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A Study Comparing JNJ-68284528, a CAR-T Therapy Directed Against B-cell Maturation Antigen (BCMA), Versus Pomalidomide, Bortezomib and Dexamethasone (PVd) or Daratumumab, Pomalidomide and Dexamethasone (DPd) in Participants With Relapsed and Lenalidomide-Refractory Multiple Myeloma

Phase 3
Active, not recruiting
Conditions
Multiple Myeloma
Interventions
Registration Number
NCT04181827
Lead Sponsor
Janssen Research & Development, LLC
Brief Summary

The purpose of this study is to compare the efficacy of ciltacabtagene autoleucel (cilta-cel) with standard therapy, either Pomalidomide, Bortezomib and Dexamethasone (PVd) or Daratumumab, Pomalidomide and Dexamethasone (DPd).

Detailed Description

Multiple myeloma is a malignant plasma cell disorder diagnosed annually in approximately 86,000 participants worldwide. JNJ-68284528 (cilta-cel) is an autologous chimeric antigen receptor T-cell (CAR-T) therapy that targets B-cell maturation antigen (BCMA), a molecule expressed on the surface of mature B lymphocytes and malignant plasma cells. The primary hypothesis for this study is that JNJ-68284528 (cilta-cel) will significantly improve progression free survival (PFS) compared with standard therapy (PVd or DPd), in participants who have previously received 1 to 3 prior line(s) of therapy, that included a proteasome inhibitor (PI) and an immunomodulatory drug (IMiD) and who are refractory to lenalidomide. This study will be conducted in 3 phases: Screening (up to 28 days before randomization), Treatment, and Follow-Up. Assessment like patient-reported outcome(s) (PROs) assessments, electrocardiogram (ECG), vital signs, pharmacokinetic will be performed during the study. Safety evaluations will include review of adverse events, laboratory test results, vital sign measurements, physical examination findings, and assessments of cardiac function, Immune Effector Cell-associated Encephalopathy (only for Arm B) and Eastern Cooperative Oncology Group performance status. Safety data will be periodically reviewed by an Independent Data Monitoring Committee (IDMC). The duration of the study is approximately 6 years.

Recruitment & Eligibility

Status
ACTIVE_NOT_RECRUITING
Sex
All
Target Recruitment
419
Inclusion Criteria
  • Measurable disease at screening as defined by any of the following: (a) Serum monoclonal paraprotein (M-protein) level greater than or equal to (>=) 0.5 gram per deciliter (g/dL) or urine M-protein level >=200 milligram (mg)/24 hours; or (b) Light chain multiple myeloma without measurable M-protein in the serum or the urine: Serum free light chain >=10 mg/dL and abnormal serum free light chain ratio

  • Have received 1 to 3 prior lines of therapy including a proteasome inhibitor (PI) and an immunomodulatory drug (IMiD)

  • Have documented evidence of PD by International Myeloma Working Group (IMWG) criteria based on investigator's determination on or within 6 months of their last regimen

  • Be refractory to lenalidomide per IMWG consensus guidelines (failure to achieve minimal response or progression on or within 60 days of completing lenalidomide therapy). Progression on or within 60 days of the last dose of lenalidomide given as maintenance will meet this criterion. For participants with more than 1 prior line of therapy, there is no requirement to be lenalidomide refractory to the most recent line of prior therapy. However, participants must be refractory to lenalidomide in at least one prior line

  • Have clinical laboratory values meeting the following criteria during the Screening Phase (re testing is allowed but the below criteria must be met in the latest test prior to randomization):

    1. Hemoglobin >=8 gram per deciliter (g/dL) (without prior RBC transfusion within 7 days before the laboratory test; recombinant human erythropoietin use is permitted);
    2. Absolute neutrophil count (ANC) >=1 * 10^9 per liter (L) (without recombinant human granulocyte colony-stimulating factor [G-CSF] within 7 days and without pegylated G-CSF within 14 days of the laboratory test);
    3. Platelet count >=75 * 10^9/L (without prior platelet transfusion within 7 days before the laboratory test) in participants in whom less than (<) 50 percent (%) of bone marrow nucleated cells are plasma cells; platelet count >=50 * 10^9/L (without prior platelet transfusion within 7 days before the laboratory test) in participants in whom >=50% of bone marrow nucleated cells are plasma cells;
    4. Lymphocyte count >=0.3 * 10^9/L;
    5. Aspartate aminotransferase (AST) less than or equal to (<=)3 * upper limit of normal (ULN);
    6. Alanine aminotransferase (ALT) <=3 * ULN;
    7. Total bilirubin <=2.0 * ULN; except in participants with congenital bilirubinemia, such as Gilbert syndrome (in which case direct bilirubin <=1.5 * ULN is required);
    8. Estimated glomerular filtration rate >=40 milliliter per minute (mL/min) per 1.73 meter square (m^2) (to be calculated using the Modification of Diet in Renal Disease [MDRD] formula)
Exclusion Criteria
  • Prior treatment with chimeric antigen receptor T-cell (CAR-T) therapy directed at any target
  • Any previous therapy that is targeted to B-cell maturation antigen (BCMA)
  • Ongoing toxicity from previous anticancer therapy that has not resolved to baseline levels or to Grade 1 or less; except for alopecia
  • Participants with Grade 1 peripheral neuropathy with pain or Grade 2 or higher peripheral neuropathy will not be permitted to receive pomalidomide, bortezomib, and dexamethasone (PVd) as standard therapy or bridging therapy; however, participants may receive daratumumab, pomalidomide, and dexamethasone (DPd) as standard therapy or bridging therapy
  • Received a cumulative dose of corticosteroids equivalent to >=70 mg of prednisone within the 7 days prior to randomization
  • Monoclonal antibody treatment within 21 days
  • Cytotoxic therapy within 14 days
  • Proteasome inhibitor therapy within 14 days
  • Immunomodulatory drug (IMiD) therapy within 7 days

