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A Study to Compare the Efficacy and Safety of JCAR017 to Standard of Care in Adult Subjects With High-risk, Transplant-eligible Relapsed or Refractory Aggressive B-cell Non-Hodgkin Lymphomas

Phase 3
Completed
Conditions
Lymphoma, Non-Hodgkin
Interventions
Drug: Standard of Care
Genetic: JCAR017
Registration Number
NCT03575351
Lead Sponsor
Celgene
Brief Summary

The study will be conducted in compliance with the International Council for Harmonisation (ICH) of Technical Requirements for Registration of Pharmaceuticals for Human Use/Good Clinical Practice (GCP) and applicable regulatory requirements.

This is a randomized, open-label, parallel-group, multi-center trial in adult subjects with Relapsed or refractory (R/R) aggressive Non-Hodgkin lymphoma (NHL) to compare safety and efficacy between the standard of care (SOC) strategy versus JCAR017 (also known as lisocabtagene maraleucel or liso-cel). Subjects will be randomized to either receive SOC (Arm A) or to receive JCAR017 (Arm B).

All subjects randomized to Arm A will receive Standard of care (SOC) salvage therapy (R-DHAP, RICE or R-GDP) as per physician's choice before proceeding to High dose chemotherapy (HDCT) and Hematopoietic stem cell transplant (HSCT).

Subjects from Arm A may be allowed to cross over and receive JCAR017 upon confirmation of an EFS event.

Subjects randomized to Arm B will receive Lymphodepleting (LD) chemotherapy followed by JCAR017 infusion.

Detailed Description

Not available

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
184
Inclusion Criteria
  1. Subject is ≥ 18 years and ≤ 75 years of age at the time of signing the informed consent form (ICF).
  2. Eastern Cooperative Oncology Group (ECOG) performance status ≤ 1.
  3. Histologically proven diffuse large B-cell lymphoma (DLBCL) NOS (de novo or transformed indolent NHL), high grade B-cell lymphoma with MYC and BCL2 and/or BCL6 rearrangements with DLBCL histology (double/triple-hit lymphoma [DHL/THL]), primary mediastinal (thymic) large B-cell lymphoma (PMBCL), T cell/histiocyte-rich large B-cell lymphoma (THRBCL) or follicular lymphoma grade 3B. Enough tumor material must be available for confirmation by central pathology.
  4. Refractory or relapsed within 12 months from CD20 antibody and anthracycline containing first line therapy.
  5. [18F] fluorodeoxyglucose (FDG) positron emission tomography (PET) positive lesion at screening. (Deauville score 4 or 5)
  6. Adequate organ function
  7. Participants must agree to use effective contraception
Exclusion Criteria
  1. Subjects not eligible for hematopoietic stem cell transplantation (HSCT).

  2. Subjects planned to undergo allogeneic stem cell transplantation.

  3. Subjects with, primary cutaneous large B-cell lymphoma, EBV (Epstein-Barr virus) positive DLBCL, Burkitt lymphoma or transformation from chronic lymphocytic leukemia/small lymphocytic lymphoma (Richter transformation).

  4. Subjects with prior history of malignancies, other than aggressive R/R NHL, unless the subject has been free of the disease for ≥ 2 years with the exception of the following noninvasive 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
  5. Treatment with any prior gene therapy product.

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

  7. Subjects with active hepatitis B, or active hepatitis C are excluded. Subjects with 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. Subjects with a history of or active human immunodeficiency virus (HIV) are excluded.

  8. Subjects with uncontrolled systemic fungal, bacterial, viral or other infection (including tuberculosis) despite appropriate antibiotics or other treatment.

  9. Active autoimmune disease requiring immunosuppressive therapy.

  10. History of any one of the following cardiovascular conditions within the past 6 months prior to signing the ICF: Class III or IV heart failure as defined by the New York Heart Association (NYHA), cardiac angioplasty or stenting, myocardial infarction, unstable angina, or other clinically significant cardiac disease.

  11. History or presence of clinically relevant central nervous system (CNS) pathology

  12. Pregnant or nursing (lactating) women.

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
Arm A - Standard of Care (SOC)Standard of CareSubjects should receive SOC (R-DHAP, R-ICE or R-GDP) followed by HDCT (BEAM) and HSCT. Standard of care regimen will be administered as per investigator decision.
Arm B - JCAR017JCAR017Lymphodepleting chemotherapy with intravenous (IV) fludarabine (30 mg/m2/day for 3 days) plus cyclophosphamide IV (300 mg/m2/day for 3 days) (flu/cy) concurrently followed by JCAR017 infusion.
Primary Outcome Measures
NameTimeMethod
Event-free survival (EFS)Approximately 3 years

Time from randomization to death from any cause, progressive disease (PD), failure to achieve complete response (CR) or partial response (PR), or start of new antineoplastic therapy due to efficacy concerns, whichever occurs first

Secondary Outcome Measures
NameTimeMethod
Rate of hematopoietic stem cell transplant (HSCT)Approximately 3 years

Rate of completion of HDCT and HSCT

Frequency of hospital resource utilizationApproximately 3 years

Will be assessed based on frequency of hospitalizations calculated as, inpatient days, intensive care unit (ICU) days, outpatient visits days

PFS on next line of treatment (PFS-2)Approximately 3 years

Time from randomization to second objective disease progression or death from any cause, whichever is first.

Reasons for hospital resource utilizationApproximately 3 years

Will be assessed based on reasons for hospitalization

Complete response rate (CRR)Approximately 3 years

Percentage of subjects achieving a complete response (CR)

HRQoL using European Organisation for Research and Treatment of Cancer - Quality of Life C30 questionnaire (EORTC-QLQ-C30)Approximately 3 years

European Organisation for Research and Treatment of Cancer - Quality of Life C30 questionnaire: The EORTC QLQ-C30 questionnaire will be used as a measure of health-related quality of life, fatigue, physical and cognitive functions.

HRQoL parameters assessed by FACT-Lym "Additional concerns" subscaleApproximately 3 years

Functional Assessment of Cancer Therapy-Lymphoma "Additional concerns" subscale: Only the LYM subscale will be administered in this study. This scale addresses symptoms and functional limitations (15 item) that are important to lymphoma patients.

Hospital resource utilization (HRU)Approximately 3 years

Will be assessed based on frequency of hospitalizations calculated as, inpatient days, intensive care unit (ICU) days, outpatient visits days and reasons for hospitalization

Progression-free survival (PFS)Approximately 3 years

Time from randomization to PD or death from any cause, whichever occurs first

Overall survival (OS)Approximately 4.5 years

Time from randomization to time of death due to any cause

Overall response rate (ORR)Approximately 3 years

Percentage of subjects achieving an objective response of partial response (PR) or better according to the Lugano Classification as assessed by IRC review

Duration of response (DOR)Approximately 3 years

Time from first response to disease progression, start of new antineoplastic therapy due to efficacy concerns or death from any cause

Adverse Events (AEs)Approximately 3 years

Type, frequency and severity of adverse events (AEs), serious adverse events (SAE), and laboratory abnormalities (overall and in clinical, histological and molecular subgroups)

Trial Locations

Locations (54)

Loyola University Medical Center Cardinal Bernardin Cancer Center

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Maywood, Illinois, United States

Local Institution - 109

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Dallas, Texas, United States

Local Institution - 350

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Gent, Belgium

Local Institution - UNK 25

🇫🇮

Helsinki, Finland

Local Institution - 122

🇺🇸

Chicago, Illinois, United States

Local Institution - 104

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Boston, Massachusetts, United States

Local Institution - 123

🇺🇸

Pittsburgh, Pennsylvania, United States

Mayo Clinic - Jacksonville

🇺🇸

Jacksonville, Florida, United States

Local Institution - 108

🇺🇸

Atlanta, Georgia, United States

Local Institution - 102

🇺🇸

Boston, Massachusetts, United States

Local Institution - 111

🇺🇸

Buffalo, New York, United States

Local Institution - 125

🇺🇸

Charlotte, North Carolina, United States

Local Institution - 124

🇺🇸

Houston, Texas, United States

Local Institution - 114

🇺🇸

Richmond, Virginia, United States

Local Institution - 403

🇫🇷

Pierre Benite, France

Local Institution - 451

🇩🇪

Berlin, Germany

Local Institution - 200

🇯🇵

Chuo-ku, Tokyo, Japan

Local Institution - 203

🇯🇵

Osaka, Osaka-shi, Japan

Local Institution - 550

🇳🇱

Rotterdam, Netherlands

Local Institution - 700

🇨🇭

Bern, Switzerland

Local Institution - 600

🇪🇸

Barcelona, Spain

Local Institution - 601

🇪🇸

Madrid, Spain

Local Institution - 452

🇩🇪

Hamburg, Germany

Local Institution - 453

🇩🇪

München, Germany

Local Institution - 502

🇮🇹

Torino, Italy

Local Institution - 107

🇺🇸

Atlanta, Georgia, United States

Local Institution - 129

🇺🇸

Scottsdale, Arizona, United States

Local Institution - 116

🇺🇸

Scottsdale, Arizona, United States

Local Institution - 121

🇺🇸

Hackensack, New Jersey, United States

Local Institution - 117

🇺🇸

New York, New York, United States

Local Institution - 110

🇺🇸

Seattle, Washington, United States

Local Institution - 401

🇫🇷

Lille, France

Local Institution - 400

🇫🇷

Marseille cedex, France

Local Institution - 402

🇫🇷

Villejuif CEDEX, France

Local Institution - 455

🇩🇪

Dresden, Saxony, Germany

Local Institution - 454

🇩🇪

Muenster, Germany

Local Institution - 450

🇩🇪

Köln, Germany

Local Institution - 501

🇮🇹

Rozzano (MI), Italy

Local Institution - 500

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Rome, Italy

Local Institution - 201

🇯🇵

Minato-ku, Tokyo, Japan

Local Institution - 202

🇯🇵

Bunkyo-ku, Japan

Local Institution - 650

🇸🇪

Stockholm, Sweden

Local Institution - 750

🇬🇧

London, United Kingdom

Local Institution - 112

🇺🇸

Minneapolis, Minnesota, United States

Local Institution - 101

🇺🇸

Portland, Oregon, United States

Local Institution - 115

🇺🇸

San Francisco, California, United States

Local Institution - 119

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Detroit, Michigan, United States

Local Institution - 106

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Aurora, Colorado, United States

Local Institution - 126

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Tampa, Florida, United States

Local Institution - 120

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Ann Arbor, Michigan, United States

Local Institution - 103

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Rochester, Minnesota, United States

Local Institution - 100

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Omaha, Nebraska, United States

Local Institution - 127

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Oklahoma City, Oklahoma, United States

Local Institution - 751

🇬🇧

Southampton, Hampshire, United Kingdom

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