Clinical trial NCT03091127: Real-world Use of Carfilzomib Among Multiple Myeloma Patients in Europe
Official title: Real-world Use of Carfilzomib Among Multiple Myeloma Patients in Europe Who Have Received at Least One Prior Therapy.
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Multiple Myeloma
Lead sponsor: Amgen
Sponsor class: INDUSTRY
First posted: 2017-03-27
Enrollment: 705
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: OTHER
Sampling method: PROBABILITY_SAMPLE
Study population: Patients with Multiple Myeloma

Brief summary: With the recent addition of carfilzomib as a treatment option for multiple myeloma, no data is available yet on how the drug is being used outside of the clinical trial setting.

This study will therefore provide essential data to demonstrate the real world utilization of carfilzomib in routine clinical practice, including dosage, administration schedule, regimen, duration of treatment and reason for discontinuation in Europe.

Detailed description: With the recent addition of carfilzomib as a treatment option for multiple myeloma, no data is available yet on how the drug is being used outside of the clinical trial setting.

The Primary Objective is to describe carfilzomib utilisation in routine clinical practice, including dosage, administration schedule, regimen, duration of treatment and reason for discontinuation.

* Secondary Objectives:
* Describe the population treated with carfilzomib in terms of demographics, multiple myeloma (MM) disease characteristics, treatment history, and comorbidities.
* Describe the safety profile of carfilzomib in routine clinical practice.
* Describe response to treatment as assessed by the physician and recorded in the medical file.
* Describe healthcare resource utilisation of subjects treated with carfilzomib, in terms of unplanned hospitalisations.
* Describe the reasons for choosing carfilzomib as the MM treatment of choice.
* Describe specific concomitant therapy (bisphosphonates, thromboprophylaxis, antihypertensive treatment, anti-infective treatment) and whether these therapies were used as prophylaxis or as treatment.
* Describe a cardiovascular assessment at carfilzomib regimen initiation and at occurrence of cardiac adverse events, where available per routine care (electrocardiogram \[ECG\], echocardiography, left ventricular ejection fraction).

Eligibility criteria: Inclusion Criteria:

* Age 18 years or older at the time of carfilzomib initiation
* At least one prior line of MM treatment has been received
* Carfilzomib treatment has been initiated per routine practice and is currently ongoing
* At least one administration of carfilzomib in a combination regimen (ie, not monotherapy) has been received
* Provided written informed consent prior to abstraction of any data, in countries where written informed consent is required.
* Subjects who previously completed treatment with carfilzomib in a clinical trial, a compassionate use program or through routine practice, are eligible to take part in the study.
* Subjects who receive radiotherapy concurrently with carfilzomib treatment are also eligible to take part in the study.
* Subjects who initiate carfilzomib treatment on a combination regimen, subsequently discontinue all concomitant medications but remain on carfilzomib monotherapy in later cycles, remain eligible for participation in the study.
* Subjects who are also enrolled in other observational studies in which standard of care is not altered are eligible to take part in the study,

Exclusion Criteria:

* Subjects who are enrolled in a carfilzomib clinical trial will not be eligible to additionally take part in this observational study.
* Subjects who are receiving carfilzomib treatment within a compassionate use program will not be eligible to take part in this observational study. If a subject who has enrolled into this observational study, also enrolls in a clinical trial in which MM treatment and/or disease management is protocol-specified, the subject becomes ineligible and the subject's data will be censored from the time the subject enrolled the clinical trial.

