An Observational Study of Presentation, Treatment Patterns, and Outcomes in Multiple Myeloma Participants
- Conditions
- Multiple Myeloma
- Interventions
- Other: No Intervention
- Registration Number
- NCT02761187
- Lead Sponsor
- Takeda
- Brief Summary
The purpose of this study is to describe contemporary, real-world patterns of participant characteristics, clinical disease presentation, therapeutic regimen chosen, and clinical outcomes in participants with newly diagnosed \[ND\] multiple myeloma (MM) and participants with relapsed/refractory \[R/R\] MM.
- Detailed Description
This is a prospective, non-interventional, observational study. This study will look at contemporary, real-world patterns of participant characteristics, clinical disease presentation, therapeutic regimen chosen, and clinical outcomes in participants with MM. Participants will not be asked to change their routine clinical treatment. Participants will have to complete patient reported outcomes (PROs) surveys during on-site routine office visits.
The study will enroll approximately 4200 participants. Participants will be assigned to one of the following cohorts based upon the diagnosis of MM:
* ND MM within 3 months from initiation of treatment
* R/R MM who have received 1 to 3 prior lines of therapy
This multi-center trial will be conducted worldwide. The overall time to participate in this study is up to 8 years. Participants will be evaluated and followed-up for a period of at least 5 years, until death, are lost to follow-up, or the end of the study, whichever comes first.
Recruitment & Eligibility
- Status
- TERMINATED
- Sex
- All
- Target Recruitment
- 4253
Is 18 years of age or older.
Is experiencing the following:
- Newly diagnosed MM within 3 months from initiation of treatment with documented month and year of diagnosis, criteria met for diagnosis, stage, and MM-directed treatment history, including duration, or
- Relapsed/refractory MM who have received 1 to 3 prior lines of therapy with documented data in the medical record regarding diagnosis (month and year), the regimens used in 1st, 2nd, and 3rd line as applicable, whether stem cell transplant was part of 1st, 2nd, and 3rd line of therapy, whether consolidation/maintenance was part of 1st, 2nd, and 3rd line of therapy, also whether investigational therapy/treated on a clinical trial was part of any of these regimens.
Is willing and able to sign informed consent to participate. Is willing and able to complete patient-reported outcomes (PROs) in accordance with local regulatory and data protection requirements.
Is reporting to a site in this study for a second opinion (consultation only) or participants whose frequency of consult and follow-up are not adequate for quarterly electronic case report form (eCRF) completion.
Has participated in another study (observational or interventional) that prohibits participation in this study.
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Arm && Interventions
Group Intervention Description Relapsed/refractory (R/R) MM No Intervention Patients who have received 1 to 3 prior lines of therapy Newly diagnosed (ND) MM No Intervention Patients within 3 months from initiation of treatment
- Primary Outcome Measures
Name Time Method Number of Participants With Co-morbidities Baseline up to 5 years Charlson Comorbidity Index (CCI) was used to represent number of participants with co-morbidities. CCI is a method of categorizing comorbidities of participants. Each comorbidity category has an associated weight (from 1 to 6), based on the adjusted risk of mortality or resource use, and the sum of all the weights results in a single comorbidity score for a participant. A score of 0 = no comorbidities found, 1 = not ill, 2 = mildly ill, 3 = moderately ill, 4 = severely ill, and ≥5 = moribund. The higher the score, the more likely the predicted outcome resulted in mortality or higher resource use.
Number of Participants Diagnosed With Newly Diagnosed Multiple Myeloma (NDMM) and Relapsed/Refractory Multiple Myeloma (R/RMM) At Baseline Participants diagnosed with NDMM and R/RMM were determined at the start of the study.
Number of Participants Diagnosed With Symptoms of ND MM and R/R MM During the Study Baseline up to 5 years Sites of Disease Diagnosed With ND MM and R/R MM Baseline up to 5 years Number of Participants With ECOG (Eastern Cooperative Oncology Group) Performance Status At Baseline ECOG-PS measured on-therapy (time between first dose and last dose date with a 30-day lag) assessed participant's performance status on 6 point scale: 0=Fully active/able to carry on all pre-disease activities without restriction; 1=restricted in physically strenuous activity, ambulatory/able to carry out light or sedentary work; 2=ambulatory (\>50% of waking hours), capable of all self care, unable to carry out any work activities; 3=capable of only limited self care, confined to bed/chair \>50% of waking hours; 4=completely disabled, cannot carry on any self care, totally confined to bed/chair; 5=dead. The line of Therapy was determined at study entry.
Number of Participants With Myeloma Frailty Index At Baseline Frailty is defined as the combination of unintentional weight loss, exhaustion, low physical activity, slow walking speed, and muscular weakness. The Myeloma Frailty Index is a composite index that was calculated using the points system, which produces a range of values from 0 to 5. Participants with score 0= fit, score 1= intermediate, and score ≥2= frail. Higher score indicates likeliness that the predicted outcome will result in frailty. The line of Therapy was determined at study entry.
Number of Participants Evaluated for Minimal Residual Disease (MRD) Baseline up to 5 years Number of Participants Evaluated for Gene Expression Profiling (GEP) Baseline up to 5 years Number of Participants Evaluated for Cytogenetics Using Fluorescence in Situ Hybridization (FISH) At Baseline FISH methodology was reported with Yes/No results for the following tests: deletion (17p)/p53 \[Del(17p)/p53\], translocation (4,14) \[t(4,14)\], and translocation (14,16) \[t(14,16)\].
Overall Survival (OS) Baseline up to 5 years Overall Survival was defined as the number of months from the index regimen start date within each line of therapy, starting with the line during study entry, until the date of death. The Kaplan Meier estimates was used for the analysis.
Response to Each Regimen Baseline up to 5 years Number of Participants Evaluated for International Staging System (ISS)/ Revised (R)-ISS Stage At Baseline ISS disease stages were defined as I:low risk, β2-Microglobulin\<3.5mg/L, albumin≥3.5g/dL, II:not stage I or III, III:high risk,β2-Microglobulin≥5.5mg/L). R-ISS is based on ISS, chromosomal abnormalities (CA), and lactate dehydrogenase (LDH). R-ISS disease stages were defined as I: ISS Stage I and standard risk CA by FISH and normal LDH (i.e. \<=300 U/L), II: Neither R-ISS Stage I nor Stage III, III: ISS Stage III and either high risk CA by FISH or high LDH (i.e. \>300 U/L).
Duration of Treatment for Participants With and Without Stem Cell Transplant Baseline up to 5 years Data was analyzed for participants with and without stem cell transplant for all enrolled population, included all participants who signed the inform consent form, out of which 990 participants were excluded during the final analysis due to concerns around robustness of data.
Disease Progression Status on Each Regimen Baseline up to 5 years Disease progression status was assessed by physician interpretation of IMWG Response criteria.
Time to Next Therapy Baseline up to 5 years The line of Therapy was determined at study entry. The Kaplan Meier estimates was used for the analysis.
Number of Participants With Stem Cell Transplant Baseline up to 5 years Number of Participants With Global Health Status Scale/Quality of Life (QoL) Among MM Participants Baseline up to 5 years The Global Health Status scale/QoL scale included 2 questions measured with a 7-point numeric rating scale (very poor to excellent). Raw scores are converted into scale scores ranging from 0 to 100. A higher score represents better HRQoL.
- Secondary Outcome Measures
Name Time Method Number of Participants Receiving Different Treatment Combinations Baseline up to 5 years Number of Treatment Sequencing Baseline up to 5 years Drug classes were based on the earliest regimen in each corresponding Line of Therapy. The data for this outcome measure was analyzed as per line of therapy.
Number of Participants in the Treatment Rechallenge Baseline up to 5 years Number of Clinical Outcomes for Different Strategies Baseline up to 5 years Number of Clinical Outcomes Between Continuous Treatment and Intermittent Treatment Strategy Baseline up to 5 years Triggers of Treatment Initiation at Relapse Including Biochemical Progression or Symptomatic Progression Baseline up to 5 years Reasons for Treatment Modifications Baseline up to 5 years Healthcare Resource Utilization (HRU) Among MM Participants Baseline up to 5 years Associations Between Presentation and Disease Characteristics Baseline up to 5 years Associations Between Choice Of Therapy and Clinical Outcomes Baseline up to 5 years Number of Participants With Atleast One Treatment-emergent Adverse Events Leading to Treatment Discontinuation Baseline up to 5 years An Adverse Event (AE) is defined as any untoward medical occurrence in a clinical investigation participant administered a drug; it does not necessarily have to have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (e.g., a clinically significant abnormal laboratory finding), symptom, or disease temporally associated with the use of a drug, whether or not it is considered related to the drug. A treatment-emergent adverse event (TEAE) is defined as an adverse event with an onset that occurs after receiving study drug. Treatment discontinuation includes temporary and permanent discontinuation, drug modification, and second primary malignancies.
