Clinical trial NCT04814186: A Study to Assess the Safety and Efficacy Of Tafamidis In Chinese Participants With Transthyretin Amyloid Cardiomyopathy (ATTR-CM)
Official title: A Study to Characterize the Safety and Efficacy of Tafamidis Once Daily in the Treatment of Transthyretin Amyloid Cardiomyopathy in Chinese Participants
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE4
Conditions: Transthyretin Amyloid Cardiomyopathy
Keywords: Tafamidis
Lead sponsor: Pfizer
Sponsor class: INDUSTRY
First posted: 2021-03-24
Enrollment: 53
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: 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).


Eligibility criteria: Inclusion Criteria:

1. Subject has documented ATTR-CM.
2. For the reproductive criteria for male and female participants, please refer to relevant protocol sections.

Exclusion Criteria:

1. 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.
2. Participants who have prior liver and/or heart transplant.
3. Participants with primary (light chain) or secondary amyloidosis.
4. 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).

Primary outcomes:
measure: Number of Participants With All-Causality Treatment-Emergent Adverse Events (TEAEs)
timeFrame: 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)
measure: Number of Participants With Treatment-Related TEAEs
timeFrame: 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)
Secondary outcomes:
measure: Change From Baseline (CFB) for Distance Walked During 6 Minute Walk Test (6MWT) at Months 6 and 12
timeFrame: Baseline, months 6 and 12
measure: Change From Baseline for N Terminal Prohormone B Type Natriuretic Peptide (NT-proBNP) at Months 6 and 12
timeFrame: Baseline, months 6 and 12
measure: Percentage of Responders in Transthyretin (TTR) Stabilization Post Dose at Months 1, 6, and 12
timeFrame: 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
measure: TTR Concentration at Baseline, Months 1, 6, and 12
timeFrame: 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
measure: Change From Baseline for Kansas City Cardiomyopathy Questionnaire Overall Summary (KCCQ-OS) Scores at Months 6 and 12
timeFrame: Day 1 (baseline), Months 6, and 12
measure: Change From Baseline for EuroQol 5 Dimensions 5 Levels (EQ-5D-5L) Index Values at Months 6 and 12
timeFrame: Day 1 (baseline), Months 6, and 12
measure: Change From Baseline for EuroQol VAS at Months 6 and 12
timeFrame: Day 1 (baseline), Months 6, and 12
measure: Change From Baseline for Physical Component Summary (PCS) for Short-Form Survey 12 (SF-12) at Months 6 and 12
timeFrame: Day 1 (baseline), Months 6, and 12
measure: Change From Baseline for Mental Component Summary (MCS) for SF-12 at Months 6 and 12
timeFrame: Day 1 (baseline), Months 6, and 12
measure: Plasma Concentrations of Tafamidis at Months 1, 6, and 12
timeFrame: 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
Other outcomes:
Locations:
city: Beijing
country: China
facility: Peking University First Hospital
state: Beijing
city: Beijing
country: China
facility: Peking University Third Hospital
state: Beijing
city: Beijing
country: China
facility: Peking Union Medical College Hospital
state: Beijing
city: Wuhan
country: China
facility: Tongji Hospital, Tongji Medical College, Huazhong University of Science & Technology
state: Hubei
city: Changsha
country: China
facility: The Second Xiangya Hospital of Central South University
state: Hunan
city: Dalian
country: China
facility: The First Affiliated Hospital of Dalian Medical University
state: Liaoning
city: Chengdu
country: China
facility: West China Hospital of Sichuan University
state: Sichuan
city: Hangzhou
country: China
facility: The Second Affiliated Hospital of Zhejiang University School of Medicine
state: Zhejiang
city: Shanghai
country: China
facility: Ruijin Hospital, Shanghai Jiaotong University School of Medicine
Study officials:
affiliation: Pfizer
name: Pfizer CT.gov Call Center
role: STUDY_DIRECTOR
References: