Clinical and Sociodemographic Characterization of Multiple Myeloma Participants With Symptomatic Relapse and/or Refractory Disease in Spain (CharisMMa Study)

• ClinicalTrials.gov Identifier: NCT03188536
• Recruitment Status: Completed
• First Posted: June 15, 2017
• Results First Posted: October 12, 2020
• Last Update Posted: October 12, 2020
• Sponsor: Takeda
• Information provided by (Responsible Party): Takeda

Tracking Information

• First Submitted Date: June 12, 2017
• First Posted Date: June 15, 2017
• Results First Submitted Date: August 17, 2020
• Results First Posted Date: October 12, 2020
• Last Update Posted Date: October 12, 2020
• Actual Study Start Date: July 26, 2017
• Actual Primary Completion Date: November 15, 2018 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: September 14, 2020)

• Percentage of MM Participants Categorized by Sociodemographic Variables at Diagnosis [ Time Frame: Day 1 ]
  Sociodemographic variables included age(in years), sex(male or female), body mass index(BMI), residence(rural or urban), educational level(illiterate,no studies(can only read/write),primary studies,secondary studies,higher studies), work status(unemployed,active employment,temporarily/ permanently disabled, retired, other), cohabitation(lives-alone,with relatives,alone with help from a caregiver), need for financial assistance (yes/no), degree of dependence(independent,dependent:grade I-requires help to perform activities of daily living [ADL] at least once a day,grade II-needs help to perform ADLs 2 or 3 times a day or grade III-needs help to perform ADLs several times a day), healthy habits (high[jogging;climbing;cycling;swimming;sports;intensive work;moving >20 kg loads], moderate[walking;dancing;domestic chores;sports with children,walking house pets;general construction work;moving <20 kg loads] physical activity or inactive, smoking habit (yes/no), alcohol use (yes/no).
• Percentage of MM Participants Categorized by Clinical Variables at Diagnosis [ Time Frame: Day 1 ]
  Clinical variables at Diagnosis included age,MM type(heavy/light chain/Bence-Jones protein), international staging system(ISS),disease stage(I:low risk,β2-Microglobulin<3.5mg/L and albumin≥3.5g/dL,II:not stage I or III,III:high risk,β2-Microglobulin≥5.5mg/L),calcium,renal insufficiency,anemia or bone lesions(CRAB) signs(serum calcium>0.25mmol/L upper limit of normal,renal failure-creatinine clearance<40mL/min/serum creatinine>117μmol/L, anemia:reduction of hemoglobin(Hb)>2g/dL below lower limit of normal or Hb<10g/dL,bone lesions 1/more osteolytic lesion,cytogenetic abnormalities(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(standard:trisomies,t[11;14];t[6;14],intermediate:t[4;14],g[1q], high:d[17p],t[14;16],t[14;20],other), eastern cooperative oncology group(ECOG) status (0:fully active,1:restricted physical activity,2:ambulatory,unable to carry out any work,3:capable of limited selfcare,4:completely disabled,5:dead).
• Percentage of MM Participants Categorized by Clinical Variables at Latest Symptomatic Relapse and/or Refractory Episode [ Time Frame: Day 1 ]
  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, concomitant diseases like diabetes, neuropathy, chronic obstructive pulmonary disease (COPD), cardiovascular disease, liver failure, psychiatric and/or neurological disorders, any other secondary disorders, cytogenetic abnormalities, risk according to cytogenetic profile at relapse(standard:trisomies,t[11;14];t[6;14],intermediate:t[4;14],g[1q], high:d[17p],t[14;16],t[14;20],other), treatment started after latest symptomatic relapse and/or refractory episode.

