Transthyretin (TTR) Amyloid Cardiomyopathy
Conditions
Keywords
transthyretin amyloid cardiomyopathy;, TTR Cardiomyopathy;, Transthyretin (TTR) Amyloid Cardiomyopathy;, Vyndaqel;, Tafamidis meglumine;, ATTR-CM;, Vynmac;
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.
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.
Interventions
DRUGTreatment for TTR amyloidosis
The usual adult dose is 80 mg of Tafamidis Meglumine orally once daily. The dose may be reduced if not tolerated.
Sponsors
Pfizer
Study design
Observational model
CASE_ONLY
Time perspective
OTHER
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Patients administered Vyndaqel for the treatment of ATTR-CM
Exclusion criteria
* N/A
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants With Adverse Drug Reactions | 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
| Measure | Time frame | Description |
|---|---|---|
| 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. | The proportion of participants who died from any cause and from cardiovascular-related causes during the observation period was assessed. |
| 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. | 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. |
Countries
Japan
Contacts
STUDY_DIRECTORPfizer CT.gov Call Center
Pfizer
Baseline characteristics
| Characteristic | — |
|---|---|
| Age, Customized ≥15 and <65 years | 36 Participants |
| Age, Customized <15 years | 0 Participants |
| Age, Customized ≥65 years | 837 Participants |
| Sex: Female, Male Female | 95 Participants |
| Sex: Female, Male Male | 778 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 31 / 873 |
| other Total, other adverse events | 101 / 873 |
| serious Total, serious adverse events | 202 / 873 |
Outcome results
None listed