About this trial
The study design is a prospective registry including asymptomatic and symptomatic patients who carry a pathogenic TTR mutation. The study will enroll patients who meet the inclusion criteria and none of the exclusion criteria until 1000 patients are enrolled, at which point in time the study investigators will evaluate whether further patient accrual is meaningful.
Eligibility criteria
Qualifiers
Over the age of 18 years
Carrier of a pathogenic hATTR mutation confirmed on whole blood gene testing or mass spectrometry
Willing to return for required follow-up visits
Disqualifiers
Patient having undergone heart transplantation or implantation of mechanical circulatory support
Patients unable to provide informed consent
Patients having undergone liver transplantation
Patients have evidence of light chain amyloidosis
Trial design
Treatments tested in this trial
- Registry