Clinical trial NCT04937777: A Study on the Management and Outcome of Patients With Systemic AL Amyloidosis in Europe
Official title: A Retrospective Observational Multicenter Study on the Management and Outcome of Patients With Systemic AL Amyloidosis in Europe
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: AL Amyloidosis
Lead sponsor: European Myeloma Network B.V.
Sponsor class: NETWORK
First posted: 2021-06-24
Enrollment: 4481
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: RETROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: The study will include adult (male and female) patients diagnosed with systemic AL-AMY and symptomatic organ involvement who have initiated first line treatment in the period 2004-2018.

Patients who have received treatment for AL-AMY as part of an interventional Clinical Trial will be identified but will not be included in the analysis due to the controlled environment of a clinical trial (not RWE data).

Patients with comorbid multiple myeloma will not be excluded.

Brief summary: This is a retrospective, observational, multicenter study to collect Real-World Evidence (RWE) data on systemic AL-AMY patients in Europe. Data from paper/electronic medical records and/or electronic databases from key reference centers in Europe will be used. Data will either be entered by the site staff in the electronic Case Report Form (eCRF) or, where feasible, transferred directly, always in accordance to local regulations.

Detailed description: The lack of regulatory approved treatment options for AL amyloidosis (AL-AMY) justifies the need to understand the current treatment practice and outcomes of this disease. There is a need for objective Real-World Evidence (RWE) that reflects how treatments are initiated, combined and sequenced, and how their relative effectiveness and safety profiles emerge outside a clinical trial setting. Therefore, the aim is to generate RWE on systemic AL-AMY patients in Europe, including patient characteristics, resource use, treatments and associated patient outcomes.

Eligibility criteria: Inclusion Criteria:

* Age\>18 years
* Confirmed diagnosis of AL-AMY and symptomatic organ involvement.
* Initiated first line treatment for AL-AMY in the period 2004-2018.
* Patients who have signed a participation agreement/ICF allowing data collection and source data verification in accordance with local requirements.

The inclusion of deceased subjects in the study is permitted under the condition that consent waiver has been granted by the Scientific Committee and/or Administrative Board of the participating sites and/or any applicable regulatory body, as per local regulations, to either implement a hospital informed consent form (ICF) already in place or provide written approval of this study-specific waiver. In the occasion that waiver of consent is not granted deceased subjects will not be enrolled in the study.

Exclusion Criteria:

* Patients under the age of 18 will not be considered eligible for this study

Primary outcomes:
measure: Patient and disease characteristics
timeFrame: 2004-2018
measure: Treatment patterns
timeFrame: 2004-2018
measure: Response evaluation and efficacy outcomes
timeFrame: 2004-2018
measure: Healthcare resource utilization: hospitalizations
timeFrame: 2004-2018
measure: Healthcare resource utilization: concomitant therapy
timeFrame: 2004-2018
measure: Healthcare resource utilization: imaging and cardiac assessments
timeFrame: 2004-2018
measure: Healthcare resource utilization: dialysis
timeFrame: 2004-2018
measure: Safety management
timeFrame: 2004-2018
Secondary outcomes:
Other outcomes:
Locations:
city: Wien
country: Austria
facility: Allgemeines Krankenhaus Der Stadt Wien (General Hopsital), Universitätsklinik Für Innere Medizin I Klinische Abteilung Für Onkologie (Clinic of Internal Medicine I), Medizinische Universität Wien
city: Ostrava
country: Czechia
facility: University Hospital Ostrava, Clinic of Hematooncology
city: Limoges
country: France
facility: Centre Hospitalier Universitaire de Limoges, Department of Clinical Hematology and Cell Therapy
city: Poitiers
country: France
facility: Service de Néphrologie, Hémodialyse Et Transplantation Rénale, Hôpital Jean Bernard, Chu Poitiers
city: Heidelberg
country: Germany
facility: Universität Heidelberg, Department of Internal Medicine V: Hematology, Oncology and Rheumatology
city: Athens
country: Greece
facility: Alexandra Hospital, University of Athens School of Medicine, Department of Clinical Therapeutics
city: Pavia
country: Italy
facility: Centro Per Lo Studio E La Cura Delle Amiloidosi Sistemiche Padiglione Forlanini
city: Groningen
country: Netherlands
facility: University Medical Center Groningen
city: Utrecht
country: Netherlands
facility: University Medical Center Utrecht, Umc Utrecht, Department of Hematology
city: Lisboa
country: Portugal
facility: Fundação Champalimaud, Hematology Research, Imunology - Medical School, Nova University
city: Porto
country: Portugal
facility: Centro Hospitalar E Universitário São João
city: Barcelona
country: Spain
facility: Servicio de Hematología, Hospital Clínic de Barcelona
city: London
country: United Kingdom
facility: University College London Medical School, Uk Royal Free Hospital
Study officials:
References: