AL AmyloidosisNCT04937777
A Study on the Management and Outcome of Patients With Systemic AL Amyloidosis in Europe
An observational study of AL Amyloidosis, run by European Myeloma Network B.V. at 13 sites in 10 countries.
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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- AL Amyloidosis
- How many people
- 4,481 participants
- Where
- 13 sitesin 10 countries
- Study dates
- 23 Apr 2019to 30 Apr 2021
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
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.
Who may be able to join
- 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.
Who cannot join
- Patients under the age of 18 will not be considered eligible for this study
Trial locations and contacts
13 sites in 10 countries.
13 sites in 10 countries
France2 sites
Centre Hospitalier Universitaire de Limoges, Department of Clinical Hematology and Cell Therapy
Limoges
Service de Néphrologie, Hémodialyse Et Transplantation Rénale, Hôpital Jean Bernard, Chu Poitiers
Poitiers
Netherlands2 sites
University Medical Center Groningen
Groningen
University Medical Center Utrecht, Umc Utrecht, Department of Hematology
Utrecht
Portugal2 sites
Fundação Champalimaud, Hematology Research, Imunology - Medical School, Nova University
Lisbon
Centro Hospitalar E Universitário São João
Porto
Austria1 site
Allgemeines Krankenhaus Der Stadt Wien (General Hopsital), Universitätsklinik Für Innere Medizin I Klinische Abteilung Für Onkologie (Clinic of Internal Medicine I), Medizinische Universität Wien
Vienna
Germany1 site
Universität Heidelberg, Department of Internal Medicine V: Hematology, Oncology and Rheumatology
Heidelberg
Greece1 site
Alexandra Hospital, University of Athens School of Medicine, Department of Clinical Therapeutics
Athens
Italy1 site
Centro Per Lo Studio E La Cura Delle Amiloidosi Sistemiche Padiglione Forlanini
Pavia
United Kingdom1 site
University College London Medical School, Uk Royal Free Hospital
London
About this study
Official title: A Retrospective Observational Multicenter Study on the Management and Outcome of Patients With Systemic AL Amyloidosis in Europe
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.
Read the 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.
- Conditions
- AL Amyloidosis
- Medical subject headings
- Immunoglobulin Light-chain Amyloidosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Group pre-2010 | Patients who initiated first-line treatment between 2004 and 2010 (pre-2010). | |
| Group post-2010 | Patients who initiated first-line treatment between 2011 and 2018 (post-2010). |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 2004-2018
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.
Measured: 2004-2018
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.
Measured: 2004-2018
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.
Measured: 2004-2018
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.
Measured: 2004-2018
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.
Measured: 2004-2018
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.
Measured: 2004-2018
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.
Measured: 2004-2018
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Jun 2021
- Study start23 Apr 2019
- Primary completion30 Apr 2021
- Study completion30 Apr 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for AL AmyloidosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Retrospective
- Enrolment
- 4,481 participants (actual)
Sponsor and investigators
- Lead sponsor
- European Myeloma Network B.V.
- Sponsor type
- Research network
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04937777
- Sponsor's protocol ID
- EMN23
- Status last verified
- Sep 2022
- First submitted
- 6 Apr 2021
- Last updated
- 27 Sep 2022
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 27 Sep 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Retrospective Observational Multicenter Study on the Management and Outcome of Patients With Systemic AL Amyloidosis in Europe
- ClinicalTrials.gov ID
- NCT04937777
- Acronym / sponsor ID
- EMN23
- Conditions
- AL Amyloidosis
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 4,481 participants (actual)
- Lead sponsor
- European Myeloma Network B.V.Research network
- Study start
- 23 Apr 2019 (actual)
- Primary completion
- 30 Apr 2021 (actual)
- Study completion
- 30 Apr 2021 (actual)
- First posted
- 24 Jun 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Group pre-2010 | – | –Patients who initiated first-line treatment between 2004 and 2010 (pre-2010). |
| Group post-2010 | – | –Patients who initiated first-line treatment between 2011 and 2018 (post-2010). |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- 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.
Key inclusion criteria (5 listed)
- 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.
Key exclusion criteria (1 listed)
- Patients under the age of 18 will not be considered eligible for this study
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Patient and disease characteristics
Time frame: 2004-2018
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.
Treatment patterns
Time frame: 2004-2018
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.
Response evaluation and efficacy outcomes
Time frame: 2004-2018
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.
Healthcare resource utilization: hospitalizations
Time frame: 2004-2018
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.
Healthcare resource utilization: concomitant therapy
Time frame: 2004-2018
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.
Healthcare resource utilization: imaging and cardiac assessments
Time frame: 2004-2018
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.
Healthcare resource utilization: dialysis
Time frame: 2004-2018
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.
Safety management
Time frame: 2004-2018
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.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Sep 2022.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
13 sites in 10 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of AL Amyloidosis, run by European Myeloma Network B.V. at 13 sites in 10 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 27 Sep 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 13 sites in France, Netherlands, Portugal, Austria, Czechia and 5 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Patient and disease characteristics (time frame: 2004-2018). The study lists 8 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 27 Sep 2022.