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    Clinical Trial Results:
    A phase 1 / 2, open-label, single arm, multicohort, multicenter trial to evaluate the safety and efficacy of JCAR017 in pediatric subjects with relapsed/refractory B-ALL and B-NHL (TRANSCEND PEDALL)

    Summary
    EudraCT number
    		 2018-001246-34
    Trial protocol
    		 FR   DE   ES   IT   BE   NL  
    Global end of trial date
    26 Jan 2024

    Results information
    Results version number
    		 v1(current)
    This version publication date
    07 Aug 2024
    First version publication date
    07 Aug 2024
    Other versions

    Trial information

    		 Close Top of page
    Trial identification
    Sponsor protocol code
    JCAR017-BCM-004
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT03743246
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Bristol-Myers Squibb
    Sponsor organisation address
    Chaussee de la Hulpe 185, Brussels, Belgium, 1170
    Public contact
    EU Study Start-Up Unit, Bristol-Myers Squibb International Corporation, Clinical.Trials@bms.com
    Scientific contact
    Bristol-Myers Squibb Study Director, Bristol-Myers Squibb, Clinical.Trials@bms.com
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    Yes
    EMA paediatric investigation plan number(s)
    		 EMEA-001995-PIP01-02
    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
    26 Jan 2024
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    26 Jan 2024
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    The primary objective of Phase 1 is to identify the recommended Phase 2 dose (RP2D) in pediatric subjects with relapsed/refractory (r/r) B-cell acute lymphoblastic leukemia (B-ALL).
    Protection of trial subjects
    Patient Confidentiality, Personal Data Protection and Biomarker Consent
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    17 Oct 2018
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    No
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    Netherlands: 1
    Country: Number of subjects enrolled
    Germany: 1
    Country: Number of subjects enrolled
    Spain: 10
    Country: Number of subjects enrolled
    Italy: 9
    Worldwide total number of subjects
    21
    EEA total number of subjects
    21
    Number of subjects enrolled per age group
    In utero
    0
    Preterm newborn - gestational age < 37 wk
    0
    Newborns (0-27 days)
    0
    Infants and toddlers (28 days-23 months)
    3
    Children (2-11 years)
    15
    Adolescents (12-17 years)
    3
    Adults (18-64 years)
    0
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 -

    Pre-assignment
    Screening details
    		 The study was terminated and hence participants were not enrolled in Phase 2 cohorts (r/r B-ALL; MRD+ B-ALL; r/r B-NHL).

    Period 1
    Period 1 title
    Pre-Treatment Period
    Is this the baseline period?
    		 Yes
    Allocation method
    Randomised - controlled
    Blinding used
    		 Not blinded

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 0.05 x 10^6 CAR+ T cells/kg
    Arm description
    		 Participants with CD19+ relapsed/refractory (r/r) B-cell acute lymphoblastic leukemia (B-ALL) with 0.05 x 10^6 CAR+ T cells per kilogram (kg) post leukapheresis.
    Arm type
    		 Experimental

    Investigational medicinal product name
    JCAR017 Infusion
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Injection
    Routes of administration
    Intravenous use
    Dosage and administration details
    JCAR017 Infusion was administered on Day 1 intravenously

    Arm title
    		 0.15 x 10^6 CAR+ T cells/kg
    Arm description
    		 Participants with CD19+ relapsed/refractory (r/r) B-cell acute lymphoblastic leukemia (B-ALL) were infused with 0.15 x 106 CAR+ T cells/kg post leukapheresis.
    Arm type
    		 Experimental

    Investigational medicinal product name
    JCAR017 Infusion
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Injection
    Routes of administration
    Intravenous use
    Dosage and administration details
    JCAR017 Infusion was administered on Day 1 intravenously

    Arm title
    		 0.50 x 106 CAR+T cells/kg
    Arm description
    		 Participants with CD19+ relapsed/refractory (r/r) B-cell acute lymphoblastic leukemia (B-ALL) with 0.50 x 106 CAR+T cells/kg post leukapheresis.
    Arm type
    		 Experimental

    Investigational medicinal product name
    JCAR017 Infusion
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Injection
    Routes of administration
    Intravenous use
    Dosage and administration details
    JCAR017 Infusion was administered on Day 1 intravenously

    Arm title
    		 Not Assigned
    Arm description
    		 Participants only underwent leukapheresis.
    Arm type
    		 No intervention

    Investigational medicinal product name
    No investigational medicinal product assigned in this arm
    Number of subjects in period 1
    		0.05 x 10^6 CAR+ T cells/kg 0.15 x 10^6 CAR+ T cells/kg 0.50 x 106 CAR+T cells/kg Not Assigned
    Started
    9
    8
    3
    1
    Completed
    8
    7
    1
    0
    Not completed
    1
    1
    2
    1
         Adverse event, serious fatal
    -
    1
    -
    -
         Failure to meet treatment criteria
    -
    -
    2
    -
         Death
    -
    -
    -
    1
         Study Drug Manufacturing Failure
    1
    -
    -
    -
    Period 2
    Period 2 title
    Lymphodepleting chemotherapy
    Is this the baseline period?
    		 No
    Allocation method
    Randomised - controlled
    Blinding used
    		 Not blinded

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 0.05 x 10^6 CAR+ T cells/kg
    Arm description
    		 Participants with CD19+ relapsed/refractory (r/r) B-cell acute lymphoblastic leukemia (B-ALL) were infused with 0.05 x 10^6 CAR+ T cells per kilogram (kg) post leukapheresis.
    Arm type
    		 Experimental

    Investigational medicinal product name
    JCAR017 Infusion
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Injection
    Routes of administration
    Intravenous use
    Dosage and administration details
    JCAR017 Infusion was administered on Day 1 intravenously

    Arm title
    		 0.15 x 10^6 CAR+ T cells/kg
    Arm description
    		 Participants with CD19+ relapsed/refractory (r/r) B-cell acute lymphoblastic leukemia (B-ALL) were infused with 0.15 x 106 CAR+ T cells/kg post leukapheresis.
    Arm type
    		 Experimental

    Investigational medicinal product name
    JCAR017 Infusion
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Injection
    Routes of administration
    Intravenous use
    Dosage and administration details
    JCAR017 Infusion was administered on Day 1 intravenously

    Arm title
    		 0.50 x 106 CAR+T cells/kg
    Arm description
    		 Participants with CD19+ relapsed/refractory (r/r) B-cell acute lymphoblastic leukemia (B-ALL) were infused with 0.50 x 106 CAR+T cells/kg post leukapheresis.
    Arm type
    		 Experimental

    Investigational medicinal product name
    JCAR017 Infusion
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Injection
    Routes of administration
    Intravenous use
    Dosage and administration details
    JCAR017 Infusion was administered on Day 1 intravenously

    Number of subjects in period 2
    		0.05 x 10^6 CAR+ T cells/kg 0.15 x 10^6 CAR+ T cells/kg 0.50 x 106 CAR+T cells/kg
    Started
    8
    7
    1
    Completed
    7
    7
    1
    Not completed
    1
    0
    0
         Progressive Disease
    1
    -
    -
    Period 3
    Period 3 title
    Treatment Period (JCAR017 Infusion)
    Is this the baseline period?
    		 No
    Allocation method
    Randomised - controlled
    Blinding used
    		 Not blinded

    Arms
    Are arms mutually exclusive
    No

    Arm title
    		 0.05 x 10^6 CAR+ T cells/kg
    Arm description
    		 Participants withCD19+ relapsed/refractory (r/r) B-cell acute lymphoblastic leukemia (B-ALL) were infused with with 0.05 x 10^6 CAR+ T cells per kilogram (kg) post leukapheresis.
    Arm type
    		 Experimental

    Investigational medicinal product name
    JCAR017 Infusion
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Injection
    Routes of administration
    Intravenous use
    Dosage and administration details
    JCAR017 Infusion was administered on Day 1 intravenously

    Arm title
    		 0.15 x 10^6 CAR+ T cells/kg
    Arm description
    		 Participants with CD19+ relapsed/refractory (r/r) B-cell acute lymphoblastic leukemia (B-ALL) were infused with 0.15 x 10^6 CAR+ T cells/kg post leukapheresis.
    Arm type
    		 Experimental

    Investigational medicinal product name
    JCAR017 Infusion
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Injection
    Routes of administration
    Intravenous use
    Dosage and administration details
    JCAR017 Infusion was administered on Day 1 intravenously

    Arm title
    		 0.50 x 10^6 CAR+T cells/kg
    Arm description
    		 Participants with CD19+ relapsed/refractory (r/r) B-cell acute lymphoblastic leukemia (B-ALL) were infused with 0.50 x 106 CAR+T cells/kg post leukapheresis.
    Arm type
    		 Experimental

    Investigational medicinal product name
    JCAR017 Infusion
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Injection
    Routes of administration
    Intravenous use
    Dosage and administration details
    JCAR017 Infusion was administered on Day 1 intravenously

    Number of subjects in period 3
    		0.05 x 10^6 CAR+ T cells/kg 0.15 x 10^6 CAR+ T cells/kg 0.50 x 10^6 CAR+T cells/kg
    Started
    7
    7
    1
    Completed
    3
    6
    1
    Not completed
    4
    1
    0
         Adverse event, serious fatal
    1
    -
    -
         Study Terminated by sponsor
    1
    -
    -
         Progressive Disease
    1
    -
    -
         Other Reason
    1
    -
    -
         Lost to follow-up
    -
    1
    -

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    0.05 x 10^6 CAR+ T cells/kg
    Reporting group description
    		 Participants with CD19+ relapsed/refractory (r/r) B-cell acute lymphoblastic leukemia (B-ALL) with 0.05 x 10^6 CAR+ T cells per kilogram (kg) post leukapheresis.

    Reporting group title
    0.15 x 10^6 CAR+ T cells/kg
    Reporting group description
    		 Participants with CD19+ relapsed/refractory (r/r) B-cell acute lymphoblastic leukemia (B-ALL) were infused with 0.15 x 106 CAR+ T cells/kg post leukapheresis.

    Reporting group title
    0.50 x 106 CAR+T cells/kg
    Reporting group description
    		 Participants with CD19+ relapsed/refractory (r/r) B-cell acute lymphoblastic leukemia (B-ALL) with 0.50 x 106 CAR+T cells/kg post leukapheresis.

    Reporting group title
    Not Assigned
    Reporting group description
    		 Participants only underwent leukapheresis.

    Reporting group values
    		 0.05 x 10^6 CAR+ T cells/kg 0.15 x 10^6 CAR+ T cells/kg 0.50 x 106 CAR+T cells/kg Not Assigned Total
    Number of subjects
    		 9 8 3 1 21
    Age Categorical
    Units: participants
        < 6 years
    		 3 4 2 0 9
        >= 6 to < 12 years
    		 5 3 1 0 9
        >= 12 to < 18 years
    		 1 1 0 1 3
    Sex: Female, Male
    Units: participants
        Female
    		 5 4 2 0 11
        Male
    		 4 4 1 1 10
    Race (NIH/OMB)
    Units: Subjects
        American Indian or Alaska Native
    		 1 1 0 1 3
        Asian
    		 0 0 0 0 0
        Native Hawaiian or Other Pacific Islander
    		 0 0 0 0 0
        Black or African American
    		 0 0 0 0 0
        White
    		 6 5 3 0 14
        More than one race
    		 0 0 0 0 0
        Unknown or Not Reported
    		 2 2 0 0 4
    Ethnicity (NIH/OMB)
    Units: Subjects
        Hispanic or Latino
    		 4 3 1 1 9
        Not Hispanic or Latino
    		 4 4 2 0 10
        Unknown or Not Reported
    		 1 1 0 0 2

    End points

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    End points reporting groups
    Reporting group title
    0.05 x 10^6 CAR+ T cells/kg
    Reporting group description
    		 Participants with CD19+ relapsed/refractory (r/r) B-cell acute lymphoblastic leukemia (B-ALL) with 0.05 x 10^6 CAR+ T cells per kilogram (kg) post leukapheresis.

    Reporting group title
    0.15 x 10^6 CAR+ T cells/kg
    Reporting group description
    		 Participants with CD19+ relapsed/refractory (r/r) B-cell acute lymphoblastic leukemia (B-ALL) were infused with 0.15 x 106 CAR+ T cells/kg post leukapheresis.

    Reporting group title
    0.50 x 106 CAR+T cells/kg
    Reporting group description
    		 Participants with CD19+ relapsed/refractory (r/r) B-cell acute lymphoblastic leukemia (B-ALL) with 0.50 x 106 CAR+T cells/kg post leukapheresis.

    Reporting group title
    Not Assigned
    Reporting group description
    		 Participants only underwent leukapheresis.
    Reporting group title
    0.05 x 10^6 CAR+ T cells/kg
    Reporting group description
    		 Participants with CD19+ relapsed/refractory (r/r) B-cell acute lymphoblastic leukemia (B-ALL) were infused with 0.05 x 10^6 CAR+ T cells per kilogram (kg) post leukapheresis.

    Reporting group title
    0.15 x 10^6 CAR+ T cells/kg
    Reporting group description
    		 Participants with CD19+ relapsed/refractory (r/r) B-cell acute lymphoblastic leukemia (B-ALL) were infused with 0.15 x 106 CAR+ T cells/kg post leukapheresis.

    Reporting group title
    0.50 x 106 CAR+T cells/kg
    Reporting group description
    		 Participants with CD19+ relapsed/refractory (r/r) B-cell acute lymphoblastic leukemia (B-ALL) were infused with 0.50 x 106 CAR+T cells/kg post leukapheresis.
    Reporting group title
    0.05 x 10^6 CAR+ T cells/kg
    Reporting group description
    		 Participants withCD19+ relapsed/refractory (r/r) B-cell acute lymphoblastic leukemia (B-ALL) were infused with with 0.05 x 10^6 CAR+ T cells per kilogram (kg) post leukapheresis.

    Reporting group title
    0.15 x 10^6 CAR+ T cells/kg
    Reporting group description
    		 Participants with CD19+ relapsed/refractory (r/r) B-cell acute lymphoblastic leukemia (B-ALL) were infused with 0.15 x 10^6 CAR+ T cells/kg post leukapheresis.

    Reporting group title
    0.50 x 10^6 CAR+T cells/kg
    Reporting group description
    		 Participants with CD19+ relapsed/refractory (r/r) B-cell acute lymphoblastic leukemia (B-ALL) were infused with 0.50 x 106 CAR+T cells/kg post leukapheresis.

