ATTR-CM: A Multi-country, Non-interventional Disease Registry

Real-World Characteristics and Treatment Patterns of Patients With Transthyretin Amyloid Cardiomyopathy (ATTR-CM): A Multi-Country, Non-Interventional, Disease Registry

This study is a multi-center, non-interventional, disease registry to characterize the natural history of ATTR-CM and treatment patterns in clinically diagnosed ATTR-CM patients. Data will be collected in the course of routine clinical practice or following local standard practice guidelines. No procedures or treatments will be mandated by this study, patients will receive usual clinical care.

The patient population will include all adult patients with a confirmed diagnosis of ATTR-CM after 01 June 2019 and who meet eligibility criteria. The index date of each patient will be the date of the first documented ATTR-CM diagnosis. The observation period for each patient will range from the index date to whichever occurs first of death, patient withdrawal of consent, loss to follow-up, or end of data collection. The end of data collection (ie, the end of study) is planned for 12 months after the end of the enrollment period.

As this study is descriptive in nature with no hypothesis testing, the study size will be based on the number of eligible ATTR-CM cases identified in the medical records and meeting the eligibility criteria. Approximately 350 patients diagnosed with ATTR-CM across approximately 17 sites in Taiwan, Hong Kong, and Malaysia are planned to be enrolled.

Study Overview

• Status: Active, not recruiting
• Conditions: Transthyretin Amyloid Cardiopathy
• Intervention / Treatment: Other: Observational

Detailed Description

Study variables will include demographic and clinical characteristics of the ATTR-CM patients at diagnosis, including age, sex, physical measurement, relevant medical history and comorbidities, New York Heart Association (NYHA) functional class, prior prescription of heart and CV medication, type of ATTR-CM (ATTRwt or ATTRm), and diagnosis parameters. Exposure variables will include treatment type, treatment regimen/patterns, and concomitant medications. Outcome variables will provide information on death (status and reasons), hospitalizations (status and reasons), imaging and lab assessments, Kansas City Cardiomyopathy Questionnaire (KCCQ), and healthcare resource utilization (eg, days of hospitalization, frequency of emergency department visits). All relevant data will be collected from the index date to the end of the observation period.

Data will be obtained from all information that is available at the site in the patients' electronic medical records and medical charts (eg, consultation notes, discharge summaries, laboratory test results, recorded prescription data, and any other documentation of communication with other health care providers). All data will be collected by trained site personnel and entered directly into web-based electronic case report forms (eCRFs).

• Study Type: Observational
• Enrollment (Actual): 350

Contacts and Locations

Study Locations

• Taiwan
  • Taipei, Taiwan, 100225
    • National Taiwan University

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

The patient population will include all adult patients with a confirmed diagnosis of ATTR-CM after 01 June 2019 and who meet eligibility criteria. The index date of each patient will be the date of the first documented ATTR-CM diagnosis. The observation period for each patient will range from the index date to whichever occurs first of death, patient withdrawal of consent, loss to follow-up, or end of data collection. The end of data collection (ie, the end of study) is planned for 12 months after the end of the enrollment period.

Description

Inclusion Criteria:

1. Evidence of a personally signed and dated informed consent document indicating that the patient (or a legally acceptable representative) has been informed of all pertinent aspects of the study. Waivers of informed consent for deceased patients will be pursued if permitted by local regulation
2. Patients aged ≥18 years at first ATTR-CM diagnosis
3. Patients with confirmed diagnosis of ATTR-CM after 01 June 2019

Exclusion Criteria:

• None

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
ATTR-CM patients in Taiwan, Hong Kong, and Malaysia; case-only	Other: Observational; There is no intervention involved in the study. It is purely observational.; Other Names: Non-interventional, Observational

