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

• ClinicalTrials.gov Identifier: NCT02761187
• Recruitment Status: Active, not recruiting
• First Posted: May 4, 2016
• Last Update Posted: August 13, 2019
• Sponsor: Takeda
• Information provided by (Responsible Party): Takeda

Tracking Information

• First Submitted Date: April 28, 2016
• First Posted Date: May 4, 2016
• Last Update Posted Date: August 13, 2019
• Actual Study Start Date: July 1, 2016
• Estimated Primary Completion Date: July 1, 2024 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: May 8, 2018)

• Number of Participants with Co-morbidities [ Time Frame: Baseline up to 8 years ]
• Number of Participants Diagnosed with ND MM and R/R MM [ Time Frame: Baseline up to 8 years ]
• Number of Participants Presenting Symptoms of ND MM and R/R MM [ Time Frame: Baseline up to 8 years ]
• Sites of Disease [ Time Frame: Baseline up to 8 years ]
• ECOG (Eastern Cooperative Oncology Group) Performance Status [ Time Frame: Baseline up to 8 years ]
  ECOG-PS measured on-therapy (time between first dose and last dose date with a 30-day lag) assessed participant's performance status on 5 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 hrs), 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 hrs; 4=completely disabled, cannot carry on any self care, totally confined to bed/chair; 5=dead.
• Frailty Index [ Time Frame: Baseline up to 8 years ]
  Frailty is defined as the combination of unintentional weight loss, exhaustion, low physical activity, slow walking speed, and muscular weakness.
• Number of Participants Evaluated for Laboratory Test [ Time Frame: Baseline up to 8 years ]
  Laboratory tests include minimal residual disease (MRD), gene expression profiling (GEP), fluorescence in situ hybridization (FISH), and cytogenetic results, international staging system (ISS)/R-ISS stage, imaging results.
• Duration for Treatment With Stem Cell Transplant [ Time Frame: Baseline up to 8 years ]
• Number of Participants Reporting Overall Survival [ Time Frame: Baseline up to 8 years ]
• Progression Status on Each Regimen [ Time Frame: Baseline up to 8 years ]
  Disease progression status was assessed by IMWG Response criteria
• Response to Each Regimen [ Time Frame: Baseline up to 8 years ]
  Disease progression status was assessed by IMWG Response criteria
• Time to Next therapy [ Time Frame: Baseline up to 8 years ]
• Number of Participants With Stem Cell Transplant [ Time Frame: Baseline up to 8 years ]

Original Primary Outcome Measures (submitted: May 2, 2016)

• Age of Participants at Baseline [ Time Frame: Baseline ]
• Gender of Participants at Baseline [ Time Frame: Baseline ]
• Race of Participants at Baseline [ Time Frame: Baseline ]
• Presence or Absence of Co-morbidities in Participants at Baseline [ Time Frame: Baseline up to 8 years ]
• ECOG (Eastern Cooperative Oncology Group) Performance Status of Participants at Baseline [ Time Frame: Baseline up to 8 years ]
• Frailty Index [ Time Frame: Baseline up to 8 years ]
  The frailty index will be based on the Charlson Co-morbidity Index, the Katz Index of Independence in ADL, and the Lawton IADL scales.
• International Staging System (ISS)/Revised ISS (R-ISS) of Participants at Diagnosis of Multiple Myeloma [ Time Frame: Baseline ]
• Number of Participants With Symptoms or Sites of Disease [ Time Frame: Baseline up to 8 years ]
• Treatment Regimens Prescribed to Participants Throughout the Study [ Time Frame: Baseline up to 8 years ]
• Number of Participants Who Receive a Stem Cell Transplant [ Time Frame: Baseline up to 8 years ]
• Number of Participants who undergo Cytogenetic, FISH, MRD, and GEP Laboratory Analysis [ Time Frame: Baseline up to 8 years ]
  Laboratory tests include minimal residual disease (MRD), gene expression profiling (GEP), fluorescence in situ hybridization (FISH), and cytogenetic results.
• Participant Response to Different Treatment Regimens Throughout the Study [ Time Frame: Baseline up to 8 years ]
• Progression Status on Different Treatment Regimens [ Time Frame: Baseline up to 8 years ]
• Time to Next therapy [ Time Frame: Baseline up to 8 years ]
• Overall Survival [ Time Frame: Baseline up to 8 years ]
• Progression Free Survival [ Time Frame: Baseline up to 8 years ]

Current Secondary Outcome Measures (submitted: May 8, 2018)

• Number of Treatment Combinations [ Time Frame: Baseline up to 8 years ]
• Number of Treatment Sequencing [ Time Frame: Baseline up to 8 years ]
• Number of Treatment Rechallenge [ Time Frame: Baseline up to 8 years ]
• Number of Clinical Outcomes for Different Strategies [ Time Frame: Baseline up to 8 years ]
• Number of Clinical Outcomes Between Continuous Treatment and Intermittent Treatment Strategy [ Time Frame: Baseline up to 8 years ]
• Triggers of Treatment Initiation at Relapse Including Biochemical Progression or Symptomatic Progression [ Time Frame: Baseline up to 8 years ]
• Reasons for Treatment Modifications [ Time Frame: Baseline up to 8 years ]
• Health Related Quality of Life (HRQoL) Among MM Participants [ Time Frame: Baseline up to 8 years ]
• Healthcare Resource Utilization (HRU) Among MM Participants [ Time Frame: Baseline up to 8 years ]
• Associations Between Presentation and Disease Characteristics [ Time Frame: Baseline up to 8 years ]
• Associations Between Choice Of Therapy and Clinical Outcomes [ Time Frame: Baseline up to 8 years ]
• Number of Participants Reporting Discontinuation One or More Treatment-emergent Adverse Events [ Time Frame: Baseline up to 8 years ]
  Treatment discontinuation includes temporary and permanent discontinuation, drug modification, and second primary malignancies.

