Flag of the European Union EU Clinical Trials Register Help

Clinical trials

The European Union Clinical Trials Register   allows you to search for protocol and results information on:
  • interventional clinical trials that were approved in the European Union (EU)/European Economic Area (EEA) under the Clinical Trials Directive 2001/20/EC
  • clinical trials conducted outside the EU/EEA that are linked to European paediatric-medicine development

  • EU/EEA interventional clinical trials approved under or transitioned to the Clinical Trial Regulation 536/2014 are publicly accessible through the
    Clinical Trials Information System (CTIS).

    The EU Clinical Trials Register currently displays   43974   clinical trials with a EudraCT protocol, of which   7311   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

    Phase 1 trials conducted solely on adults and that are not part of an agreed paediatric investigation plan (PIP) are not publicly available (see Frequently Asked Questions ).  
    Examples: Cancer AND drug name. Pneumonia AND sponsor name.
    How to search [pdf]
    Search Tips: Under advanced search you can use filters for Country, Age Group, Gender, Trial Phase, Trial Status, Date Range, Rare Diseases and Orphan Designation. For these items you should use the filters and not add them to your search terms in the text field.
    Advanced Search: Search tools

    < Back to search results

    Print Download

    EudraCT Number:2017-000106-38
    Sponsor's Protocol Code Number:JCAR017-BCM-001
    National Competent Authority:Austria - BASG
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2018-03-22
    Trial results
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedAustria - BASG
    A.2EudraCT number2017-000106-38
    A.3Full title of the trial
    A Phase 2, Single-arm, Multi-cohort, Multi-center Trial to Determine the Efficacy and Safety of JCAR017 in Adult Subjects with Aggressive B-Cell Non-Hodgkin Lymphoma (TRANSCEND WORLD)
    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 adults with aggressive B-Cell Non-Hodgkin Lymphoma
    A.4.1Sponsor's protocol code numberJCAR017-BCM-001
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    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 Address86 Morris Avenue
    B.5.3.2Town/ citySummit
    B.5.3.3Post codeNJ 07901
    B.5.3.4CountryUnited States
    B.5.4Telephone number+19137096862
    B.5.5Fax number+19088974074
    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, EU/3/18/2018, EU/3/18/2099
    D.3 Description of the IMP
    D.3.1Product nameLisocabtagene maraleucel
    D.3.2Product code JCAR017
    D.3.4Pharmaceutical form Suspension for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.9.2Current sponsor codeJCAR017
    D.3.9.4EV Substance CodeSUB191306
    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. cell therapy medicinal product No
    D. therapy medical product Yes
    D. Engineered Product No
    D. ATIMP (i.e. one involving a medical device) No
    D. 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
    Aggressive 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 23.0
    E.1.2Level LLT
    E.1.2Classification code 10029593
    E.1.2Term Non-Hodgkin's lymphoma NOS
    E.1.2System Organ Class 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    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.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The primary objectives are:
    Cohorts 1, 2, 3, 4, and 5
    - To determine the efficacy, defined as overall response rate (ORR), of JCAR017 in subjects with aggressive B-cell non-Hodgkin lymphoma

    Cohort 7
    - To evaluate the safety of JCAR017 treatment in subjects intended to be treated as outpatients
    E.2.2Secondary objectives of the trial
    - To evaluate the safety and feasibility of administering JCAR017 (Cohorts 1, 2, 3, 4, 5)

    - To determine the efficacy, defined as ORR of JCAR017 intended to be treated as outpatients (Cohort 7)

    - To evaluate other measures of efficacy of JCAR017 ( e.g., complete response rate [CRR], event-free survival [EFS], progression-free survival [PFS], overall survival [OS], duration of response [DOR])

    - To characterize the pharmacokinetic (PK) profile of JCAR017 in the peripheral blood measured using qPCR detection for the JCAR017 vector sequence

    - To describe changes in health-related quality of life (HRQoL) using the global health/QoL, fatigue, physical and cognitive functioning subscales of the European Organisation for Research and Treatment of Cancer – Quality of Life C30 questionnaire (EORTC QLQ-C30), and the Functional Assessment of Cancer Therapy - Lymphoma "Additional concerns" (FACT-LymS)
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Subject is ≥ 18 years of age at the time of signing the informed consent form (ICF).

    2. Subject must understand and voluntarily sign an ICF prior to any study-related assessments/procedures being conducted.