Trial Locations
- Locations (133)
San Juan Oncology Associates
🇺🇸Farmington, New Mexico, United States
University of Wisconsin Carbone Cancer Center
🇺🇸Madison, Wisconsin, United States
Poudre Valley Health System
🇺🇸Fort Collins, Colorado, United States
Illinois Cancer Specialists (Niles) - USOR
🇺🇸Niles, Illinois, United States
SCRI Florida Cancer Specialists North
🇺🇸Saint Petersburg, Florida, United States
St Joseph Heritage Healthcare
🇺🇸Santa Rosa, California, United States
Washington University in St. Louis
🇺🇸Saint Louis, Missouri, United States
Saint Francis Hospital
🇺🇸East Hills, New York, United States
Azienda Ospedaliero Universitaria Ospedali Riuniti di Ancona-Umberto I G.M. Lancisi G. Salesi
🇮🇹Ancona, Italy
Levine Cancer Center
🇺🇸Charlotte, North Carolina, United States
Centre Hospitalier de La Cote Basque
🇫🇷Bayonne, France
Azienda Ospedaliero Universitaria Policlinico Vittorio Emanuele
🇮🇹Catania, Italy
Centre Hospitalier de Perigueux
🇫🇷Perigueux, France
Clinica Sao Germano
🇧🇷Sao Paulo, Brazil
Kansas City VA Medical Center
🇺🇸Kansas City, Missouri, United States
Hospital Israelita Albert Einstein
🇧🇷Sao Paulo, Brazil
OnkoNet Marburg GmbH
🇩🇪Marburg, Germany
Hospital Universitario 12 de Octubre
🇪🇸Madrid, Spain
Mount Sinai Medical Center
🇺🇸New York, New York, United States
St Vincent Hospital
🇺🇸Green Bay, Wisconsin, United States
Azienda Ospedaliero Universitaria Di Bologna - Policlinico S Orsola Malpighi
🇮🇹Bologna, Italy
CHRU de Poitiers La Miletrie
🇫🇷Poitiers, France
Centre Hospitalier Departemental de Vendee
🇫🇷Roche-sur-Yon, France
Istanbul Universitesi Istanbul Tip Fakultesi Hastanesi
🇹🇷Istanbul, Turkey
Karadeniz Technical University Faculty of Medicine
🇹🇷Trabzon, Turkey
Hospital Clinic de Barcelona
🇪🇸Barcelona, Spain
Royal United Hospital
🇬🇧Bath, United Kingdom
Birmingham Heartlands Hospital
🇬🇧Birmingham, United Kingdom
Klinikum Chemnitz gGmbH
🇩🇪Chemnitz, Germany
Gefos - Gesellschaft fur onkologische Studien mbH
🇩🇪Dortmund, Germany
Universitatsklinikum Tubingen
🇩🇪Tubingen, Germany
Universidade Federal Do Rio de Janeiro Hospital Universitario Clementino Fraga Filho
🇧🇷Rio de Janeiro, Brazil
Azienda Ospedaliera Universitaria Federico II
🇮🇹Napoli, Italy
Internistisch Hamatologische und Internistische Praxis
🇩🇪Herrsching am Ammersee, Germany
Yakima Valley Memorial Hospital North Star Lodge - USOR
🇺🇸Yakima, Washington, United States
Unicamp Universidade Estadual de Campinas
🇧🇷Campinas, Brazil
Universitatsklinikum Heidelberg
🇩🇪Heidelberg, Germany
Hospital Universitario de Salamanca
🇪🇸Salamanca, Spain
CHU de Nancy-Hopital Brabois Adulte
🇫🇷Vandoeuvre-les-nancy, France
Ankara University Medical Faculty Cebeci Hospital
🇹🇷Ankara, Turkey
Erciyes Universitesi Tip Fakultesi Hastanesi
🇹🇷Kayseri, Turkey
Azienda Policlinico Umberto I
🇮🇹Roma, Italy
Mannheimer Onkologie Praxis
🇩🇪Mannheim, Germany
Nucleo Oncologico de Occidente S.C.
🇲🇽Guadalajara, Mexico
China Medical University Hospital
🇨🇳Taichung City, Taiwan
Royal Bournemouth Hospital
🇬🇧Bournemouth, United Kingdom
Leeds Teaching Hospitals NHS Trust
🇬🇧Leeds, West Yorkshire, United Kingdom
Cukurova Universitesi Tip Fakultesi Balcali Hastanesi
🇹🇷Adana, Turkey
Dokuz Eylul University Medical Faculty
🇹🇷Izmir, Turkey
Ninewells Hospital - PPDS
🇬🇧Dundee, United Kingdom
Nottingham University Hospitals NHS Trust
🇬🇧Nottingham, United Kingdom
The Royal Marsden NHS Foundation Trust
🇬🇧Sutton, United Kingdom
Akdeniz University Medical Faculty
🇹🇷Antalya, Turkey
Ondokuz Mayis Universitesi Tip Fakultesi Hastanesi
🇹🇷Samsun, Turkey
University Hospital Birmingham
🇬🇧Birmingham, United Kingdom
Pinderfields General Hospital
🇬🇧Wakefield, United Kingdom
Peking Union Medical College Hospital
🇨🇳Beijing, China
Peking University Peoples Hospital
🇨🇳Beijing, China
The First Affiliated Hospital of College of Medicine, Zhejiang University
🇨🇳Hangzhou, China
First Affiliated Hospital of Soochow University
🇨🇳Suzhou, China
SCRI Florida Cancer Specialists East
🇺🇸Daytona Beach, Florida, United States
Onkologische Gemeinschaftspraxis Siegburg
🇩🇪Siegburg, Germany
Universitatsmedizin der Johannes Gutenberg-Universitat Mainz
🇩🇪Mainz, Germany
Shaare Zedek Medical Center
🇮🇱Jerusalem, Israel
Azienda Sanitaria Universitaria Integrata di Udine
🇮🇹Udine, Italy
Hospital Universitario Dr. Jose Eleuterio Gonzalez
🇲🇽Monterrey, Mexico
Hospital Universitario Virgen de Las Nieves
🇪🇸Granada, Spain
Complejo Asistencial Universitario de Leon
🇪🇸Leon, Spain
Johns Hopkins Medicine - Anadolu Saglik Merkezi
🇹🇷Gebze, Turkey
Ege Universitesi Tip Fakultesi Hastanesi
🇹🇷Istanbul, Turkey
Hematology Oncology Associates - USOR
🇺🇸Medford, Oregon, United States
Veterans Affairs Pittsburgh Healthcare System
🇺🇸Pittsburgh, Pennsylvania, United States
Central Care Cancer Center
🇺🇸Bolivar, Missouri, United States
SCRI Florida Cancer Specialists South
🇺🇸Fort Myers, Florida, United States
Indiana University
🇺🇸Indianapolis, Indiana, United States
Maryland Oncology Hematology (Columbia) - USOR
🇺🇸Columbia, Maryland, United States
Barbara Ann Karmanos Cancer Center
🇺🇸Detroit, Michigan, United States
Texas Oncology (Loop) - USOR
🇺🇸San Antonio, Texas, United States
Park Nicollet Institute
🇺🇸Saint Louis Park, Minnesota, United States
University General Hospital of Patras
🇬🇷Patras, Greece
HaEmek Medical Center
🇮🇱Afula, Israel
Lady Davis Carmel Medical Center
🇮🇱Haifa, Israel
George Washington University
🇺🇸Washington, District of Columbia, United States
SCRI Tennessee Oncology Nashville
🇺🇸Nashville, Tennessee, United States
Hunterdon Hematology Oncology
🇺🇸Flemington, New Jersey, United States
CARTI Cancer Center
🇺🇸Little Rock, Arkansas, United States
University of California San Diego
🇺🇸La Jolla, California, United States
Grand Hopital de Charleroi asbl
🇧🇪Charleroi, Belgium
UZ Gent
🇧🇪Gent, Belgium
CHU de Liege
🇧🇪Liege, Belgium
CHU UCL Namur asbl - Site Godinne
🇧🇪Yvoir, Belgium
University of Arkansas For Medical Sciences
🇺🇸Little Rock, Arkansas, United States
University of Cincinnati
🇺🇸Cincinnati, Ohio, United States
Central Maine Medical Center
🇺🇸Lewiston, Maine, United States
Fox Chase Cancer Center
🇺🇸Philadelphia, Pennsylvania, United States
Oncomedica SA
🇨🇴Monteria, Colombia
Greenville Health System Cancer Institute
🇺🇸Greenville, South Carolina, United States
Hopital de Jolimont
🇧🇪Haine-Saint-Paul, Belgium
UZ Leuven
🇧🇪Leuven, Belgium
Fundacion Oftalmologica de Santander Foscal
🇨🇴Floridablanca, Colombia
Hospital Pablo Tobon Uribe
🇨🇴Medellin, Colombia
Alexandra Hospital
🇬🇷Athens, Greece
Fundacion Santa Fe de Bogota
🇨🇴Bogota, Colombia
Berkeley Medical Center
🇺🇸Martinsburg, West Virginia, United States
Kaohsiung Medical University Hospital
🇨🇳Kaohsiung, Taiwan
University Hospital of Alexandroupolis
🇬🇷Alexandroupoli, Greece
Evangelismos General Hospital of Athens
🇬🇷Athens, Greece
University General Hospital of Ioannina
🇬🇷Ioannina, Greece
Meir Medical Center
🇮🇱Kefar-Sava, Israel
University General Hospital of Larissa
🇬🇷Larisa, Greece
Hematologica Alta Especialidad S.C.
🇲🇽Huixquilucan, Mexico
Tel Aviv Sourasky Medical Center
🇮🇱Tel Aviv, Israel
Assuta Medical Centers
🇮🇱Tel Aviv, Israel
National Cheng Kung University Hospital
🇨🇳Tainan, Taiwan
National Taiwan University Hospital
🇨🇳Taipei, Taiwan
Instituto Nacional de Ciencias Medicas y Nutricion Salvador Zubiran
🇲🇽Mexico City, Mexico
Chang Gung Medical Foundation Chiayi Chang Gung Memorial Hospital
🇨🇳Puzi, Chiayi County, Taiwan
Chang Gung Medical Foundation Kaohsiung Chang Gung Memorial Hospital
🇨🇳Kaohsiung, Taiwan
Centre Hospitalier Le Mans
🇫🇷Le Mans, France
Rocky Mountain Cancer Centers (Williams) - USOR
🇺🇸Denver, Colorado, United States
Northwest Cancer Specialists (Broadway) - USOR
🇺🇸Portland, Oregon, United States
Azienda Ospedaliera Universitaria Careggi
🇮🇹Firenze, Italy
Azienda Ospedaliera Citta della Salute e della Scienza di Torino
🇮🇹Torino, Italy
Leicester Royal Infirmary
🇬🇧Leicester, United Kingdom
Centro de Pesquisas Oncologicas
🇧🇷Florianopolis, Brazil
Hospital Das Clinicas Da Universidade Federal de Goias
🇧🇷Goiania, Brazil
Hospital das Clinicas da Faculdade de Medicina da Universidade de Sao Paulo
🇧🇷Sao Paulo, Brazil
CEHON - Centro de Hematologia e Oncologia da Bahia Ltda
🇧🇷Salvador, Brazil
Hospital Universitari i Politecnic La Fe de Valencia
🇪🇸Valencia, Spain
Institut fur Versorgungsforschung in der Onkologie GbR
🇩🇪Koblenz, Germany
Aurora Health Care, Aurora Cancer Care
🇺🇸Milwaukee, Wisconsin, United States
University of Texas MD Anderson Cancer Center
🇺🇸Houston, Texas, United States
East Jefferson General Hospital
🇺🇸Metairie, Louisiana, United States