Transthyretin (TTR) Amyloid CardiomyopathyNCT04108091

Vyndaqel Capsules Special Investigation (ATTR-CM)

An observational study of Transthyretin (TTR) Amyloid Cardiomyopathy, run by Pfizer at 1 site in Japan.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Trial 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. Read more about this study

Who is it for?: Ages 18 years and older
Conditions the study is for: Transthyretin (TTR) Amyloid Cardiomyopathy
What is being tested?: Treatment for TTR amyloidosis
Type of treatment: Pill or oral medicine
How many people: 1,214 participants
Where: 1 siteJapan
Study dates: 3 Oct 2019to 27 Mar 2025
Phase: Observational
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 18 years and older
All sexes
No healthy volunteers
Adult, Older adult

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

Who may be able to join

Patients administered Vyndaqel for the treatment of ATTR-CM

Who cannot join

Trial locations and contacts

1 site in 1 country.

Japan1 site

Pfizer
Tokyo

Other trials in Japan

About this study

Official title: Vyndaqel Capsules Special Investigation - Investigation on Patients With Transthyretin Amyloid Cardiomyopathy

Read the 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.

Conditions: Transthyretin (TTR) Amyloid Cardiomyopathy
Being tested: Treatment for TTR amyloidosis
Keywords: transthyretin amyloid cardiomyopathy;, TTR Cardiomyopathy;, Transthyretin (TTR) Amyloid Cardiomyopathy;, Vyndaqel;, Tafamidis meglumine;, ATTR-CM;, Vynmac;
Medical subject headings: Amyloid Neuropathies, Familial

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

Treatment for TTR amyloidosisDrug
Also called Vyndaqel
The usual adult dose is 80 mg of Tafamidis Meglumine orally once daily. The dose may be reduced if not tolerated.
Treatment for TTR amyloidosisDrug
Also called Vynmac
The usual adult dose is 61 mg of tafamidis orally once daily.

Study groups in NCT04108091
Study group	Role	What they receive
Treatment for TTR amyloidosis		Transthyretin amyloid cardiomyopathy (wild-type or variants) patients administered Vyndaqel and Vynmac. Receives: Drug: Treatment for TTR amyloidosis

What is being measured

The primary outcome is the main question the study is designed to answer.

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.
Measured: 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
The proportion of participants who died from any cause and from cardiovascular-related causes during the observation period was assessed.
Measured: 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.
Measured: 30 months,Participants who discontinued this drug during the observation period, the observation period was until the first visit after the date of discontinuation.

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

3 Oct 201927 Mar 2025

First posted27 Sep 2019
Study start3 Oct 2019
Primary completion27 Mar 2025
Study completion27 Mar 2025

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Other trials for Transthyretin (TTR) Amyloid CardiomyopathyStudies of the same condition, open and under way.
More trials run by PfizerThe sponsor's other registered studies.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Observational
Observational model: Case only
Time perspective: Other
Enrolment: 1,214 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT04108091
Sponsor's protocol ID: B3461064
Other IDs: NCT04108091
Status last verified: Apr 2026
First submitted: 26 Sep 2019
Last updated: 18 May 2026
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No
Individual data sharing: No: 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.

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

Active, not recruitingObservational study
A Retrospective Observational Study of Epidemiology and Outcomes in Transthyretin Amyloid Cardiomyopathy in Germany
ATTR-CM (Transthyretin Amyloid Cardiomyopathy)
Active, not recruitingPhase 3
EPIC-ATTR: A Study to Evaluate the Effect of Eplontersen on the Transthyretin Reduction and Long-term Safety in Chinese Subjects With Transthyretin Amyloid Cardiomyopathy
Transthyretin Amyloid Cardiomyopathy
RecruitingObservational study
Essen Amyloidosis Registry
Amyloidosis; Systemic, Amyloidosis Cardiac
Active, not recruitingPhase 3
A Phase 3 Study of NTLA-2001 in ATTRv-PN
Neuromuscular Disease, Neuromuscular Diseases (NMD)
RecruitingPhase 3
MAGNITUDE: A Phase 3 Study of NTLA-2001 in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR-CM)
Transthyretin Amyloidosis (ATTR) With Cardiomyopathy

All Transthyretin (TTR) Amyloid Cardiomyopathy trials

Study at a glance

From the registry record, last updated by the sponsor on 18 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Vyndaqel Capsules Special Investigation - Investigation on Patients With Transthyretin Amyloid Cardiomyopathy
ClinicalTrials.gov ID: NCT04108091
Acronym / sponsor ID: B3461064
Conditions: Transthyretin (TTR) Amyloid Cardiomyopathy
Keywords: transthyretin amyloid cardiomyopathy;, TTR Cardiomyopathy;, Transthyretin (TTR) Amyloid Cardiomyopathy;, Vyndaqel;, Tafamidis meglumine;, ATTR-CM;, Vynmac;
Study type: Observational
Recruitment status: Completed
Enrolment: 1,214 participants (actual)
Lead sponsor: PfizerIndustry (pharma, biotech, devices)
Study start: 3 Oct 2019 (actual)
Primary completion: 27 Mar 2025 (actual)
Study completion: 27 Mar 2025 (actual)
First posted: 27 Sep 2019 (actual)
Results: Posted 18 May 2026

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Detailed description

Study design

Observational model: Case only
Time perspective: Other
Number of arms: 1

Arm	Type	Receives
Treatment for TTR amyloidosis	–	Drug: Treatment for TTR amyloidosisTransthyretin amyloid cardiomyopathy (wild-type or variants) patients administered Vyndaqel and Vynmac.

Who may qualify

Ages: 18 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult
Study population: Patients who are administered with this drug for the treatment of ATTR-CM

Key inclusion criteria (1 listed)

Patients administered Vyndaqel for the treatment of ATTR-CM

Key exclusion criteria (1 listed)

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
Treatment for TTR amyloidosisAlso: VyndaqelThe usual adult dose is 80 mg of Tafamidis Meglumine orally once daily. The dose may be reduced if not tolerated.	Drug	Treatment for TTR amyloidosis
Treatment for TTR amyloidosisAlso: VynmacThe usual adult dose is 61 mg of tafamidis orally once daily.	Drug	Treatment for TTR amyloidosis

Primary outcome

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.

2 secondary outcomes

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.
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.

Results and safety

Results posted to the registry on 18 May 2026, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Vyndaqel Capsules 20mg (Tafamidis Meglumine) and Vynmac Capsules 61mg(Tafamidis)	1,214	873

Primary outcome: 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.. Count of participants. Unit: Participants

Measure	Vyndaqel Capsules 20mg (Tafamidis Meglumine)and Vynmac Capsules 61mg(Tafamidis) n = 873
ADR	45
Serious ADR	7

Population analysed: The safety analysis set comprised of participants who satisfied the inclusion criteria and had received Vyndaqel capsules 20mg or Vynmac capsules 61mg at least once. Participants with protocol violation, or lacking informed consent for publication of study results, adverse event information (no revisits), or administration information were excluded.

2 secondary outcomes are reported in the full results.

Adverse events

Group	Serious adverse events	Other adverse events	Deaths
Vyndaqel Capsules 20mg (Tafamidis Meglumine)and Vynmac Capsules 61mg(Tafamidis)	202 of 873 (23.1%)	101 of 873 (11.6%)	31 of 873

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 18 May 2026.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
To obtain contact information for a study center near you, click here.Link provided by the sponsor.
Individual participant dataSharing planned: No. 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.

Related trials for Transthyretin (TTR) Amyloid Cardiomyopathy

Active, not recruitingObservational study
A Retrospective Observational Study of Epidemiology and Outcomes in Transthyretin Amyloid Cardiomyopathy in Germany
ATTR-CM (Transthyretin Amyloid Cardiomyopathy)
Active, not recruitingPhase 3
EPIC-ATTR: A Study to Evaluate the Effect of Eplontersen on the Transthyretin Reduction and Long-term Safety in Chinese Subjects With Transthyretin Amyloid Cardiomyopathy
Transthyretin Amyloid Cardiomyopathy
RecruitingObservational study
Essen Amyloidosis Registry
Amyloidosis; Systemic, Amyloidosis Cardiac
Active, not recruitingPhase 3
A Phase 3 Study of NTLA-2001 in ATTRv-PN
Neuromuscular Disease, Neuromuscular Diseases (NMD)
RecruitingPhase 3
MAGNITUDE: A Phase 3 Study of NTLA-2001 in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR-CM)
Transthyretin Amyloidosis (ATTR) With Cardiomyopathy

Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Pfizer CT.gov Call Center study director

Pfizer

1 site in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

An observational study of Transthyretin (TTR) Amyloid Cardiomyopathy, run by Pfizer at 1 site in Japan.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 1 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 18 May 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in Japan. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

Are results available for this study?

Yes. Results are posted in the registry (first posted 18 May 2026). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

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.). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04108091.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 May 2026.