Transthyretin Amyloid CardiopathyNCT06651073
ATTR-CM: A Multi-country, Non-interventional Disease Registry
An observational study of Transthyretin Amyloid Cardiopathy, run by Pfizer at 17 sites in 3 countries.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Transthyretin Amyloid Cardiopathy
- What is being tested?
- Observational
- How many people
- 350 participants
- Where
- 17 sitesin 3 countries
- Study dates
- 1 Oct 2024to 28 Apr 2028
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
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.
Who may be able to join
- 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
- Patients aged ≥18 years at first ATTR-CM diagnosis
- Patients with confirmed diagnosis of ATTR-CM after 01 June 2019
Who cannot join
- None
Trial locations and contacts
17 sites in 3 countries.
17 sites in 3 countries
Taiwan10 sites
Chang Gung Memorial Hospital, Linkou
Gueishan Township, Taoyuan, 333
Kaohsiung Medical University Chung-Ho Memorial Hospital
Kaohsiung City, 807
Kaohsiung Chang Gung Memorial Hospital
Kaohsiung City, 833
Far Eastern Memorial Hospital
New Taipei City, 220
Taichung Veterans General Hospital
Taichung, Taipei, 40705
China Medical University Hospital
Taichung, Taiwan R O C, 40447
Chi Mei Medical Center
Tainan
National Taiwan University
Taipei, 100225
MacKay Memorial Hospital
Taipei, 10449
Taipei Veterans General Hospital
Taipei, 11217
Hong Kong4 sites
Queen Elizabeth Hospital
Hong Kong
Princess Margaret Hospital
Kwai Chung
Queen Mary Hospital
Pok Fu Lam
Prince of Wales Hospital
Shatin
Malaysia3 sites
Institut Jantung Negara
Kuala Lumpur
Hospital Serdang
Kuala Selangor
Sarawak Heart Centre
Sarawak
About this study
Official title: 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.
Read the 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).
- Conditions
- Transthyretin Amyloid Cardiopathy
- Being tested
- Observational
- Keywords
- Transthyretin amyloid cardiopathy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ObservationalOther
Also called Non-interventional, Observational
There is no intervention involved in the study. It is purely observational.
| Study group | Role | What they receive |
|---|---|---|
| ATTR-CM patients in Taiwan, Hong Kong, and Malaysia | case-only Receives: Other: Observational |
What is being measured
The primary outcome is the main question the study is designed to answer.
Demographic data
Age (measured in years) at enrollment, sex/gender (male/female), race/ethnicity
Measured: 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)
Measured: 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.
Measured: 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.
Measured: 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
Measured: 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)
Measured: 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)
Measured: 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
Measured: enrollment/baseline
Secondary outcomes (2)
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
Measured: 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.
Measured: from index date until 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Oct 2024
- Study start1 Oct 2024
- Primary completion28 Apr 2028 (estimated)
- Study completion28 Apr 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Transthyretin Amyloid CardiopathyStudies of the same condition, open and under way.
- More trials run by PfizerThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Other
- Time perspective
- Other
- Enrolment
- 350 participants (actual)
Sponsor and investigators
- Lead sponsor
- Pfizer
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Pfizer CT.gov Call Center, Study director, Pfizer
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06651073
- Sponsor's protocol ID
- B3461110
- Other IDs
- NCT06651073
- Status last verified
- Oct 2026
- First submitted
- 4 Oct 2024
- Last updated
- 6 Oct 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: 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.
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.
PubMed 40479720
Study at a glance
From the registry record, last updated by the sponsor on 6 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Real-World Characteristics and Treatment Patterns of Patients With Transthyretin Amyloid Cardiomyopathy (ATTR-CM): A Multi-Country, Non-Interventional, Disease Registry
- ClinicalTrials.gov ID
- NCT06651073
- Acronym / sponsor ID
- B3461110
- Conditions
- Transthyretin Amyloid Cardiopathy
- Keywords
- Transthyretin amyloid cardiopathy
- Study type
- Observational
- Recruitment status
- Active, not recruiting
- Enrolment
- 350 participants (actual)
- Lead sponsor
- PfizerIndustry (pharma, biotech, devices)
- Study start
- 1 Oct 2024 (actual)
- Primary completion
- 28 Apr 2028 (estimated)
- Study completion
- 28 Apr 2028 (estimated)
- First posted
- 21 Oct 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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 design
- Observational model
- Other
- Time perspective
- Other
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| ATTR-CM patients in Taiwan, Hong Kong, and Malaysia | – | Other: Observationalcase-only |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- 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.
Key inclusion criteria (3 listed)
- 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
- Patients aged ≥18 years at first ATTR-CM diagnosis
- Patients with confirmed diagnosis of ATTR-CM after 01 June 2019
Key exclusion criteria (1 listed)
- None
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ObservationalAlso: Non-interventional, ObservationalThere is no intervention involved in the study. It is purely observational. | Other | ATTR-CM patients in Taiwan, Hong Kong, and Malaysia |
Primary outcome
Demographic data
Time frame: From Enrollment/Baseline to 12 months
Age (measured in years) at enrollment, sex/gender (male/female), race/ethnicity
Physical measurements (in all patients whether receiving treatment or not)
Time frame: index date (closest available measurement) to 12 months
Height (in cm), weight (in kg), smoking status (never/prior/current), blood pressure (diastolic/systolic [in mmHg], heart rate (in beats/minute)
Relevant medical history and comorbidities
Time frame: enrollment/baseline to 12 months
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.
NYHA Functional Class
Time frame: index date (closest available measurement to enrollment)
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.
Prescription of heart and cardiovascular medication
Time frame: within 6 months prior to the index date to 12 months
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
Type of ATTR-CM
Time frame: enrollment/index date
described by whether it is wild type or hereditary (including genetic variant if identified)
Family History
Time frame: index date/baseline
Describe whether the patient has family history of known cardiomyopathy, polyneuropathy, and sudden cardiac death (among parents, siblings, and 2nd/3rd grade family)
Diagnosis of ATTR-CM
Time frame: enrollment/baseline
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
2 secondary outcomes
Treatment for ATTR-CM
Time frame: enrollment, every 3 month or as per standard of care, 12 months
Concomitant medications (including heart and CV medications and other concomitant medications)
Time frame: from index date until 12 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Oct 2026.
- Derived from this study: PubMed 40479720Lin 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.
- To obtain contact information for a study center near you, click here.Link provided by the sponsor.
- Individual participant dataSharing planned: No. 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Pfizer CT.gov Call Center study director
Pfizer
17 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Transthyretin Amyloid Cardiopathy, run by Pfizer at 17 sites in 3 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 6 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 17 sites in Taiwan, Hong Kong, Malaysia. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Demographic data (time frame: From Enrollment/Baseline to 12 months). The study lists 8 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT06651073, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 Oct 2026.