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
Arm A: PVd or DPd (Standard Therapy)PomalidomideParticipants will receive either PVd or DPd as a standard therapy. In PVd treatment, participants will receive oral pomalidomide 4 mg on Days 1 to 14 in each cycle; bortezomib 1.3 mg/meter square (m\^2) SC on Days 1, 4, 8 and 11 (Cycles 1 to 8) and on Days 1 and 8 (Cycle 9 onwards) and oral dexamethasone 20 mg on Days 1, 2, 4, 5, 8, 9, 11 and 12 (Cycles 1 to 8) and Days 1, 2, 8 and 9 (Cycle 9 onwards). Each cycle will consist of 21 days. In DPd treatment, participants will receive daratumumab SC 1800 mg weekly on Days 1, 8, 15, and 22 (Cycles 1 and 2), every 2 weeks on Days 1 and 15 (Cycles 3 to 6) and every 4 weeks on Day 1 (Cycle 7 onwards); oral pomalidomide 4 mg on Days 1 to 21 (Cycle 1 onwards); dexamethasone 40 mg oral or IV weekly on Days 1, 8, 15, and 22 (Cycle 1 onwards). Each cycle will consist of 28 days. Participants will continue to receive PVd or DPd until confirmed PD, death, intolerable toxicity, withdrawal of consent, or end of the study, whichever occurs earlier.
Arm A: PVd or DPd (Standard Therapy)BortezomibParticipants will receive either PVd or DPd as a standard therapy. In PVd treatment, participants will receive oral pomalidomide 4 mg on Days 1 to 14 in each cycle; bortezomib 1.3 mg/meter square (m\^2) SC on Days 1, 4, 8 and 11 (Cycles 1 to 8) and on Days 1 and 8 (Cycle 9 onwards) and oral dexamethasone 20 mg on Days 1, 2, 4, 5, 8, 9, 11 and 12 (Cycles 1 to 8) and Days 1, 2, 8 and 9 (Cycle 9 onwards). Each cycle will consist of 21 days. In DPd treatment, participants will receive daratumumab SC 1800 mg weekly on Days 1, 8, 15, and 22 (Cycles 1 and 2), every 2 weeks on Days 1 and 15 (Cycles 3 to 6) and every 4 weeks on Day 1 (Cycle 7 onwards); oral pomalidomide 4 mg on Days 1 to 21 (Cycle 1 onwards); dexamethasone 40 mg oral or IV weekly on Days 1, 8, 15, and 22 (Cycle 1 onwards). Each cycle will consist of 28 days. Participants will continue to receive PVd or DPd until confirmed PD, death, intolerable toxicity, withdrawal of consent, or end of the study, whichever occurs earlier.
Arm A: PVd or DPd (Standard Therapy)DexamethasoneParticipants will receive either PVd or DPd as a standard therapy. In PVd treatment, participants will receive oral pomalidomide 4 mg on Days 1 to 14 in each cycle; bortezomib 1.3 mg/meter square (m\^2) SC on Days 1, 4, 8 and 11 (Cycles 1 to 8) and on Days 1 and 8 (Cycle 9 onwards) and oral dexamethasone 20 mg on Days 1, 2, 4, 5, 8, 9, 11 and 12 (Cycles 1 to 8) and Days 1, 2, 8 and 9 (Cycle 9 onwards). Each cycle will consist of 21 days. In DPd treatment, participants will receive daratumumab SC 1800 mg weekly on Days 1, 8, 15, and 22 (Cycles 1 and 2), every 2 weeks on Days 1 and 15 (Cycles 3 to 6) and every 4 weeks on Day 1 (Cycle 7 onwards); oral pomalidomide 4 mg on Days 1 to 21 (Cycle 1 onwards); dexamethasone 40 mg oral or IV weekly on Days 1, 8, 15, and 22 (Cycle 1 onwards). Each cycle will consist of 28 days. Participants will continue to receive PVd or DPd until confirmed PD, death, intolerable toxicity, withdrawal of consent, or end of the study, whichever occurs earlier.
Arm A: PVd or DPd (Standard Therapy)DaratumumabParticipants will receive either PVd or DPd as a standard therapy. In PVd treatment, participants will receive oral pomalidomide 4 mg on Days 1 to 14 in each cycle; bortezomib 1.3 mg/meter square (m\^2) SC on Days 1, 4, 8 and 11 (Cycles 1 to 8) and on Days 1 and 8 (Cycle 9 onwards) and oral dexamethasone 20 mg on Days 1, 2, 4, 5, 8, 9, 11 and 12 (Cycles 1 to 8) and Days 1, 2, 8 and 9 (Cycle 9 onwards). Each cycle will consist of 21 days. In DPd treatment, participants will receive daratumumab SC 1800 mg weekly on Days 1, 8, 15, and 22 (Cycles 1 and 2), every 2 weeks on Days 1 and 15 (Cycles 3 to 6) and every 4 weeks on Day 1 (Cycle 7 onwards); oral pomalidomide 4 mg on Days 1 to 21 (Cycle 1 onwards); dexamethasone 40 mg oral or IV weekly on Days 1, 8, 15, and 22 (Cycle 1 onwards). Each cycle will consist of 28 days. Participants will continue to receive PVd or DPd until confirmed PD, death, intolerable toxicity, withdrawal of consent, or end of the study, whichever occurs earlier.
Arm B: (Ciltacabtagene Autoleucel [Cilta-cel])Cilta-celParticipants will receive at least one cycle of bridging therapy (PVd or DPd) and additional cycles of bridging therapy may be considered based on participant's clinical status and timing of availability of cilta-cel along with conditioning regimen (cyclophosphamide 300 milligram \[mg\]/m\^2 intravenous \[IV\] and fludarabine 30 mg/m\^2 IV daily, for 3 days), and cilta-cel infusion 0.75 \* 10\^6 chimeric antigen receptor (CAR)-positive viable T cells/ kilogram (kg).
Primary Outcome Measures
NameTimeMethod
Progression Free Survival (PFS)From randomization (Day 1) to either progressive disease or death, whichever occurred first (up to 3.9 years)

