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

NCT ID: NCT06651073

Last Updated: 2025-05-08

Basic Information

• Recruitment Status: RECRUITING
• Total Enrollment: 350 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2024-10-01
• Study Completion Date: 2025-10-03

Brief 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. 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).

Conditions

Transthyretin Amyloid Cardiopathy

Study Design

• Observational Model Type: OTHER
• Study Time Perspective: OTHER

Study Groups

ATTR-CM patients in Taiwan, Hong Kong, and Malaysia

case-only

Observational

Intervention Type: OTHER

There is no intervention involved in the study. It is purely observational.

Interventions

Observational

There is no intervention involved in the study. It is purely observational.

Intervention Type: OTHER

Other Intervention Names

Non-interventional, Observational

Eligibility Criteria

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

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Pfizer

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Pfizer CT.gov Call Center

Role: STUDY_DIRECTOR

Pfizer

Locations

National Taiwan University

Taipei, , Taiwan

Site Status: RECRUITING

Countries

Taiwan

Central Contacts

Pfizer CT.gov Call Center

Role: CONTACT

ClinicalTrials.gov_Inquiries@pfizer.com

1-800-718-1021

References

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.

Reference Type: DERIVED

PMID: 40479720 (View on PubMed)

Related Links

https://pmiform.com/clinical-trial-info-request?StudyID=B3461110

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

Other Identifiers

NCT06651073

Identifier Type: REGISTRY

Identifier Source: secondary_id

B3461110

Identifier Type: -

Identifier Source: org_study_id