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)

November 18, 2022 updated by: Celgene

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

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.

Study Overview

Status

Completed

Conditions

Multiple Myeloma

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:

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).
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).
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

Enrollment (Actual)

355

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

Spain
- - Madrid, Spain, 28031
    - Hospital Infanta Leonor
  - Madrid, Spain, 28046
    - Hospital La Paz
  - Madrid, Spain, 28031
    - Hospital Ramon y Cajal
  - Navarra, Spain, 31008
    - Complejo Hospitalario de Navarra
  - Valencia, Spain, 46026
    - Hospital Universitario La Fe
  - Zaragoza, Spain, 50009
    - Hospital Miguel Servet
  - Ávila, Spain, 05004
    - Complejo Hospitalario de Ávila
- Andalusia
  - Algeciras, Andalusia, Spain, 11207
    - Hospital Punta de Europa
  - Antequera, Andalusia, Spain, 29200
    - Hospital de Antequera
  - Córdoba, Andalusia, Spain, 14004
    - Hospital Reina Sofía
  - Huercal Olvera, Andalusia, Spain, 04600
    - Hospital Huercal Olvera
  - Jaén, Andalusia, Spain, 23007
    - Hospital General Jaén
  - Jerez de la Frontera, Andalusia, Spain, 11407
    - Hospital Jerez de la Frontera
  - Motril, Andalusia, Spain, 18600
    - Hospital de Motril
  - Málaga, Andalusia, Spain, 29010
    - Clinico Univ. Virgen de la Victoria
  - Málaga, Andalusia, Spain, 29010
    - Hospital Carlos Haya
  - Málaga, Andalusia, Spain, 29603
    - Hospital de Costa del Sol
  - Pozoblanco, Andalusia, Spain, 14400
    - Hospital Valle de los Pedroches Pozoblanco
  - Puerto Real, Andalusia, Spain, 11510
    - Hospital Puerto Real
  - Sevilla, Andalusia, Spain, 29200
    - Hospital de Macarena
- Aragón
  - Zaragoza, Aragón, Spain, 50009
    - Hospital Miguel Servet
- Barcelona
  - Hospitalet de Llobregat, Barcelona, Spain, 08908
    - Hospital ICO Durán y Reynals
  - Hospitalet de Llobregat, Barcelona, Spain, 08908
    - ICO Duran i Reynals
- Canarias
  - Las Palmas de Gran Canaria, Canarias, Spain, 35010
    - Hospital Universitario Dr. Negrin
- Castilla Y León
  - Burgos, Castilla Y León, Spain, 09006
    - Hospital Universitario de Burgos
  - Salamanca, Castilla Y León, Spain, 37007
    - Hospital Clinico Universitario de Salamanca
  - Valladolid, Castilla Y León, Spain, 47003
    - Hospital Clínico de Valladolid
- Cataluña
  - Barcelona, Cataluña, Spain, 08003
    - Hospital Del Mar
  - Barcelona, Cataluña, Spain, 08026
    - Hospital Santa Creu i Sant Pau
  - Barcelona, Cataluña, Spain, 08035
    - Hospital Vall Hebron
  - Granollers, Cataluña, Spain, 08402
    - Hospital General de Granollers
  - Lleida, Cataluña, Spain, 25198
    - Hospital Arnau Vilanova de Lleida
  - Manresa, Cataluña, Spain, 08243
    - Fundació Althaia
  - Sabadell, Cataluña, Spain, 08208
    - Parc Taulí
  - Terrassa, Cataluña, Spain, 08221
    - Mutua Terrassa
- Comunidad De Madrid
  - Getafe, Comunidad De Madrid, Spain, 28905
    - Hospital Universitario de Getafe
  - Madrid, Comunidad De Madrid, Spain, 28040
    - Hospital Clinico San Carlos
  - Madrid, Comunidad De Madrid, Spain, 28006
    - Hospital de La Princesa
  - Madrid, Comunidad De Madrid, Spain, 28007
    - Hospital General Universitario Gregorio Marañón
  - Madrid, Comunidad De Madrid, Spain, 28040
    - Hospital Universitario Fundación Jiménez Díaz
  - Madrid, Comunidad De Madrid, Spain, 28041
    - Hospital 12 de Octubre
- Comunidad Valenciana
  - Castellón de la Plana, Comunidad Valenciana, Spain, 12004
    - Hospital General Castellón
  - Valencia, Comunidad Valenciana, Spain, 46015
    - Hospital Arnau Vilanova Valencia
  - Valencia, Comunidad Valenciana, Spain, 46017
    - Hospital Doctor Peset
  - Valencia, Comunidad Valenciana, Spain, 46026
    - Hospital La Fe Valencia
  - Valencia, Comunidad Valenciana, Spain, 46600
    - Hospital La Ribera
- Extremadura
  - Llerena, Extremadura, Spain, 06900
    - Complejo Hospitalario Llerena-Zafra
  - Mérida, Extremadura, Spain, 06800
    - Hospital de Mérida
- Galicia
  - A Coruña, Galicia, Spain, 15006
    - Complexo Hospitalario Universitario de A Coruña
  - Lugo, Galicia, Spain, 27003
    - Hospital Lucus Augusti
  - Ourense, Galicia, Spain, 32005
    - Complexo Hospitalario Universitario de Ourense
  - Pontevedra, Galicia, Spain, 36071
    - Complexo Hospitalario de Pontevedra
  - Vigo, Galicia, Spain, 36312
    - Complexo Hospitalario Universitario de Vigo
- Islas Baleares
  - Palma de Mallorca, Islas Baleares, Spain, 07198
    - Hospital Son Llatzer
  - Palma de Mallorca, Islas Baleares, Spain, 07120
    - Hospital Son Espases
- Islas Canaria
  - Santa Cruz de Tenerife, Islas Canaria, Spain, 38320
    - Hospital Universitario Canarias
- Islas Canarias
  - Las Palmas de Gran Canaria, Islas Canarias, Spain, 38010
    - Hospital Dr. Negrín
  - Santa Cruz de Tenerife, Islas Canarias, Spain, 38010
    - Hospital Universitario Nuestra Señora de Candelaria
- Madrid
  - Pozuelo de Alarcón, Madrid, Spain, 28223
    - Hospital Quirón
  - San Sebastián de los Reyes, Madrid, Spain, 28703
    - Hospital Infanta Sofía
- Navarra
  - Pamplona, Navarra, Spain, 31008
    - Complejo Hospitalario Navarra
- País Vasco
  - Bilbao, País Vasco, Spain, 48013
    - Hospital de Basurto
  - Usansolo, País Vasco, Spain, 48960
    - Hospital de Galadakao
  - Vitoria-Gasteiz, País Vasco, Spain, 01009
    - Hospital de Txagorritxu
- Valencia
  - Alzira, Valencia, Spain, 46600
    - Hospital de la Ribera

