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

NCT ID: NCT04814186

Last Updated: 2024-12-18

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

View full results

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

COMPLETED

Clinical Phase

PHASE4

Total Enrollment

53 participants

Study Classification

INTERVENTIONAL

Study Start Date

2021-07-22

Study Completion Date

2023-10-16

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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).

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Transthyretin Amyloid Cardiomyopathy

Keywords

Explore important study keywords that can help with search, categorization, and topic discovery.

Tafamidis

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

Chinese participants treated with Tafamidis

treatment group with tafamidis

Group Type EXPERIMENTAL

Tafamidis

Intervention Type DRUG

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

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

Tafamidis

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

Intervention Type DRUG

Other Intervention Names

Discover alternative or legacy names that may be used to describe the listed interventions across different sources.

Vyndamax, Tafamidis free acid, PF 06291826

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

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).
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

Pfizer

INDUSTRY

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Responsibility Role SPONSOR

Principal Investigators

Learn about the lead researchers overseeing the trial and their institutional affiliations.

Pfizer CT.gov Call Center

Role: STUDY_DIRECTOR

Pfizer

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

Peking University First Hospital

Beijing, Beijing Municipality, China

Site Status

Peking University Third Hospital

Beijing, Beijing Municipality, China

Site Status

Peking Union Medical College Hospital

Beijing, Beijing Municipality, China

Site Status

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

Wuhan, Hubei, China

Site Status

The Second Xiangya Hospital of Central South University

Changsha, Hunan, China

Site Status

The First Affiliated Hospital of Dalian Medical University

Dalian, Liaoning, China

Site Status

West China Hospital of Sichuan University

Chengdu, Sichuan, China

Site Status

The Second Affiliated Hospital of Zhejiang University School of Medicine

Hangzhou, Zhejiang, China

Site Status

Ruijin Hospital, Shanghai Jiaotong University School of Medicine

Shanghai, , China

Site Status

Countries

Review the countries where the study has at least one active or historical site.

China

References

Explore related publications, articles, or registry entries linked to this study.

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.

Reference Type DERIVED
PMID: 40410537 (View on PubMed)

Provided Documents

Download supplemental materials such as informed consent forms, study protocols, or participant manuals.

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Related Links

Access external resources that provide additional context or updates about the study.

https://pmiform.com/clinical-trial-info-request?StudyID=B3461077

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

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

B3461077

Identifier Type: -

Identifier Source: org_study_id