MedPath

A Study to Evaluate the Efficacy and Safety of Daratumumab in Combination With Cyclophosphamide, Bortezomib and Dexamethasone (CyBorD) Compared to CyBorD Alone in Newly Diagnosed Systemic Amyloid Light-chain (AL) Amyloidosis

Phase 3
Completed
Conditions
Amyloidosis
Interventions
Registration Number
NCT03201965
Lead Sponsor
Janssen Research & Development, LLC
Brief Summary

The purpose of this study is to evaluate the efficacy and safety of daratumumab plus cyclophosphamide, bortezomib and dexamethasone (CyBorD) compared with CyBorD alone in treatment of newly diagnosed amyloid light chain (AL) amyloidosis participants.

Detailed Description

Participant involved in study for approx. 8 years duration includes Screening Phase (complete clinical evaluation will be done), Treatment Phase (monitoring of adverse events (AEs), laboratory abnormalities and clinical response), Post-Treatment Observation Phase (disease evaluations will be done) and a Long-term Follow-up Phase (Subsequent anticancer treatment, response to subsequent treatment, date of progression and survival status will be obtained every 16 weeks).The primary hypothesis is that daratumumab in combination with CyBorD will improve the overall complete hematological response rate compared to CyBorD alone in AL amyloidosis participants. Safety will be assessed by AEs, laboratory test results, electrocardiogram, vital sign measurements, physical examination, and Eastern Cooperative Oncology Group (ECOG) performance status.

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
416
Inclusion Criteria
  • Histopathological diagnosis of amyloidosis based on detection by immunohistochemistry and polarizing light microscopy of green bi-refringent material in congo red stained tissue specimens (in an organ other than bone marrow) or characteristic electron microscopy appearance

  • Measurable disease of amyloid light-chain (AL) amyloidosis as defined by at least one of the following:

    1. serum monoclonal (M)-protein greater than or equal (>=) 0.5 grams/deciliter (g/dL) by protein electrophoresis (routine serum protein electrophoresis and immunofixation [IFE] performed at a central laboratory)
    2. serum free light chain greater than or equal to (>=) 50 milligram/Liter (mg/L) with an abnormal kappa:lambda ratio or the difference between involved and uninvolved free light chains (dFLC) >= 50 mg/L
  • One or more organs impacted by AL amyloidosis according to consensus guidelines

  • Eastern Cooperative Oncology Group (ECOG) Performance Status (PS) 0, 1 or 2

Exclusion Criteria
  • Prior therapy for AL amyloidosis or multiple myeloma including medications that target CD38, with the exception of 160 mg dexamethasone (or equivalent corticosteroid) maximum exposure prior to randomization

  • Previous or current diagnosis of symptomatic multiple myeloma, including the presence of lytic bone disease, plasmacytomas, >= 60 percent (%) plasma cells in the bone marrow, or hypercalcemia

  • Evidence of significant cardiovascular conditions as specified below:

    1. NT-ProBNP > 8500 nanogram per liter (ng/L)
    2. New York Heart Association (NYHA) classification IIIB or IV heart failure
    3. Heart failure that in the opinion of the investigator is on the basis of ischemic heart disease (eg, prior myocardial infarction with documented history of cardiac enzyme elevation and electrocardiogram [ECG] changes) or uncorrected valvular disease and not primarily due to AL amyloid cardiomyopathy
    4. Inpatient admission to a hospital for unstable angina or myocardial infarction within the last 6 months prior to first dose or percutaneous cardiac intervention with recent stent within 6 months or coronary artery bypass grafting within 6 months
    5. For participants with congestive heart failure, cardiovascular-related hospitalizations within 4 weeks prior to randomization
    6. Participants with a history of sustained ventricular tachycardia or aborted ventricular fibrillation or with a history of atrioventricular (AV) nodal or sinoatrial (SA) nodal dysfunction for which a pacemaker/implantable cardioverter-defibrillators [ICD] is indicated but not placed (participants who do have a pacemaker/ICD are allowed on study)
    7. Screening 12-lead ECG showing a baseline QT interval as corrected by Fridericia's formula (QTcF) > 500 milliseconds (msec). Participants who have a pacemaker may be included regardless of calculated QTc interval
    8. Supine systolic blood pressure < 90 millimeter of mercury (mmHg), or symptomatic orthostatic hypotension, defined as a decrease in systolic blood pressure upon standing of > 20 mmHg despite medical management (eg, midodrine, fludrocortisones) in the absence of volume depletion
  • Planned stem cell transplant during the first 6 cycles of protocol therapy are excluded. Stem cell collection during the first 6 cycles of protocol therapy is permitted

