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Completed

NCT Number: NCT04108091

Vyndaqel Capsules Special Investigation (ATTR-CM)

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.

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Key information

Conditions

Transthyretin (TTR) Amyloid Cardiomyopathy Amyloid Neuropathies Amyloid Neuropathies, Familial Amyloidosis Amyloidosis, Familial Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Heredodegenerative Disorders, Nervous System Metabolic Diseases Metabolism, Inborn Errors Nervous System Diseases Neurodegenerative Diseases Neuromuscular Diseases Nutritional and Metabolic Diseases Peripheral Nervous System Diseases Proteostasis Deficiencies

Age range

18 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

Pfizer

Tokyo, Japan

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Patients administered Vyndaqel for the treatment of ATTR-CM

Exclusion criteria

  • N/A

Treatment and study plan

Treatment for TTR amyloidosis

Drug

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

Other names: Vyndaqel

Primary outcomes

  1. 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.

Secondary outcomes

  1. 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.

  2. 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.

Sponsors and collaborators

Lead sponsor

Pfizer

Industry

Registry information

Official study title

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

Important dates

Study start
2019
Primary completion
2025
Study completion
2025
First posted
Sep 27, 2019
Registry last updated
May 18, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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