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

AL Amyloidosis Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04937777
• Other study ID #: EMN23
• Status: Completed
• Start date: April 23, 2019
• Est. completion date: April 30, 2021

Study information

• Verified date: September 2022
• Source: European Myeloma Network
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 4481
• Est. completion date: April 30, 2021
• Est. primary completion date: April 30, 2021
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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

Related Conditions & MeSH terms

• AL Amyloidosis
• Amyloidosis
• Immunoglobulin Light-chain Amyloidosis

Locations

Country	Name	City	State
Austria	Allgemeines Krankenhaus Der Stadt Wien (General Hopsital), Universitätsklinik Für Innere Medizin I Klinische Abteilung Für Onkologie (Clinic of Internal Medicine I), Medizinische Universität Wien	Wien
Czechia	University Hospital Ostrava, Clinic of Hematooncology	Ostrava
France	Centre Hospitalier Universitaire de Limoges, Department of Clinical Hematology and Cell Therapy	Limoges
France	Service de Néphrologie, Hémodialyse Et Transplantation Rénale, Hôpital Jean Bernard, Chu Poitiers	Poitiers
Germany	Universität Heidelberg, Department of Internal Medicine V: Hematology, Oncology and Rheumatology	Heidelberg
Greece	Alexandra Hospital, University of Athens School of Medicine, Department of Clinical Therapeutics	Athens
Italy	Centro Per Lo Studio E La Cura Delle Amiloidosi Sistemiche Padiglione Forlanini	Pavia
Netherlands	University Medical Center Groningen	Groningen
Netherlands	University Medical Center Utrecht, Umc Utrecht, Department of Hematology	Utrecht
Portugal	Fundação Champalimaud, Hematology Research, Imunology - Medical School, Nova University	Lisboa
Portugal	Centro Hospitalar E Universitário São João	Porto
Spain	Servicio de Hematología, Hospital Clínic de Barcelona	Barcelona
United Kingdom	University College London Medical School, Uk Royal Free Hospital	London

Sponsors (1)

Lead Sponsor	Collaborator
European Myeloma Network

Countries where clinical trial is conducted

Austria,  Czechia,  France,  Germany,  Greece,  Italy,  Netherlands,  Portugal,  Spain,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Patient and disease characteristics	To describe the proportion of patients belonging to different age groups, and the proportion of patients per gender, stage, organ involvement, performance status, and hematologic biomarkers at screening or at baseline; by first-line treatment initiation period (pre-2010 or post-2010) and overall.	2004-2018
Primary	Treatment patterns	To describe the proportion of patients who had an autologous stem cell transplantation by first-line initiation period (pre-2010 or post-2010) and overall; the proportion of regimen combinations used by line of treatment, and by first-line initiation period; the proportion of patients receiving specific regimen combinations following different first-line regimens, by first-line initiation period and overall.	2004-2018
Primary	Response evaluation and efficacy outcomes	To describe the treatment effectiveness in terms of hematologic response rates at 3, 6, 12, and 24 months after first-line initiation, by regimen and first-line initiation period (pre-2010 or post-2010); overall survival, progression-free survival, and time on treatment, overall, by first-line treatment and by first-line initiation period.	2004-2018
Primary	Healthcare resource utilization: hospitalizations	To describe the healthcare resource utilization patterns during the treatment of AL amyloidosis by first-line treatment initiation period (pre-2010 or post-2010) and overall, in terms of hospitalization information (number of hospitalizations, duration per hospitalization); use of concomitant treatment (frequency of regimens used, proportion of patients receiving concomitant treatment); proportion of patients undergoing imaging exams; proportion of patients undergoing cardiac exams; proportion of patients receiving dialysis.	2004-2018
Primary	Healthcare resource utilization: concomitant therapy	To describe the healthcare resource utilization patterns during the treatment of AL amyloidosis, by first-line treatment initiation period (pre-2010 or post-2010) and overall, in terms of the proportion of patients receiving concomitant therapy.	2004-2018
Primary	Healthcare resource utilization: imaging and cardiac assessments	To describe the healthcare resource utilization patterns during the treatment of AL amyloidosis, by first-line treatment initiation period (pre-2010 or post-2010) and overall, in terms of the proportion of patients undergoing imaging and cardiac exams.	2004-2018
Primary	Healthcare resource utilization: dialysis	To describe the healthcare resource utilization patterns during the treatment of AL amyloidosis, by first-line treatment initiation period (pre-2010 or post-2010) and overall, in terms of the proportion of patients receiving dialysis.	2004-2018
Primary	Safety management	To describe the proportion of patients having serious adverse events or adverse events of special interest, by first-line treatment initiation period (pre-2010 or post-2010) and overall.	2004-2018