Active — Not RecruitingNot Applicable

A Retrospective Observational Study of Epidemiology and Outcomes in Transthyretin Amyloid Cardiomyopathy in Germany

Germany1 participantsStarted 2026-04-02

Plain-language summary

This observational study aims to describe the characteristics, health outcomes, and epidemiology of individuals diagnosed with transthyretin amyloid cardiomyopathy (ATTR-CM) in Germany. ATTR-CM is a rare and serious heart disease caused by the buildup of an abnormal protein called transthyretin in the heart muscle. This can lead to heart failure and other cardiovascular complications. This is a retrospective, non-interventional study based on anonymized statutory health insurance claims data from Germany. The study includes adults with a new diagnosis of ATTR-CM identified between 2020 and 2022. Data from both inpatient and outpatient healthcare settings are analyzed. No participants are recruited, and no medical treatments, procedures, or study visits are assigned as part of this study. The study will describe overall survival, cardiovascular-related hospitalizations, and the incidence of ATTR-CM in Germany. A predefined subgroup of individuals with at least one documented prescription for tafamidis will be analyzed separately to describe outcomes in this population. In addition, the study will examine the use of genetic testing after diagnosis and describe patient characteristics, as observed in routine clinical care. By using nationwide healthcare data, this study aims to provide a comprehensive and recent real-world overview of ATTR-CM in Germany and to support a better understanding of the disease.

Who can participate

Age range

18 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: Adults aged 18 years or older at the time of the index date Individuals with a newly identified transthyretin amyloid cardiomyopathy (ATTR-CM) diagnosis between January 2020 and December 2022, identified using a validated claims-based algorithm Evidence of amyloidosis, defined as: At least one inpatient diagnosis or At least two outpatient diagnoses in separate quarters Evidence of cardiac involvement, defined as at least one diagnosis of cardiomyopathy or heart failure occurring within a defined temporal window before or after the amyloidosis diagnosis Continuous enrollment in German statutory health insurance for: At least 8 quarters prior to the index date, and At least 4 consecutive quarters after the index date (or until death) Exclusion Criteria: Evidence of light-chain (AL) amyloidosis, including: AL-specific treatment prescriptions Chemotherapy associated with amyloidosis Diagnosis of multiple myeloma autologous stem cell transplantation Diagnosis of amyloid angiopathy Prior amyloidosis diagnosis during the 2-year wash-out period before the index date Missing or implausible information on sex Additional Criteria for Tafamidis Subgroup At least one prescription of tafamidis 61 mg during follow-up No prescriptions for other disease-modifying therapies for transthyretin amyloidosis with polyneuropathy

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Overall Survival in Individuals With Incident Transthyretin Amyloid Cardiomyopathy (ATTR-CM), Overall and in Those With at Least One Tafamidis Prescription

Timeframe: From incident ATTR-CM diagnosis (Index 1) until death, end of SHI enrollment, or end of data availability (December 31, 2023). For those with at least one tafamidis prescription, additionally from first tafamidis prescription (Index 2).

Cardiovascular-Related Hospitalizations in Individuals With Incident Transthyretin Amyloid Cardiomyopathy (ATTR-CM), Overall and in Those With at Least One Tafamidis Prescription

Trial details

NCT IDNCT07494656

SponsorPfizer

Sponsor typeINDUSTRY

Study typeOBSERVATIONAL

Primary completion2026-06-30

View on ClinicalTrials.gov More ATTR-CM (Transthyretin Amyloid Cardiomyopathy) trials