  • Known to be seropositive for human immunodeficiency virus (HIV)

  • Any one of the following:

    1. Seropositive for hepatitis B (defined by a positive test for hepatitis B surface antigen [HBsAg]). Participants with resolved infection (ie, participants who are HBsAg negative but positive for antibodies to hepatitis B core antigen [anti-HBc] and/or antibodies to hepatitis B surface antigen [anti-HBs]) must be screened using real-time polymerase chain reaction (PCR) measurement of hepatitis B virus (HBV) deoxyribonucleic acid (DNA) levels. Those who are PCR positive will be excluded
    2. Known to be seropositive for hepatitis C (except in the setting of a sustained virologic response [SVR], defined as aviremia at least 12 weeks after completion of antiviral therapy)
  • Grade 2 sensory or Grade 1 painful peripheral neuropathy

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
CyBorD alone (cyclophosphamide/bortezomib/dexamethasone)CyclophosphamideParticipants will receive dexamethasone (40 milligrams \[mg\] orally or intravenous \[IV\] dose), followed by cyclophosphamide (300 milligram per meter square \[mg/m\^2\] orally or IV dose), then bortezomib (1.3 mg/m\^2 subcutaneous injection) weekly on Days 1, 8, 15, 22 in every 28-day cycle for a maximum of 6 cycles.
CyBorD alone (cyclophosphamide/bortezomib/dexamethasone)BortezomibParticipants will receive dexamethasone (40 milligrams \[mg\] orally or intravenous \[IV\] dose), followed by cyclophosphamide (300 milligram per meter square \[mg/m\^2\] orally or IV dose), then bortezomib (1.3 mg/m\^2 subcutaneous injection) weekly on Days 1, 8, 15, 22 in every 28-day cycle for a maximum of 6 cycles.
CyBorD alone (cyclophosphamide/bortezomib/dexamethasone)Dexamethasone, 40 mgParticipants will receive dexamethasone (40 milligrams \[mg\] orally or intravenous \[IV\] dose), followed by cyclophosphamide (300 milligram per meter square \[mg/m\^2\] orally or IV dose), then bortezomib (1.3 mg/m\^2 subcutaneous injection) weekly on Days 1, 8, 15, 22 in every 28-day cycle for a maximum of 6 cycles.
CyBorD plus DaratumumabDaratumumabParticipants will receive dexamethasone (20 mg orally or IV dose as premedication and 20 mg on the day after daratumumab dosing) followed by 1800 mg of daratumumab subcutaneously followed by cyclophosphamide (300 mg/m\^2 orally or IV dose weekly) and bortezomib (1.3 mg/m\^2 subcutaneous injection weekly) on Days 1, 8, 15, 22 in every 28-day cycle for a maximum of 6 cycles. Daratumumab will be administered weekly for the first 8 weeks (2 cycles), then every 2 weeks for 4 cycles (cycles 3-6), and then every 4 weeks until progression of disease or subsequent therapy for a maximum of 2 years.
Primary Outcome Measures
NameTimeMethod
Percentage of Participants With Overall Complete Hematologic Response (CHR)Up to 2.4 years

Overall CHR rate was defined as percentage of participants who achieved CHR, according to the International Amyloidosis Consensus Criteria. CHR: normalization of free light chain levels and ratio, negative serum, and urine immunofixation. If involved free light chain (iFLC) is less than (\<) upper limit of normal (ULN) and serum and urine Immunofixation electrophoresis (IFE) are negative, then neither a normal uninvolved free light chain (uFLC) level nor a normal free light chain (FLC) ratio are required for complete response (CR).

Secondary Outcome Measures
NameTimeMethod

Trial Locations

Locations (139)

CHU De Poitiers

🇫🇷

Poitiers, France

Institut Paoli Calmettes

🇫🇷

Marseille, France

Chu Hotel Dieu

🇫🇷

Nantes cedex 01, France

Hopital Saint Louis

🇫🇷

PARIS cedex 10, France

Centre hospitalier Lyon-Sud

🇫🇷

Pierre - Bénite cedex, France

CHU Rangueil

🇫🇷

Toulouse, France

Universitatsklinikum Essen

🇩🇪

Essen, Germany

HOPA-Hämatologisch-Onkologische Praxis Altona MVZ GmbH

🇩🇪

Hamburg, Germany

CHU Bretonneau

🇫🇷

Tours cedex, France

CHU de Nancy_ Hopital Brabois

🇫🇷

Vandoeuvre les Nancy, France

Charite Campus Benjamin Franklin

🇩🇪

Berlin, Germany

Heinrich-Heine-Universität Düsseldorf

🇩🇪

Düsseldorf, Germany

Universitaetsklinikum Heidelberg Medizinische Klinik V

🇩🇪

Heidelberg, Germany

Hosp. Univ. de Canarias

🇪🇸

San Cristóbal de La Laguna, Spain

Hosp. Univ. Dr. Peset

🇪🇸

Valencia, Spain

South Elvsborg Hospital

🇸🇪

Boras, Sweden

Skanes universitetssjukhus

🇸🇪

Lund, Sweden

Ankara Universitesi Tip Fakultesi Cebeci Hastanesi

🇹🇷

Ankara, Turkey

Akdeniz University Medical Faculty

🇹🇷

Antalya, Turkey

Ondokuz Mayis Universitesi Tip Fakultesiy

🇹🇷

Atakum, Turkey

Istanbul University Istanbul Medical Faculty

🇹🇷

Istanbul, Turkey

Dokuz Eylul Universitesi Tip Fakultesi

🇹🇷

Izmir, Turkey

Erciyes University Medical Faculty

🇹🇷

Talas, Turkey

University Hospitals Birmingham NHS Trust,

🇬🇧

Birmingham, United Kingdom

University College Hospital

🇬🇧

London, United Kingdom

Box Hill Hospital

🇦🇺

Box Hill, Australia

CHU Dijon

🇫🇷

Dijon, France

Hopital Claude Huriez

🇫🇷

Lille cedex, France

CHU de Limoges - Fédération Hépatologie

🇫🇷

Limoges, France

Hosp Clinico Univ de Salamanca

🇪🇸

Salamanca, Spain

Arthur J E Child Comprehensive Cancer Centre

🇨🇦

Calgary, Alberta, Canada

Mayo Clinic Arizona

🇺🇸

Phoenix, Arizona, United States

City of Hope

🇺🇸

Duarte, California, United States

University of California San Francisco

🇺🇸

San Francisco, California, United States

Stanford University

🇺🇸

Stanford, California, United States

University of Colorado

🇺🇸

Aurora, Colorado, United States

Colorado Blood Cancer Institute

🇺🇸

Denver, Colorado, United States

Mayo Clinic

🇺🇸

Jacksonville, Florida, United States

Winship Cancer Institute Emory University

🇺🇸

Atlanta, Georgia, United States

University of Maryland

🇺🇸

Baltimore, Maryland, United States

Tufts Medical Center

🇺🇸

Boston, Massachusetts, United States

Boston University Medical Center

🇺🇸

Boston, Massachusetts, United States

Dana Farber Cancer Institute

🇺🇸

Boston, Massachusetts, United States

Barbara Ann Karmanos Cancer Institute

🇺🇸

Detroit, Michigan, United States

Mayo Clinic Rochester

🇺🇸

Rochester, Minnesota, United States

Washington University School Of Medicine

🇺🇸

Saint Louis, Missouri, United States

Columbia University Medical Center

🇺🇸

New York, New York, United States

Weill Cornell Medical College

🇺🇸

New York, New York, United States

University of Rochester Medical Center

🇺🇸

Rochester, New York, United States

Levine Cancer Institute

🇺🇸

Charlotte, North Carolina, United States

Wake Forest University Health Sciences - Cardiovascular Medicine

🇺🇸

Winston-Salem, North Carolina, United States

Cleveland Clinic

🇺🇸

Cleveland, Ohio, United States

The Ohio State University

🇺🇸

Columbus, Ohio, United States

Oregon Health And Science University

🇺🇸

Portland, Oregon, United States

University of Pennsylvania Medical Center

🇺🇸

Philadelphia, Pennsylvania, United States

Sarah Cannon Research Institute

🇺🇸

Nashville, Tennessee, United States

Vanderbilt University Medical Center

🇺🇸

Nashville, Tennessee, United States

University of Texas, MD Anderson Cancer Center

🇺🇸

Houston, Texas, United States

Huntsman Cancer Institute

🇺🇸

Salt Lake City, Utah, United States

Seattle Cancer Care Alliance

🇺🇸

Seattle, Washington, United States

Sir Charles Gairdner Hospital

🇦🇺

Nedlands, Australia

Westmead Hospital

🇦🇺

Westmead, Australia

Princess Alexandra Hospital

🇦🇺

Woolloongabba, Australia

Institut Jules Bordet

🇧🇪

Anderlecht, Belgium

UZ Gent

🇧🇪

Gent, Belgium

Az Groeninge

🇧🇪

Kortrijk, Belgium

Universitair Ziekenhuis Leuven

🇧🇪

Leuven, Belgium

Hospital das Clinicas de Porto Alegre

🇧🇷

Porto Alegre, Brazil

Sociedade Pernambucana de Combate ao Cancer

🇧🇷

Recife, Brazil

Instituto de Educacao, Pesquisa e Gestao em Saude Instituto Americas (COI)

🇧🇷

Rio de Janeiro, Brazil

Hospital Sao Rafael

🇧🇷

Salvador, Brazil

Fundacao Faculdade Regional de Medicina de Sao Jose do Rio Preto Hospital de Base

🇧🇷

Sao Jose do Rio Preto, Brazil

Instituto de Assistencia Medica ao Servidor Publico Estadual IAMSPE

🇧🇷

Sao Paulo, Brazil

Clinica Sao Germano

🇧🇷

São Paulo, Brazil

Hospital Das Clinicas Da Faculdade De Medicina Da USP

🇧🇷

São Paulo, Brazil

Alberta Health Services

🇨🇦

Edmonton, Alberta, Canada

Vancouver General Hospital

🇨🇦

Vancouver, British Columbia, Canada

London Health Sciences Center

🇨🇦

London, Ontario, Canada

University Health Network UHN Princess Margaret Cancer Centre

🇨🇦

Toronto, Ontario, Canada

McGill University Health Centre

🇨🇦

Montreal, Quebec, Canada

Peking University First Hospital

🇨🇳

Beijing, China

Peking University People s Hospital

🇨🇳

Beijing, China

First affiliated Hospital of Zhejiang University

🇨🇳

Hangzhou, China

Ruijin Hospital Shanghai Jiao Tong University

🇨🇳

Shanghai, China

The First Affiliated Hospital of Wenzhou Medical University

🇨🇳

Wenzhou, China

Aarhus University Hospital

🇩🇰

Aarhus N, Denmark

Dep. of Hematology, Rigshospitalet

🇩🇰

Copenhagen, Denmark

Odense Universitets Hospital

🇩🇰

Odense, Denmark

Universitaetsklinikum Tuebingen der Eberhard-Karls-Universitaet, Abteilung fuer Innere Medizin II,

🇩🇪

Tübingen, Germany

Universitätsklinikum Würzburg Med. Klinik U. Poliklinik Ii

🇩🇪

Würzburg, Germany

Alexandra General Hospital of Athens

🇬🇷

Athens, Greece

University General Hospital of Rio

🇬🇷

Patra, Greece

Semmelweis Egyetem I.Belgyogyaszati Klinika

🇭🇺

Budapest, Hungary

Del Pesti Centrumkorhaz Orszagos Hematologiai es Infektologiai Intezet Szent Laszlo Telephely

🇭🇺

Budapest, Hungary

Carmel Hospital

🇮🇱

Haifa, Israel

Hadassah Medical Center

🇮🇱

Jerusalem, Israel

Sheba Medical Center

🇮🇱

Ramat-Gan, Israel

Sourasky Medical Center

🇮🇱

Tel-Aviv, Israel

Assaf Ha'Rofeh Medical Center

🇮🇱

Zerifin, Israel

Policlinico di Bari

🇮🇹

Bari, Italy

Istituto di Ematologia Seràgnoli azienda ospedaliera univeristaria Policlinico S.Orsola-Malpighi

🇮🇹

Bologna, Italy

Casa di Cura La Maddalena

🇮🇹

Palermo, Italy

Amyloidosis Research and Treatment Center, Fondazione IRCCS Policlinico San Matteo

🇮🇹

Pavia, Italy

Dipartimento Di Biotecnologie Cellulari Ed Ematologia-Università ''La Sapienza'',Policlinico Umberto I

🇮🇹

Roma, Italy

A.O.U. Città della Salute e della Scienza

🇮🇹

Torino, Italy

Fukushima Medical University Hospital

🇯🇵

Fukushima, Japan

Hiroshima Red Cross Hospital & Atomic-bomb Survivors Hospital

🇯🇵

Hiroshima, Japan

Teine Keijinkai Hospital

🇯🇵

Hokkaido, Japan

Kanazawa University Hospital

🇯🇵

Kanazawa, Japan

Kumamoto University Hospital

🇯🇵

Kumamoto-City, Japan

Kyoto Kuramaguchi Medical Center

🇯🇵

Kyoto, Japan

Shinshu University Hospital

🇯🇵

Matsumoto, Japan

Matsuyama Red Cross Hospital

🇯🇵

Matsuyama, Japan

Nagoya City University Hospital

🇯🇵

Nagoya, Japan

National Hospital Organization Okayama Medical Center

🇯🇵

Okayama, Japan

Japanese Red Cross Medical Center

🇯🇵

Shibuya, Japan

Tokushima University Hospital

🇯🇵

Tokushima, Japan

Pusan National University Hospital

🇰🇷

Busan, Korea, Republic of

Seoul National University Hospital

🇰🇷

Seoul, Korea, Republic of

Severance Hospital Yonsei University Health System

🇰🇷

Seoul, Korea, Republic of

Samsung Medical Center

🇰🇷

Seoul, Korea, Republic of

The Catholic University of Korea Seoul St Marys Hospital

🇰🇷

Seoul, Korea, Republic of

Centro de Investigación Farmacéutica Especializada

🇲🇽

Guadalajara, Mexico

Hospital Universitario Dr Jose Eleuterio Gonzalez

🇲🇽

Monterrey, Mexico

Haga ziekenhuis

🇳🇱

Den Haag, Netherlands

UMCG

🇳🇱

Groningen, Netherlands

Erasmus MC

🇳🇱

Rotterdam, Netherlands

UMC Utrecht

🇳🇱

Utrecht, Netherlands

Maxima Medisch Centrum

🇳🇱

Veldhoven, Netherlands

Samodzielny Publiczny Zaklad Opieki Zdrowotnej Zespol Szpitali Miejskich

🇵🇱

Chorzow, Poland

SKPP UM w Poznaniu

🇵🇱

Poznan, Poland

Instytut Hematologii i Transfuzjologii

🇵🇱

Warszawa, Poland

Inst. Cat. D'Oncologia-Badalona

🇪🇸

Badalona, Spain

Hosp Univ Vall D Hebron

🇪🇸

Barcelona, Spain

Hosp Clinic de Barcelona

🇪🇸

Barcelona, Spain

Hosp. Univ. Ramon Y Cajal

🇪🇸

Madrid, Spain

Hosp Univ Fund Jimenez Diaz

🇪🇸

Madrid, Spain

Hosp. Univ. 12 de Octubre

🇪🇸

Madrid, Spain

Clinica Univ. de Navarra

🇪🇸

Pamplona, Spain

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