Transthyretin Amyloidosis Clinical Trial
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
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.
MaesTTRo is an international, longitudinal, non-interventional study of adult patients with transthyretin (ATTR) amyloidosis. The study plans to enroll a minimum of 1,600 patients with ATTR amyloidosis, including a minimum of 1,500 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 (e.g., 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. ;
| Status | Clinical Trial | Phase | |
|---|---|---|---|
| Completed |
NCT03190577 -
Assessment of the Prevalence of TTR Amyloid Neuropathy in a Population of Patients With Neuropathy of Unknown Aetiology
|
N/A | |
| Completed |
NCT03588468 -
Expanding the Biomarkers in Familial Amyloid Neuropathy: MRI and Motor Unit Estimation by Electrophysiological Study
|
N/A | |
| Completed |
NCT03591757 -
Short-term Effects of TOLCAPONE on Transthyretin Stability in Subjects With Leptomeningeal TTR Amyloidosis (ATTR)
|
Early Phase 1 | |
| Completed |
NCT05075798 -
Study of Cerebral MRI Anomalies in Mutated Transthyretin Amyloidosis Patients
|
||
| Recruiting |
NCT05577819 -
Prevalence and Prediction of ATTR in Ambulatory Patients With HFpEF
|
N/A | |
| Terminated |
NCT04611204 -
Transthyretin Cardiac Amyloidosis in Patients With Idiopathic Carpal Tunnel Syndrome Referred for Release Surgery
|
||
| Completed |
NCT03352089 -
Positron Emission Tomography / Magnetic Resonance Imaging in Aortic Stenosis
|
||
| Recruiting |
NCT05814380 -
The Regional Scintigraphic DPD Uptake in Cardiac Transthyretin Amyloidosis.
|
||
| Completed |
NCT03886155 -
Cardiac Amyloidosis Screening at Trigger Finger Release
|
||
| Completed |
NCT03923920 -
Screening for Systemic Amyloidosis Via the Ligamentum Flavum
|
||
| Completed |
NCT03862807 -
Patisiran in Patients With Hereditary Transthyretin-mediated Amyloidosis (hATTR Amyloidosis) Disease Progression Post-Liver Transplant
|
Phase 3 | |
| Completed |
NCT02792790 -
Carpal Tunnel Syndrome and Amyloid Cardiomyopathy
|
||
| Completed |
NCT03860935 -
Efficacy and Safety of AG10 in Subjects With Transthyretin Amyloid Cardiomyopathy
|
Phase 3 | |
| Recruiting |
NCT04963985 -
The Effect of Tafamidis on Transthyretin Stabilization, Safety, Tolerability and Efficacy in Transthyretin Amyloid Polyneuropathy Patients
|
Phase 4 | |
| Recruiting |
NCT05409833 -
Systemic Transthyretin Amyloidosis: Carpal Tunnel Syndrome in a Portuguese Population
|
||
| Recruiting |
NCT05635045 -
Evuzamitide in PET/CT to Measure Potential Therapeutic Response in ATTR
|
Phase 2 | |
| Completed |
NCT01171859 -
Safety, Efficacy and Pharmacokinetics of Doxycycline Plus Tauroursodeoxycholic Acid in Transthyretin Amyloidosis
|
Phase 2 | |
| Recruiting |
NCT03237494 -
TRAMmoniTTR Study Genetic Screening of an At-risk Population for hATTR and Monitoring of TTR Positive Subjects
|
||
| Recruiting |
NCT04899180 -
Prevalence of Transthyretin Cardiac Amyloidosis in Clinically Significant Aortic Stenosis
|
Early Phase 1 | |
| Recruiting |
NCT06345235 -
New Biomarkers and Plasma Prothrombotic Potential in Cardiac Transthyretin Amyloidosis
|