A Study to Assess the Safety and Efficacy Of Tafamidis In Chinese Participants With Transthyretin Amyloid Cardiomyopathy (ATTR-CM)

Study Overview
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Brief Summary
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This is a national, multi-center, single-arm study, open-label to patients with symptomatic Transthyretin amyloid cardiomyopathy (ATTR-CM) who are tafamidis naïve. This study is to obtain safety, descriptive efficacy, Pharmacokinetics (PK) and Pharmacodynamics (PD) data for tafamidis orally once daily. Subject eligibility for participation in the study will receive tafamidis once daily or 12 months following the assessment as the screening and baseline, month 1, 3, 6, 9 and 12 visits (or Early Study Discontinuation).

Official Title
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A Study to Characterize the Safety and Efficacy of Tafamidis Once Daily in the Treatment of Transthyretin Amyloid Cardiomyopathy in Chinese Participants

Conditions
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Transthyretin Amyloid Cardiomyopathy

Intervention / Treatment
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* Drug: Tafamidis


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Subject has documented ATTR-CM. For the reproductive criteria for male and female participants, please refer to relevant protocol sections. Exclusion Criteria: Other acute or chronic medical or psychiatric condition including recent or active suicidal ideation or behavior or laboratory abnormality, in the judgment of the investigator, would make the participant inappropriate for entry into this study. Participants who have prior liver and/or heart transplant. Participants with primary (light chain) or secondary amyloidosis. Previous administration with an investigational drug within 30 days or 5 half lives preceding the first dose of study intervention used in this study (whichever is longer).

Ages Eligible for Study
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Minimum Age: 18 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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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Chinese participants treated with Tafamidis<br>treatment group with tafamidis | Drug: Tafamidis<br>* 61 mg, once daily, oral administration, for 12 months.<br>* Other names: Vyndamax, Tafamidis free acid, PF 06291826;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Incidence of treatment-emergent adverse events (TEAEs) during the study |  | Baseline up to 28 days after the last dose (approximately 13 months) | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change from baseline in the 6 Minute Walk Distance (6MWD) at Month 6 and Month 12 |  | Baseline, Month 6, Month 12 | 
| The percentage of participants with TTR stabilization at each collection visit | TTR stabilization is a Pharmacodynamic (PD) parameter, the blood samples will be analyzed using validated analytical methods. | Baseline, Months 1, Month 6, and Month 12 | 
| Change from baseline in Kansas City Cardiomyopathy Questionnaire overall summary scores at Month 6 and Month 12 | Domain scores are transformed to a 0 to 100 range; higher scores indicate better health status | Baseline, Months 6, Month 12 | 
| Plasma concentrations of tafamidis at each collection visit |  | Baseline, Month 1, Month 6, Month 12 | 
| Change from baseline in N Terminal prohormone B type Natriuretic Peptide (NT-pro BNP) at Month 6 and Month 12 |  | Baseline, Month 6, Month 12 | 
| TTR concentration at each collection visit |  | Baseline, Months 1, Month 6, and Month 12 | 
| Change from baseline in EuroQoL 5 Dimensions visual analogue scale (EQ-5D VAS) scores at Month 6 and Month 12 | the score range is from 0-100, higher scores indicate better health status | Baseline, Months 6, Month 12 | 
| Change from baseline in 12-Item Short Form Survey (SF-12) scores at Month 6 and Month 12 |  | Baseline, Months 6, Month 12 | 
| Change from baseline in Change from baseline in EuroQol 5 Dimensions 5 Levels (EQ-5D-5L) index score at Month 6 and Month 12 |  | Baseline, Months 6, Month 12 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Tafamidis