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

Transthyretin Amyloid Cardiomyopathy Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04814186
• Other study ID #: B3461077
• Status: Completed
• Phase: Phase 4
• Start date: July 22, 2021
• Est. completion date: October 16, 2023

Study information

• Verified date: November 2023
• Source: Pfizer
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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).

Recruitment information / eligibility

• Status: Completed
• Enrollment: 53
• Est. completion date: October 16, 2023
• Est. primary completion date: October 16, 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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).

Related Conditions & MeSH terms

• Amyloidosis
• Cardiomyopathies
• Transthyretin Amyloid Cardiomyopathy

Intervention

Drug:
• Tafamidis
61 mg, once daily, oral administration, for 12 months.

Locations

Country	Name	City	State
China	Peking Union Medical College Hospital	Beijing	Beijing
China	Peking University First Hospital	Beijing	Beijing
China	Peking University Third Hospital	Beijing	Beijing
China	The Second Xiangya Hospital of Central South University	Changsha	Hunan
China	West China Hospital of Sichuan University	Chengdu	Sichuan
China	The First Affiliated Hospital of Dalian Medical University	Dalian	Liaoning
China	The Second Affiliated Hospital of Zhejiang University School of Medicine	Hangzhou	Zhejiang
China	Ruijin Hospital, Shanghai Jiaotong University School of Medicine	Shanghai
China	Tongji Hospital, Tongji Medical College, Huazhong University of Science & Technology	Wuhan	Hubei

Sponsors (1)

Lead Sponsor	Collaborator
Pfizer

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Incidence of treatment-emergent adverse events (TEAEs) during the study		Baseline up to 28 days after the last dose (approximately 13 months)
Secondary	Change from baseline in the 6 Minute Walk Distance (6MWD) at Month 6 and Month 12		Baseline, Month 6, Month 12
Secondary	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
Secondary	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
Secondary	Plasma concentrations of tafamidis at each collection visit		Baseline, Month 1, Month 6, Month 12
Secondary	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
Secondary	TTR concentration at each collection visit		Baseline, Months 1, Month 6, and Month 12
Secondary	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
Secondary	Change from baseline in 12-Item Short Form Survey (SF-12) scores at Month 6 and Month 12		Baseline, Months 6, Month 12
Secondary	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

External Links

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