AL Amyloidosis, Amyloidosis, Amyloidosis Cardiac
Conditions
Brief summary
The goal of this study is to collect and store human body material (HBM) of patients with amyloidosis in a biobank "BE.Amycon biobank" for future research and to collect clinical data of patients with amyloidosis in a database "BE.Amycon data registry".
Detailed description
With the support of VIB (Vlaams Instituut voor Biotechnologie) Grand Challenges Program, this project aims to establish the BElgian AMYloidosis CONsortium (BE.AMYCON) by joining forces of VIB researchers, a state-of-the-art diagnostic platform, and clinicians from various disciplines and different institutes. Such a consortium will address patient needs and improve outcomes on many levels. The aim is is to establish a GDPR (General Data Protection Regulation)-proof HBM biobank in the context of amyloidosis. HBM of patients with suspected or confirmed amyloidosis will be prospectively collected and stored for future scientific research. In addition to the HBM collection, a database with personal and health data of patients diagnosed with amyloidosis (any subtype) will be established in order to get better insights on the disease presentation, disease evolution pattern, treatment plans and responses and survival.
Interventions
None listed
Sponsors
Universitaire Ziekenhuizen KU Leuven
Vlaams Instituut voor Biotechnologie
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
Yes
Inclusion criteria
* Provide consent and sign informed consent form * Age 18 years or older * Diagnosis of amyloidosis (suspected or confirmed, any subtypes) * For the prospective sample collection only: newly diagnosed (any subtype) or at relapse (AL amyloidosis)
Exclusion criteria
* Not willing to sign informed consent * Not able to sign informed consent
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Establishment of a data registry: participant baseline demographics | Baseline | Demographic characteristics of amyloidosis participants will be assessed at baseline. |
| Establishment of a data registry: description of the disease characteristics of patients with amyloidosis at diagnosis | Baseline | Disease characteristics of amyloidosis (disease presentation and symptoms, type of organ involvment, results of diagnostic tests (lab values, imaging, biopsy)) will be collected at moment of diagnosis. |
| Establishment of a data registry: treatment in participants with amyloidosis | From enrollment of the patient until death, until loss to follow-up or withdrawal of informed consent, whichever comes first, up to 10 years | Description of treatment (type of treatment per line of treatment) of participants with amyloidosis within routine clinical care. |
| Establishment of a biobank with biological samples (blood, urine, tissue) from patients with amyloidosis | From enrollment of the patient until death, until loss to follow-up or withdrawal of informed consent, whichever comes first, up to 2 years | Biological samples (blood, urine, tissue) will be collected from patients with amyloidosis. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Best Response | From enrollment of the patient until death, until loss to follow-up or withdrawal of informed consent, whichever comes first, up to 10 years | Documentation of response rates per line of treatment |
| Duration of response | From enrollment of the patient until death, until loss to follow-up or withdrawal of informed consent, whichever comes first, up to 10 years. | Duration of response is defined as the time from the date of initial documentation of a response to the date of first documented evidence of progressive disease (or relapse for participants who experience CR during the study) or death. |
| Time to Next Treatment (TTNT) | From enrollment of the patient until death, until loss to follow-up or withdrawal of informed consent, whichever comes first, up to 10 years. | TTNT is defined as the time from the date of initiation of regimen to the initiation of next regimen for each successive therapy received. |
| Overall Survival (OS) | From enrollment of the patient until death, until loss to follow-up or withdrawal of informed consent, whichever comes first, up to 10 years | OS is defined as the time from the date of initiation of therapy to the date of death from any cause (or last documented follow-up). |
| Progression-free survival (PFS) | From enrollment of the patient until death, until loss to follow-up or withdrawal of informed consent, whichever comes first, up to 10 years | PFS is defined as the time from the date of initiation of therapy to the date of progression or death of any cause, whichever occurs first (or last documented follow-up). |
Countries
Belgium
Contacts
CONTACTMichel Delforge
PRINCIPAL_INVESTIGATORMichel Delforge
UZ Leuven Gasthuisberg
PRINCIPAL_INVESTIGATORJoost Schymkowitz, Prof.
VIB - Switch Lab
PRINCIPAL_INVESTIGATORFrederic Rousseau, Prof.
VIB - Switch Lab
Outcome results
None listed