Clinical trial NCT03190525: Clinical Decision-making, Prognosis, Quality of Life and Satisfaction in Relapsed/Refractory Multiple Myeloma
Official title: CLinical Decision-making, Prognosis, quAlity of Life and Satisfaction With caRe in patIents With Relapsed/refracTory Multiple mYeloma (CLARITY)
Study type: OBSERVATIONAL
Overall status: ACTIVE_NOT_RECRUITING
Conditions: Multiple Myeloma
Lead sponsor: Gruppo Italiano Malattie EMatologiche dell'Adulto
Sponsor class: OTHER
First posted: 2017-06-16
Enrollment: 530
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Observational model: CASE_ONLY
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Considering the observational nature of study and fact that we aim to provide data on RRMM patients from the real-world setting, we have set up very broad inclusion criteria. Patients enrolled in clinical trials are still eligible for this protocol. Possible participation in a clinical trial at the time of entry in this study will be recorded and used for sensitivity analysis.

Brief summary: The primary objective is overall survival (OS) as predicted by baseline self-reported EORTC QLQ-C30 (European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire-Core 30) fatigue scale ratings, independently from other prognostic factors for OS in multiple myeloma (MM), including the clinically-based prognostic frailty score.

Detailed description: While quality of life (QoL) and other types of patient-reported Outcomes (PROs) can be crucial in the management of RRMM patients, a perusal of the literature indicates a dearth of information in this area. PRO is defined by the US Food and Drug Administration (FDA) as "a measurement based on a report that comes directly from the patient (i.e., study subject) about the status of a patient's health condition without amendment or interpretation of the patient's response by a clinician or anyone else. A PRO can be measured by self-report or by interview provided that the interviewer records only the patient's response. Until now the few studies that have included PRO data have been conducted in newly diagnosed MM patients enrolled in randomized controlled trials (RCTs).

For the purpose of this protocol, we have conducted a systematic review of PRO studies conducted in RRMM patients receiving treatments with either Imunomodulatory agents (IMiDs) or Proteasome inhibitors (PIs). The search was conducted for studies published from January, 1990 to July, 2015 and yielded only eight studies published within this timeframe. Three studies dealt with bortezomib based regimens, two with thalidomide, and the remaining studies with carfilzomib, pomalidomide, and lenalidomide. In all these studies PROs were considered as secondary outcomes and in four this was analyzed in the context of RCTs.

Despite enhanced disease control, none of the current novel agents, either IMiDs or PIs are free of significant toxicities, which frequently persist after completing treatment and continue to impair patient's daily functioning over the long-term period. Also, it is important to consider the poor prognosis for many of these patients. For example, patients who fail first-line PIs or IMiDs have been shown to report an average life expectancy of 9 months from the time of becoming refractory to PIs and IMiDs. Maintaining a "good" or "acceptable" level of QoL and lowering symptom burden over the longest possible period of time is a main goal of treatment for RRMM patients. Therefore, considering the paucity of QoL evidence-base data, CLARITY is designed to expand on some key understudied areas in this population that are broadly summarized in the next two paragraphs.

Eligibility criteria: Inclusion Criteria:

* MM patients who have received at least 1 prior line of therapy and are considered as RRMM according to IMWG criteria.
* Adult patients (≥ 18 years old).
* Written informed consent provided.
* Patients who have been enrolled onto other study therapy protocols are also eligible.
* Having a full baseline PRO evaluation completed.
* All data a available to calculate the frailty score.

Exclusion Criteria:

* Having any kind of psychiatric disorder or major cognitive dysfunction hampering the provision of informed consent.
* Having reported any grade ≥3 adverse event within two weeks prior to study entry.
* Having received more than 5 lines of therapies.

