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

NCT07494656 | Active Not Recruiting

• 1 other identifier: B3461130
• Study Type: observational
• Target: 1
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

ATTR-CM (Transthyretin Amyloid Cardiomyopathy)

Outcome Measures

Primary Outcomes (2)

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

  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

  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).

Secondary Outcomes (1)

• Annual Incidence of Transthyretin Amyloid Cardiomyopathy (ATTR-CM) in Germany

  Calendar years 2020, 2021, and 2022

Study Arms (1)

Patients with incident transthyretin amyloid cardiomyopathy (ATTR-CM) in Germany

Adult patients (≥18 years) with incident transthyretin amyloid cardiomyopathy (ATTR-CM) in Germany, identified using a claims-based algorithm in nationwide statutory health insurance data. Incident cases are defined based on a washout period of at least two years without prior amyloidosis diagnosis. Patients are followed from the date of ATTR-CM diagnosis. Subset of the incident ATTR-CM population with at least one documented prescription of tafamidis 61 mg during follow-up will be included.

Other: tafamidis 61mg (based on prescription data)

Interventions

tafamidis 61mg (based on prescription data) OTHER

As provided in real world practice

Patients with incident transthyretin amyloid cardiomyopathy (ATTR-CM) in Germany

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

The study population consists of adults insured under the German statutory health insurance system who were newly identified with transthyretin amyloid cardiomyopathy (ATTR-CM) between 2020 and 2022.

You may qualify if:

• 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)

You may not qualify if:

• 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

Sponsors & Collaborators

• Pfizer: lead

Study Sites (1)

Pfizer

Berlin, 10117, Germany

Location

Related Links

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

MeSH Terms

Interventions

tafamidis

Study Officials

• Pfizer CT.gov Call Center

  Pfizer

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: OTHER
• Time Perspective: RETROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 20, 2026
• First Posted: March 27, 2026
• Study Start: April 2, 2026
• Primary Completion: June 30, 2026
• Study Completion: June 30, 2026
• Last Updated: May 22, 2026

Record last verified: 2026-05

Data Sharing

• IPD Sharing: Will not share

Locations

DE (1)