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

Primary Purpose

Transthyretin Amyloid Cardiomyopathy

Status
Active
Phase
Phase 4
Locations
China
Study Type
Interventional
Intervention
Tafamidis
Sponsored by
Pfizer
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Transthyretin Amyloid Cardiomyopathy focused on measuring Tafamidis

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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).

Sites / Locations

  • Peking University First Hospital
  • Peking University Third Hospital
  • Peking Union Medical College Hospital
  • Tongji Hospital, Tongji Medical College, Huazhong University of Science & Technology
  • The Second Xiangya Hospital of Central South University
  • The First Affiliated Hospital of Dalian Medical University
  • West China Hospital of Sichuan University
  • The Second Affiliated Hospital of Zhejiang University School of Medicine
  • Ruijin Hospital, Shanghai Jiaotong University School of Medicine

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Chinese participants treated with Tafamidis

Arm Description

treatment group with tafamidis

Outcomes

Primary Outcome Measures

Incidence of treatment-emergent adverse events (TEAEs) during the study

Secondary Outcome Measures

Change from baseline in the 6 Minute Walk Distance (6MWD) at Month 6 and 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.
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
Plasma concentrations of tafamidis at each collection visit
Change from baseline in N Terminal prohormone B type Natriuretic Peptide (NT-pro BNP) at Month 6 and Month 12
TTR concentration at each collection visit
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
Change from baseline in 12-Item Short Form Survey (SF-12) scores at Month 6 and 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

Full Information

First Posted
March 10, 2021
Last Updated
February 22, 2023
Sponsor
Pfizer
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1. Study Identification

Unique Protocol Identification Number
NCT04814186
Brief Title
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

2. Study Status

Record Verification Date
February 2023
Overall Recruitment Status
Active, not recruiting
Study Start Date
July 22, 2021 (Actual)
Primary Completion Date
October 16, 2023 (Anticipated)
Study Completion Date
October 16, 2023 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Pfizer

4. Oversight

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
Data Monitoring Committee
No

5. Study Description

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).

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Transthyretin Amyloid Cardiomyopathy
Keywords
Tafamidis

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 4
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
53 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Chinese participants treated with Tafamidis
Arm Type
Experimental
Arm Description
treatment group with tafamidis
Intervention Type
Drug
Intervention Name(s)
Tafamidis
Other Intervention Name(s)
Vyndamax, Tafamidis free acid, PF 06291826
Intervention Description
61 mg, once daily, oral administration, for 12 months.
Primary Outcome Measure Information:
Title
Incidence of treatment-emergent adverse events (TEAEs) during the study
Time Frame
Baseline up to 28 days after the last dose (approximately 13 months)
Secondary Outcome Measure Information:
Title
Change from baseline in the 6 Minute Walk Distance (6MWD) at Month 6 and Month 12
Time Frame
Baseline, Month 6, Month 12
Title
The percentage of participants with TTR stabilization at each collection visit
Description
TTR stabilization is a Pharmacodynamic (PD) parameter, the blood samples will be analyzed using validated analytical methods.
Time Frame
Baseline, Months 1, Month 6, and Month 12
Title
Change from baseline in Kansas City Cardiomyopathy Questionnaire overall summary scores at Month 6 and Month 12
Description
Domain scores are transformed to a 0 to 100 range; higher scores indicate better health status
Time Frame
Baseline, Months 6, Month 12
Title
Plasma concentrations of tafamidis at each collection visit
Time Frame
Baseline, Month 1, Month 6, Month 12
Title
Change from baseline in N Terminal prohormone B type Natriuretic Peptide (NT-pro BNP) at Month 6 and Month 12
Time Frame
Baseline, Month 6, Month 12
Title
TTR concentration at each collection visit
Time Frame
Baseline, Months 1, Month 6, and Month 12
Title
Change from baseline in EuroQoL 5 Dimensions visual analogue scale (EQ-5D VAS) scores at Month 6 and Month 12
Description
the score range is from 0-100, higher scores indicate better health status
Time Frame
Baseline, Months 6, Month 12
Title
Change from baseline in 12-Item Short Form Survey (SF-12) scores at Month 6 and Month 12
Time Frame
Baseline, Months 6, Month 12
Title
Change from baseline in Change from baseline in EuroQol 5 Dimensions 5 Levels (EQ-5D-5L) index score at Month 6 and Month 12
Time Frame
Baseline, Months 6, Month 12

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Subject has documented ATTR-CM. For the reproductive criteria for male and female participants, please refer to relevant protocol sections. Exclusion Criteria: 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).
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Pfizer CT.gov Call Center
Organizational Affiliation
Pfizer
Official's Role
Study Director
Facility Information:
Facility Name
Peking University First Hospital
City
Beijing
State/Province
Beijing
ZIP/Postal Code
100034
Country
China
Facility Name
Peking University Third Hospital
City
Beijing
State/Province
Beijing
ZIP/Postal Code
100191
Country
China
Facility Name
Peking Union Medical College Hospital
City
Beijing
State/Province
Beijing
ZIP/Postal Code
100730
Country
China
Facility Name
Tongji Hospital, Tongji Medical College, Huazhong University of Science & Technology
City
Wuhan
State/Province
Hubei
ZIP/Postal Code
430030
Country
China
Facility Name
The Second Xiangya Hospital of Central South University
City
Changsha
State/Province
Hunan
ZIP/Postal Code
410011
Country
China
Facility Name
The First Affiliated Hospital of Dalian Medical University
City
Dalian
State/Province
Liaoning
ZIP/Postal Code
116014
Country
China
Facility Name
West China Hospital of Sichuan University
City
Chengdu
State/Province
Sichuan
ZIP/Postal Code
610041
Country
China
Facility Name
The Second Affiliated Hospital of Zhejiang University School of Medicine
City
Hangzhou
State/Province
Zhejiang
ZIP/Postal Code
310009
Country
China
Facility Name
Ruijin Hospital, Shanghai Jiaotong University School of Medicine
City
Shanghai
ZIP/Postal Code
200025
Country
China

12. IPD Sharing Statement

Plan to Share IPD
Yes
IPD Sharing 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.
IPD Sharing URL
https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests
Links:
URL
https://pmiform.com/clinical-trial-info-request?StudyID=B3461077
Description
To obtain contact information for a study center near you, click here.

Learn more about this trial

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

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