Ignite Creation Date:
2024-07-17 @ 11:59 AM
Last Modification Date:
2024-10-26 @ 3:32 PM
Study NCT ID:
NCT06465810
Status:
RECRUITING
Last Update Posted:
2024-06-20
First Post:
2024-05-13
Brief Title:
Non-interventional Study of Patients With Transthyretin ATTR Amyloidosis
Sponsor:
AstraZeneca
Organization:
AstraZeneca
Study Overview
Official Title:
A Non-interventional Prospective Multi-country Study Collecting Real-world Data on the Characteristics Treatment Patterns and Outcomes of Patients With Transthyretin ATTR Amyloidosis
Status:
RECRUITING
Status Verified Date:
2024-09
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
MaesTTRo
Brief Summary:
The MaesTTRo study aims to enroll a global cohort of patients with transthyretin ATTR amyloidosis to longitudinally observe the natural course of the disease and describe real-world treatment patterns and outcomes In addition information on the effectiveness of ATTR amyloidosis treatments including eplontersen which is a ligand-conjugated antisense oligonucleotide gene silencing treatment targeting activity against both the mutant and wild-type TTR protein will be collected
Detailed Description:
MaesTTRo is an international longitudinal non-interventional study of adult patients with transthyretin ATTR amyloidosis
The study plans to enroll a minimum of 1600 patients with ATTR amyloidosis including a minimum of 1500 patients with ATTR cardiomyopathy ATTR-CM and a minimum of 100 patients with ATTRv-PN hereditary polyneuropathy
The enrollment period is expected to last approximately 4 years The duration of follow-up for each patient will be at least 3 years and up to 7 years depending on the date when the patient is enrolled
This study design will include both primary and secondary data Primary data will consist of patient-reported outcome PRO questionnaires Patients will be asked to complete electronic PRO questionnaires at enrollment and every 6 months 3 months only during routine visits Secondary data will consist of demographic clinical and treatment information and will be collected as per routine clinical practice These data will be abstracted directly from the electronic health record or review of paper charts for each patient and entered in the electronic data capture system No site visits are required for this study and patients will not be contacted for data collection outside of routine clinic visits
For patients enrolled in the United States a tokenization process creation of a unique encrypted identifier called a token in place of personal identifiable information will be used to collect additional de-identified data eg healthcare resource use healthcare costs from other sources that are part of patients routine medical care electronic medical hospital or pharmacy records Only de-identified data will be analyzed Patients will be given a choice within the informed consent form to opt in or opt out of participating in the tokenization process
The study plans to enroll a minimum of 1600 patients with ATTR amyloidosis including a minimum of 1500 patients with ATTR cardiomyopathy ATTR-CM and a minimum of 100 patients with ATTRv-PN hereditary polyneuropathy
The enrollment period is expected to last approximately 4 years The duration of follow-up for each patient will be at least 3 years and up to 7 years depending on the date when the patient is enrolled
This study design will include both primary and secondary data Primary data will consist of patient-reported outcome PRO questionnaires Patients will be asked to complete electronic PRO questionnaires at enrollment and every 6 months 3 months only during routine visits Secondary data will consist of demographic clinical and treatment information and will be collected as per routine clinical practice These data will be abstracted directly from the electronic health record or review of paper charts for each patient and entered in the electronic data capture system No site visits are required for this study and patients will not be contacted for data collection outside of routine clinic visits
For patients enrolled in the United States a tokenization process creation of a unique encrypted identifier called a token in place of personal identifiable information will be used to collect additional de-identified data eg healthcare resource use healthcare costs from other sources that are part of patients routine medical care electronic medical hospital or pharmacy records Only de-identified data will be analyzed Patients will be given a choice within the informed consent form to opt in or opt out of participating in the tokenization process
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None