    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. Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1. Subjects not eligible for transplant (TNE) in Cohorts 2 and 3 and subjects in Cohort 5 may be enrolled with ECOG of 2 only if they meet all other inclusion/exclusion criteria.

    6. Subjects with one of the following:
    • Cohort 1: Subjects with DLBCL NOS (de novo or tFL), HGBL and FL3B per WHO 2016 classification (Swerdlow, 2016), after ≥ 2 lines of therapy*, including an anthracycline and rituximab (or other CD20-targeted agent).
    • Cohort 2: Transplant not eligible subjects with DLBCL NOS (de novo or tFL), HGBL and FL3B per WHO 2016 classification (Swerdlow, 2016), who failed first line therapy* including an anthracycline and rituximab (or other CD-20-targeted agent).
    - Transplant not eligible subjects will include those who are deemed ineligible for high-dose chemotherapy and HSCT due to age, performance status or comorbidity, while also having adequate organ function for CAR T cell treatment. At the very least, subjects have to meet one of the following criteria:
    a)age ≥ 70 years
    b) ECOG performance status ≥ 2
    c) impaired pulmonary function (DLCO ≤ 60% adjusted for hemoglobin concentration using the Dinakara equation)
    d) impaired cardiac function (LVEF < 50%)
    e) impaired renal function (CrCl < 60 mL/min)
    f) Impaired hepatic function (AST/ALT > 2x ULN, bilirubin > 2 mg/dL or cirrhosis Child-Pugh B or C).
    - Subjects must fulfil all other in- and exclusion criteria
    • Cohort 3: (Japan only): Subjects meeting eligibility criteria for either Cohort 1 or 2.
    • Cohort 4: Subjects with newly diagnosed HGBL. Subjects must be eligible for anthracycline and rituximab (or other CD20-targeted agent) containing regimen as induction prior to consolidation with JCAR017**
    • Cohort 5: Subjects with PCNSL who failed first line therapy with HDCT and ASCT or who failed to proceed to HDCT and ASCT due to failure of
    PBSC mobilization or insufficient response at the completion of induction
    therapy with high-dose methotrexate-based polychemotherapy regimen
    (eg, high dose methotrexate, high dose cytarabine, rituximab and
    thiotepa [MATRix regimen])
    • Cohort 6: (REMOVED)
    • Cohort 7: Subjects meeting eligibility criteria for Cohort 1 and suitable for outpatient treatment***
    *For subjects with transformed disease, the subject should have had at least 2 lines of systemic therapy for his/her transformed disease (i.e. DLBCL) for Cohort 1 and 1 line for Cohort 2 to be eligible. Lines of therapy do not include those given for a previously indolent condition (i.e. follicular lymphoma). Subjects do NOT have to have anthracycline for their DLBCL if received for indolent disease.
    ** For subjects already undergoing anthracycline and rituximab containing regimen, eligibility is to be discussed with Medical Monitor. Subjects with complete metabolic response after 2 cycles of induction will proceed with JCAR017 infusion only at time of relapse, if applicable.
    *** Subjects must meet the conditions for outpatient treatment and monitoring as outlined in the Outpatient Administration and Monitoring Guidance for Lisocabtagene Maraleucel.

    Note: Subjects with secondary CNS lymphoma involvement may enroll in Cohorts 1 to 4 and 7; subjects with PCNSL are eligible for Cohort 5. Subject selection must consider clinical risk factors for severe adverse events (AEs) and alternative treatment options. Subjects should only be enrolled if the Investigator considers the potential benefit outweighs the risk for the subject. For Cohort 5 and to not compromise safety, subject selection has been restricted to those fit enough to be considered for HDCT and ASCT as their prior therapy.

    7. Histological confirmation of diagnosis at last relapse. Enough tumor material must be available for central confirmation of diagnosis, otherwise a new tumor biopsy is mandated. Note: If the subject did not experience CR since last biopsy, the most recent biopsy will be considered adequate to participate in the trial. For subjects with PCNSL, at a minimum, corresponding pathology report is required if archival tumor material is not available and repeated biopsy not feasible.

    8. For subjects with NHL (except Cohort 5): Subjects must have positron emission tomography (PET)-positive disease as per Lugano Classification

    Please refer to protocol section 4.2 for other 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 would place the subject at unacceptable risk if participating in the study.

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

    4. Subjects with T cell rich/histiocyte rich large B-cell lymphoma (THRBCL), primary cutaneous large B-cell lymphoma, primary mediastinal B-cell lymphoma (PMBCL), Epstein-Barr virus (EBV) positive DLBCL of the elderly, Burkitt lymphoma, and intraocular lymphoma.

    5. Subjects with prior history of malignancies, other than aggressive r/r NHL, unless the subject has been in remission for 2 years with the exception of the following non-invasive malignancies:
    • Basal cell carcinoma of the skin
    • Squamous cell carcinoma of the skin
    • Carcinoma in situ of the cervix
    • Carcinoma in situ of the breast
    • Incidental histologic finding of prostate cancer (T1a or T1b using the TNM [tumor, nodes, metastasis] clinical staging system) or prostate cancer that is curative
    •Other completely resected stage 1 solid tumor with low risk for recurrence

    6. Treatment with any prior gene therapy product.

    7. Subjects who have received previous CD19-targeted therapy.

    8. Human immunodeficiency virus (HIV) infection , hepatitis B, or hepatitis C:
    • Subjects with a history of or active HIV are excluded
    • Subjects with active hepatitis B, or active hepatitis C are excluded
    - Subjects with a negative polymerase chain reaction (PCR) assay for viral load for hepatitis B or C are permitted. Subjects positive for hepatitis B surface antigen and/or anti-hepatitis B core antibody with negative viral load are eligible and should be considered for prophylactic antiviral therapy

    9. 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.

    10. Presence of acute or chronic graft-versus-host disease (GVHD).

    11. Active autoimmune disease requiring immunosuppressive therapy.

    12.History of any one of the following cardiovascular conditions within the past 6 months:
    • Heart failure class III or IV as defined by the New York Heart Association (NYHA)
    • Cardiac angioplasty or stenting
    • Myocardial infarction
    • Unstable angina
    • Other clinically significant cardiac disease

    13. History or presence of clinically relevant CNS pathology not related to disease under study such as epilepsy, seizure, aphasia, stroke, cerebral edema, severe brain injuries, dementia, Parkinson's disease, cerebellar disease, organic brain syndrome, or psychosis.

    14. Pregnant or nursing women.

    15. Treatment with alemtuzumab within 6 months of leukapheresis, or treatment with fludarabine or cladribine within 3 months of leukapheresis

    16. Use of the following (see Section 8.2 for full details):
    • 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 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 > 300mg/m2, ifosfamide, bendamustine) within 2 weeks prior to leukapheresis.
    • Experimental agents within 4 weeks prior to leukapheresis unless no response or progressive disease (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 anti-tumor 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 progressive disease in irradiated lesions or have additional non-irradiated, PET-positive lesions to be eligible. Radiation to a single lesion, if additional non-irradiated, measurable PET-positive lesions are present, is allowed up to 2 weeks prior to leukapheresis . Prior WBRT for subjects enrolled in Cohort 5 is not allowed
    • Allogenic HSCT within 90 days prior to leukapheresis.

    See protocol for additional exclusion criteria
    E.5 End points
    E.5.1Primary end point(s)
    - Non-Hodgkin lymphoma (NHL; including secondary central nervous system [CNS] involvement): Overall Response Rate (ORR)
    Cohorts 1, 2, 3 and 4

    - Relapsed/refractory (r/r) primary central nervous system lymphoma (PCNSL): ORR
    Cohort 5

    - Safety in subjects intended to be treated as outpatients
    Cohort 7
    E.5.1.1Timepoint(s) of evaluation of this end point
    Up to 2 years after JCAR017 infusion
    E.5.2Secondary end point(s)
    - Safety

    - Safety in subjects treated as outpatients

    - ORR in subjects intended to be treated as outpatients Cohort7

    - Complete response rate (CRR)

    - Event-free survival (EFS)

    - Progression-free survival (PFS)

    - Overall survival (OS)

    - Duration of response (DOR)

    - Pharmacokinetics (PK) by qPCR

    - Health Related Quality of Life (domain of interest)
    E.5.2.1Timepoint(s) of evaluation of this end point
    Up to 2 years after JCAR017 infusion

    For overall survival (OS) - up to last subject last visit
    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 Yes
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E. trial type description
    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
    United Kingdom
    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 follow-up of this study 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 years4
    E.8.9.1In the Member State concerned months5
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years4
    E.8.9.2In all countries concerned by the trial months5
    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 No
    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) No
    F.1.1.5Children (2-11years) No
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 58
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 58
    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 No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state10
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 106
    F.4.2.2In the whole clinical trial 116
    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 Decision2018-07-05
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-05-24
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2023-12-15
    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.

    European Medicines Agency © 1995-2024 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands