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A Study to Assess Disease Burden, in Terms of Health-related Quality of Life and Direct Healthcare Costs, in Patients With Newly Diagnosed Multiple Myeloma Who Are Not Candidates for Autologous Stem Cell Transplant (ASCT) in Spain (QoLMMBuS)

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ClinicalTrials.gov Identifier: NCT02946333
Recruitment Status : Recruiting
First Posted : October 27, 2016
Last Update Posted : April 17, 2019
Sponsor:
Information provided by (Responsible Party):
Celgene

Tracking Information
First Submitted Date September 30, 2016
First Posted Date October 27, 2016
Last Update Posted Date April 17, 2019
Actual Study Start Date November 25, 2016
Estimated Primary Completion Date October 10, 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: October 26, 2016)
  • Health-related quality of life (HRQoL) [ Time Frame: 4 years ]
    This questionnaire consists of 30 items grouped into 8 dimensions: number of drugs, gait velocity, mood, activities of daily living, health status, nutrition, mental state and comorbidity/habits. It is an instrument that was developed and validated to assess the health status of elderly patients diagnosed with different hematological diseases, including MM. This scale may be a useful instrument in clinical practice as a support tool to predict tolerance to treatment in advanced age. The time required to complete the questionnaire is approximately 10-12 minutes. It will be provided only at the baseline visit for the patient to fill in before starting treatment. The patient must fill it in again each time he or she starts a new line. The questionnaire will be provided by the physician during the visit.
  • EuroQoL 5-Dimension, 5-Level Questionnaire (EQ-5D-5L) [ Time Frame: 4 years ]
    This is a generic HRQoL questionnaire that contains a descriptive system and a visual analogue scale (VAS) for self-assessment of health status. The EQ-5D-5L's descriptive system contains a description of health state in five dimensions (mobility, self-care, usual activities, pain/discomfort and anxiety/depression). Each dimension has 5 possible responses. The respondent must mark the level of problems, within each dimension, that best describes his or her health state "today". It will be provided at each visit for the patient to fill in before starting treatment: at the baseline visit, approximately every 4 months in the first year (the visits must coincide with the patient's routine clinical practice visit) and after the first year every 6 months to progression. In case of progression and start of a second line of treatment, the questionnaire will start to be filled in with the same frequency initially described (baseline, every 4 months, etc.).
  • Quality of Life Questionnaire (QLQ-C30) [ Time Frame: 4 years ]
    This is a cancer-specific questionnaire that consists of 30 items structured into 5 functional scales (physical functioning, role functioning, emotional functioning, cognitive functioning and social functioning), 3 symptom scales (fatigue, nausea and vomiting, pain), a global health status scale and, finally, 6 independent items (dyspnoea, insomnia, appetite loss, constipation, diarrhea and financial difficulties). Values from 1 to 4 are assigned depending on the patient's responses to the item. Only items 29 and 30 are evaluated with scoring from 1 to 7. Scores are standardised to obtain a score from 0 to 100, which determines the level of impact of the cancer on the patient for each scale. High scores on the overall health and functional status scales indicate a better QoL, while high scores on the symptoms scale would indicate a decrease in QoL since they indicate the presence of cancer-related symptoms.
  • Quality of Life Questionnaire — Multiple Myeloma Module 20 (QLQ-MY20) [ Time Frame: 4 years ]
    This is a specific questionnaire used in combination with the QLQ-C30 generic questionnaire to evaluate quality of life in patients with MM. It consists of 20 items that address four specific domains — disease symptoms, side effects of treatment, future perspective and body image — and analyses various aspects of each dimension. Three of the four domains of the QLQ-MY20 are multi-item scales: Disease symptoms , side effects of treatment and future perspective (includes worry about death and health in the future and thinking about illness). The body image scale is a single-item scale that addresses physical attractiveness. The scores obtained are transformed linearly to a scale from 0 to 100. A high score in the disease symptoms and side effects domains represents a high level of symptoms or problems, while a high score in the future perspective and body image domains represents better outcomes.
  • Health care cost for Multiple Myeloma (MM) patients [ Time Frame: 4 years ]
    MM-related direct healthcare resources and costs will be evaluated in each line MM treatment will be evaluated. The questionnaire will be provided by the physician during the visit. The resources used since the previous study visit will be collected at each visit.
Original Primary Outcome Measures
 (submitted: October 25, 2016)
  • Health-related quality of life (HRQoL) [ Time Frame: 4 years ]
    This questionnaire consists of 30 items grouped into 8 dimensions: number of drugs, gait velocity, mood, activities of daily living, health status, nutrition, mental state and comorbidity/habits. It is an instrument that was developed and validated to assess the health status of elderly patients diagnosed with different hematological diseases, including MM. This scale may be a useful instrument in clinical practice as a support tool to predict tolerance to treatment in advanced age. The time required to complete the questionnaire is approximately 10-12 minutes. It will be provided only at the baseline visit for the patient to fill in before starting treatment. The patient must fill it in again each time he or she starts a new line. The questionnaire will be provided by the physician during the visit.
  • EuroQoL 5-Dimension, 5-Level Questionnaire (EQ-5D-5L) [ Time Frame: 4 years ]
    This is a generic HRQoL questionnaire that contains a descriptive system and a visual analogue scale (VAS) for self-assessment of health status. The EQ-5D-5L's descriptive system contains a description of health state in five dimensions (mobility, self-care, usual activities, pain/discomfort and anxiety/depression). Each dimension has 5 possible responses. The respondent must mark the level of problems, within each dimension, that best describes his or her health state "today". It will be provided at each visit for the patient to fill in before starting treatment: at the baseline visit, approximately every 4 months in the first year (the visits must coincide with the patient's routine clinical practice visit) and after the first year every 6 months to progression. In case of progression and start of a second line of treatment, the questionnaire will start to be filled in with the same frequency initially described (baseline, every 4 months, etc.).
  • Quality of Life Questionnaire (QLQ-C30) [ Time Frame: 4 years ]
    This is a cancer-specific questionnaire that consists of 30 items structured into 5 functional scales (physical functioning, role functioning, emotional functioning, cognitive functioning and social functioning), 3 symptom scales (fatigue, nausea and vomiting, pain), a global health status scale and, finally, 6 independent items (dyspnoea, insomnia, appetite loss, constipation, diarrhea and financial difficulties). Values from 1 to 4 are assigned depending on the patient's responses to the item. Only items 29 and 30 are evaluated with scoring from 1 to 7. Scores are standardised to obtain a score from 0 to 100, which determines the level of impact of the cancer on the patient for each scale. High scores on the overall health and functional status scales indicate a better QoL, while high scores on the symptoms scale would indicate a decrease in QoL since they indicate the presence of cancer-related symptoms.
  • Quality of Life Questionnaire — Multiple Myeloma Module 20 (QLQ-MY20) [ Time Frame: 4 years ]
    This is a specific questionnaire used in combination with the QLQ-C30 generic questionnaire to evaluate quality of life in patients with MM. It consists of 20 items that address four specific domains — disease symptoms, side effects of treatment, future perspective and body image — and analyses various aspects of each dimension. Three of the four domains of the QLQ-MY20 are multi-item scales: Disease symptoms , side effects of treatment and future perspective (includes worry about death and health in the future and thinking about illness). The body image scale is a single-item scale that addresses physical attractiveness. The scores obtained are transformed linearly to a scale from 0 to 100. A high score in the disease symptoms and side effects domains represents a high level of symptoms or problems, while a high score in the future perspective and body image domains represents better outcomes.
  • To evaluate health care cost for Multiple Myeloma (MM) patients [ Time Frame: 4 years ]
    MM-related direct healthcare resources and costs will be evaluated in each line MM treatment will be evaluated. The questionnaire will be provided by the physician during the visit. The resources used since the previous study visit will be collected at each visit.
Change History Complete list of historical versions of study NCT02946333 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures
 (submitted: October 25, 2016)
  • Time to progression (TTP) [ Time Frame: 4 years ]
    Time that elapses from the start of a treatment up to disease progression.
  • Progression-free survival (PFS) [ Time Frame: 4 years ]
    Time that elapses from the start of a treatment to disease progression or death, whichever occurs first.
  • Overall survival (OS) [ Time Frame: 9 years ]
    Time that elapses from the diagnosis of the disease up to the death of the patient for any reason.
  • Overall response rate [ Time Frame: 4 years ]
    Response measured through the IMWG criteria: Stringent complete response; complete response (CR); very good partial response; partial response (PR); stable disease (SD); progression (P)
  • Response duration [ Time Frame: 4 years ]
    For each line of treatment for multiple myeloma (MM) during the study period
  • Treatment-free interval [TFI] [ Time Frame: 4 years ]
    Time between relapse and start of a new treatment (second/third lines)
Original Secondary Outcome Measures Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title A Study to Assess Disease Burden, in Terms of Health-related Quality of Life and Direct Healthcare Costs, in Patients With Newly Diagnosed Multiple Myeloma Who Are Not Candidates for Autologous Stem Cell Transplant (ASCT) in Spain
Official Title Observational Study to Assess Disease Burden, in Terms of Health-related Quality of Life and Direct Healthcare Costs, in Patients With Newly Diagnosed Multiple Myeloma Who Are Not Candidates for Autologous Stem Cell Transplant (ASCT) in Spain
Brief Summary Those patients with newly diagnosed Multiple Myeloma (MM) who are not candidates for Autologous Stem Cell Transplant (ASCT) and who meet the screening criteria described in this protocol can participate. The participating patients must sign an informed consent, which the investigator who will collect the study variables in an electronic case report form (eCRF) will provide to them. The investigators must recruit at least 450 patients in a 24-month period.
Detailed Description

Following the baseline enrolment visit, the following data corresponding to the patient's visits scheduled according to routine clinical practice will be collected in accordance with the following model:

  1. Recruitment period: data collection at the baseline visit, every 4 months +/- 15 days in the first year (month 4, 8 and 12) and every 6 months +/- 1 month in the second year (month 18 and 24).
  2. Follow-up period: (study will end at 4 years following enrolment of the first patient)

    • From month 24 until up to 4 years from enrolment of the first patient, follow-ups will be performed with semi-annual data collections coinciding with the patient's routine clinical practice visits.
    • In case of progression, suspension or unexpected termination of treatment (for example due to toxicity), or death, a data collection coinciding with the visit in which any of the cases presented occurs will be performed.
    • If a new line of treatment is started, the follow-up model will be started with the same frequency of follow-ups described in point 1 above (month 4, 8, 12, 18 and 24 and semi-annually according to routine clinical practice until up to 4 years from enrolment of the first patient in the study).
  3. Observation period: Following this 4-year period, an additional 5-year follow-up will be performed only to evaluate the onset of second primary malignant neoplasms. This follow-up during the 5-year observational period will be performed semi-annually (± 1 month) according to routine clinical practice at each site.
Study Type Observational
Study Design Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration Not Provided
Biospecimen Not Provided
Sampling Method Non-Probability Sample
Study Population At least 450 patients with newly diagnosed multiple myeloma (MM) according to IMWG criteria who are not candidates for ASCT and who start drug treatment for the study disease will be enrolled.
Condition Multiple Myeloma
Intervention Not Provided
Study Groups/Cohorts Patient with MM who are not candidates for ASCT
Patients with newly diagnosed MM who are not candidates for ASCT and who are going to start drug treatment for the study disease and patient who is capable of understanding and filling in the study questionnaires At least 450 patients will be enrolled in a 2-year period. Patients must meet all of the inclusion criteria and none of the exclusion criteria and must have previously granted their informed consent in writing.
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Recruiting
Estimated Enrollment
 (submitted: October 25, 2016)
450
Original Estimated Enrollment Same as current
Estimated Study Completion Date October 30, 2025
Estimated Primary Completion Date October 10, 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria

Inclusion Criteria:

  • Patient ≥ 18 years of age.
  • Patient with newly diagnosed multiple myeloma (MM) according to IMWG criteria who are not candidates for ASCT and who start treatment for the study disease.
  • Patient who is capable of understanding and filling in the study questionnaires.
  • Patient who has granted his or her informed consent in writing.

Exclusion Criteria:

  • Patient who is participating in the active phase of treatment of any clinical trial during the study period.
Sex/Gender
Sexes Eligible for Study: All
Ages 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers No
Contacts
Contact: Associate Director Clinical Trial Disclosure 1-888-260-1599 clinicaltrialdisclosure@celgene.com
Listed Location Countries Spain
Removed Location Countries  
 
Administrative Information
NCT Number NCT02946333
Other Study ID Numbers CEL-MIE-2016-01
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement Not Provided
Responsible Party Celgene
Study Sponsor Celgene
Collaborators Not Provided
Investigators
Study Director: Marta Duran, RML Advocacy MM Celgene Corporation
PRS Account Celgene
Verification Date April 2019