    Primary: Number of Participants with Treatment Emergent Adverse Events up to 30 Days Post JCAR017 Infusion

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    End point title
    		 Number of Participants with Treatment Emergent Adverse Events up to 30 Days Post JCAR017 Infusion [1] [2]
    End point description
    An AE is defined as any noxious, unintended, or untoward medical occurrence that may appear or worsen in a participant during the course of a study. Treatment Emergent Adverse Events (TEAEs) are defined as any AE occurring or worsening on the day of the JCAR017 infusion until 30 days post-treatment (JCAR017 infusion) using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) version 4.03 or greater. Safety Analysis Set included all participants who received conforming JCAR017 infusion.
    End point type
    Primary
    End point timeframe
    From first JCAR017 infusion to 30 days after JCAR017 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: Statistical Analysis Comparison was not planned as per protocol.
    [2] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: Statistical Analysis Comparison was not planned as per protocol.
    End point values
    		 0.05 x 10^6 CAR+ T cells/kg 0.15 x 10^6 CAR+ T cells/kg 0.50 x 106 CAR+T cells/kg
    Number of subjects analysed
    7
    6
    1
    Units: participants
    7
    6
    1
    No statistical analyses for this end point

    Primary: Number of Participants with Dose Limiting Toxicities

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    End point title
    		 Number of Participants with Dose Limiting Toxicities [3] [4]
    End point description
    A DLT was defined as below: • Death not related to PD • Grade (Gr) 4 (Life-threatening) neurotoxicity • Gr 3 (Severe) neurotoxicity of greater than 7 days • Gr 3 neurotoxicity does not revert to baseline within 28 days of the start date of the Grade 3 event • Seizures of grade that do not resolve within 7 days • Gr 4 cytokine release syndrome (CRS) that does not resolve to Grade ≤ 3 within 3 days • Gr 3 CRS that does not resolve to Grade ≤ 2 within 7 days • Any increase in aspartate aminotransferase (AST) or ALT > 3 × ULN and concurrent increase in total bilirubin > 2 × ULN that is unrelated to CRS and has no other probable reason to explain the combination of increases • Any cardiac, dermatologic, gastrointestinal, hepatic, pulmonary, renal/genitourinary, or neurologic Gr 3 or 4 event not pre-existing or not due to the underlying malignancy • Any other Gr 3 or 4 event deemed unexpected by the Investigator and considered a DLT upon evaluation by the safety review committee
    End point type
    Primary
    End point timeframe
    From first JCAR017 infusion to 28 days after JCAR017 infusion
    Notes
    [3] - 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: Statistical Analysis Comparison was not planned as per protocol.
    [4] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: Statistical Analysis Comparison was not planned as per protocol.
    End point values
    		 0.05 x 10^6 CAR+ T cells/kg 0.15 x 10^6 CAR+ T cells/kg 0.50 x 106 CAR+T cells/kg
    Number of subjects analysed
    5
    5
    1
    Units: participants
    0
    2
    1
    No statistical analyses for this end point

    Primary: Concentration of JCAR017 in Peripheral Blood at Day 28 Post JCAR017 Infusion

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    End point title
    		 Concentration of JCAR017 in Peripheral Blood at Day 28 Post JCAR017 Infusion [5] [6]
    End point description
    Concentration of JCAR017 in Peripheral Blood was assessed by droplet digital polymerase chain reaction. The PK Analysis Set include all subjects who received JCAR017 infusion and have at least one measurable JCAR017 concentration. Participants available at specific timepoints have been analyzed.
    End point type
    Primary
    End point timeframe
    From first JCAR017 infusion to 28 days after JCAR017 infusion
    Notes
    [5] - 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: Statistical Analysis Comparison was not planned as per protocol.
    [6] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: Statistical Analysis Comparison was not planned as per protocol.
    End point values
    		 0.05 x 10^6 CAR+ T cells/kg 0.15 x 10^6 CAR+ T cells/kg 0.50 x 106 CAR+T cells/kg
    Number of subjects analysed
    3
    3
    1 [7]
    Units: copies per microgram
        geometric mean (geometric coefficient of variation)
    1131.821 ( 64.658 )
    1457.591 ( 75.558 )
    2847.910 ( 99999 )
    Notes
    [7] - Not calculable due to insufficient number of participants
    No statistical analyses for this end point

    Secondary: Number of Participants with Treatment-Emergent Adverse Events up to 90 Days Post JCAR017 Infusion

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    End point title
    		 Number of Participants with Treatment-Emergent Adverse Events up to 90 Days Post JCAR017 Infusion [8]
    End point description
    An AE is defined as any noxious, unintended, or untoward medical occurrence that may appear or worsen in a participant during the course of a study. Treatment Emergent Adverse Events (TEAEs) are defined as any AE occurring or worsening on the day of the JCAR017 infusion until 90 days post-treatment (JCAR017 infusion) using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) version 4.03 or greater. The following scale for grading was used - Grade 3 = Severe, Grade 4 = Life-Threatening. Safety Analysis Set included all participants who received conforming JCAR017 infusion.
    End point type
    Secondary
    End point timeframe
    From first JCAR017 infusion to 90 days after JCAR017 infusion
    Notes
    [8] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: Statistical Analysis Comparison was not planned as per protocol.
    End point values
    		 0.05 x 10^6 CAR+ T cells/kg 0.15 x 10^6 CAR+ T cells/kg 0.50 x 106 CAR+T cells/kg
    Number of subjects analysed
    7
    6
    1
    Units: participants
        At least one TEAE
    7
    6
    1
        At least one grade 3/4 TEAE
    6
    5
    1
        At Least One TEAE Related To JCAR017
    5
    5
    1
        At Least One grade 3/4 TEAE Related To JCAR017
    3
    4
    1
    No statistical analyses for this end point

    Secondary: Number of Participants with Serious Treatment-Emergent Adverse Events up to 90 Days Post JCAR017 Infusion

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    End point title
    		 Number of Participants with Serious Treatment-Emergent Adverse Events up to 90 Days Post JCAR017 Infusion [9]
    End point description
    Serious adverse events (SAE) are defined as any AE which results in death; is life-threatening; requires inpatient hospitalization or prolongation of existing hospitalization; results in persistent or significant disability/incapacity; is a congenital anomaly/ birth defect; constitutes an important medical event. Treatment Emergent Adverse Events (TEAEs) are defined as any AE occurring or worsening on the day of the JCAR017 infusion until 90 days post-treatment (JCAR017 infusion) using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) version 4.03 or greater. Safety Analysis Set included all participants who received conforming JCAR017 infusion.
    End point type
    Secondary
    End point timeframe
    From first JCAR017 infusion to 90 days after JCAR017 infusion
    Notes
    [9] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: Statistical Analysis Comparison was not planned as per protocol.
    End point values
    		 0.05 x 10^6 CAR+ T cells/kg 0.15 x 10^6 CAR+ T cells/kg 0.50 x 106 CAR+T cells/kg
    Number of subjects analysed
    7
    6
    1
    Units: participants
        AT Least one Serious TEAE
    5
    3
    1
        At Least One Serious TEAE Related To JCAR017
    1
    3
    1
    No statistical analyses for this end point

    Secondary: Change from Baseline at Day 28 in Hematology Laboratory Parameters- Basophils, Eosinophils, Leukocytes, Lymphocytes, Monocytes, Neutrophils, Platelets

