Clinical trial NCT06651073: ATTR-CM: A Multi-country, Non-interventional Disease Registry
Official title: Real-World Characteristics and Treatment Patterns of Patients With Transthyretin Amyloid Cardiomyopathy (ATTR-CM): A Multi-Country, Non-Interventional, Disease Registry
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Transthyretin Amyloid Cardiopathy
Keywords: Transthyretin amyloid cardiopathy
Lead sponsor: Pfizer
Sponsor class: INDUSTRY
First posted: 2024-10-21
Enrollment: 350
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: OTHER
Time perspective: OTHER
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.

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).

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

Primary outcomes:
measure: Demographic data
timeFrame: From Enrollment/Baseline to 12 months
measure: Physical measurements (in all patients whether receiving treatment or not)
timeFrame: index date (closest available measurement) to 12 months
measure: Relevant medical history and comorbidities
timeFrame: enrollment/baseline to 12 months
measure: NYHA Functional Class
timeFrame: index date (closest available measurement to enrollment)
measure: Prescription of heart and cardiovascular medication
timeFrame: within 6 months prior to the index date to 12 months
measure: Type of ATTR-CM
timeFrame: enrollment/index date
measure: Family History
timeFrame: index date/baseline
measure: Diagnosis of ATTR-CM
timeFrame: enrollment/baseline
Secondary outcomes:
measure: Treatment for ATTR-CM
timeFrame: enrollment, every 3 month or as per standard of care, 12 months
measure: Concomitant medications (including heart and CV medications and other concomitant medications)
timeFrame: from index date until 12 months
Other outcomes:
Locations:
city: Taipei City
country: Taiwan
facility: National Taiwan University
Study officials:
affiliation: Pfizer
name: Pfizer CT.gov Call Center
role: STUDY_DIRECTOR
References: