A Study on the Management and Outcome of Patients With Systemic AL Amyloidosis in Europe

Stichting European Myeloma Network

Status

Completed

Conditions

AL Amyloidosis

Study type

Observational

Funder types

NETWORK

Identifiers

NCT04937777

EMN23

Details and patient eligibility

About

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.

Full 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.

Enrollment

4,481 patients

Sex

All

Ages

18+ years old

Volunteers

No Healthy Volunteers

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