A Study to Characterize the Safety and Efficacy of Tafamidis Once Daily in the Treatment of Transthyretin Amyloid Cardiomyopathy in Chinese Participants
试验速览
- 阶段
- 4 期
- 状态
- 已完成
- 发起方
- Pfizer
- 入组人数
- 53
- 试验地点
- 9
- 主要终点
- Number of Participants With All-Causality Treatment-Emergent Adverse Events (TEAEs)
研究概览
简要总结
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).
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Subject has documented ATTR-CM.
- •For the reproductive criteria for male and female participants, please refer to relevant protocol sections.
排除标准
- •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).
研究组 & 干预措施
Chinese participants treated with Tafamidis
treatment group with tafamidis
干预措施: Tafamidis (Drug)
结局指标
主要结局
Number of Participants With All-Causality Treatment-Emergent Adverse Events (TEAEs)
时间窗: From first dose of study intervention on Day 1 (baseline) up to 28 days post the last dose of study intervention (up to a maximum of 393 days)
An adverse event (AE) was any untoward medical occurrence in a patient or clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention. All AEs that started after the first dosing but before the last dose plus the lag time (28 days) were TEAEs. A serious adverse event (SAE) was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience; persistent disability/incapacity; congenital anomaly/birth defect. A severe TEAE was an event that prevented normal everyday activities. Severe TEAEs were assessed by the investigator.
Number of Participants With Treatment-Related TEAEs
时间窗: From first dose of study intervention on Day 1 (baseline) up to 28 days post the last dose of study intervention (up to a maximum of 393 days)
An AE was any untoward medical occurrence in a patient or clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention. All AEs that started after the first dosing but before the last dose plus the lag time (28 days) were TEAEs. An SAE was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience; persistent disability/incapacity; congenital anomaly/birth defect. A severe TEAE was an event that prevented normal everyday activities. Severe TEAEs and treatment-related TEAEs were assessed by the investigator.
次要结局
- Change From Baseline (CFB) for Distance Walked During 6 Minute Walk Test (6MWT) at Months 6 and 12(Baseline, months 6 and 12)
- Change From Baseline for N Terminal Prohormone B Type Natriuretic Peptide (NT-proBNP) at Months 6 and 12(Baseline, months 6 and 12)
- Percentage of Responders in Transthyretin (TTR) Stabilization Post Dose at Months 1, 6, and 12(Predose on Day 1 (baseline), predose and 3 hours post dose at Month 1 visit, 7 hours post dose at Month 6 visit, and 1 hour post dose at Month 12 visit)
- TTR Concentration at Baseline, Months 1, 6, and 12(Predose on Day 1 (baseline), predose and 3 hours post dose at Month 1 visit, 7 hours post dose at Month 6 visit, and 1 hour post dose at Month 12 visit)
- Change From Baseline for Kansas City Cardiomyopathy Questionnaire Overall Summary (KCCQ-OS) Scores at Months 6 and 12(Day 1 (baseline), Months 6, and 12)
- Change From Baseline for EuroQol 5 Dimensions 5 Levels (EQ-5D-5L) Index Values at Months 6 and 12(Day 1 (baseline), Months 6, and 12)
- Change From Baseline for EuroQol VAS at Months 6 and 12(Day 1 (baseline), Months 6, and 12)
- Change From Baseline for Physical Component Summary (PCS) for Short-Form Survey 12 (SF-12) at Months 6 and 12(Day 1 (baseline), Months 6, and 12)
- Change From Baseline for Mental Component Summary (MCS) for SF-12 at Months 6 and 12(Day 1 (baseline), Months 6, and 12)
- Plasma Concentrations of Tafamidis at Months 1, 6, and 12(Predose and 3 hours post dose at Month 1 visit, 7 hours post dose at Month 6 visit, and 1 hour post at Month 12 visit)