An Observational Study of Presentation, Treatment Patterns, and Outcomes in Multiple Myeloma Participants

Terminated

Conditions: Multiple Myeloma

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

Inclusion Criteria

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.

Exclusion Criteria

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

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): CARTI Cancer Center
🇺🇸
Little Rock, Arkansas, United States
University of Arkansas For Medical Sciences
🇺🇸
Little Rock, Arkansas, United States
University of California San Diego
🇺🇸
La Jolla, California, United States
St Joseph Heritage Healthcare
🇺🇸
Santa Rosa, California, United States
Rocky Mountain Cancer Centers (Williams) - USOR
🇺🇸
Denver, Colorado, United States
Poudre Valley Health System
🇺🇸
Fort Collins, Colorado, United States
George Washington University
🇺🇸
Washington, District of Columbia, United States
SCRI Florida Cancer Specialists East
🇺🇸
Daytona Beach, Florida, United States
SCRI Florida Cancer Specialists South
🇺🇸
Fort Myers, Florida, United States
SCRI Florida Cancer Specialists North
🇺🇸
Saint Petersburg, Florida, United States
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CARTI Cancer Center
🇺🇸Little Rock, Arkansas, United States