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

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

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).

Study Overview

• Status: Completed
• Conditions: Transthyretin Amyloid Cardiomyopathy
• Intervention / Treatment: Drug: Tafamidis

• Study Type: Interventional
• Enrollment (Actual): 53
• Phase: Phase 4

Contacts and Locations

Study Locations

• China
  • Shanghai, China, 200025
    • Ruijin Hospital, Shanghai Jiaotong University School of Medicine
  • Beijing
    • Beijing, Beijing, China, 100034
      • Peking University First Hospital
    • Beijing, Beijing, China, 100191
      • Peking University Third Hospital
    • Beijing, Beijing, China, 100730
      • Peking Union Medical College Hospital
  • Hubei
    • Wuhan, Hubei, China, 430030
      • Tongji Hospital, Tongji Medical College, Huazhong University of Science & Technology
  • Hunan
    • Changsha, Hunan, China, 410011
      • The Second Xiangya Hospital of Central South University
  • Liaoning
    • Dalian, Liaoning, China, 116014
      • The First Affiliated Hospital of Dalian Medical University
  • Sichuan
    • Chengdu, Sichuan, China, 610041
      • West China Hospital of Sichuan University
  • Zhejiang
    • Hangzhou, Zhejiang, China, 310009
      • The Second Affiliated Hospital of Zhejiang University School of Medicine

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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).

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

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

What is the study measuring?

Primary Outcome Measures

Outcome Measure	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

Collaborators and Investigators

• Sponsor: Pfizer
• Investigators: Study Director: Pfizer CT.gov Call Center, Pfizer

Publications and helpful links

Helpful Links

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

Study record dates

Study Major Dates

• Study Start (Actual): July 22, 2021
• Primary Completion (Actual): October 16, 2023
• Study Completion (Actual): October 16, 2023

Study Registration Dates

• First Submitted: March 10, 2021
• First Submitted That Met QC Criteria: March 23, 2021
• First Posted (Actual): March 24, 2021

Study Record Updates

• Last Update Posted (Estimated): November 9, 2023
• Last Update Submitted That Met QC Criteria: November 7, 2023
• Last Verified: November 1, 2023

More Information

Terms related to this study

• Keywords: Tafamidis
• Additional Relevant MeSH Terms: Heart Diseases; Cardiovascular Diseases; Metabolic Diseases; Proteostasis Deficiencies; Amyloidosis; Cardiomyopathies
• Other Study ID Numbers: B3461077

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: Yes