NCT04108091

Brief Summary

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
1,214

participants targeted

Target at P75+ for all trials

Timeline
Completed

Started Oct 2019

Longer than P75 for all trials

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

September 26, 2019

Completed
1 day until next milestone

First Posted

Study publicly available on registry

September 27, 2019

Completed
6 days until next milestone

Study Start

First participant enrolled

October 3, 2019

Completed
5.5 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

March 27, 2025

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

March 27, 2025

Completed
1.1 years until next milestone

Results Posted

Study results publicly available

May 18, 2026

Completed
Last Updated

May 18, 2026

Status Verified

April 1, 2026

Enrollment Period

5.5 years

First QC Date

September 26, 2019

Results QC Date

March 19, 2026

Last Update Submit

April 24, 2026

Conditions

Transthyretin (TTR) Amyloid Cardiomyopathy

Keywords

transthyretin amyloid cardiomyopathy;TTR Cardiomyopathy;Transthyretin (TTR) Amyloid Cardiomyopathy;Vyndaqel;Tafamidis meglumine;ATTR-CM;Vynmac;

Outcome Measures

Primary Outcomes (1)

  • 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 Outcomes (2)

  • Percentage of Participants Who Died

    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

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

Study Arms (1)

Treatment for TTR amyloidosis

Transthyretin amyloid cardiomyopathy (wild-type or variants) patients administered Vyndaqel and Vynmac.

Drug: Treatment for TTR amyloidosis

Interventions

Treatment for TTR amyloidosisDRUG

The usual adult dose is 80 mg of Tafamidis Meglumine orally once daily. The dose may be reduced if not tolerated.

Also known as: Vyndaqel
Treatment for TTR amyloidosis

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)
Sampling MethodProbability Sample
Study Population

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

You may qualify if:

  • Patients administered Vyndaqel for the treatment of ATTR-CM

You may not qualify if:

  • N/A

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Pfizer

Tokyo, Japan

Location

Related Links

MeSH Terms

Conditions

Amyloid Neuropathies, Familial

Interventions

Therapeuticstafamidis

Condition Hierarchy (Ancestors)

Heredodegenerative Disorders, Nervous SystemNeurodegenerative DiseasesNervous System DiseasesAmyloid NeuropathiesPeripheral Nervous System DiseasesNeuromuscular DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesAmyloidosis, FamilialMetabolism, Inborn ErrorsMetabolic DiseasesNutritional and Metabolic DiseasesAmyloidosisProteostasis Deficiencies

Results Point of Contact

Title
Pfizer ClinicalTrials.gov Call Center
Organization
Pfizer, Inc.
ClinicalTrials.gov_Inquiries@pfizer.com
1-800-718-1021

Study Officials

  • Pfizer CT.gov Call Center

    Pfizer

    STUDY DIRECTOR

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
OTHER
Restrictive Agreement
Yes

Study Design

Study Type
observational
Observational Model
CASE ONLY
Time Perspective
OTHER
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

September 26, 2019

First Posted

September 27, 2019

Study Start

October 3, 2019

Primary Completion

March 27, 2025

Study Completion

March 27, 2025

Last Updated

May 18, 2026

Results First Posted

May 18, 2026

Record last verified: 2026-04

Data Sharing

IPD Sharing
Will not share

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.

Locations

JP(1)