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    End point title
    		 Change from Baseline at Day 28 in Hematology Laboratory Parameters- Basophils, Eosinophils, Leukocytes, Lymphocytes, Monocytes, Neutrophils, Platelets [10]
    End point description
    Baseline is defined as the last assessment with non-missing value on or prior to the first date of the lymphodepletion chemotherapy. Safety Analysis Set included all participants who received conforming JCAR017 infusion.
    End point type
    Secondary
    End point timeframe
    Baseline and at Day 28
    Notes
    [10] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: Statistical Analysis Comparison was not planned as per protocol.
    End point values
    		 0.05 x 10^6 CAR+ T cells/kg 0.15 x 10^6 CAR+ T cells/kg 0.50 x 106 CAR+T cells/kg
    Number of subjects analysed
    6
    5
    1 [11]
    Units: 10^9 cells/L
    arithmetic mean (standard deviation)
        Basophils
    0.00 ( 0.025 )
    -0.01 ( 0.023 )
    -0.01 ( 99999 )
        Eosinophils
    0.03 ( 0.048 )
    0.08 ( 0.114 )
    -0.08 ( 99999 )
        Leukocytes
    4.01 ( 8.338 )
    0.59 ( 1.753 )
    -2.00 ( 99999 )
        Lymphocytes
    -0.22 ( 0.879 )
    0.27 ( 1.286 )
    -0.57 ( 99999 )
        Monocytes
    0.11 ( 0.163 )
    0.09 ( 0.173 )
    -0.22 ( 99999 )
        Neutrophils
    0.41 ( 0.782 )
    -0.18 ( 0.735 )
    -1.50 ( 99999 )
        Platelets
    27.33 ( 132.672 )
    -68.60 ( 168.583 )
    -163.00 ( 99999 )
    Notes
    [11] - 99999 stands for Not Applicable
    No statistical analyses for this end point

    Secondary: Change from Baseline at Day 28 in Hematology Laboratory Parameters- Basophils/Leukocytes; Eosinophils/Leukocytes; Lymphocytes/Leukocytes; Neutrophils/Leukocytes; Monocytes/Leukocytes

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    End point title
    		 Change from Baseline at Day 28 in Hematology Laboratory Parameters- Basophils/Leukocytes; Eosinophils/Leukocytes; Lymphocytes/Leukocytes; Neutrophils/Leukocytes; Monocytes/Leukocytes [12]
    End point description
    Baseline is defined as the last assessment with non-missing value on or prior to the first date of the lymphodepletion chemotherapy. Safety Analysis Set included all participants who received conforming JCAR017 infusion.
    End point type
    Secondary
    End point timeframe
    Baseline and at Day 28
    Notes
    [12] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: Statistical Analysis Comparison was not planned as per protocol.
    End point values
    		 0.05 x 10^6 CAR+ T cells/kg 0.15 x 10^6 CAR+ T cells/kg 0.50 x 106 CAR+T cells/kg
    Number of subjects analysed
    6
    5
    1 [13]
    Units: Ratio
    arithmetic mean (standard deviation)
        Basophils/Leukocytes
    0.25 ( 0.885 )
    -0.24 ( 0.654 )
    -0.30 ( 99999 )
        Eosinophils/Leukocytes
    0.85 ( 1.402 )
    4.28 ( 5.389 )
    -2.90 ( 99999 )
        Lymphocytes/Leukocytes
    -29.57 ( 21.690 )
    -16.30 ( 25.140 )
    33.80 ( 99999 )
        Neutrophils/Leukocytes
    26.45 ( 26.815 )
    10.70 ( 18.797 )
    -33.90 ( 99999 )
        Monocytes/Leukocytes
    4.35 ( 10.123 )
    1.74 ( 4.515 )
    0.20 ( 99999 )
    Notes
    [13] - 99999 stands for Not Applicable
    No statistical analyses for this end point

    Secondary: Change from Baseline at Day 28 in Hematology Laboratory Parameters- Hematocrit

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    End point title
    		 Change from Baseline at Day 28 in Hematology Laboratory Parameters- Hematocrit [14]
    End point description
    Baseline is defined as the last assessment with non-missing value on or prior to the first date of the lymphodepletion chemotherapy. Safety Analysis Set included all participants who received conforming JCAR017 infusion.
    End point type
    Secondary
    End point timeframe
    Baseline and at Day 28
    Notes
    [14] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: Statistical Analysis Comparison was not planned as per protocol.
    End point values
    		 0.05 x 10^6 CAR+ T cells/kg 0.15 x 10^6 CAR+ T cells/kg 0.50 x 106 CAR+T cells/kg
    Number of subjects analysed
    6
    5
    1 [15]
    Units: proportion of red blood cells in blood
        arithmetic mean (standard deviation)
    0.00 ( 0.068 )
    0.02 ( 0.016 )
    -0.03 ( 99999 )
    Notes
    [15] - 99999 stands for Not Applicable
    No statistical analyses for this end point

    Secondary: Change from Baseline at Day 28 in Hematology Laboratory Parameters- Erythrocytes

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    End point title
    		 Change from Baseline at Day 28 in Hematology Laboratory Parameters- Erythrocytes [16]
    End point description
    Baseline is defined as the last assessment with non-missing value on or prior to the first date of the lymphodepletion chemotherapy. Safety Analysis Set included all participants who received conforming JCAR017 infusion.
    End point type
    Secondary
    End point timeframe
    Baseline and at Day 28
    Notes
    [16] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: Statistical Analysis Comparison was not planned as per protocol.
    End point values
    		 0.05 x 10^6 CAR+ T cells/kg 0.15 x 10^6 CAR+ T cells/kg 0.50 x 106 CAR+T cells/kg
    Number of subjects analysed
    6
    5
    1 [17]
    Units: 10^12 cells/L
        arithmetic mean (standard deviation)
    0.04 ( 0.781 )
    0.08 ( 0.268 )
    -0.70 ( 99999 )
    Notes
    [17] - 99999 stands for Not Applicable
    No statistical analyses for this end point

    Secondary: Change from Baseline at Day 28 in Hematology Laboratory Parameters- Hemoglobin

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    End point title
    		 Change from Baseline at Day 28 in Hematology Laboratory Parameters- Hemoglobin [18]
    End point description
    Baseline is defined as the last assessment with non-missing value on or prior to the first date of the lymphodepletion chemotherapy. Safety Analysis Set included all participants who received conforming JCAR017 infusion.
    End point type
    Secondary
    End point timeframe
    Baseline and at Day 28
    Notes
    [18] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: Statistical Analysis Comparison was not planned as per protocol.
    End point values
    		 0.05 x 10^6 CAR+ T cells/kg 0.15 x 10^6 CAR+ T cells/kg 0.50 x 106 CAR+T cells/kg
    Number of subjects analysed
    6
    5
    1 [19]
    Units: grams per liter
        arithmetic mean (standard deviation)
    2.33 ( 24.312 )
    6.20 ( 6.834 )
    -13.00 ( 99999 )
    Notes
    [19] - 99999 stands for Not Applicable
    No statistical analyses for this end point

    Secondary: Change from Baseline at Day 28 in Chemistry Laboratory Parameters- Alanine aminotransferase; Alkaline phosphatase; Aspartate aminotransferase; Lactate dehydrogenase

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    End point title
    		 Change from Baseline at Day 28 in Chemistry Laboratory Parameters- Alanine aminotransferase; Alkaline phosphatase; Aspartate aminotransferase; Lactate dehydrogenase [20]
    End point description
    Baseline is defined as the last assessment with non-missing value on or prior to the first date of the lymphodepletion chemotherapy. Safety Analysis Set included all participants who received conforming JCAR017 infusion.
    End point type
    Secondary
    End point timeframe
    Baseline and at Day 28
    Notes
    [20] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: Statistical Analysis Comparison was not planned as per protocol.
    End point values
    		 0.05 x 10^6 CAR+ T cells/kg 0.15 x 10^6 CAR+ T cells/kg 0.50 x 106 CAR+T cells/kg
    Number of subjects analysed
    6
    5
    1 [21]
    Units: units per liter
    arithmetic mean (standard deviation)
        Alanine aminotransferase
    5.33 ( 19.439 )
    4.50 ( 16.299 )
    -19.00 ( 99999 )
        Alkaline phosphatase
    53.50 ( 158.322 )
    78.75 ( 40.950 )
    -121.00 ( 99999 )
        Aspartate aminotransferase
    29.17 ( 72.204 )
    11.50 ( 13.675 )
    -1.00 ( 99999 )
        Lactate dehydrogenase
    992.33 ( 2238.888 )
    22.25 ( 43.684 )
    -36.00 ( 99999 )
    Notes
    [21] - 99999 stands for Not Applicable
    No statistical analyses for this end point