Original Secondary Outcome Measures (submitted: May 2, 2016)

• Clinical Outcome of Participants on Different Treatment Regimens [ Time Frame: Baseline up to 8 years ]
• Clinical Outcome of Participants in Different Treatment Sequences or Combinations, or Retreatment with Prior Therapy [ Time Frame: Baseline up to 8 years ]
• Clinical Outcome of Participants on Continuous Treatment [ Time Frame: Baseline up to 8 years ]
• Clinical Outcome of Participants with Intermittent/Fixed Treatment [ Time Frame: Baseline up to 8 years ]
• Reasons for Treatment Initiation or Treatment Change for Participants at Relapse Including Biochemical Progression or Symptomatic Progression Throughout the Study [ Time Frame: Baseline up to 8 years ]
• Health Related Quality of Life (HRQoL) Among Multiple Myeloma (MM) Participants [ Time Frame: Baseline up to 8 years ]
• The Effect of Prescribed Treatment on Healthcare Resource Utilization (HRU) Among MM Participants [ Time Frame: Baseline up to 8 years ]
• Clinical Outcome of Participants Associated with Participant Presentation, Disease Characteristics, and Choice Of Therapy [ Time Frame: Baseline up to 8 years ]
• Number of Participants With Serious Adverse Events [ Time Frame: Baseline up to 8 years ]
• Number of Participants with Non-serious Adverse Events Leading to treatment discontinuation or modification [ Time Frame: Baseline up to 8 years ]

• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: An Observational Study of Presentation, Treatment Patterns, and Outcomes in Multiple Myeloma Participants
• Official Title: A Global, Prospective, Non-interventional, Observational Study of Presentation, Treatment Patterns, and Outcomes in Multiple Myeloma Patients - the INSIGHT - MM Study
• Brief Summary: The purpose of this study is to describe contemporary, real-world patterns of patient 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 patient 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.

• Study Type: Observational
• Study Design: Observational Model: Case-Only; Time Perspective: Prospective
• Target Follow-Up Duration: Not Provided
• Biospecimen: Not Provided
• Sampling Method: Probability Sample
• Study Population: Participants with Multiple Myeloma
• Condition: Multiple Myeloma
• Intervention: Other: No Intervention

Study Groups/Cohorts

• Relapsed/refractory (R/R) MM
  Patients who have received 1 to 3 prior lines of therapy
  Intervention: Other: No Intervention
• Newly diagnosed (ND) MM
  Patients within 3 months from initiation of treatment
  Intervention: Other: No Intervention

• Publications *: Costello C, Davies FE, Cook G, Vela-Ojeda J, Omel J, Rifkin RM, Berdeja J, Puig N, Usmani SZ, Weisel K, Zonder JA, Terpos E, Spencer A, Leleu X, Boccadoro M, Thompson MA, Romanus D, Stull DM, Hungria V. INSIGHT MM: a large, global, prospective, non-interventional, real-world study of patients with multiple myeloma. Future Oncol. 2019 May;15(13):1411-1428. doi: 10.2217/fon-2019-0013. Epub 2019 Feb 28.

Recruitment Information

• Recruitment Status: Active, not recruiting
• Estimated Enrollment (submitted: May 8, 2018): 4200
• Original Estimated Enrollment (submitted: May 2, 2016): 5000
• Estimated Study Completion Date: July 1, 2024
• Estimated Primary Completion Date: July 1, 2024 (Final data collection date for primary outcome measure)

Eligibility Criteria

Inclusion Criteria:

Is 18 years of age or older.

Is experiencing the following:

1. 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
2. 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.

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 18 Years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: Belgium, Brazil, China, Colombia, France, Germany, Greece, Israel, Italy, Mexico, Spain, Taiwan, Turkey, United Kingdom, United States

Administrative Information

• NCT Number: NCT02761187
• Other Study ID Numbers: NSMM-5001
• Has Data Monitoring Committee: No
• U.S. FDA-regulated Product: Not Provided

IPD Sharing Statement

• Plan to Share IPD: Yes
• Plan Description: Takeda makes patient-level, de-identified data sets and associated documents available after applicable marketing approvals and commercial availability have been received, an opportunity for the primary publication of the research has been allowed, and other criteria have been met as set forth in Takeda's Data Sharing Policy (see www.TakedaClinicalTrials.com/Approach for details). To obtain access, researchers must submit a legitimate academic research proposal for adjudication by an independent review panel, who will review the scientific merit of the research and the requestor's qualifications and conflict of interest that can result in potential bias. Once approved, qualified researchers who sign a data sharing agreement are provided access to these data in a secure research environment.

• Responsible Party: Takeda
• Study Sponsor: Takeda
• Collaborators: Not Provided
• Investigators: Not Provided
• PRS Account: Takeda
• Verification Date: August 2019