PFS: defined as time from date of randomization to date of first documented progressed disease (PD) as per International Myeloma Working Group (IMWG) criteria, or death due to any cause, whichever occurred first. PD: increase of 25% from lowest response value: serum and urine M-component (absolute increase must be \>=0.5 grams per deciliter \[g/dL\] and \>=200 milligrams \[mg\] per 24 hours, respectively); only in participants without measurable serum and urine M-protein levels, difference between involved and uninvolved free light chain (FLC) levels (absolute increase of \>10 mg/dL); only in participants without measurable serum and urine M-protein levels and without measurable disease by FLC levels, bone marrow plasma cell (PC)% (absolute increase of \>=10%), appearance of new lesion; definite development of new bone lesions or definite increase in size of existing bone lesions, \>=50% increase in circulating PCs (minimum of 200 cells per microliter \[uL\]) if this was only measure of disease.

Secondary Outcome Measures
NameTimeMethod
Change From Baseline in Health-Related Quality of Life as Assessed by European Quality of Life - 5 Dimensions-5 Levels (EQ-5D-5L) Questionnaire Scale ScoreFrom baseline (Cycle 1 Day 1) up to 7 years (each cycle of 28 days)
Percentage of Participants Who Achieved Sustained MRD-negative StatusFrom randomization (Day 1) up to 7 years
Change From Baseline in Systemic Cytokine Concentrations on Participants Who Received Cilta-cel as Study Treatment (Arm B)From baseline (Cycle 1 Day 1) up to 7 years
Change From Baseline in Levels of CAR-T Cell Activation Markers on Participants Who Received Cilta-cel as Study Treatment (Arm B)From baseline (Cycle 1 Day 1) up to 7 years (each cycle of 28 days)
Number of Participants With Anti-JNJ-68284528 Antibodies on Participants Who Received Cilta-cel as Study Treatment (Arm B)From Cycle 1 Day 1 up to 7 years
Change From Baseline in Health-Related Quality of Life as Assessed by Patient Global Impression of Symptom Severity (PGIS) Scale ScoreFrom baseline (Cycle 1 Day 1) up to 7 years (each cycle of 28 days)
Change From Baseline in Levels of JNJ-68284528 T Cell Expansion (Proliferation), and Persistence on Participants Who Received Cilta-cel as Study Treatment (Arm B)From baseline (Cycle 1 Day 1) up to 7 years (each cycle of 28 days)
Change From Baseline in Health-Related Quality of Life (HRQoL) as Assessed by European Organization for Research and Treatment of Cancer Quality-of-life Questionnaire Core 30 Item (EORTC-QLQ-C30) Scale ScoreFrom baseline (Cycle 1 Day 1) up to 7 years (each cycle of 28 days)
Percentage of Participants Who Achieved Complete Response (CR) or Stringent Complete Response (sCR)From randomization (Day 1) up to 7 years
Percentage of Participants Who Achieved Overall Minimal Residual Disease (MRD) Negative Status (at 10^-5)From randomization (Day 1) up to 7 years
Percentage of Participants Who Were in CR or sCR and Achieved MRD-negative Status at 12 Months +/-3 MonthsFrom randomization (Day 1) up to 12 months +/- 3 months
Overall Response Rate (ORR)From randomization (Day 1) up to 7 years
Number of Participants With Treatment-emergent Adverse Events (AEs)From Cycle 1 Day 1 up to 7 years
Overall Survival (OS)From randomization (Day 1) up to 7 years
Time to Worsening of Symptoms Using the Multiple Myeloma Symptom and Impact Questionnaire (MySIm-Q) Total Symptom ScoreFrom randomization (Day 1) up to 7 years
Progression Free Survival on Next-line Therapy (PFS2)From randomization (Day 1) up to 7 years
Change From Baseline in Health-Related Quality of Life as Assessed by MySIm-Q Scale SoreFrom baseline (Cycle 1 Day 1) up to 7 years (each cycle of 28 days)
Number of Participants With Treatment-emergent Adverse Events (AEs) by SeverityFrom Cycle 1 Day 1 up to 7 years
Number of Participants in Health-Related Quality of Life as Assessed by The Patient-reported Outcomes Version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE) ItemFrom randomization (Day 1) up to 7 years