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Demographic data	Age (measured in years) at enrollment, sex/gender (male/female), race/ethnicity	From Enrollment/Baseline to 12 months
Physical measurements (in all patients whether receiving treatment or not)	Height (in cm), weight (in kg), smoking status (never/prior/current), blood pressure (diastolic/systolic [in mmHg], heart rate (in beats/minute)	index date (closest available measurement) to 12 months
Relevant medical history and comorbidities	The following information will be collected: First clinical cardiac symptom related to ATTR-CM, symptom onset date, age at onset of symptom; treatment received for the first symptom (with start and end date); Relevant medical history and comorbidities of interest including: amyloidosis, CV conditions (HFpEF, HFrEF, left ventricular hypertrophy/increased wall thickness, ischemic heart disease, pulmonary embolism, pericarditis, aortic stenosis, cardiac arrhythmias, atrial fibrillation and flutter, atrioventricular and left bundle branch block, conduction disorders, pacemaker or implantable cardioverter-defibrillator, other) Other vascular comorbidities: hypertensive diseases, diabetes mellitus, cerebrovascular disease, peripheral vascular disease, venous thrombosis, other. Nervous system conditions: carpal tunnel syndrome, lumbar spinal stenosis, peripheral neuropathy, autonomic neuropathy, other. Genitourinary system conditions: chronic kidney disease, erectile dysfunction, other.	enrollment/baseline to 12 months
NYHA Functional Class	Patients' Physical activities wil be graded I, II, III, IV. I being asymptomatic, II being slight limitation, III being marked limitation, and IV being symptoms at rest.	index date (closest available measurement to enrollment)
Prescription of heart and cardiovascular medication	The following information will be collected: Medication type (eg. beta-blockers, ACE inhibitors, ARBs, ARNi, digoxin, calcium channel blockers, diuretics, antiplatelets, lipid-lowering agents, anticoagulants, other), medication name, start date, end date, reason of discontinuation	within 6 months prior to the index date to 12 months
Type of ATTR-CM	described by whether it is wild type or hereditary (including genetic variant if identified)	enrollment/index date
Family History	Describe whether the patient has family history of known cardiomyopathy, polyneuropathy, and sudden cardiac death (among parents, siblings, and 2nd/3rd grade family)	index date/baseline
Diagnosis of ATTR-CM	Date of first ATTR-CM diagnosis; Age (in years) at first ATTR-CM diagnosis; Method of diagnosis of ATTR-CM including any of the following: cardiological assessments, MRI, ECG, ECHO, PYP scan with SPECT/CT (for definite confirmatory testing), nerve conduction studies, plasma neurofilament light (pNfL), and any other methods	enrollment/baseline

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Treatment for ATTR-CM	Type of Treatment including: ATTR-specific pharmacologic treatment: treatment name, dosage, frequency, start and end date, any treatment change, date and reason of treatment change.; Supportive treatment of cardiac involvement: 1. management of HF; 2. management of arrhythmias: medical therapy (eg. rate/rhythm control, cardioversion/ablation, other), device therapy, and other.; Liver or heart transplant.; Other	enrollment, every 3 month or as per standard of care, 12 months
Concomitant medications (including heart and CV medications and other concomitant medications)	The following information will be collected: Medication type (CV-related [eg. beta-blockers, ACE inhibitors, ARBs, ARNi, digoxin, calcium channel blockers, diuretics, antiplatelets, lipid-lowering agents, anticoagulants, or other] non-CV related), medication name, prescribed dose, dosing frequency, indication, start and stop dates, and reasons for discontinuation.	from index date until 12 months

Collaborators and Investigators

• Sponsor: Pfizer
• Investigators: Study Director: Pfizer CT.gov Call Center, Pfizer

Publications and helpful links

General Publications

• Lin YH, Chou HW, Tsai S, Gomez R. Real-World Characteristics and Treatment Patterns of Patients With Transthyretin Amyloid Cardiomyopathy: Protocol for a Multicountry Disease Registry Study. JMIR Res Protoc. 2025 Jun 6;14:e71314. doi: 10.2196/71314.

Helpful Links

• To obtain contact information for a study center near you, click here.

Study record dates

Study Major Dates

• Study Start (Actual): October 1, 2024
• Primary Completion (Estimated): April 28, 2028
• Study Completion (Estimated): April 28, 2028

Study Registration Dates

• First Submitted: October 4, 2024
• First Submitted That Met QC Criteria: October 18, 2024
• First Posted (Actual): October 21, 2024

Study Record Updates

• Last Update Posted (Actual): February 19, 2026
• Last Update Submitted That Met QC Criteria: February 17, 2026
• Last Verified: February 1, 2026

More Information

Terms related to this study

• Keywords: Transthyretin amyloid cardiopathy
• Additional Relevant MeSH Terms: Cardiovascular Diseases; Metabolic Diseases; Proteostasis Deficiencies; Heart Diseases; Amyloidosis; Health Services Administration; Quality of Health Care; Outcome Assessment, Health Care; Outcome and Process Assessment, Health Care; Watchful Waiting
• Other Study ID Numbers: B3461110; NCT06651073 (Registry Identifier: ClinicalTrials.gov)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO
• IPD Plan Description: Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No