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    Summary
    EudraCT Number:2018-001246-34
    Sponsor's Protocol Code Number:JCAR017-BCM-004
    National Competent Authority:Belgium - FPS Health-DGM
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2018-06-08
    Trial results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedBelgium - FPS Health-DGM
    A.2EudraCT number2018-001246-34
    A.3Full title of the trial
    A phase 1B/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
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study to assess the efficacy and safety of JCAR017, a CAR-T cell therapy, in pediatric subjects with relapsed or refractory CD19+ B-Cell Acute Lymphoblastic Leukemia and B-Cell Non-Hodgkin Lymphoma.
    A.4.1Sponsor's protocol code numberJCAR017-BCM-004
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/119/2018
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorCelgene Corporation
    B.1.3.4CountryUnited States
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportCelgene Corporation
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationCelgene Corporation
    B.5.2Functional name of contact pointClinicalTrialDisclosure
    B.5.3 Address:
    B.5.3.1Street Address9225 Indian Creek Parkway, Suite 900
    B.5.3.2Town/ cityOverland Park, Kansas
    B.5.3.3Post code66210
    B.5.3.4CountryUnited States
    B.5.4Telephone number+1888260 1599
    B.5.5Fax number+1913266 0394
    B.5.6E-mailClinicalTrialDisclosure@celgene.com
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEU/3/17/1890
    D.3 Description of the IMP
    D.3.1Product nameJCAR017
    D.3.2Product code JCAR017
    D.3.4Pharmaceutical form Suspension for injection
    D.3.4.1Specific paediatric formulation Yes
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNAutologous CD4+ and CD8+ T cells expressing a CD19-specific chimeric antigen receptor (CAR)
    D.3.9.2Current sponsor codeJCAR017
    D.3.10 Strength
    D.3.10.1Concentration unit Other
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number1
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) Yes
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product Yes
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product Yes
    D.3.11.10Medicinal product containing genetically modified organisms Yes
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Relapsed or refractory (r/r) CD19+ B-Cell Acute Lymphoblastic Leukemia (B-ALL) and B-Cell Non-Hodgkin Lymphoma (B-NHL).
    E.1.1.1Medical condition in easily understood language
    A cancer of B-cells, a type of white blood cell responsible for producing antibodies.
    E.1.1.2Therapeutic area Diseases [C] - Immune System Diseases [C20]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10029593
    E.1.2Term Non-Hodgkin's lymphoma NOS
    E.1.2System Organ Class 100000004864
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level HLGT
    E.1.2Classification code 10025320
    E.1.2Term Lymphomas non-Hodgkin's B-cell
    E.1.2System Organ Class 10005329 - Blood and lymphatic system disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10000845
    E.1.2Term Acute lymphoblastic leukemia
    E.1.2System Organ Class 100000004864
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10063625
    E.1.2Term Acute lymphoblastic leukemia recurrent
    E.1.2System Organ Class 100000004864
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Primary Objectives

    Phase 1b:
    The primary objective of the Phase 1b is to identify the recommended Phase 2 dose (RP2D) of JCAR017 in pediatric subjects with relapsed/refractory (r/r) B-cell acute lymphoblastic leukemia (B-ALL).

    Phase 2:
    The primary objective of Phase 2 is to evaluate the efficacy of JCAR017 in the following disease cohorts:
    - Cohort 1 (r/r B-ALL): Overall response rate (ORR) defined as proportion of subjects with a complete response (CR) or complete response with incomplete blood count recovery (CRi) on Day 28 that must be confirmed on Day 56

    - Cohort 2 (minimal residual disease [MRD]+ B-ALL): MRD- rate defined as proportion of subjects with a MRD- response on Day 28 that must be confirmed on Day 56

    - Cohort 3 (r/r B-cell non-Hodgkin lymphoma [B-NHL] (diffuse large B-cell lymphoma [DLBCL], Burkitt lymphoma [BL] or primary mediastinal large B-cell lymphoma [PMBCL])): ORR defined as proportion of subjects with a CR or partial response (PR) on Day 28.
    E.2.2Secondary objectives of the trial
    The secondary objectives for Phase 1b and Phase 2 are:

    - Evaluate the feasibility of manufacturing JCAR017 measured by the percentage of product generated successfully (Phase 1b only)

    - Evaluate ORR in the non-selected dose from Phase 1b

    - Evaluate safety and tolerability assessments during 24 months after JCAR017 infusion

    - To further evaluate efficacy by assessing the duration of response (DOR), relapse-free survival (RFS), event-free survival (EFS), and overall survival (OS) during 24 months after JCAR017 infusion

    - Evaluate the percentage of r/r B-ALL subjects who achieve CR or CRi with no MRD detected in bone marrow (BM) (<0.01% by a validated assay) assessed during 24 months after JCAR017 infusion

    - Assess the percentage of subjects who achieve a response after JCAR017 infusion and then proceed to hematopoietic stem cell transplant (HSCT)

    - Characterize the pharmacokinetic (PK) profile of JCAR017 assessed during 24 months after JCAR017 infusion
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Phase 1b: Subject < 18 years of age and weighs ≥ 12 kg (for subjects receiving a dose of 0.5x10^6 JCAR017 CAR+ T cells/kg) or ≥ 6 kg (for subjects receiving a dose of 1x10^6 JCAR017 CAR+ T cells/kg) at the time of signing the informed consent form (ICF)/informed assent form (IAF).
    Phase 2: Subject ≤ 25 years of age and weighs ≥ 12 kg (if RP2D is 0.5 x 10^6 JCAR017 CAR+ T cells/kg) or ≥ 6 kg (if RP2D is 1 x 10^6 JCAR017 CAR+ T cells/kg) at the time of signing the ICF/IAF.

    2. Subject (when applicable, parental/legal representative) must understand and voluntarily provide permission to the ICF/IAF prior to conducting any study-related assessments/procedures.

    3. Subject is willing and able to adhere to the study visit schedule and other protocol requirements.

    4. Investigator considers the subject is appropriate for adoptive T cell therapy.

    5. Evidence of CD19 expression via flow cytometry (peripheral blood or bone marrow) or immunohistochemistry (bone marrow biopsy)

    6. Subject has a Karnofsky score of ≥ 50 (subjects ≥ 16 years of age) or a Lansky score ≥ 50 (subjects < 16 years of age).

    7. Phase 1b: Subjects with r/r B-ALL, defined as morphological evidence of disease in BM (5% or greater lymphoblast by morphology) and either of the following:

    First or greater marrow relapse, or
    Any marrow relapse after allogeneic HSCT, or
    Primary refractory defined as not achieving a CR or a CRi after 2 or more separate induction regimens (or chemo-refractory as not achieving CR/CRi after 1 cycle of standard chemotherapy for relapsed leukemia), or
    Ineligible for allogeneic HSCT
    Note: Subjects will be included regardless of MRD status.

    Phase 2: Subjects with one of the following:

    Cohort 1: r/r B-ALL, defined as morphological evidence of disease in BM (5% or greater lymphoblast by morphology) and either:
    First or greater marrow relapse, or
    Any marrow relapse after allogeneic HSCT, or
    Primary refractory defined as not achieving a CR or a CRi after 2 or more separate induction regimens (or chemo-refractory as not achieving CR/CRi after 1 cycle of standard chemotherapy for relapsed leukemia), or
    Ineligible for allogeneic HSCT.

    Cohort 2: MRD+ B-ALL, defined as:
    < 5% lymphoblasts by morphology
    MRD detected by a validated assay at a frequency of 1 x10-4 or greater in BM cells after two lines of therapy.

    Cohort 3: r/r B-NHL, defined as measurable disease after 1 or more lines of chemotherapy and/or having failed HSCT or being ineligible for HSCT.

    8. Subjects with Philadelphia chromosome positive ALL are eligible if they are intolerant to or have failed one or more lines of tyrosine-kinase inhibitor (TKI) therapy or if TKI therapy is contraindicated.

    9. Adequate organ function, defined as:
    Adequate BM function to receive LD chemotherapy as assessed by the Investigator.
    Subject with adequate renal function, which is defined as:
    Creatinine clearance calculated using the Schwartz formula, (Schwartz, 1976) or radioisotope glomerular filtration rate (GFR) > 70 mL/min/1.73 m2.
    Alanine aminotransferase (ALT) ≤ 5 x upper limit of normal (ULN) and total bilirubin < 2.0 mg/dL (or < 3.0 mg/dL for subjects with Gilbert’s syndrome or
    lymphomatous infiltration of the liver).
    Adequate pulmonary function, defined as ≤ Grade 1 dyspnea according to Common Toxicity Criteria for Adverse Events (CTCAE) and oxygen saturation (SaO2) ≥ 92% on room air.
    Adequate cardiac function, defined as left ventricular ejection fraction (LVEF) ≥ 40% as assessed by echocardiogram (ECHO) or multi-gated acquisition scan (MUGA) within 4 weeks prior to leukapheresis.

    10. Adequate vascular access for leukapheresis procedure.

    11. Subjects must agree to not donate blood, organs, sperm or semen, and egg cells for usage in other individuals while receiving as well as within 12 months after the JCAR017 infusion and until CAR T cells are no longer present by qPCR on 2 consecutive tests, whichever occurs last.

    12. Female children of childbearing potential (FCCBP) and females of childbearing potential (FCBP) must:
    Have two negative pregnancy tests as verified by the Investigator (one negative serum beta human chorionic gonadotropin [ß-hCG] pregnancy test result at screening and one negative serum pregnancy test within 48 hours prior to the first dose of LD chemotherapy). Subjects must agree to have another pregnancy test performed 90 days post JCAR017 infusion. This applies even if the subject practices true abstinence** from heterosexual contact.

    ** True abstinence is acceptable when this is in line with the preferred and usual lifestyle of the subject. In contrast, periodic abstinence (e.g., calendar, ovulation, symptothermal, post-ovulation methods) and withdrawal are not acceptable methods of contraception.

    See section 4.2 of the protocol for further inclusion criteria.
    E.4Principal exclusion criteria
    1. Subject has any significant medical condition, laboratory abnormality, or psychiatric illness that would prevent the subject from participating in the study.

    2. Subject has any condition including the presence of laboratory abnormalities, which places the subject at unacceptable risk if he/she were to participate in the study.

    3. Subject has any condition that confounds the ability to interpret data from the study.

    4. Subject with a history of another primary malignancy that has not been in remission for at least 2 years prior to enrollment.

    5. Subjects who have received previous CD19-targeted therapy must have CD19-positive disease confirmed since completing the prior CD19-targeted therapy.

    6. Prior CAR T cell or other genetically-modified T cell therapy.

    7. Subject with a previous history of or active hepatitis B, hepatitis C, or human immunodeficiency virus (HIV) infection.

    8. Subjects with uncontrolled systemic fungal, bacterial, viral or other infection (including tuberculosis) despite appropriate antibiotics or other treatment at the time of leukapheresis or JCAR017 infusion.

    9. Subject has presence of acute or chronic graft-versus-host disease (GVHD).

    10. Subject with active autoimmune disease requiring immunosuppressive therapy.

    11. Subject has cardiac disorders (CTCAE version 4.03 Grade 3 or 4) within the past 6 months.

    12. Subject with a concomitant genetic syndrome, with the exception of Down’s syndrome.

    13. Subject with active CNS disease and significant neurological deterioration. Subjects with CNS-2 or CNS-3 involvement are eligible provided they are asymptomatic and do not have significant neurological deterioration and, in the opinion of the study investigator, the CNS disease burden can be controlled until JCAR017 infusion.

    14. Subject with a history or presence of clinically relevant CNS pathology such as epilepsy, seizure, paresis, aphasia, stroke, severe brain injuries, dementia, Parkinson’s disease, cerebellar disease, organic brain syndrome, or psychosis.

    15. Subject is pregnant or nursing.

    16. Subject has used the following:
    Therapeutic doses of corticosteroids (defined as > 20 mg/day prednisone or equivalent) within 7 days prior to leukapheresis or 72 hours prior to JCAR017 infusion. Physiologic replacement, topical, and inhaled steroids are permitted.
    Low-dose chemotherapy (eg, vincristine, rituximab, cyclophosphamide ≤ 300 mg/m2) given after leukapheresis to maintain disease control must be stopped ≥ 7 days prior to LD chemotherapy.
    Cytotoxic chemotherapeutic agents that are not considered lymphotoxic (see below) within 1 week prior to leukapheresis. Oral anticancer agents, including lenalidomide and ibrutinib, are allowed if at least 3 half-lives have elapsed prior to leukapheresis.
    Lymphotoxic chemotherapeutic agents (eg, cyclophosphamide, ifosfamide, bendamustine) within 2 weeks prior to leukapheresis.
    Experimental agents within 4 weeks prior to leukapheresis unless no response or PD is documented on the experimental therapy and at least 3 half-lives have elapsed prior to leukapheresis.
    Immunosuppressive therapies within 4 weeks prior to leukapheresis and JCAR017 infusion (eg, calcineurin inhibitors, methotrexate or other chemotherapeutics, mycophenolate, rapamycin, thalidomide, immunosuppressive antibodies such as antitumor necrosis factor [TNF], anti-IL-6, or anti-IL-6R).
    Donor lymphocyte infusions (DLI) within 6 weeks prior to JCAR017 infusion.
    Radiation within 6 weeks prior to leukapheresis. Subjects must have PD in irradiated lesions or have additional non-irradiated lesions to be eligible. Radiation to a single lesion, if additional non-irradiated, measurable lesions are present, is allowed up to 2
    weeks prior to leukapheresis.
    Allogeneic HSCT within 90 days prior to leukapheresis.
    E.5 End points
    E.5.1Primary end point(s)
    Phase 1b
    Recommended Phase 2 dose (RP2D). Identify pharmacokinetic (PK), and number of subjects experiencing a dose-limiting toxicity (DLT).

    Phase 2 - Cohort 1
    (relapsed/refractory [r/r] B-cell acute lymphoblastic leukemia [B-ALL]): Overall response rate (ORR). Percentage of subjects achieving a confirmed complete response (CR) or complete response with incomplete blood count recovery (CRi)

    Phase 2 - Cohort 2
    (MRD+ BALL): Minimal residual disease (MRD) negative (MRD-) rate. Percentage of subjects achieving a confirmed MRD negative rate

    Phase 2 - Cohort 3
    (r/r B-cell non-Hodgkin lymphoma [BNHL]): Overall response rate (ORR). Percentage of subjects achieving CR or partial response (PR)
    E.5.1.1Timepoint(s) of evaluation of this end point
    Phase 1b
    Up to 28 days after JCAR017 infusion

    Phase 2 - Cohort 1
    Day 28 and must be confirmed on Day 56

    Phase 2 - Cohort 2
    Day 28 and must be confirmed on Day 56

    Phase 2 - Cohort 3
    Day 28
    E.5.2Secondary end point(s)
    1. Safety - Type, frequency, and severity of adverse events (AEs), including serious adverse events (SAEs) and laboratory abnormalities

    2. Feasibility of manufacturing JCAR017 (Phase 1b only) - Percentage of JCAR017 product generated successfully

    3. Overall response rate (ORR) in the non-selected dose from Phase 1b - Percentage of subjects achieving a confirmed CR or CRi

    4. Duration of response (DOR) - Time from first response until progressive disease (PD), disease relapse, or death from any cause, whichever occurs first

    5. Relapse-free survival (RFS) - Time from first response to documentation of PD, disease relapse, or death due to any cause, whichever occurs first

    6. Event-free survival (EFS) - Time from JCAR017 infusion to PD, disease relapse, start of a new anticancer therapy, or death from any cause, whichever occurs first

    7. Overall survival (OS) - Time from JCAR017 infusion to time of death due to any cause

    8. MRD response rate (non-selected RP2D cohort in Phase 1b and Cohort 1 in Phase 2 only) - Percentage of subjects achieving a CR or CRi and a negative MRD bone marrow

    9. Rate of hematopoietic stem cell transplant (HSCT) after response to JCAR017 infusion - Percentage of subjects who achieve a response after JCAR017 infusion and then proceed to HSCT

    10. Pharmacokinetics (PK) - Maximum concentration (Cmax), time to peak concentration (Tmax), area under the curve (AUC), including maximum expansion and duration of persistence of JCAR017
    E.5.2.1Timepoint(s) of evaluation of this end point
    Timeframe for secondary endpoints:

    1, 4, 5, 6, 7, 8, 9 and 10. During 2 years after JCAR017 infusion

    2. During Part A (leukapheresis and JCAR017 generation)

    3. On Day 28 and must be confirmed on Day 56
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic Yes
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic Yes
    E.6.12Pharmacoeconomic No
    E.6.13Others Yes
    E.6.13.1Other scope of the trial description
    dose confirmation for use in phase 2
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) Yes
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other Yes
    E.7.1.3.1Other trial type description
    To identify the recommended Phase 2 dose (RP2D) of JCAR017 in pediatric subjects
    E.7.2Therapeutic exploratory (Phase II) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised No
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) Information not present in EudraCT
    E.8.2.2Placebo Information not present in EudraCT
    E.8.2.3Other Information not present in EudraCT
    E.8.2.4Number of treatment arms in the trial1
    E.8.3 The trial involves single site in the Member State concerned Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA16
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Belgium
    France
    Germany
    Italy
    Spain
    United States
    E.8.7Trial has a data monitoring committee Yes
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    The End of Trial is defined as either the date of the last visit of the last subject completing the post-treatment period or the date when the last subject enters the LTFU study, or the date of receipt of the last data point from the last subject that is required for primary, secondary and/or exploratory analysis, as pre-specified in the protocol, whichever is the later date.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years3
    E.8.9.1In the Member State concerned months0
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months0
    E.8.9.2In all countries concerned by the trial days0
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1Number of subjects for this age range: 91
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) Yes
    F.1.1.4.1Number of subjects for this age range: 5
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 25
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 61
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 20
    F.1.3Elderly (>=65 years) No
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception Yes
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Informed consent/assent of a subject and/or a subject’s legal representative must be obtained by the Investigator prior to any study related procedures.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state3
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 50
    F.4.2.2In the whole clinical trial 111
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    Because this protocol involves gene transfer, long-term follow-up for lentiviral vector safety, disease status, and survival will continue on this protocol until 24 months after last infusion of JCAR017, regardless of disease status, and rolled over to a separate LTFU protocol thereafter for up to 15 years after JCAR017 infusion.
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2018-07-18
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-08-20
    P. End of Trial
    P.End of Trial StatusOngoing
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