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

NCT04814186 | Completed Phase 4 Results FDA Drug

• 1 other identifier: B3461077
• Study Type: interventional
• Target: 53
• Locations: 1 country
• Sites: 9

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).

Conditions

Transthyretin Amyloid Cardiomyopathy

Keywords

Tafamidis

Outcome Measures

Primary Outcomes (2)

• Number of Participants With All-Causality Treatment-Emergent Adverse Events (TEAEs)

  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.

  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)

• Number of Participants With Treatment-Related TEAEs

  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.

  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 (10)

• 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

Study Arms (1)

Chinese participants treated with Tafamidis

EXPERIMENTAL

treatment group with tafamidis

Drug: Tafamidis

Interventions

Tafamidis DRUG

61 mg, once daily, oral administration, for 12 months.

Also known as: Vyndamax, Tafamidis free acid, PF 06291826

Chinese participants treated with Tafamidis

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

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

You may not qualify if:

• 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).

Sponsors & Collaborators

• Pfizer: lead

Study Sites (9)

Peking University First Hospital

Beijing, Beijing Municipality, 100034, China

Location

Peking University Third Hospital

Beijing, Beijing Municipality, 100191, China

Location

Peking Union Medical College Hospital

Beijing, Beijing Municipality, 100730, China

Location

Tongji Hospital, Tongji Medical College, Huazhong University of Science & Technology

Wuhan, Hubei, 430030, China

Location

The Second Xiangya Hospital of Central South University

Changsha, Hunan, 410011, China

Location

The First Affiliated Hospital of Dalian Medical University

Dalian, Liaoning, 116014, China

Location

West China Hospital of Sichuan University

Chengdu, Sichuan, 610041, China

Location

The Second Affiliated Hospital of Zhejiang University School of Medicine

Hangzhou, Zhejiang, 310009, China

Location

Ruijin Hospital, Shanghai Jiaotong University School of Medicine

Shanghai, 200025, China

Location

Related Publications (1)

• Tian Z, Peng D, Ma W, Yan J, Wang J, Tang Y, Jin W, Liu Y, Jia C, Gao Y, Gong Y, Sun X, Chen N, Zhu S, Zhang S. Safety and Efficacy of Tafamidis in Chinese Patients with Transthyretin Amyloid Cardiomyopathy. Cardiol Ther. 2025 Sep;14(3):439-452. doi: 10.1007/s40119-025-00408-6. Epub 2025 May 23.

  PMID: 40410537 DERIVED

Related Links

• To obtain contact information for a study center near you, click here.

MeSH Terms

Interventions

tafamidis

Results Point of Contact

• Title: Pfizer ClinicalTrials.gov Call Center
• Organization: Pfizer Inc.

ClinicalTrials.gov_Inquiries@pfizer.com

1-800-718-1021

Study Officials

• Pfizer CT.gov Call Center

  Pfizer

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 10, 2021
• First Posted: March 24, 2021
• Study Start: July 22, 2021
• Primary Completion: October 16, 2023
• Study Completion: October 16, 2023
• Last Updated: December 18, 2024
• Results First Posted: December 18, 2024

Record last verified: 2024-11

Data Sharing

• IPD Sharing: Will share

Locations

CN (9)