Trial Locations

Locations (88)

Kings College Hospital

🇬🇧

London, United Kingdom

Christie Hospital

🇬🇧

Manchester, United Kingdom

Freeman Hospital

🇬🇧

Newcastle Upon Tyne, United Kingdom

Narodowy Instytut Onkologii im Marii Sklodowskiej Curie Panstwowy Instytut BadawczyOddz w Gliwicach

🇵🇱

Gliwice, Poland

University of Alabama at Birmingham

🇺🇸

Birmingham, Alabama, United States

Mayo Clinic Cancer Center-Scottsdale

🇺🇸

Phoenix, Arizona, United States

Stanford University Medical Center

🇺🇸

Stanford, California, United States

Colorado Blood Cancer Institute

🇺🇸

Denver, Colorado, United States

Yale New Haven Hospital

🇺🇸

New Haven, Connecticut, United States

University Of Miami Leonard M Mille School Of Medicine SCCC

🇺🇸

Miami, Florida, United States

University of Iowa Hospitals and Clinics

🇺🇸

Iowa City, Iowa, United States

University of Kansas

🇺🇸

Westwood, Kansas, United States

University Of Maryland Medical Center

🇺🇸

Baltimore, Maryland, United States

Mayo Clinic - Rochester

🇺🇸

Rochester, Minnesota, United States

Washington University School Of Medicine

🇺🇸

Saint Louis, Missouri, United States

Hackensack University Medical Center

🇺🇸

Hackensack, New Jersey, United States

Memorial Sloan-Kettering Cancer Center

🇺🇸

New York, New York, United States

University of Rochester Medical Center

🇺🇸

Rochester, New York, United States

Duke University Medical Center

🇺🇸

Durham, North Carolina, United States

The Ohio State University

🇺🇸

Columbus, Ohio, United States

Huntsman Cancer Institute

🇺🇸

Salt Lake City, Utah, United States

Wisconsin Institutes for Medical Research

🇺🇸

Madison, Wisconsin, United States

Medical College Of Wisconsin

🇺🇸

Milwaukee, Wisconsin, United States

Royal Adelaide Hospital

🇦🇺

Adelaide, Australia

Royal Prince Alfred Hospital

🇦🇺

Camperdown, Australia

Royal Brisbane and Womens Hospital

🇦🇺

Herston, Australia

Peter MacCallum Cancer Centre

🇦🇺

Melbourne, Australia

Alfred Health

🇦🇺

Melbourne, Australia

Fiona Stanley Hospital

🇦🇺

Murdoch, Australia

Universitair Ziekenhuis - Antwerpen

🇧🇪

Antwerp, Belgium

UZ Gent

🇧🇪

Gent, Belgium

UZ Leuven

🇧🇪

Leuven, Belgium

Centre Hospitalier Universitaire de Liege Domaine Universitaire du Sart Tilman

🇧🇪

Liege, Belgium

Rigshospitalet

🇩🇰

Copenhagen, Denmark

CHRU de Lille Hopital Claude Huriez

🇫🇷

LILLE Cedex, France

CHU de Montpellier Hopital Saint Eloi

🇫🇷

Montpellier, France

C.H.U. Hotel Dieu - France

🇫🇷

Nantes, France

Hopital Saint Louis

🇫🇷

Paris cedex 10, France

Centre hospitalier Lyon-Sud

🇫🇷

Pierre Benite cedex, France

CHU De Poitiers

🇫🇷

Poitiers, France

Institut Universitaire du cancer de Toulouse-Oncopole

🇫🇷

Toulouse cedex 9, France

Universitatsklinikum Carl Gustvav Carus Dresden an der Technischen Universitat Dresden

