DemonsTTRate: A Global, Observational, Multicenter, Long-term Study of Patients With ATTR-CM in a Real-World Setting
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- This is an observational study: the protocol does not assign a study treatment.
- Who it may be relevant to
- Registry conditions: Transthyretin Amyloidosis With Cardiomyopathy. Basic parameters: from 18 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
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Official title
DemonsTTRate: A Global, Observational, Multicenter, Long-term Study of Patients With Transthyretin-Mediated Amyloidosis With Cardiomyopathy in a Real-World Setting
Overview
The purpose of this study is to: * Describe the clinical characteristics of adult patients with transthyretin-mediated amyloidosis with cardiomyopathy (ATTR-CM) treated with vutrisiran in routine clinical care * Describe treatment patterns of adult patients with ATTR-CM treated with vutrisiran in routine clinical care * Assess health-related quality of life (HRQOL) in adult patients with ATTR-CM treated with vutrisiran in routine clinical care * Assess healthcare resource use (HCRU) in adult patients with ATTR-CM treated with vutrisiran in routine clinical care Compare the long-term effectiveness of vutrisiran versus other approved ATTR-CM treatments in routine clinical care
Detailed description
Each site will invite all eligible patients to participate. Patients will be followed for up to 5 years or until patient withdrawal of consent, loss to follow-up, death, or the end of the study, whichever comes first.
Primary outcome measures
- New York Heart Association (NYHA) Class [Time frame: From time of ATTR-CM diagnosis up to 5 years]
- Outpatient Diuretic Intensification [Time frame: From time of enrollment for up to 5 years]
- Norfolk Quality of Life - Diabetic Neuropathy (QOL-DN) Total Score [Time frame: From time of enrollment (if available) or ATTR-CM diagnosis up to 5 years]
- Kansas City Cardiomyopathy Questionnaire (KCCQ) [Time frame: From time of enrollment (if available) or ATTR-CM diagnosis up to 5 years]
- Patient Global Impressions - Change (PGI-C) [Time frame: From time of enrollment (if available) or ATTR-CM diagnosis up to 5 years]
- Patient Global Impressions - Severity (PGI-S); [Time frame: From time of enrollment (if available) or ATTR-CM diagnosis up to 5 years]
- Treatment Satisfaction Questionnaire for Medication version 9 (TSQM-9) [Time frame: From time of enrollment (if available) or ATTR-CM diagnosis up to 5 years]
Eligibility criteria
Inclusion criteria
- Has a diagnosis of ATTR-CM
- Is initiating treatment with vutrisiran or another approved ATTR-CM therapy
Exclusion criteria
- Is enrolled in an interventional clinical trial
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Observational model
- Cohort
Study locations
United States · 4 centers
- Clinical Trial Site — Naples
- Clinical Trial Site — Boston
- Clinical Trial Site — New Brunswick
- Clinical Trial Site — Memphis
Identifiers
NCT: NCT07358078 · ALN-TTRSC02-008