Primary outcomes:
measure: The primary objective is overall survival (OS) as predicted by baseline self-reported EORTC QLQ-C30 fatigue scale ratings, independently from other prognostic factors for OS in MM, including the clinically-based prognostic frailty score.
timeFrame: 30 months from study entry
Secondary outcomes:
measure: To devise a patient-centered frailty score for RRMM patients.
timeFrame: 30 months from study entry
measure: To investigate the prognostic value of the frailty score in the setting of RRMM.
timeFrame: 30 months from study entry
measure: To investigate QoL over time (outcome measures: EORTC QLQ-C30 and QLQ-MY20) by type of treatment and examine factors that contribute the most in maintaining baseline QoL levels.
timeFrame: 30 months from study entry
measure: To investigate relationship between satisfaction with information provision (outcome measure: EORTC INFO-25) and QoL outcomes (outcome measures: EORTC QLQ-C30 and QLQ-MY20).
timeFrame: 30 months from study entry
measure: To assess patients' preferences for involvement in treatment decision-making and the relationships between preferences and patient characteristics.
timeFrame: 30 months from study entry
measure: To assess and compare the baseline self-reported EORTC QLQ-C30 fatigue scale ratings, between those RRMM patients who had received only one line of treatment (1 line) versus more than one line of treatment (>1 line) at study entry
timeFrame: 30 months from study entry
Other outcomes:
Locations:
city: Ancona
country: Italy
facility: Azienda Ospedaliero - Universitaria Ospedali Riuniti Umberto I - G.M. LANCISI - G. SALESI
city: Bologna
country: Italy
facility: S. Orsola Malpighi
city: Brindisi
country: Italy
facility: Divisione di Ematologia Ospedale A. Perrino
city: Cagliari
country: Italy
facility: ASL N.8 - Ospedale "A. Businco" - Struttura Complessa di Ematologia e CTMO
city: Catania
country: Italy
facility: Unità di Onco-Ematologia - Azienda Ospedaliera - Garibaldi
city: Catania
country: Italy
facility: Università di Catania - Cattedra di Ematologia - Ospedale "Ferrarotto"
city: Catanzaro
country: Italy
facility: Unità Operativa Oncologia Medica - A.O. Pugliese Ciaccio
city: Cona
country: Italy
facility: Azienda Ospedaliero Universitaria Arcispedale Sant'Anna Dipartimento di Scienze Mediche Sezione di Ematologia e Fisiopatologia dell'Emostasi
city: Cosenza
country: Italy
facility: U.O. Ematologia - P.O. Annunziata - A.O. di Cosenza
city: Firenze
country: Italy
facility: Unità di Ricerca e di Malattie del sangue - Ematologia San Luca Vecchio Pad. 16 - 1° Piano
city: Lecce
country: Italy
facility: ASL Le/1 P.O. Vito Fazzi - U.O. di Ematologia ed UTIE
city: Messina
country: Italy
facility: Azienda Ospedaliera Universitaria - Policlinico G. Martino Dipartimento di Medicina Interna - U.O. Messina
city: Messina
country: Italy
facility: U.O. Ematologia - P.O. Annunziata - A.O. di Cosenza
city: Modena
country: Italy
facility: UO Ematologia - AOU Policlinico di Modena
city: Novara
country: Italy
facility: S.C.D.U. Ematologia - DIMECS e Dipartimento Oncologico - Università del Piemonte Orientale Amedeo Avogadro
city: Pagani
country: Italy
facility: U.O. di Oncoematologia -plesso ospedaliero "A. Tortora" di Pagani
city: Palermo
country: Italy
facility: U.O. di Ematologia con trapianto - Centro di Riferimento Regionale per le coagulopatie rare nel bambino e nell'adulto Dipart. Biomedico di Medicina Interna - A.U. Policlinico "Paolo Giaccone"
city: Parma
country: Italy
facility: Day Hospital dell'U.O.C di Ematologia e CTMO Padiglione 1 TORRE DELLE MEDICINE, 6° piano
city: Ragusa
country: Italy
facility: UOS di Ematologia Servizio di Immunoematologia e medicina Trasfusionale Azienda Sanitaria Provinciale 7
city: Reggio Emilia
country: Italy
facility: Unità Operativa Complessa di Ematologia - Arcispedale S. Maria Nuova
city: Rimini
country: Italy
facility: Ospedale "Infermi"
city: Roma
country: Italy
facility: Asl Roma 2, Ospedale S. Eugenio- Ospedale S.Eugenio - Uoc Ematologia
city: Roma
country: Italy
facility: Az. Ospedaliera "Sant' Andrea"-Università la Sapienza Seconda Facoltà di Medicina e Chirurgia
city: Roma
country: Italy
facility: Divisione Ematologia - Università Campus Bio-Medico
city: Roma
country: Italy
facility: Università degli Studi "Sapienza" - Dip Biotecnologie Cellulari ed Ematologia - Divisione di Ematologia
city: Roma
country: Italy
facility: Università degli Studi - Policlinico di Tor Vergata
city: Rossano
country: Italy
facility: Unità Operativa di Oncologia - Presidio Ospedaliero N. Giannetasio - Azienda ASL 3
city: San Giovanni Rotondo
country: Italy
facility: Istituto di Ematologia - IRCCS Ospedale Casa Sollievo della Sofferenza
city: Sassari
country: Italy
facility: Ematologia - Dipartimento di Medicina Clinica e Sperimentale
city: Siena
country: Italy
facility: U.O.C. Ematologia - A.O. Senese - Policlinico " Le Scotte"
city: Taranto
country: Italy
facility: U.O.C. di Ematolgia - A.O. " SS Annunziata" - P.O. S.G. Moscati
city: Terni
country: Italy
facility: A.O. Santa Maria - Terni S.C Oncoematologia
city: Torino
country: Italy
facility: Divisione di Ematologia dell' Università degli Studi di Torino - "Città della Salute e della Scienza di Torino"
city: Trieste
country: Italy
facility: Struttura Complessa II Medicina - Ematologia - Centro di Riferimento Ematologico - Ospedale Maggiore
city: London
country: United Kingdom
facility: London North West Healthcare Trust
Study officials:
affiliation: Roma Sapienza
name: Maria Teresa Petrucci, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: GIMEMA Foundation
name: Fabio Efficace
role: STUDY_CHAIR
References:
citation: Efficace F, Boccadoro M, Palumbo A, Petrucci MT, Cottone F, Cannella L, Zamagni E, Niscola P, Kyriakou C, Caravita T, Offidani M, Mandelli F, Cavo M. A prospective observational study to assess clinical decision-making, prognosis, quality of life and satisfaction with care in patients with relapsed/refractory multiple myeloma: the CLARITY study protocol. Health Qual Life Outcomes. 2018 Jun 18;16(1):127. doi: 10.1186/s12955-018-0953-4.