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.
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Demographic characteristics (overall and in patients initiating a treatment with eplontersen)
Timeframe: From time of enrollment for up to 7 years
Treatment patterns (overall and in patients initiating a treatment with eplontersen)
Timeframe: From time of enrollment for up to 7 years
Clinical characteristics (overall and in patients initiating a treatment with eplontersen)
Timeframe: From time of enrollment for up to 7 years
Findings from biopsy (overall and in patients initiating a treatment with eplontersen)
Timeframe: From time of enrollment for up to 7 years
Findings from Cardiovascular magnetic resonance imaging (CMR) (overall and in patients initiating a treatment with eplontersen)
Timeframe: From time of enrollment for up to 7 years
Findings from Bone tracer cardiac scintigraphy (overall and in patients initiating a treatment with eplontersen)
Timeframe: From time of enrollment for up to 7 years
Findings from Echocardiography (overall and in patients initiating a treatment with eplontersen)
Timeframe: From time of enrollment for up to 7 years
AstraZeneca Clinical Study Information Center
ECG variables (overall and in patients initiating a treatment with eplontersen)
Timeframe: From time of enrollment for up to 7 years
Sural nerve and tibial nerve amplitude (overall and in patients initiating a treatment with eplontersen)
Timeframe: From time of enrollment for up to 7 years
Biomarker results (overall and in patients initiating a treatment with eplontersen)
Timeframe: From time of enrollment for up to 7 years
Urine test results (overall and in patients initiating a treatment with eplontersen)
Timeframe: From time of enrollment for up to 7 years
Clinical manifestations (signs and symptoms) of ATTR amyloidosis (overall and in patients initiating a treatment with eplontersen)
Timeframe: From time of enrollment for up to 7 years
36-Item Short Form Health Survey Version 2 (SF-36v2) Physical Component Summary score (overall and in patients initiating a treatment with eplontersen)
Timeframe: From time of enrollment for up to 7 years
Norfolk Quality of Life-Diabetic Neuropathy total score (overall and in patients initiating a treatment with eplontersen)
Timeframe: From time of enrollment for up to 7 years
Kansas City Cardiomyopathy Questionnaire (KCCQ) overall summary score (overall and in patients initiating a treatment with eplontersen)
Timeframe: From time of enrollment for up to 7 years
New York Heart Association (NYHA) classification (overall and in patients initiating a treatment with eplontersen)
Timeframe: From time of enrollment for up to 7 years
National Amyloidosis Centre (NAC) ATTR staging or Mayo staging (overall and in patients initiating a treatment with eplontersen)
Timeframe: From time of enrollment for up to 7 years
Familial amyloid polyneuropathy (FAP) (Coutinho) staging (overall and in patients initiating a treatment with eplontersen)
Timeframe: From time of enrollment for up to 7 years
Polyneuropathy disability (PND) score (overall and in patients initiating a treatment with eplontersen)
Timeframe: From time of enrollment for up to 7 years
Left Ventricular Ejection Fraction (overall and in patients initiating a treatment with eplontersen)
Timeframe: From time of enrollment for up to 7 years
6-minute walk test (overall and in patients initiating a treatment with eplontersen)
Timeframe: From time of enrollment for up to 7 years
Charlson comorbidity index (CCI) and CCI components (overall and in patients initiating a treatment with eplontersen)
Timeframe: From time of enrollment for up to 7 years
Other comorbidities of interest (overall and in patients initiating a treatment with eplontersen)
Timeframe: From time of enrollment for up to 7 years
Healthcare resource utilization (overall and in patients initiating a treatment with eplontersen)
Timeframe: From time of enrollment for up to 7 years
Mortality (overall and in patients initiating a treatment with eplontersen)
Timeframe: Throughout study follow-up (up to 7 years)
Liver Disease and Live Transplant
Timeframe: From time of Enrollment for up to 7 Years