🇩🇪

Dresden, Germany

Universitaetsklinikum Hamburg Eppendorf

🇩🇪

Hamburg, Germany

Universitaetsklinikum Koeln

🇩🇪

Koeln, Germany

Universitaetsklinikum Leipzig

🇩🇪

Leipzig, Germany

Klinikum der Eberhard Karls Universitaet Abt fur innere Med II Haematologie Onkologie Germany

🇩🇪

Tubingen, Germany

Universitatsklinikum Wurzburg

🇩🇪

Wuerzburg, Germany

Attikon University General Hospital of Attica

🇬🇷

Athens, Greece

Hadassah University Hospita Ein Kerem

🇮🇱

Jerusalem, Israel

Sheba Medical Center

🇮🇱

Ramat Gan, Israel

Sourasky Medical Center

🇮🇱

Tel Aviv, Israel

Azienda Opedaliero-Universitaria Policlinico Sant'orsola Malpighi di Bologna

🇮🇹

Bologna, Italy

IRCCS Ospedale San Raffaele HSR

🇮🇹

Milano, Italy

Fondazione IRCCS Istituto Nazionale dei Tumori

🇮🇹

Milano, Italy

Policlinico Universitario Agostino Gemelli

🇮🇹

Roma, Italy

A.O.U. Citta della Salute e della Scienza di Torino - Presidio Molinette

🇮🇹

Turin, Italy

Kyushu University Hospital

🇯🇵

Fukuoka, Japan

Kanazawa University Hospital

🇯🇵

Kanazawa, Japan

University Hospital Kyoto Prefectural University of Medicine

🇯🇵

Kyoto, Japan

Nagoya City University Hospital

🇯🇵

Nagoya, Japan

Okayama University Hospital

🇯🇵

Okayama, Japan

Hokkaido University Hospital

🇯🇵

Sapporo, Japan

Tohoku University Hospital

🇯🇵

Sendai, Japan

Japanese Red Cross Medical Center

🇯🇵

Shibuya, Japan

Seoul National University Hospital

🇰🇷

Seoul, Korea, Republic of

Samsung Medical Center

🇰🇷

Seoul, Korea, Republic of

The Catholic University of Korea Seoul St Marys Hospital

🇰🇷

Seoul, Korea, Republic of

VU Medisch Centrum

🇳🇱

Amsterdam, Netherlands

University Medical Center Groningen

🇳🇱

Groningen, Netherlands

Erasmus MC

🇳🇱

Rotterdam, Netherlands

UMC Utrecht

🇳🇱

Utrecht, Netherlands

Uniwersyteckie Centrum Kliniczne

🇵🇱

Gdansk, Poland

Uniwersytecki Szpital Kliniczny w Poznaniu

🇵🇱

Poznan, Poland

Instytut Hematologii i Transfuzjologii

🇵🇱

Warszawa, Poland

Inst. Cat. D'Oncologia-Badalona

🇪🇸

Badalona, Spain

Hosp Clinic de Barcelona

🇪🇸

Barcelona, Spain

Hosp. Gral. Univ. Gregorio Maranon

🇪🇸

Madrid, Spain

Hosp. Univ. 12 de Octubre

🇪🇸

Madrid, Spain

Clinica Univ. de Navarra

🇪🇸

Pamplona, Spain

Hosp Clinico Univ de Salamanca

🇪🇸

Salamanca, Spain

Hosp. Virgen Del Rocio

🇪🇸

Sevilla, Spain

Skane University Hospital

🇸🇪

Lund, Sweden

Karolinska Universitetssjukhuset Huddinge, Hematologiska Kliniken, Huddinge

🇸🇪

Stockholm, Sweden

Akademiska Sjukhuset

🇸🇪

Uppsala, Sweden

Queen Elizabeth Hospital

🇬🇧

Birmingham, United Kingdom

Bristol Haematology and Oncology Centre

🇬🇧

Bristol, United Kingdom

University Hospital Wales

🇬🇧

Cardiff, United Kingdom

University College Hospital

🇬🇧

London, United Kingdom

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