Vyndaqel Capsules Special Investigation (ATTR-CM)

April 24, 2026 updated by: Pfizer

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

Intervention / Treatment

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

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Japan
      • Tokyo, Japan
        • Pfizer

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

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

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

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

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
Time Frame: 30 months,Participants who discontinued this drug during the observation period, the observation period was until the first visit after the date of discontinuation.
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
Time Frame: 30 months,Participants who discontinued this drug during the observation period, the observation period was until the first visit after the date of discontinuation.
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
Time Frame: 30 months,Participants who discontinued this drug during the observation period, the observation period was until the first visit after the date of discontinuation.

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

This is where you will find people and organizations involved with this study.

Sponsor

Pfizer

Investigators

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

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

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

Additional Relevant MeSH Terms

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

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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