QoLMMBuS: 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

Sponsor

Celgene (Industry)

Overall Status

Active, not recruiting

CT.gov ID

NCT02946333

Collaborator

(none)

365

Enrollment

Locations

71.2

Anticipated Duration (Months)

5.6

Patients Per Site

0.1

Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
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 Design

Study Type:

Observational

Anticipated Enrollment :

365 participants

Observational Model:

Cohort

Time Perspective:

Prospective

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

Actual Study Start Date :

Nov 25, 2016

Anticipated Primary Completion Date :

Oct 31, 2022

Anticipated Study Completion Date :

Oct 31, 2022

Arms and Interventions

Arm	Intervention/Treatment
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.

Arm

Intervention/Treatment

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.

Outcome Measures

Primary Outcome Measures

Health-related quality of life (HRQoL) [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) [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) [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) [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 [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.

Secondary Outcome Measures

Time to progression (TTP) [4 years]
Time that elapses from the start of a treatment up to disease progression.
Progression-free survival (PFS) [4 years]
Time that elapses from the start of a treatment to disease progression or death, whichever occurs first.
Overall survival (OS) [9 years]
Time that elapses from the diagnosis of the disease up to the death of the patient for any reason.
Overall response rate [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 [4 years]
For each line of treatment for multiple myeloma (MM) during the study period
Treatment-free interval [TFI] [4 years]
Time between relapse and start of a new treatment (second/third lines)

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Hospital Punta de Europa	Algeciras	Andalusia	Spain	11207
2	Hospital de Antequera	Antequera	Andalusia	Spain	29200
3	Hospital Reina Sofía	Córdoba	Andalusia	Spain	14004
4	Hospital Huercal Olvera	Huercal Olvera	Andalusia	Spain	04600
5	Hospital General Jaén	Jaén	Andalusia	Spain	23007
6	Hospital Jerez de la Frontera	Jerez de la Frontera	Andalusia	Spain	11407
7	Hospital de Motril	Motril	Andalusia	Spain	18600
8	Clinico Univ. Virgen de la Victoria	Málaga	Andalusia	Spain	29010
9	Hospital Carlos Haya	Málaga	Andalusia	Spain	29010
10	Hospital de Costa del Sol	Málaga	Andalusia	Spain	29603
11	Hospital Valle de los Pedroches Pozoblanco	Pozoblanco	Andalusia	Spain	14400
12	Hospital Puerto Real	Puerto Real	Andalusia	Spain	11510
13	Hospital de Macarena	Sevilla	Andalusia	Spain	29200
14	Hospital Miguel Servet	Zaragoza	Aragón	Spain	50009
15	Hospital ICO Durán y Reynals	Hospitalet de Llobregat	Barcelona	Spain	08908
16	ICO Duran i Reynals	Hospitalet de Llobregat	Barcelona	Spain	08908
17	Hospital Universitario Dr. Negrín	Las Palmas de Gran Canaria	Canarias	Spain	35010
18	Hospital Universitario de Burgos	Burgos	Castilla Y León	Spain	09006
19	Hospital Clínico Universitario de Salamanca	Salamanca	Castilla Y León	Spain	37007
20	Hospital Clínico de Valladolid	Valladolid	Castilla Y León	Spain	47003
21	Hospital del Mar	Barcelona	Cataluña	Spain	08003
22	Hospital Santa Creu i Sant Pau	Barcelona	Cataluña	Spain	08026
23	Hospital Vall Hebrón	Barcelona	Cataluña	Spain	08035
24	Hospital General de Granollers	Granollers	Cataluña	Spain	08402
25	Hospital Arnau Vilanova de Lleida	Lleida	Cataluña	Spain	25198
26	Fundació Althaia	Manresa	Cataluña	Spain	08243
27	Parc Taulí	Sabadell	Cataluña	Spain	08208
28	Mutua Terrassa	Terrassa	Cataluña	Spain	08221
29	Hospital Universitario de Getafe	Getafe	Comunidad De Madrid	Spain	28905
30	Hospital de la Princesa	Madrid	Comunidad De Madrid	Spain	28006
31	Hospital General Universitario Gregorio Marañon	Madrid	Comunidad De Madrid	Spain	28007
32	Hospital Clínico San Carlos	Madrid	Comunidad De Madrid	Spain	28040
33	Hospital Universitario Fundación Jiménez Díaz	Madrid	Comunidad De Madrid	Spain	28040
34	Hospital 12 de Octubre	Madrid	Comunidad De Madrid	Spain	28041
35	Hospital General Castellón	Castellón de la Plana	Comunidad Valenciana	Spain	12004
36	Hospital Arnau Vilanova Valencia	Valencia	Comunidad Valenciana	Spain	46015
37	Hospital Doctor Peset	Valencia	Comunidad Valenciana	Spain	46017
38	Hospital La Fe Valencia	Valencia	Comunidad Valenciana	Spain	46026
39	Hospital La Ribera	Valencia	Comunidad Valenciana	Spain	46600
40	Complejo Hospitalario Llerena-Zafra	Llerena	Extremadura	Spain	06900
41	Hospital de Mérida	Mérida	Extremadura	Spain	06800
42	Complexo Hospitalario Universitario de A Coruña	A Coruña	Galicia	Spain	15006
43	Hospital Lucus Augustí	Lugo	Galicia	Spain	27003
44	Complexo Hospitalario Universitario de Ourense	Ourense	Galicia	Spain	32005
45	Complexo Hospitalario de Pontevedra	Pontevedra	Galicia	Spain	36071
46	Complexo Hospitalario Universitario de Vigo	Vigo	Galicia	Spain	36312
47	Hospital Son Espases	Palma de Mallorca	Islas Baleares	Spain	07120
48	Hospital Son Llàtzer	Palma de Mallorca	Islas Baleares	Spain	07198
49	Hospital Dr. Negrín	Las Palmas de Gran Canaria	Islas Canarias	Spain	38010
50	Hospital Universitario Nuestra Señora de Candelaria	Santa Cruz de Tenerife	Islas Canarias	Spain	38010
51	Hospital Universitario Canarias	Santa Cruz de Tenerife	Islas Canaria	Spain	38320
52	Hospital Quirón	Pozuelo de Alarcón	Madrid	Spain	28223
53	Hospital Infanta Sofía	San Sebastián de los Reyes	Madrid	Spain	28703
54	Complejo Hospitalario Navarra	Pamplona	Navarra	Spain	31008
55	Hospital de Basurto	Bilbao	País Vasco	Spain	48013
56	Hospital de Galadakao	Usansolo	País Vasco	Spain	48960
57	Hospital de Txagorritxu	Vitoria-Gasteiz	País Vasco	Spain	01009
58	Hospital de La Ribera	Alzira	Valencia	Spain	46600
59	Hospital Infanta Leonor	Madrid		Spain	28031
60	Hospital Ramón y Cajal	Madrid		Spain	28031
61	Hospital La Paz	Madrid		Spain	28046
62	Complejo Hospitalario de Navarra	Navarra		Spain	31008
63	Hospital Universitario La Fe	Valencia		Spain	46026
64	Hospital Miguel Servet	Zaragoza		Spain	50009
65	Complejo Hospitalario de Ávila	Ávila		Spain	05004