrences all number
    0
    0
    0
    1
    1
    1
    0
    3
    Vomiting
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    0 / 1 (0.00%)
    1 / 3 (33.33%)
    0 / 1 (0.00%)
    2 / 15 (13.33%)
         occurrences all number
    2
    0
    0
    0
    0
    1
    0
    3
    Abdominal pain
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 2 (50.00%)
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    0 / 1 (0.00%)
    0 / 3 (0.00%)
    0 / 1 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    1
    0
    0
    0
    0
    0
    1
    Ascites
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
    1 / 4 (25.00%)
    0 / 1 (0.00%)
    0 / 3 (0.00%)
    0 / 1 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    0
    1
    Constipation
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    0 / 1 (0.00%)
    0 / 3 (0.00%)
    0 / 1 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    0
    1
    Dyspepsia
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    0 / 1 (0.00%)
    0 / 3 (0.00%)
    0 / 1 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    0
    1
    Toothache
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 2 (0.00%)
    1 / 1 (100.00%)
    0 / 4 (0.00%)
    0 / 1 (0.00%)
    0 / 3 (0.00%)
    0 / 1 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    2
    0
    0
    0
    0
    2
    Skin and subcutaneous tissue disorders
    Rash maculo-papular
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
    2 / 4 (50.00%)
    0 / 1 (0.00%)
    0 / 3 (0.00%)
    0 / 1 (0.00%)
    3 / 15 (20.00%)
         occurrences all number
    1
    0
    0
    2
    0
    0
    0
    3
    Night sweats
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    0 / 1 (0.00%)
    1 / 3 (33.33%)
    0 / 1 (0.00%)
    2 / 15 (13.33%)
         occurrences all number
    1
    0
    0
    0
    0
    1
    0
    2
    Skin lesion
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 2 (50.00%)
    1 / 1 (100.00%)
    0 / 4 (0.00%)
    0 / 1 (0.00%)
    0 / 3 (0.00%)
    0 / 1 (0.00%)
    2 / 15 (13.33%)
         occurrences all number
    0
    1
    1
    0
    0
    0
    0
    2
    Alopecia
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 2 (50.00%)
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    0 / 1 (0.00%)
    0 / 3 (0.00%)
    0 / 1 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    1
    0
    0
    0
    0
    0
    1
    Dry skin
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 2 (50.00%)
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    0 / 1 (0.00%)
    0 / 3 (0.00%)
    0 / 1 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    1
    0
    0
    0
    0
    0
    1
    Hyperhidrosis
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 2 (50.00%)
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    0 / 1 (0.00%)
    0 / 3 (0.00%)
    0 / 1 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    1
    0
    0
    0
    0
    0
    1
    Livedo reticularis
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    0 / 1 (0.00%)
    0 / 3 (0.00%)
    0 / 1 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    0
    1
    Rash
         subjects affected / exposed
    2 / 3 (66.67%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    0 / 1 (0.00%)
    0 / 3 (0.00%)
    0 / 1 (0.00%)
    2 / 15 (13.33%)
         occurrences all number
    2
    0
    0
    0
    0
    0
    0
    2
    Musculoskeletal and connective tissue disorders
    Muscle spasms
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    0 / 1 (0.00%)
    0 / 3 (0.00%)
    0 / 1 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    0
    1
    Infections and infestations
    Epstein-Barr virus infection reactivation
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    0 / 1 (0.00%)
    1 / 3 (33.33%)
    0 / 1 (0.00%)
    2 / 15 (13.33%)
         occurrences all number
    1
    0
    0
    0
    0
    1
    0
    2
    COVID-19
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
    1 / 4 (25.00%)
    0 / 1 (0.00%)
    0 / 3 (0.00%)
    0 / 1 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    0
    1
    Candida infection
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 2 (0.00%)
    1 / 1 (100.00%)
    0 / 4 (0.00%)
    0 / 1 (0.00%)
    0 / 3 (0.00%)
    0 / 1 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    0
    1
    Cytomegalovirus infection reactivation
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    0 / 1 (0.00%)
    0 / 3 (0.00%)
    0 / 1 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    2
    0
    0
    0
    0
    0
    0
    2
    Device related infection
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    0 / 1 (0.00%)
    0 / 3 (0.00%)
    1 / 1 (100.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    1
    1
    Gingivitis
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 2 (50.00%)
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    0 / 1 (0.00%)
    0 / 3 (0.00%)
    0 / 1 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    1
    0
    0
    0
    0
    0
    1
    Onychomycosis
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    0 / 1 (0.00%)
    0 / 3 (0.00%)
    0 / 1 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    0
    1
    Peritonitis
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    0 / 1 (0.00%)
    0 / 3 (0.00%)
    1 / 1 (100.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    1
    1
    Respiratory syncytial virus infection
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    0 / 1 (0.00%)
    0 / 3 (0.00%)
    0 / 1 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    0
    1
    Staphylococcal infection
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    0 / 1 (0.00%)
    0 / 3 (0.00%)
    0 / 1 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    0
    1
    Toxoplasmosis
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    0 / 1 (0.00%)
    0 / 3 (0.00%)
    0 / 1 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    0
    1
    Upper respiratory tract infection
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    0 / 1 (0.00%)
    0 / 3 (0.00%)
    0 / 1 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    0
    1
    Vulvovaginal candidiasis
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    0 / 1 (0.00%)
    0 / 3 (0.00%)
    0 / 1 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    0
    1
    Wound infection
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    1 / 1 (100.00%)
    0 / 3 (0.00%)
    0 / 1 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    0
    1
    0
    0
    1
    Metabolism and nutrition disorders
    Hypokalaemia
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    0 / 1 (0.00%)
    0 / 3 (0.00%)
    0 / 1 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    0
    1
    Hypoproteinaemia
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    0 / 1 (0.00%)
    0 / 3 (0.00%)
    0 / 1 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    0
    1

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    30 Jun 2022
    Version 6 (the final amended version) was a substantial amendment to version 5 (dated 07 July 2021) to introduce new dose escalation cohorts using the modified manufacturing process.

    Interruptions (globally)
    Were there any global interruptions to the trial? Yes
    Date
    Interruption
    Restart date
    07 Mar 2024
    Study terminated. Only Phase I of the study was conducted.
    -

    Limitations and caveats
    Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.
    Phase 2 was not started and therefore no planned Phase 2 endpoints were available for analysis.

    Online references
    http://www.ncbi.nlm.nih.gov/pubmed/39528665
    For support, Contact us.
    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

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