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (ADULT, OLDER_ADULT)

Accepts Healthy Volunteers

Genders Eligible for Study

All

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.

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Number of groups / cohorts

Cohorts and Interventions

Group / Cohort
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.

Group / Cohort

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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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.	4 years
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.).	4 years
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.	4 years
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.	4 years
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.	4 years

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Time to progression (TTP) Time Frame: 4 years	Time that elapses from the start of a treatment up to disease progression.	4 years
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.	4 years
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.	9 years
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)	4 years
Response duration Time Frame: 4 years	For each line of treatment for multiple myeloma (MM) during the study period	4 years
Treatment-free interval [TFI] Time Frame: 4 years	Time between relapse and start of a new treatment (second/third lines)	4 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Celgene

Investigators

Study Director: Giselle Lostaunau, Senior Medical Advisor MM, Celgene

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Engelhardt M, Ihorst G, Singh M, Rieth A, Saba G, Pellan M, Lebioda A. Real-World Evaluation of Health-Related Quality of Life in Patients With Multiple Myeloma From Germany. Clin Lymphoma Myeloma Leuk. 2021 Feb;21(2):e160-e175. doi: 10.1016/j.clml.2020.10.002. Epub 2020 Oct 24.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (ACTUAL)

December 25, 2016

Primary Completion (ACTUAL)

October 31, 2022

Study Completion (ACTUAL)