    Secondary: Change from Baseline at Day 28 in Chemistry Laboratory Parameters- Albumin; Protein

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    End point title
    		 Change from Baseline at Day 28 in Chemistry Laboratory Parameters- Albumin; Protein [22]
    End point description
    Baseline is defined as the last assessment with non-missing value on or prior to the first date of the lymphodepletion chemotherapy. Safety Analysis Set included all participants who received conforming JCAR017 infusion.
    End point type
    Secondary
    End point timeframe
    Baseline and at Day 28
    Notes
    [22] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: Statistical Analysis Comparison was not planned as per protocol.
    End point values
    		 0.05 x 10^6 CAR+ T cells/kg 0.15 x 10^6 CAR+ T cells/kg 0.50 x 106 CAR+T cells/kg
    Number of subjects analysed
    6
    4
    1 [23]
    Units: grams per liter
    arithmetic mean (standard deviation)
        Albumin
    5.83 ( 9.663 )
    6.75 ( 2.062 )
    -3.00 ( 99999 )
        Protein
    4.00 ( 12.602 )
    5.00 ( 6.683 )
    -6.00 ( 99999 )
    Notes
    [23] - 99999 stands for Not Applicable
    No statistical analyses for this end point

    Secondary: Change from Baseline at Day 28 in Chemistry Laboratory Parameters- Bicarbonate; Blood Urea Nitrogen; Calcium; Chloride; Glucose; Magnesium; Phosphate; Potassium; Sodium, Triglyceride

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    End point title
    		 Change from Baseline at Day 28 in Chemistry Laboratory Parameters- Bicarbonate; Blood Urea Nitrogen; Calcium; Chloride; Glucose; Magnesium; Phosphate; Potassium; Sodium, Triglyceride [24]
    End point description
    Baseline is defined as the last assessment with non-missing value on or prior to the first date of the lymphodepletion chemotherapy. Safety Analysis Set included all participants who received conforming JCAR017 infusion.
    End point type
    Secondary
    End point timeframe
    Baseline and at Day 28
    Notes
    [24] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: Statistical Analysis Comparison was not planned as per protocol.
    End point values
    		 0.05 x 10^6 CAR+ T cells/kg 0.15 x 10^6 CAR+ T cells/kg 0.50 x 106 CAR+T cells/kg
    Number of subjects analysed
    6
    4
    1 [25]
    Units: mmol/L
    arithmetic mean (standard deviation)
        Bicarbonate
    2.53 ( 4.453 )
    -2.65 ( 1.919 )
    4.10 ( 99999 )
        Blood Urea Nitrogen
    3.60 ( 3.542 )
    2.43 ( 0.826 )
    0.20 ( 99999 )
        Calcium
    0.13 ( 0.157 )
    0.04 ( 0.058 )
    -0.17 ( 99999 )
        Chloride
    -1.50 ( 4.324 )
    1.50 ( 1.291 )
    1.00 ( 99999 )
        Glucose
    0.32 ( 0.641 )
    -0.08 ( 0.472 )
    14.90 ( 99999 )
        Magnesium
    0.06 ( 0.107 )
    0.06 ( 0.109 )
    0.00 ( 99999 )
        Phosphate
    0.02 ( 0.281 )
    -0.02 ( 0.336 )
    -0.16 ( 99999 )
        Potassium
    -0.05 ( 0.672 )
    0.20 ( 0.535 )
    1.50 ( 99999 )
        Sodium
    1.00 ( 2.608 )
    2.25 ( 2.217 )
    2.00 ( 99999 )
        Triglyceride
    0.45 ( 1.652 )
    0.29 ( 0.320 )
    0.33 ( 99999 )
    Notes
    [25] - 99999 stands for Not Applicable
    No statistical analyses for this end point

    Secondary: Change from Baseline at Day 28 in Chemistry Laboratory Parameters- Bilirubin; Creatinine; Direct Bilirubin; Urate

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    End point title
    		 Change from Baseline at Day 28 in Chemistry Laboratory Parameters- Bilirubin; Creatinine; Direct Bilirubin; Urate [26]
    End point description
    Baseline is defined as the last assessment with non-missing value on or prior to the first date of the lymphodepletion chemotherapy. Safety Analysis Set included all participants who received conforming JCAR017 infusion.
    End point type
    Secondary
    End point timeframe
    Baseline and at Day 28
    Notes
    [26] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: Statistical Analysis Comparison was not planned as per protocol.
    End point values
    		 0.05 x 10^6 CAR+ T cells/kg 0.15 x 10^6 CAR+ T cells/kg 0.50 x 106 CAR+T cells/kg
    Number of subjects analysed
    6
    5
    1 [27]
    Units: umol/L
    arithmetic mean (standard error)
        Bilirubin
    20.26 ( 41.498 )
    -1.00 ( 6.928 )
    2.00 ( 99999 )
        Creatinine
    9.84 ( 14.218 )
    1.50 ( 9.000 )
    -2.00 ( 99999 )
        Direct Bilirubin
    13.44 ( 33.601 )
    -0.25 ( 0.500 )
    0.00 ( 99999 )
        Urate
    43.51 ( 94.081 )
    85.00 ( 71.615 )
    9.00 ( 99999 )
    Notes
    [27] - 99999 stands for Not Applicable
    No statistical analyses for this end point

    Secondary: Number of Participants with Manufacturing Success of JCAR017 product

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    End point title
    		 Number of Participants with Manufacturing Success of JCAR017 product
    End point description
    Successful product was defined as JCAR017 product was generated and able to be QC released (including nonconforming product) for infusion. Unsuccessful product is defined as no JCAR017 product could be generated after two manufacturing attempts using a single apheresis product for starting material or product was unable to be QC released for infusion. Pre-Treatment Set included all participants who have screened successfully into the study and underwent leukapheresis.
    End point type
    Secondary
    End point timeframe
    From screening to JCAR017 infusion (day -35 to day 1)
    End point values
    		 0.05 x 10^6 CAR+ T cells/kg 0.15 x 10^6 CAR+ T cells/kg 0.50 x 106 CAR+T cells/kg Not Assigned
    Number of subjects analysed
    9
    8
    3
    1
    Units: participants
    7
    7
    2
    1
    No statistical analyses for this end point

    Secondary: Overall Response Rate (ORR)

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    End point title
    		 Overall Response Rate (ORR) [28]
    End point description
    ORR is defined as the percentage of participants who achieved either a complete response (CR) or complete response with incomplete blood recovery (CRi) on Day 28 that is confirmed on Day 56. Response assessment was performed according to the 2019 Comprehensive Cancer Network (NCCN) response criteria guidelines for pediatric acute lymphoblastic leukemia (ALL). CR is defined as absence of circulating blasts, extramedullary disease, lymphadenopathy, splenomegaly, skin/gum infiltration/testicular mass/CNS involvement, Trilineage hematopoiesis (TLH) and < 5%, Absolute neutrophil count (ANC) > 1000 per microliter, Platelets > 100,000 per microliter and no recurrence for 4 weeks. CRi is defined as meeting all criteria for CR except platelets < 100,000/μL or ANC is < 1000/μL. Efficacy Analysis Set.
    End point type
    Secondary
    End point timeframe
    Up to Day 56
    Notes
    [28] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: Statistical Analysis Comparison was not planned as per protocol.
    End point values
    		 0.05 x 10^6 CAR+ T cells/kg 0.15 x 10^6 CAR+ T cells/kg 0.50 x 106 CAR+T cells/kg
    Number of subjects analysed
    6
    4
    1
    Units: percentage of participants
        number (confidence interval 95%)
    50.0 (11.8 to 88.2)
    25.0 (0.6 to 80.6)
    100.0 (2.5 to 100.0)
    No statistical analyses for this end point