Original Primary Outcome Measures (submitted: June 13, 2017)

• Percentage of MM Participants Categorized by Sociodemographic Variables [ Time Frame: Day 1 ]
  Sociodemographic variables 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 activities of daily living (ADL) at least once a day, dependent grade II - needs help to perform ADLs 2 or 3 times a day or dependent grade III - needs help to perform ADLs several times a day), working situation (unemployed, in active employment, full time, part time, temporarily/ permanently disabled, retired, student or other), need for financial assistance (yes/no), healthy habits (high/moderate physical activity or inactive, smoking habit (yes/no), alcohol use (yes/no).
• Percentage of MM Participants Categorized by Clinical Variables on Diagnosis and During Previous Relapses [ Time Frame: Day 1 ]
  Clinical variables include age (at diagnosis), MM type (heavy/light chain/ Bence-Jones protein), ISS (international staging system) disease stage (stage I-low risk, β2-Microglobulin <3.5 mg/L and albumin ≥3.5 g/dL, stage II-not ISS stage I or III, stage III-high risk, β2-Microglobulin ≥5.5 mg/L), CRAB (calcium, renal insufficiency, anemia or bone lesions) signs (serum calcium >0.25 mmol/L upper limit of normal, renal failure-creatinine clearance <40 mL/min/ serum creatinine >117 μmol/L, anemia:reduction of hemoglobin (Hb) >2 g/dL below lower limit of normal or Hb <10 g/dL, bone lesions 1/more osteolytic lesion, cytogenetic abnormalities (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 (standard risk:trisomies, t[11;14];t[6;14], intermediate risk:t[4;14],g[1q], high risk: d[17p],t[14;16],t[14;20]), previous relapses (before the latest), received stem cell transplant, eastern cooperative oncology group (ECOG:0-5).
• Percentage of MM Participants Categorized by Clinical Variables at Latest Symptomatic Relapse and/or Refractory Episode [ Time Frame: Day 1 ]
  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, risk according to cytogenetic profile at relapse, treatment started after latest symptomatic relapse and/or refractory episode, concomitant diseases at time of latest symptomatic relapse and/or refractory (R/R) episode.

Current Secondary Outcome Measures (submitted: September 14, 2020)

• Percentage of MM Participants With Clinical and Sociodemographic Characteristics Categorized by Treatment Selection at the Latest Symptomatic Relapse and/or Refractory Episode [ Time Frame: Day 1 ]
  Clinical and Sociodemographic variables categorized by treatment selection(immunomodulators[IMiDs],proteasome inhibitor[PI]+IMiDs,PI,monoclonal antibodies[mAb]): Age,sex(male/female),prior treatment lines(0,1,2,3 or more),prior relapses(0,1,2,3 or more),ISS disease stage(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),CRAB signs(serum calcium>0.25mmol/L upper limit of normal,renal failure-creatinine clearance<40mL/min/serum creatinine>117μmol/L,anemia:reduction of hemoglobin(Hb)>2g/dL below lower limit of normal or Hb<10g/dL,bone lesions 1/more osteolytic lesion),risk according to cytogenetic profile(standard:trisomies,t[11;14];t[6;14];high:d[17p],t[14;16],t[14;20],other),other clinical variables:medullary/extramedullary plasmacytomas,osteopenia,fractures,neurological symptoms,infections,concomitant diseases:diabetes, neuropathy, COPD, cardiovascular disease, liver failure, psychiatric, neurological disorders.
• Number of New Relevant Variables That Are Not Currently Collected in Clinical Records and That Could Influence in the Disease Management at Relapse [ Time Frame: Day 1 ]
• Health-Related Quality of Life (HRQOL) Based on European Organization for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire- Core 30 [ Time Frame: Day 1 ]
  The EORTC QLQ-C30 contains 30 items across 5 functional scales (physical, role, cognitive, emotional, and social), 9 symptom scales (fatigue, nausea and vomiting, pain, dyspnea, sleep disturbance, appetite loss, constipation, diarrhea, and financial difficulties) and a global health status/QOL scale. Most of the 30 items have 4 response levels (not at all, a little, quite a bit, and very much), with 2 questions relying on a 7-point numeric rating scale with a recall period of the previous week. Raw scores are converted into scale scores ranging from 0 to 100. For the functional scales and the global health status/QOL scale, higher scores represent better QOL; for the symptom scales, lower scores represent better QOL.
• HRQOL Based on EORTC Multiple Myeloma Module (EORTC QLQ-MY20) Subscale Score [ Time Frame: Day 1 ]
  The EORTC QLQ-MY20 has 20 items across 4 independent subscales, 2 functional subscales (body image, future perspective), and 2 symptoms scales (disease symptoms, and side effects of treatment) with a recall period of previous week. Scores from each subscale were transformed from 0 to 100. For the functional scales, high scores represent improvement. For the symptom scales, higher scores represent worsening.
• HRQOL Based on the Spanish Translated Version of EORTC Multiple Myeloma Module (EORTC QLQ-MY20) [ Time Frame: Day 1 ]
  The EORTC QLQ-MY20 has 20 items across 4 independent subscales, 2 functional subscales (body image, future perspective), and 2 symptoms scales (disease symptoms, and side effects of treatment) with a recall period of previous week. Scores from each subscale were transformed from 0 to 100. For the functional scales, high scores represent improvement. For the symptom scales, higher scores represent worsening. Spanish Translated Version of EORTC Multiple Myeloma Module included 4 factors, each factor corresponds to questions related to the particular component of the scale. Factor I indicate symptoms. Factor II indicates future perspectives. Factor III indicate adverse effects of the treatment. Factor IV indicates adverse effects of the treatment and body image. Each of these factors were used for the clinical validity of the scale.
• Percentage of Participants With Health Care Resource Utilization (HU) [ Time Frame: Day 1 ]
  Healthcare resources used during medical encounters include intensive care unit (ICU) admissions, hospital admissions, visits to the emergency room, days admitted.

Original Secondary Outcome Measures (submitted: June 13, 2017)

• Percentage of MM Participants with Clinical and Sociodemographic Characteristics Categorized by Treatment Selection at the Latest Symptomatic Relapse and/or Refractory Episode [ Time Frame: Day 1 ]
  Treatment selection includes treatment line and therapeutic group.
• Number of new Relevant Variables that are not Currently Collected in Clinical Records and that Could Influence in the Disease Management at Relapse [ Time Frame: Day 1 ]
• Health-Related Quality of Life (HRQOL) based on European Organization for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire- Core 30 (EORTC QLQ-C30) [ Time Frame: Day 7 ]
  The EORTC QLQ-C30 contains 30 items across 5 functional scales (physical, role, cognitive, emotional, and social), 9 symptom scales (fatigue, nausea and vomiting, pain, dyspnea, sleep disturbance, appetite loss, constipation, diarrhea, and financial difficulties) and a global health status/QOL scale. Most of the 30 items have 4 response levels (not at all, a little, quite a bit, and very much), with 2 questions relying on a 7-point numeric rating scale. Raw scores are converted into scale scores ranging from 0 to 100. For the functional scales and the global health status/QOL scale, higher scores represent better QOL; for the symptom scales, lower scores represent better QOL. The questionnaire is completed with assessment over the previous 7 days.
• HRQOL based on EORTC Multiple Myeloma Module (EORTC QLQ-MY20) Score [ Time Frame: Day 7 ]
  The EORTC QLQ-MY20 has 20 items across 4 independent subscales, 2 functional subscales (body image, future perspective), and 2 symptoms scales (disease symptoms, and side effects of treatment). Scores are averaged, and transformed scores ranging from 0 to 100. For the future perspective scale, higher score = better perspective of the future. For the body image scale, higher scores = better body image. Higher score for the disease symptoms scale = higher level of symptomatology. The questionnaire is completed with assessment over the previous 7 days.
• HRQOL based on the Spanish Translated Version of EORTC Multiple Myeloma Module (EORTC QLQ-MY20) [ Time Frame: Day 7 ]
  The Spanish version of EORTC QLQ-MY20 has 20 items across 4 independent subscales, 2 functional subscales (body image, future perspective), and 2 symptoms scales (disease symptoms, and side effects of treatment). Scores are averaged, and transformed scores ranging from 0 to 100. For the future perspective scale, higher score = better perspective of the future. For the body image scale, higher scores = better body image. Higher score for the disease symptoms scale = higher level of symptomatology. The questionnaire is completed with assessment over the previous 7 days.
• Health Care Resource Utilization (HU): Number of Medical Encounters [ Time Frame: Day 1 ]
  Healthcare resources used during medical encounters include transport use, hospital visits, intensive care unit (ICU) admissions, hospital admissions, visits to the emergency room, days admitted, visits to specialists, test use, accompanying person in active employment.

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

Descriptive Information

• Brief Title: Clinical and Sociodemographic Characterization of Multiple Myeloma Participants With Symptomatic Relapse and/or Refractory Disease in Spain (CharisMMa Study)
• Official Title: Clinical and Sociodemographic Characterization of Multiple Myeloma Patients With Symptomatic Relapse and/or Refractory Disease in Spain
• Brief Summary: The purpose of this study is to characterize the multiple myeloma (MM) participants with symptomatic relapse and/or refractory disease in Spain.

Detailed Description

Adult participants with a diagnosis of MM who have received at least one previous treatment line and have experienced symptomatic relapse and/or refractory disease in the previous 6 months, who are still in follow-up at the time of the study visit will be observed in this study.

The study will look into sociodemographic data, current clinical and therapeutic data, clinical data relative to the latest relapse and clinical data at diagnosis and previous relapses will be collected.

The study will enroll approximately 350 patients.

This multi-center trial will be conducted in a total of 30 public sites in Spain. The overall time to collect data will be approximately 1 year from June 2017 to May 2018.

• Study Type: Observational
• Study Design: Observational Model: Cohort; Time Perspective: Retrospective
• Target Follow-Up Duration: Not Provided
• Biospecimen: Not Provided
• Sampling Method: Non-Probability Sample
• Study Population: Participants with a diagnosis of MM who have received at least one previous treatment line and have experienced symptomatic relapse and/or refractory disease in the previous 6 months, who are still in follow-up at the time of the study visit will be observed in this study.
• Condition: Multiple Myeloma

Intervention

Other: No Intervention

As it was an observational study, no intervention was administered in this study.

Study Groups/Cohorts

Multiple Myeloma (MM) Participants

Adult participants with a diagnosis of MM who received at least one previous treatment line (standard care of treatment) and experienced symptomatic relapse and/or refractory disease in the previous 6 months, who were followed-up at the time of the study visit. No intervention was administered in this study.

Intervention: Other: No Intervention

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: September 14, 2020): 282
• Original Estimated Enrollment (submitted: June 13, 2017): 350
• Actual Study Completion Date: January 30, 2019
• Actual Primary Completion Date: November 15, 2018 (Final data collection date for primary outcome measure)

Eligibility Criteria

Inclusion Criteria:

• Have a diagnosis of MM and has received at least one previous treatment line.
• Have experienced symptomatic relapse and/or refractory disease in the 6 months before the study.
• Has continued in follow-up at the time of the study visit.
• Is currently treated in the site who have clinical records available.
• Is capable of understanding and completing the questions in the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire-Core 30 (EORTC QLQ-C30), and EORTC Multiple Myeloma Module (QLQ-MY20) questionnaires.

Exclusion Criteria:

• Participants who do not agree to participate in the study or who do not give written informed consent.

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: Spain

Administrative Information

• NCT Number: NCT03188536
• Other Study ID Numbers: RRMM-5012; TAK-MMR-2017-01 ( Registry Identifier: Spanish health authority )
• Has Data Monitoring Committee: No

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No

IPD Sharing Statement

• Plan to Share IPD: Yes
• Plan Description: Takeda makes patient-level, de-identified data sets and associated documents available for all interventional studies after applicable marketing approvals and commercial availability have been received (or program is completely terminated), an opportunity for the primary publication of the research and final report development has been allowed, and other criteria have been met as set forth in Takeda's Data Sharing Policy (see www.TakedaClinicalTrials.com 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.

• Current Responsible Party: Takeda
• Original Responsible Party: Same as current
• Current Study Sponsor: Takeda
• Original Study Sponsor: Same as current
• Collaborators: Not Provided

Investigators

• Study Director: Medical Monitor Clinical Science; Takeda

• PRS Account: Takeda
• Verification Date: September 2020