Primary outcomes:
measure: Carfilzomib starting dose
timeFrame: 18 months
measure: Carfilzomib dose
timeFrame: 18 months
measure: Carfilzomib dose modification
timeFrame: 18 months
measure: Time to carfilzomib dose modification
timeFrame: 18 months
measure: Reason for dose modification
timeFrame: 18 months
measure: Number of cycles started
timeFrame: 18 months
measure: Carfilzomib regimen
timeFrame: 18 months
measure: Carfilzomib dosing frequency
timeFrame: 18 months
measure: Carfilzomib dosing schedule
timeFrame: 18 months
measure: Carfilzomib duration of treatment
timeFrame: 18 months
measure: Starting dose of concomitant anti-myeloma agents
timeFrame: 18 months
measure: Dose modification for concomitant anti-myeloma agents
timeFrame: 18 months
measure: Reason for frequency modification
timeFrame: 18 months
measure: Reason for change in frequency of concomitant multiple myeloma therapies
timeFrame: 18 months
Secondary outcomes:
measure: International Staging System (ISS) score and revised ISS stage at diagnosis and carfilzomib regimen initation
timeFrame: 18 months
measure: Eastern Cooperative Oncology Group (ECOG) performance status
timeFrame: 18 months
measure: Cytogenetic risk profile at diagnosis
timeFrame: 18 months
measure: Presence of CRAB features (i.e. hypercalcemia, renal insufficiency, anemia and/or bone pain)
timeFrame: 18 months
measure: Presence of comorbidities
timeFrame: 18 months
measure: Previously received anti-myeloma treatment
timeFrame: 18 months
measure: Response to prior treatment
timeFrame: 18 months
measure: Number of prior relapses
timeFrame: 18 months
measure: Adverse event
timeFrame: 18 months
measure: Time to adverse event
timeFrame: 18 months
measure: Electrocardiogram (ECG) changes
timeFrame: 18 months
measure: Decrease in left ventricular ejection fraction (LVEF)
timeFrame: 18 months
measure: Initiation or dose increase of antihypertensive treatment
timeFrame: 18 months
measure: Initiation or dose increase of existing heart failure treatment
timeFrame: 18 months
measure: Response to carfilzomib treatment
timeFrame: 18 months
measure: Type of relapse
timeFrame: 18 months
measure: Number of unplanned hospitalisations
timeFrame: 18 months
measure: Concomitant therapy not part of the carfilzomib regimen
timeFrame: 18 months
measure: Planned subsequent treatment regimen
timeFrame: 18 months
measure: Patient age
timeFrame: 18 months
measure: Patient sex
timeFrame: 18 months
measure: Patient height
timeFrame: 18 months
measure: Patient weight
timeFrame: 18 months
measure: MRI (magnetic resonance imaging) performed at MM diagnosis and carfilzomib regimen initiation.
timeFrame: 18 months
measure: PET-CT (positron emission tomography-computed tomography) performed at MM diagnosis and carfilzomib regimen initiation.
timeFrame: 18 months
measure: Measurement of Serum M component at MM diagnosis and carfilzomib regimen initiation.
timeFrame: 18 months
measure: Measurement of Urine M component at MM diagnosis and carfilzomib regimen initiation.
timeFrame: 18 months
measure: Measurement of serum albumin at MM diagnosis and carfilzomib regimen initiation.
timeFrame: 18 months
measure: Measurement of serum beta-2-microglobulin at MM diagnosis and carfilzomib regimen initiation.
timeFrame: 18 months
measure: Measurement of percent of plasma cells in bone marrow at MM diagnosis and carfilzomib regimen initiation.
timeFrame: 18 months
measure: Baseline measurement of lactate dehydrogenase at MM diagnosis and carfilzomib regimen initiation.
timeFrame: 18 months
measure: ECG (electrocardiogram)
