A Study of People With Multiple Myeloma (MM) in Portugal (CharisMMa-Portugal)

Clinical and Sociodemographic Characterization of Multiple Myeloma Patients With Symptomatic Relapse and/or Refractory Disease in Portugal: an Observational, Multicenter Study

The main aim of this study is to see how people with MM respond to previous or current treatment.

Participants will be treated according to their clinic's standard practice. Each participant will fill out a study questionnaire during a routine doctor visit. Information collected from past medical records will also be used.

Study Overview

• Status: Completed
• Conditions: Relapse and/or Refractory Multiple Myeloma

Detailed Description

This is a non- interventional, retrospective epidemiological study of participants with MM. The study will characterize MM participants with symptomatic relapse and/or refractory (R/R) disease.

The study will enroll approximately 151 participants. The study will have a retrospective data collection referring to previous 5 years from participants' records and medical charts regarding diagnosis, disease activity, treatment patterns, and healthcare resources while quality of life (QoL) will be obtained from European Organization for Research and Treatment of Cancer Quality of Life Questionnaire (EORTC QLQ)-C30 and EORTC QLQ-MY20 questionnaires. All participants will be enrolled in one observational group.

This multi-center trial will be conducted in Portugal. The overall time to participate in this study is limited to completion of a questionnaire at the time of joining the study.

• Study Type: Observational
• Enrollment (Actual): 62

Contacts and Locations

Study Locations

• Portugal
  • Braga, Portugal, 4710-243
    • Centro Clinico Academico Hospital de Braga
  • Coimbra, Portugal, 3004-561
    • Centro Hospitalar e Universitario de Coimbra. EPE
  • Lisboa, Portugal, 1099-023
    • Instituto Portugues de Oncologia de Lisboa Francisco Gentil (IPO)
  • Lisboa, Portugal, 1649-028
    • Centro Hospitalar Lisboa Norte EPE - Hospital de Santa Maria
  • Porto, Portugal, 4099-001
    • Centro Hospitalar Universitario do Porto, E.P.E.
  • Porto, Portugal, 4200-072
    • Instituto Português de Oncologia do Porto Francisco Gentil, E.P.E.
  • Porto, Portugal, 4200-319
    • Centro Hospitalar Universitario de Sao Joao, EPE,
  • Vila Nova de Gaia, Portugal, 4434-502
    • Centro Hospitalar de Vila Nova de Gaia/Espinho, EPE,

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Participants with a diagnosis of MM with symptomatic R/R disease receiving treatment in Portugal.

Description

Inclusion Criteria:

1. At least one previous treatment line.
2. Symptomatic R/R disease in the previous 6 months to study enrolment.
3. Receiving treatment with an accurate and thorough data fulfilled in their participant's medical records available at the study site.
4. On regular follow-up for relapse and/or refractory disease during the recruitment period at the study site.
5. Capable of understanding and completing both QoL questionnaires (EORTC QLQ-C30 and EORTC QLQ-MY20).

Exclusion Criteria:

1. Diagnosed with MM more than 5 years previous to inclusion in this study.
2. Diagnosed with any malignancy other than MM or its complications within the past 5 years.
3. Currently participating in a clinical trial for his MM or having participated in a clinical trial within 5 years before inclusion.
4. Diagnosed with any hematological disease other than MM or its complications.

Study Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Retrospective

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort
Participants with MM; Participants diagnosed with MM from 8 investigative sites will be observed retrospectively for previous 5 years before enrollment until Day 1.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of MM Participants Categorized by Sociodemographic Variables	The sociodemographic variables will include age (in years), sex (male or female), body mass index (BMI), area of residence (rural or urban), educational level (illiterate, no studies (can only read/write), primary studies, secondary studies, or university studies), cohabitation (lives alone, lives with the family, lives alone with help from a caregiver, daily, 2-3 times/week, permanent or night-time), degree of dependence (independent, dependent grade I- requires help to perform instrumental activities of daily living (IADL) at least once a day, dependent grade II- needs help to perform IADLs 2 or 3 times a day or dependent grade III- needs help to perform IADLs several times a day),working situation (unemployed, in active employment, temporarily/permanently disabled, retired, student, other, need for financial assistance (yes/no), physical activity (high, moderate/low, inactive), smoking habit (yes/no), alcohol use (yes/no).	Day 1
Number of MM Participants Categorized by Clinical Variables on Diagnosis and During Previous Relapses	Clinical variables will include age (at diagnosis); MM type; international staging system (ISS) disease stage; Revised-ISS (R-ISS) disease stage; SliMCRAB signs at time of diagnosis: plasma cell bone marrow infiltration greater than or equal to (>=) 60 percent (%), serum free light chain (FLC) ratio >=100, >1 focal lesion visible on magnetic resonance imaging (MRI) with 5 millimeter (mm) or greater, increased blood calcium, renal failure, anemia, bone lesions: one or more osteolytic lesion on standard radiograph, computed tomography (CT) or Positron emission tomography-CT (PET-CT); cytogenetic abnormalities at time of diagnosis (t[4;14], t[11;14], t[14;16], t[14;20], t[6;14], trisomies, d[17p], g[1q]/ others); risk according to cytogenetic profile at diagnosis (mSMART v2 and mSMART v3); previous relapses (before the latest); stem cell transplant (yes/no); eastern cooperative oncology group (ECOG: 0-4) on diagnosis.	Day 1
Number of MM Participants Categorized by Clinical Variables at Last R/R Episode	Clinical variables include date of latest symptomatic relapse and/or refractory episode; ISS disease stage, CRAB signs; other clinical variables (plasmacytomas: medullary or extramedullary, diffuse osteopenia, fractures, neurological symptoms, infections, lactate dehydrogenase levels, paraprotein levels, free light chain levels); concomitant diseases (diabetes, neuropathy, chronic obstructive pulmonary disease, cardiovascular disease, liver failure, psychiatric and/or neurological disorders, any other secondary disorders); cytogenetic abnormalities at diagnosis; risk according to cytogenetic profile at relapse; treatment started after latest symptomatic relapse and/or refractory episode.	Day 1
Number of Participants Categorized by Current Treatment	Type of treatment will include therapeutic group.	Day 1

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of Participants Categorized by Sociodemographic Characteristics at the Latest Symptomatic R/R Episode per Type of Treatment	Sociodemographic variables will include age (in years), sex (male or female), BMI, area of residence (rural or urban), educational level (illiterate, no studies (can only read/write), primary studies, secondary studies, or university studies), cohabitation (lives alone, lives with the family, lives alone with help from a caregiver, daily, 2-3 times/week, permanent or night-time), degree of dependence (independent, dependent grade I - requires help to perform IADL at least once a day, dependent grade II - needs help to perform IADLs 2 or 3 times a day or dependent grade III - needs help to perform IADLs several times a day), need for financial assistance (yes/no), smoking habit (yes/no), alcohol use (yes/no).	Day 1
Number of MM Participants Categorized by Clinical Variables at Latest Symptomatic R/R Episode per Type of MM	Clinical variables will include- date of latest symptomatic R/R episode; SLiMCRAB signs at latest symptomatic R/R episode: plasma cell bone marrow infiltration >=60 %, serum FLC ratio >=100, >1 focal lesion visible on MRI with 5 mm or greater, increased blood calcium, renal failure, anemia, bone lesions: one or more osteolytic lesion on standard radiograph, CT or PET-CT; other clinical variables at latest symptomatic R/R episode include- plasmacytomas (medullary/extramedullary); diffuse osteopenia; fractures; neurological symptoms (MM related); infections; lactose dehydrogenase (LDH); paraprotein levels (gram per liter [g/L]) (serum and urine) free light chain levels (g/L) (serum and urine).	Day 1
Number of Participants Categorized by Relevant Variables that are not Currently Collected in Clinical Record and Could Influence in the Disease Management at Relapse	New relevant variables will include factors mentioned by the participant as relevant to the disease management (example, family support, employment status); type of transportation used to receive treatment; mean cost in Euros per month with transportation to receive treatment, mean of hospital visits per month since last treatment was initiated, mean scheduled and non-scheduled participant's visits to the hospital, days of hospitalization, and specialists consulted; consultations, days of hospitalization, medical appointments and specialists consulted by type of treatment.	Day 1

Collaborators and Investigators

• Sponsor: Takeda
• Investigators: Study Director: Study Director, Takeda

Publications and helpful links

Helpful Links

• To obtain more information about this study, click this link.

Study record dates

Study Major Dates

• Study Start (Actual): July 31, 2020
• Primary Completion (Actual): September 30, 2022
• Study Completion (Actual): September 30, 2022

Study Registration Dates

• First Submitted: October 21, 2019
• First Submitted That Met QC Criteria: October 21, 2019
• First Posted (Actual): October 23, 2019

Study Record Updates

• Last Update Posted (Actual): October 23, 2023
• Last Update Submitted That Met QC Criteria: October 20, 2023
• Last Verified: October 1, 2023

More Information

Terms related to this study

• Keywords: Multiple Myeloma
• Additional Relevant MeSH Terms: Pathologic Processes; Cardiovascular Diseases; Vascular Diseases; Immune System Diseases; Neoplasms by Histologic Type; Neoplasms; Lymphoproliferative Disorders; Immunoproliferative Disorders; Disease Attributes; Hematologic Diseases; Hemorrhagic Disorders; Hemostatic Disorders; Paraproteinemias; Blood Protein Disorders; Multiple Myeloma; Neoplasms, Plasma Cell; Recurrence
• Other Study ID Numbers: RRMM-5017; U1111-1234-4861 (Registry Identifier: WHO)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.
• IPD Sharing Access Criteria: IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.
• IPD Sharing Supporting Information Type: STUDY_PROTOCOL; SAP; ICF; CSR

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No