    Secondary: Duration of Response (DOR)

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    End point title
    		 Duration of Response (DOR) [29]
    End point description
    DOR is defined as time from first response (either CR or CRi) until progressive disease (PD), disease relapse, or death from any cause, whichever occurs first. Response assessment was performed according to the 2019 Comprehensive Cancer Network (NCCN) response criteria guidelines for pediatric ALL. CR is defined as absence of circulating blasts, extramedullary disease, lymphadenopathy, splenomegaly, skin/gum infiltration/testicular mass/CNS involvement, Trilineage hematopoiesis (TLH) and < 5%, Absolute neutrophil count (ANC) > 1000 per microliter, Platelets > 100,000 per microliter and no recurrence for 4 weeks. CRi is defined as meeting all criteria for CR except platelets < 100,000/μL or ANC is < 1000/μL. Disease progression is defined as increase of at least 25% in the absolute number of circulating or bone marrow blasts or development of extramedullary disease. Efficacy Analysis Set.
    End point type
    Secondary
    End point timeframe
    From first response (either CR or CRi) until progressive disease (PD), disease relapse, or death from any cause, whichever occurs first (Up to approximately 14 months)
    Notes
    [29] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: Statistical Analysis Comparison was not planned as per protocol.
    End point values
    		 0.05 x 10^6 CAR+ T cells/kg 0.15 x 10^6 CAR+ T cells/kg 0.50 x 106 CAR+T cells/kg
    Number of subjects analysed
    3
    1 [30]
    1 [31]
    Units: months
        median (full range (min-max))
    13.70 (2.46 to 13.70)
    99999 (99999 to 99999)
    99999 (99999 to 99999)
    Notes
    [30] - 99999 = Data not calculable (insufficient number of participants with events)
    [31] - 99999 = Data not calculable (insufficient number of participants with events)
    No statistical analyses for this end point

    Secondary: Relapse Free Survival (RFS)

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    End point title
    		 Relapse Free Survival (RFS) [32]
    End point description
    RFS is defined as time from conforming JCAR017 infusion to the first progressive disease (PD), relapsed disease or death from any cause, whichever occurs first. Disease progression is defined as increase of at least 25% in the absolute number of circulating or bone marrow blasts or development of extramedullary disease. Relapsed disease is defined as Reappearance of blasts in the blood or bone marrow (> 5%) or > 1% with previous/supportive molecular findings or in any extramedullary site after a CR. CR is defined as absence of circulating blasts, extramedullary disease, lymphadenopathy, splenomegaly, skin/gum infiltration/testicular mass/CNS involvement, Trilineage hematopoiesis (TLH) and < 5%, Absolute neutrophil count (ANC) > 1000 per microliter, Platelets > 100,000 per microliter and no recurrence for 4 weeks. Efficacy Analysis Set.
    End point type
    Secondary
    End point timeframe
    Up to approximately 15 months
    Notes
    [32] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: Statistical Analysis Comparison was not planned as per protocol.
    End point values
    		 0.05 x 10^6 CAR+ T cells/kg 0.15 x 10^6 CAR+ T cells/kg 0.50 x 106 CAR+T cells/kg
    Number of subjects analysed
    6
    4
    1 [33]
    Units: months
        median (full range (min-max))
    7.77 (0.46 to 14.62)
    6.98 (1.15 to 8.97)
    99999 (99999 to 99999)
    Notes
    [33] - 99999 stands for Not Applicable
    No statistical analyses for this end point

    Secondary: Event Free Survival (EFS)

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    End point title
    		 Event Free Survival (EFS) [34]
    End point description
    EFS is defined as time from conforming JCAR017 infusion to progressive disease (PD), relapsed disease, start of a new anticancer therapy including HSCT or death from any cause, whichever occurs first. Disease progression is defined as increase of at least 25% in the absolute number of circulating or bone marrow blasts or development of extramedullary disease. Relapsed disease is defined as Reappearance of blasts in the blood or bone marrow (> 5%) or > 1% with previous/supportive molecular findings or in any extramedullary site after a CR. CR is defined as absence of circulating blasts, extramedullary disease, lymphadenopathy, splenomegaly, skin/gum infiltration/testicular mass/CNS involvement, Trilineage hematopoiesis (TLH) and < 5%, Absolute neutrophil count (ANC) > 1000 per microliter, Platelets > 100,000 per microliter and no recurrence for 4 weeks. Only responders are included in he analysis. Censored participants were also analyzed. Efficacy Analysis Set.
    End point type
    Secondary
    End point timeframe
    From conforming JCAR017 infusion to PD, relapsed disease, start of a new anticancer therapy including HSCT or death from any cause, whichever occurs first (Up to approximately 15 months)
    Notes
    [34] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: Statistical Analysis Comparison was not planned as per protocol.
    End point values
    		 0.05 x 10^6 CAR+ T cells/kg 0.15 x 10^6 CAR+ T cells/kg 0.50 x 106 CAR+T cells/kg
    Number of subjects analysed
    5
    4
    1
    Units: months
        median (full range (min-max))
    3.24 (0.46 to 14.62)
    5.42 (1.15 to 8.97)
    3.12 (3.12 to 3.12)
    No statistical analyses for this end point

    Secondary: Overall Survival (OS)

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    End point title
    		 Overall Survival (OS) [35]
    End point description
    OS is defined as the interval from the date of first confirming JCAR017 infusion to the date of death due to any reason. Efficacy Analysis Set. The Efficacy Analysis Set included all participants who fulfill all study eligibility criteria (prospectively and retrospectively) and receive JCAR017 infusion in accordance with drug product release specifications (i.e., conforming JCAR017 product).
    End point type
    Secondary
    End point timeframe
    From the date of first confirming JCAR017 infusion to the date of death due to any reason (Up to approximately 63 months)
    Notes
    [35] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: Statistical Analysis Comparison was not planned as per protocol.
    End point values
    		 0.05 x 10^6 CAR+ T cells/kg 0.15 x 10^6 CAR+ T cells/kg 0.50 x 106 CAR+T cells/kg
    Number of subjects analysed
    6 [36]
    4 [37]
    1 [38]
    Units: months
        median (confidence interval 95%)
    7.13 (0.76 to 99999)
    7.08 (4.99 to 99999)
    99999 (99999 to 99999)
    Notes
    [36] - 99999 stands for Not Applicable
    [37] - 99999 stands for Not Applicable
    [38] - 99999 stands for Not Applicable (Due to low number of events)
    No statistical analyses for this end point

    Secondary: Minimal Residual Response (MRD) Negative Response Rate

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    End point title
    		 Minimal Residual Response (MRD) Negative Response Rate [39]
    End point description
    Minimal Residual Disease (MRD) Negative Response Rate is defined as the percentage of participants achieving either a CR or CRi with a MRD negative bone marrow on Day 28, confirmed on Day 56. CR is defined as absence of circulating blasts, extramedullary disease, lymphadenopathy, splenomegaly, skin/gum infiltration/testicular mass/CNS involvement, Trilineage hematopoiesis (TLH) and < 5%, Absolute neutrophil count (ANC) > 1000 per microliter, Platelets > 100,000 per microliter and no recurrence for 4 weeks. CRi is defined as meeting all criteria for CR except platelets < 100,000/μL or ANC is < 1000/μL. Disease progression is defined as increase of at least 25% in the absolute number of circulating or bone marrow blasts or development of extramedullary disease. Disease assessments recorded on or after start of a new anticancer therapy, including HSCT, will not be considered, nor will disease assessments reported after a PD or relapse has been observed. Efficacy Analysis Set.
    End point type
    Secondary
    End point timeframe
    Up to Day 56
    Notes
    [39] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: Statistical Analysis Comparison was not planned as per protocol.
    End point values
    		 0.05 x 10^6 CAR+ T cells/kg 0.15 x 10^6 CAR+ T cells/kg 0.50 x 106 CAR+T cells/kg
    Number of subjects analysed
    6
    4
    1
    Units: percentage of participants
        number (confidence interval 95%)
    16.7 (0.4 to 64.1)
    25.0 (0.6 to 80.6)
    100.0 (2.5 to 100.0)
    No statistical analyses for this end point

    Secondary: Number of Participants who Achieved a Response after JCAR017 Infusion and then Proceeded to Hematopoietic Stem Cell Transplant

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    End point title
    		 Number of Participants who Achieved a Response after JCAR017 Infusion and then Proceeded to Hematopoietic Stem Cell Transplant [40]
    End point description
    Number of participants who undergo HSCT after receiving a JCAR017 infusion and achieving a response are presented. The time of proceeding to HSCT is defined as the time of commencing the conditioning regimen as required for HSCT. CR is defined as absence of circulating blasts, extramedullary disease, lymphadenopathy, splenomegaly, skin/gum infiltration/testicular mass/CNS involvement, Trilineage hematopoiesis (TLH) and < 5%, Absolute neutrophil count (ANC) > 1000 per microliter, Platelets > 100,000 per microliter and no recurrence for 4 weeks. CRi is defined as meeting all criteria for CR except platelets < 100,000/μL or ANC is < 1000/μL. Disease progression is defined as increase of at least 25% in the absolute number of circulating or bone marrow blasts or development of extramedullary disease.
    End point type
    Secondary
    End point timeframe
    Up to approximately 24 months
    Notes
    [40] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: Statistical Analysis Comparison was not planned as per protocol.
    End point values
    		 0.05 x 10^6 CAR+ T cells/kg 0.15 x 10^6 CAR+ T cells/kg 0.50 x 106 CAR+T cells/kg
    Number of subjects analysed
    7
    6
    1
    Units: participants
    1
    1
    1
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    All-cause mortality was collected from JCAR017 infusion until their study completion (up to approximately 63 months). Serious and non-serious adverse events were collected from first JCAR017 infusion to 90 days post JCAR017 infusion
    Adverse event reporting additional description
    All-cause mortality was collected for all participants that underwent leukapheresis and were assigned to dose level. Serious and non-serious adverse events were collected for all participants who received conforming JCAR017 infusion.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    23.1
    Reporting groups
    Reporting group title
    0.05 x 10^6 CAR+ T cells/kg
    Reporting group description
    		 Participants with Relapsed and Refractory Multiple Myeloma were infused with 0.05 x 10^6 CAR+ T cells per kilogram (kg) post leukapheresis.

    Reporting group title
    0.50 x 106 CAR+T cells/kg
    Reporting group description
    		 Participants with RRMM were infused with 0.50 x 106 CAR+T cells/kg post leukapheresis.

    Reporting group title
    0.15 x 10^6 CAR+ T cells/kg
    Reporting group description
    		 Participants with RRMM were infused with 0.15 x 106 CAR+ T cells/kg post leukapheresis.

    Serious adverse events
    		 0.05 x 10^6 CAR+ T cells/kg 0.50 x 106 CAR+T cells/kg 0.15 x 10^6 CAR+ T cells/kg
    Total subjects affected by serious adverse events
         subjects affected / exposed
    5 / 7 (71.43%)
    1 / 1 (100.00%)
    3 / 6 (50.00%)
         number of deaths (all causes)
    4
    1
    3
         number of deaths resulting from adverse events
    0
    0
    0
    Injury, poisoning and procedural complications
    Extradural haematoma
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 1 (0.00%)
    0 / 6 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Infusion related reaction
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 1 (0.00%)
    1 / 6 (16.67%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Nervous system disorders
    Neurotoxicity
         subjects affected / exposed
    1 / 7 (14.29%)
    1 / 1 (100.00%)
    1 / 6 (16.67%)
         occurrences causally related to treatment / all
    1 / 1
    1 / 1
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Brain oedema
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 1 (100.00%)
    0 / 6 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Immune system disorders
    Cytokine release syndrome
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 1 (0.00%)
    3 / 6 (50.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    3 / 3
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Infections and infestations
    Sepsis
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 1 (0.00%)
    0 / 6 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Pneumonia
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 1 (0.00%)
    0 / 6 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Viraemia
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 1 (0.00%)
    0 / 6 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 0.05 x 10^6 CAR+ T cells/kg 0.50 x 106 CAR+T cells/kg 0.15 x 10^6 CAR+ T cells/kg
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    7 / 7 (100.00%)
    1 / 1 (100.00%)
    6 / 6 (100.00%)
    Vascular disorders
    Cyanosis
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 1 (0.00%)
    1 / 6 (16.67%)
         occurrences all number
    0
    0
    1
    Hypertension
         subjects affected / exposed
    1 / 7 (14.29%)
    1 / 1 (100.00%)
    0 / 6 (0.00%)
         occurrences all number
    1
    1
    0
    General disorders and administration site conditions
    Pyrexia
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 1 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    3
    0
    0
    Immune system disorders
    Cytokine release syndrome
         subjects affected / exposed
    4 / 7 (57.14%)
    1 / 1 (100.00%)
    1 / 6 (16.67%)
         occurrences all number
    4
    1
    1
    Haemophagocytic lymphohistiocytosis
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 1 (0.00%)
    1 / 6 (16.67%)
         occurrences all number
    0
    0
    1
    Hypogammaglobulinaemia
         subjects affected / exposed
    1 / 7 (14.29%)
    1 / 1 (100.00%)
    1 / 6 (16.67%)
         occurrences all number
    1
    1
    1
    Respiratory, thoracic and mediastinal disorders
    Pleural effusion
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 1 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    1
    0
    0
    Cough
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 1 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    1
    0
    0
    Psychiatric disorders
    Insomnia
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 1 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    1
    0
    0
    Investigations
    Alanine aminotransferase increased
         subjects affected / exposed
    4 / 7 (57.14%)
    1 / 1 (100.00%)
    0 / 6 (0.00%)
         occurrences all number
    4
    1
    0
    Aspartate aminotransferase increased
         subjects affected / exposed
    4 / 7 (57.14%)
    0 / 1 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    12
    0
    0
    Blood fibrinogen decreased
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 1 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    1
    0
    0
    Gamma-glutamyltransferase increased
         subjects affected / exposed
    4 / 7 (57.14%)
    0 / 1 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    5
    0
    0
    Interleukin level increased
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 1 (0.00%)
    1 / 6 (16.67%)
         occurrences all number
    0
    0
    1
    International normalised ratio increased
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 1 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    1
    0
    0
    Oxygen saturation decreased
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 1 (0.00%)
    2 / 6 (33.33%)
         occurrences all number
    0
    0
    2
    Serum ferritin increased
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 1 (0.00%)
    1 / 6 (16.67%)
         occurrences all number
    0
    0
    1
    Blood uric acid increased
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 1 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    1
    0
    0
    Blood triglycerides increased
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 1 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    1
    0
    0
    Bilirubin conjugated increased
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 1 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    1
    0
    0
    Blood bilirubin increased
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 1 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    3
    0
    0
    Blood creatinine increased
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 1 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    5
    0
    0
    Injury, poisoning and procedural complications
    Refractoriness to platelet transfusion
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 1 (0.00%)
    1 / 6 (16.67%)
         occurrences all number
    0
    0
    1
    Cardiac disorders
    Tachycardia
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 1 (100.00%)
    0 / 6 (0.00%)
         occurrences all number
    0
    1
    0
    Bradycardia
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 1 (100.00%)
    1 / 6 (16.67%)
         occurrences all number
    0
    1
    2
    Nervous system disorders
    Dizziness
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 1 (0.00%)
    1 / 6 (16.67%)
         occurrences all number
    0
    0
    1
    Hemiplegia
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 1 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    1
    0
    0
    Neurotoxicity
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 1 (100.00%)
    1 / 6 (16.67%)
         occurrences all number
    0
    1
    1
    Blood and lymphatic system disorders
    Anaemia
         subjects affected / exposed
    5 / 7 (71.43%)
    1 / 1 (100.00%)
    5 / 6 (83.33%)
         occurrences all number
    10
    2
    14
    Thrombocytopenia
         subjects affected / exposed
    4 / 7 (57.14%)
    1 / 1 (100.00%)
    3 / 6 (50.00%)
         occurrences all number
    4
    2
    6
    Neutropenia
         subjects affected / exposed
    3 / 7 (42.86%)
    1 / 1 (100.00%)
    2 / 6 (33.33%)
         occurrences all number
    9
    2
    2
    Leukopenia
         subjects affected / exposed
    4 / 7 (57.14%)
    1 / 1 (100.00%)
    1 / 6 (16.67%)
         occurrences all number
    6
    2
    1
    Febrile neutropenia
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 1 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    1
    0
    0
    Coagulopathy
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 1 (100.00%)
    0 / 6 (0.00%)
         occurrences all number
    0
    3
    0
    Ear and labyrinth disorders
    Ear pain
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 1 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    1
    0
    0
    Gastrointestinal disorders
    Colitis
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 1 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    1
    0
    0
    Anal fissure
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 1 (0.00%)
    1 / 6 (16.67%)
         occurrences all number
    1
    0
    1
    Anaesthesia oral
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 1 (0.00%)
    1 / 6 (16.67%)
         occurrences all number
    0
    0
    1
    Abdominal pain
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 1 (0.00%)
    1 / 6 (16.67%)
         occurrences all number
    1
    0
    1
    Constipation
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 1 (0.00%)
    1 / 6 (16.67%)
         occurrences all number
    0
    0
    1
    Diarrhoea
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 1 (100.00%)
    1 / 6 (16.67%)
         occurrences all number
    0
    2
    1
    Intestinal haemorrhage
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 1 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    1
    0
    0
    Lip dry
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 1 (0.00%)
    1 / 6 (16.67%)
         occurrences all number
    0
    0
    1
    Nausea
         subjects affected / exposed
    2 / 7 (28.57%)
    0 / 1 (0.00%)
    1 / 6 (16.67%)
         occurrences all number
    2
    0
    3
    Oral mucosal erythema
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 1 (0.00%)
    1 / 6 (16.67%)
         occurrences all number
    0
    0
    1
    Vomiting
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 1 (0.00%)
    3 / 6 (50.00%)
         occurrences all number
    1
    0
    4
    Hepatobiliary disorders
    Hyperbilirubinaemia
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 1 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    1
    0
    0
    Cholecystitis
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 1 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    1
    0
    0
    Skin and subcutaneous tissue disorders
    Skin erosion
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 1 (0.00%)
    1 / 6 (16.67%)
         occurrences all number
    0
    0
    1
    Dermatitis atopic
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 1 (0.00%)
    1 / 6 (16.67%)
         occurrences all number
    0
    0
    1
    Erythema
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 1 (0.00%)
    1 / 6 (16.67%)
         occurrences all number
    0
    0
    1
    Night sweats
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 1 (100.00%)
    0 / 6 (0.00%)
         occurrences all number
    0
    1
    0
    Rash
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 1 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    1
    0
    0
    Rash maculo-papular
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 1 (0.00%)
    1 / 6 (16.67%)
         occurrences all number
    0
    0
    1
    Skin exfoliation
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 1 (0.00%)
    1 / 6 (16.67%)
         occurrences all number
    0
    0
    1
    Renal and urinary disorders
    Haematuria
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 1 (0.00%)
    1 / 6 (16.67%)
         occurrences all number
    0
    0
    1
    Musculoskeletal and connective tissue disorders
    Myalgia
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 1 (100.00%)
    0 / 6 (0.00%)
         occurrences all number
    0
    1
    0
    Infections and infestations
    Clostridium difficile infection
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 1 (100.00%)
    0 / 6 (0.00%)
         occurrences all number
    0
    2
    0
    Bacteraemia
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 1 (0.00%)
    1 / 6 (16.67%)
         occurrences all number
    1
    0
    1
    Staphylococcal bacteraemia
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 1 (100.00%)
    0 / 6 (0.00%)
         occurrences all number
    0
    1
    0
    Cytomegalovirus infection
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 1 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    1
    0
    0
    Paronychia
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 1 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    1
    0
    0
    Pneumonia fungal
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 1 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    1
    0
    0
    Upper respiratory tract infection
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 1 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    1
    0
    0
    Metabolism and nutrition disorders
    Decreased appetite
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 1 (0.00%)
    1 / 6 (16.67%)
         occurrences all number
    1
    0
    1
    Fluid retention
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 1 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    1
    0
    0
    Hyperamylasaemia
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 1 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    1
    0
    0
    Hyperlipidaemia
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 1 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    1
    0
    0
    Hypernatraemia
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 1 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    1
    0
    0
    Hyperphosphataemia
         subjects affected / exposed
    1 / 7 (14.29%)
    1 / 1 (100.00%)
    0 / 6 (0.00%)
         occurrences all number
    1
    1
    0
    Hypertriglyceridaemia
         subjects affected / exposed
    3 / 7 (42.86%)
    0 / 1 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    3
    0
    0
    Hypoglycaemia
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 1 (0.00%)
    1 / 6 (16.67%)
         occurrences all number
    0
    0
    1
    Hypophosphataemia
         subjects affected / exposed
    1 / 7 (14.29%)
    1 / 1 (100.00%)
    0 / 6 (0.00%)
         occurrences all number
    1
    2
    0
    Tumour lysis syndrome
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 1 (0.00%)
    1 / 6 (16.67%)
         occurrences all number
    0
    0
    1
    Hypoalbuminaemia
         subjects affected / exposed
    2 / 7 (28.57%)
    0 / 1 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    3
    0
    0
    Hypermagnesaemia
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 1 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    1
    0
    0
    Hypomagnesaemia
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 1 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    2
    0
    0
    Hypokalaemia
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 1 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    3
    0
    0

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    07 Jan 2019
    PA01 was was implemented to amend inclusion and exclusion criteria following an Urgent Safety Measure on another liso-cel clinical trial.
    10 Sep 2019
    PA 02 was implemented to amend the initial starting dose of liso-cel and Phase 1 study design.

    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.
    Study was terminated early on the grounds that liso-cel did not represent a significant therapeutic benefit over existing therapies for the treatment of pediatric B-cell malignancies.
    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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