timeFrame: 18 months
measure: Echocardiogram
timeFrame: 18 months
measure: LVEF (left ventricular ejection fraction) assessment
timeFrame: 18 months
measure: Computed Tomography (CT) performed at MM diagnosis and carfilzomib regiment initiation.
timeFrame: 18 Months
measure: Myeloma/Osteolytic lesions detected by MRI, PET-CT, and X-ray at MM diagnosis and carfilzomib regimen initiation
timeFrame: 18 months
Other outcomes:
Locations:
city: Braunau
country: Austria
facility: Krankenhaus Sankt Josef Braunau
city: Innsbruck
country: Austria
facility: Medizinische Universitaet Innsbruck
city: Leoben
country: Austria
facility: Landeskrankenhaus Hochsteiermark
city: Linz
country: Austria
facility: Ordensklinikum Linz Elisabethinen
city: Rankweil
country: Austria
facility: Landeskrankenhaus Rankweil
city: Salzburg
country: Austria
facility: Landeskrankenhaus Salzburg
city: Schwarzach im Pongau
country: Austria
facility: Kardinal Schwarzenbergsches Krankenhaus
city: Steyr
country: Austria
facility: Landeskrankenhaus Steyr
city: Waidhofen an der Ybbs
country: Austria
facility: Landesklinikum Waidhofen an der Ybbs
city: Wels
country: Austria
facility: Klinikum Wels - Grieskirchen GmbH
city: Wiener Neustadt
country: Austria
facility: Krankenhaus Wiener Neustadt
city: Wien
country: Austria
facility: Universitaetsklinikum Allgemeines Krankenhaus Wien
city: Bonheiden
country: Belgium
facility: Imelda Ziekenhuis Vzw
city: Brussels
country: Belgium
facility: Hopital Delta
city: Edegem
country: Belgium
facility: Universitair Ziekenhuis Antwerpen
city: Geel
country: Belgium
facility: Algemeen Ziekenhuis Sint-Dimpna
city: Gent
country: Belgium
facility: Algemeen Ziekenhuis Sint Lucas
city: Haine Saint Paul - La Louviere
country: Belgium
facility: Centres Hospitaliers Jolimont - Hopital de Jolimont
city: Ieper
country: Belgium
facility: Jan Yperman Ziekenhuis
city: Liege
country: Belgium
facility: Centre Hospitalier Regional de la Citadelle
city: Liege
country: Belgium
facility: Centre Hospitalier Universitaire de Liege - Sart Tilman
city: Sint-Niklaas
country: Belgium
facility: Algemeen Ziekenhuis Nikolaas Campus Sint-Niklaas
city: Tournai
country: Belgium
facility: Centre Hospitalier Wallonie Picarde - site imc
city: Verviers
country: Belgium
facility: Centre Hospitalier Regional Verviers
city: Sofia
country: Bulgaria
facility: University Multiprofile Hospital for Active Treatment Alexandrovska
city: Sofia
country: Bulgaria
facility: University Multiprofile Hospital for Active Treatment Sveti Ivan Rilski EAD
city: Sofia
country: Bulgaria
facility: Military Medical Academy Multiprofile Hospital for Active Treatment - Sofia
city: Sofia
country: Bulgaria
facility: Specialized Hospital for Active Treatment of Hematology Diseases EAD
city: Hradec Kralove
country: Czechia
facility: Fakultni nemocnice Hradec Kralove
city: Olomouc
country: Czechia
facility: Fakultni nemocnice Olomouc
city: Plzen
country: Czechia
facility: Fakultni nemocnice Plzen
city: Praha 2
country: Czechia
facility: Vseobecna fakultni nemocnice v Praze
city: Amiens Cedex 1
country: France
facility: Centre Hospitalier Rene Dubos
city: Avignon Cedex 9
country: France
facility: Centre Hospitalier Henri Duffaut
city: Bayonne
country: France
facility: Centre Hospitalier de la Cote Basque
city: Creteil
country: France
facility: Hopital Henri Mondor
city: Grenoble Cedex 9
country: France
facility: Centre Hospitalier Universitaire de Grenoble
city: Limoges Cedex
country: France
facility: Centre Hospitalier Regional Universitaire de Limoges - Hopital Dupuytren
city: Montpellier cedex 5
country: France
facility: Centre Hospitalier Universitaire de Montpellier - Hopital Saint Eloi
city: Nimes cedex 09
country: France
facility: Groupe Hospitalo Universitaire de Nimes - Hopital Caremeau
city: Paris
country: France
facility: Hopital Pitie-Salpetriere
city: Paris
country: France
facility: HÃ´pital Cochin
city: Paris
country: France
facility: Groupe Hospitalier Necker - Enfants Malades
city: Poitiers Cedex
country: France
facility: Centre Hospitalier Universitaire de Poitiers - Hopital la Miletrie
city: Reims Cedex
country: France
facility: Centre Hospitalier Universitaire de Reims - Hopital Robert Debre
city: Saint Quentin
country: France
facility: Centre Hospitalier de Saint Quentin
city: Strasbourg
country: France
facility: Clinique Sainte Anne
city: Alexandroupoli
country: Greece
facility: University General Hospital of Evros-Alexandroupolis District
city: Athens
country: Greece
facility: General Hospital Evangelismos
city: Athens
country: Greece
facility: Errikos Dunant Hospital Center
city: Athens
country: Greece
facility: General Hospital of Athens Georgios Gennimatas
city: Athens
country: Greece
facility: General Hospital of Athens Laiko
city: Athens
country: Greece
facility: Laikon University Hospital
city: Athens
country: Greece
facility: University of Athens School of Medicine Alexandra Hospital
city: Athens
country: Greece
facility: University General Hospital Attikon
city: Athens
country: Greece
facility: Metropolitan General
city: Athens
country: Greece
facility: Metropolitan Hospital
city: Heraklion
country: Greece
facility: University Hospital of Heraklion
city: Larissa
country: Greece
facility: University Hospital of Larissa
city: Patra
country: Greece
facility: General Hospital of Patras Agios Andreas
city: Patra
country: Greece
facility: General University Hospital of Patras Panagia i Voithia
city: Piraeus
country: Greece
facility: Special Anticancer Hospital of Piraeus Metaxa
city: Thessaloniki
country: Greece
facility: Theagenion Cancer Hospital of Thessaloniki
city: Thessaloniki
country: Greece
facility: General Hospital of Thessaloniki Georgios Papanikolaou
city: Petah Tiqva
country: Israel
facility: Rabin Medical Center - Beilinson Hospital
city: Tel Aviv
country: Israel
facility: Tel Aviv Sourasky Medical Center
city: Alessandria
country: Italy
facility: Azienda Ospedaliera Santi Antonio e Biagio e Cesare Arrigo
city: Bagno A Ripoli (FI)
country: Italy
facility: Azienda Unita Sanitaria Locale Toscana Centro
city: Barletta
country: Italy
facility: Ospedale Monsignor Raffaele Dimiccoli
city: Benevento
country: Italy
facility: Azienda Ospedaliera G Rummo
city: Bergamo
country: Italy
facility: Azienda Socio Sanitaria Territoriale Papa Giovanni XXIII
city: Brindisi
country: Italy
facility: Presidio Ospedaliero Di Summa Perrino
city: Cagliari
country: Italy
facility: Azienda Ospedaliera Brotzu Presidio Ospedaliero A Businco Centro di Riferimento Oncologico Regionale
city: Cuneo
country: Italy
facility: Azienda Ospedaliera Santa Croce e Carle
city: Lecco
country: Italy
facility: Azienda Ospedaliera di Alessandro Manzoni Lecco
city: Livorno
country: Italy
facility: Spedali Riuniti di Livorno
city: Messina
country: Italy
facility: Azienda Ospedaliera Papardo
city: Milano
country: Italy
facility: IRCCS Istituto Europeo di Oncologia
city: Padova
country: Italy
facility: Azienda Ospedaliera di Padova
city: Pagani (SA)
country: Italy
facility: Presidio Ospedaliero Andrea Tortora
city: Palermo
country: Italy
facility: Azienda Ospedaliera Ospedali Riuniti Villa Sofia Cervello
city: Perugia
country: Italy
facility: Azienda Ospedaliera di Perugia Ospedale Santa Maria della Misericordia
city: Pescara
country: Italy
facility: Ospedale Civile Spirito Santo
city: Potenza
country: Italy
facility: Azienda Ospedaliera San Carlo
city: Reggio Calabria
country: Italy
facility: Grande Ospedale Metropolitano Bianchi Melacrino Morelli
city: Reggio Emilia
country: Italy
facility: Azienda Unita Sanitaria Locale Istituto di Ricovero di Reggio Emilia Arcispedale Santa Maria Nuova
city: Roma
country: Italy
facility: Fondazione Policlinico Tor Vergata
city: Roma
country: Italy
facility: Ospedale Sant Eugenio
city: Roma
country: Italy
facility: Azienda Ospedaliera Policlinico Umberto I
city: Roma
country: Italy
facility: Azienda Ospedaliera Sant Andrea
city: Salerno
country: Italy
facility: Azienda Ospedaliera Universitaria Ospedale San Giovanni di Dio e Ruggi D Aragona
city: Taranto
country: Italy
facility: Presidio Ospedaliero San Giuseppe Moscati
city: Torino
country: Italy
facility: Azienda Ospedaliera Citta della Salute e della Scienza di Torino Ospedale Molinette
city: Udine
country: Italy
facility: Azienda Ospedaliero Universitaria Integrata di Udine
city: Verona
country: Italy
facility: Azienda Ospedaliera Universitaria Integrata di Verona Ospedale G B Rossi Borgo Roma
city: Beverwijk
country: Netherlands
facility: Rode Kruis Ziekenhuis
city: Breda
country: Netherlands
facility: Amphia Ziekenhuis, locatie Langendijk
city: Den Haag
country: Netherlands
facility: HagaZiekenhuis, locatie Leyweg
city: Nieuwegein
country: Netherlands
facility: Sint Antonius Ziekenhuis, locatie Nieuwegein
city: Schiedam
country: Netherlands
facility: Franciscus Vlietland
city: Venlo
country: Netherlands
facility: VieCuri Medisch Centrum
city: Oslo
country: Norway
facility: Oslo Universitetssykehus HF
city: Baia Mare
country: Romania
facility: Spitalul Judetean de Urgenta Dr Constantin Opris Baia Mare
city: Brasov
country: Romania
facility: Policlinica de Diagnostic Rapid
city: Bucharest
country: Romania
facility: Spitalul Clinic Colentina
city: Bucharest
country: Romania
facility: Fundeni Clinical Institute
city: Bucharest
country: Romania
facility: Coltea Clinical Hospital
city: Bucuresti
country: Romania
facility: Spitalul Universitar de Urgenta Militar Central "Dr. Carola Davila"
city: Bucuresti
country: Romania
facility: Fundeni Clinical Institute
city: Cluj-Napoca
country: Romania
facility: Profesor Dr Ion Chiricuta Institut of Oncology
city: Craiova
country: Romania
facility: Spitalul Clinic Municipal Filantropia Craiova
city: Iasi
country: Romania
facility: Iasi Regional Oncology Institute
city: Oradea
country: Romania
facility: Spitalul Clinic Dr Gavril Curteanu Oradea
city: Targu Mures
country: Romania
facility: Targu-Mures County Emergency Clinical Hospital
city: Timisoara
country: Romania
facility: SC Oncomed SRL
Study officials:
affiliation: Amgen
name: MD
role: STUDY_DIRECTOR
References:
citation: Leleu X, Katodritou E, Kuehr T, Terpos E, Caers J, Zambello R, Brescianini A, Liang T, Wetten S, Badelita SN. Real-world use of carfilzomib combined with lenalidomide and dexamethasone in patients with multiple myeloma in Europe and Israel. EJHaem. 2022 Nov 6;4(1):174-183. doi: 10.1002/jha2.595. eCollection 2023 Feb.
citation: Terpos E, Zambello R, Leleu X, Kuehr T, Badelita SN, Katodritou E, Brescianini A, Liang T, Wetten S, Caers J. Real-World Use and Effectiveness of Carfilzomib Plus Dexamethasone in Relapsed/Refractory Multiple Myeloma in Europe. Cancers (Basel). 2022 Oct 28;14(21):5311. doi: 10.3390/cancers14215311.