Korean Post-marketing Surveillance Vyndamax® Capsules for the Treatment of Transthyretin Amyloid Cardiomyopathy

Study Overview
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Brief Summary
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This non-interventioal study will be performed by design of post-marketing surveillance (PMS) as an additional pharmacovigilance activity of the Risk Management Plan (RMP) for Vyndamax® Capsules, which is required by the Ministry of Food and Drug Safety (MFDS) according to the local regulation. This post-marketing surveillance will investigate the safety and effectiveness of Vyndamax® Capsules as the treatment of transthyretin amyloid cardiomyopathy during 10 years under the setting of routine practice in Korea.

Official Title
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KOREAN POST-MARKETING SURVEILLANCE VYNDAMAX (Registered) CAPSULES FOR THE TREATMENT OF TRANSTHYRETIN AMYLOID CARDIOMYOPATHY (ATTR-CM)

Conditions
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ATTR-CM (Transthyretin Amyloid Cardiomyopathy)

Intervention / Treatment
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* Drug: Vyndamax (tafamidis 61mg)


Participation Criteria
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Eligibility Criteria
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*Inclusion Criteria: Patients must meet all of the following inclusion criteria to be eligible for inclusion in the study: 1. Adult patients with the cardiomyopathy of wild type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) 2. Patients to whom Vyndamax® Capsules is prescribed for the treatment of the cardiomyopathy of wild type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) 3. 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. Exclusion criteria Patients meeting any of the following criteria will not be included in the study according to the local product label: 1. Patient with hypersensitivity or case history to tafamidis or to any of the excipients in the product 2. This product contains sorbitol (E420). Patients with rare hereditary problems of fructose intolerance should not take this medicine. 3. Patient who has a contraindication to Vyndamax® Capsules according to the approved local product label

Ages Eligible for Study
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Minimum Age: 19 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Adults diagnosed with transthyretin-mediated amyloidosis (ATTR-CM)<br> | Drug: Vyndamax (tafamidis 61mg)<br>* As prescribed in real world practice<br>* Other names: Tafamadis;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| The incidence of Adverse Events (AEs) | The primary interest of this study is the incidence of AEs investigated during administration period and within 28 days after discontinuation of Vyndamax® Capsules. Incidence of AEs, 95% confidence interval and the number of AEs will be presented. Serious AEs/ADRs, unexpected AEs/ADRs, unexpected serious AEs/ADRs will be summarized separately table. | Baseline through 6 months of treatment | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change from baseline in the New York Heart Association (NYHA) class at Month 6 |  | Baseline, month 6 | 
| Change from baseline in the 6 Minute Walk Distance (6MWD) at Month 6 |  | Baseline, month 6 | 
| Global assessment by investigator at Month 6 |  | Baseline, month 6 |