Vyndaqel Capsules Special Investigation (ATTR-CM)

Vyndaqel Capsules Special Investigation - Investigation on Patients With Transthyretin Amyloid Cardiomyopathy

Secondary Data Collection : To confirm the safety and effectiveness profiles under the actual medical practice of Vyndaqel in Japan. This study is conducted in accordance with the protocol even when Vynmac is used, and information the use of Vynmac during the observation period is also collected.

Study Overview

• Status: Completed
• Conditions: Transthyretin (TTR) Amyloid Cardiomyopathy
• Intervention / Treatment: Drug: Treatment for TTR amyloidosis; Drug: Treatment for TTR amyloidosis

Detailed Description

To comprehend information on the long-term safety (e.g., onset status of adverse reactions), etc. of patients who are treated with Vyndaqel for the treatment of transthyretin amyloid cardiomyopathy. When Vynmac is used, conduct the study to grasp information on safety (e.g., onset status of adverse reactions),etc. during the observation period.

• Study Type: Observational
• Enrollment (Actual): 1214

Contacts and Locations

Study Locations

• Japan
  • Tokyo, Japan
    • Pfizer

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 1 second and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

• Sampling Method: Probability Sample

Study Population

Patients who are administered with this drug for the treatment of ATTR-CM

Description

Inclusion Criteria:

• Patients administered Vyndaqel for the treatment of ATTR-CM

Exclusion Criteria:

• N/A

Study Plan

How is the study designed?

Design Details

• Observational Models: Case-Only
• Time Perspectives: Other

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Treatment for TTR amyloidosis; Transthyretin amyloid cardiomyopathy (wild-type or variants) patients administered Vyndaqel and Vynmac.	Drug: Treatment for TTR amyloidosis; The usual adult dose is 80 mg of Tafamidis Meglumine orally once daily. The dose may be reduced if not tolerated.; Other Names: Vyndaqel; Drug: Treatment for TTR amyloidosis; The usual adult dose is 61 mg of tafamidis orally once daily.; Other Names: Vynmac

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of Participants With Adverse Drug Reactions	An ADR was any untoward medical occurrence attributed to Vyndaqel capsules 20mg or Vynmac capsules 61mg in a participant who received this drug. A serious ADR was ADR resulting in any of the following outcomes or deemed significant for any other reason: death; life-threatening; inpatient hospitalization or prolongation of hospitalization; persistent or significant disability/incapacity; and congenital anomaly/birth defect. Relatedness to Vyndaqel capsules 20mg or Vynmac capsules 61mg was assessed without distinction.	30 months,Participants who discontinued this drug during the observation period, the observation period was until the first visit after the date of discontinuation.

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Percentage of Participants Who Died	The proportion of participants who died from any cause and from cardiovascular-related causes during the observation period was assessed.	30 months,Participants who discontinued this drug during the observation period, the observation period was until the first visit after the date of discontinuation.
NYHA Classification Changes	Changes in NYHA functional classification at Month 30 compared with baseline were summarized using cross-tabulation and expressed as percentages, with the number of participants in each NYHA class at baseline as the denominator. The NYHA class ranges from Class 1 to 4, with lower class numbers indicating better functional status; Class 1 indicates no limitation of physical activity, whereas Class 4 indicates symptoms at rest.	30 months,Participants who discontinued this drug during the observation period, the observation period was until the first visit after the date of discontinuation.

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): October 3, 2019
• Primary Completion (Actual): March 27, 2025
• Study Completion (Actual): March 27, 2025

Study Registration Dates

• First Submitted: September 26, 2019
• First Submitted That Met QC Criteria: September 26, 2019
• First Posted (Actual): September 27, 2019

Study Record Updates

• Last Update Posted (Actual): May 18, 2026
• Last Update Submitted That Met QC Criteria: April 24, 2026
• Last Verified: April 1, 2026

More Information

Terms related to this study

• Keywords: transthyretin amyloid cardiomyopathy;; TTR Cardiomyopathy;; Transthyretin (TTR) Amyloid Cardiomyopathy;; Vyndaqel;; Tafamidis meglumine;; ATTR-CM;; Vynmac
• Additional Relevant MeSH Terms: Nervous System Diseases; Neuromuscular Diseases; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Metabolic Diseases; Peripheral Nervous System Diseases; Neurodegenerative Diseases; Heredodegenerative Disorders, Nervous System; Proteostasis Deficiencies; Amyloid Neuropathies; Amyloidosis, Familial; Amyloidosis; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Nutritional and Metabolic Diseases; Amyloid Neuropathies, Familial; Therapeutics; tafamidis
• Other Study ID Numbers: B3461064; NCT04108091 (Registry Identifier: ClinicalTrials.gov)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO
• 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: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No