N/A
N=1,214
Vyndaqel Capsules Special Investigation (ATTR-CM)
Transthyretin (TTR) Amyloid Cardiomyopathy
Bottom Line
View on ClinicalTrials.gov: NCT04108091 ↗Enrolled (actual)
1,214
Serious AEs
23.1%
Results posted
May 2026
Primary outcome: Primary: Number of Participants With Adverse Drug Reactions — 45; 7 Participants
Study Design & Population
- Study type
- Observational
- Phase
- N/A
- Interventions
- Treatment for TTR amyloidosis (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Pfizer
- Primary completion
- Mar 2025
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With Adverse Drug Reactions |
45; 7 | — |
| SECONDARY Percentage of Participants Who Died |
3.6; 1.8 | — |
| SECONDARY NYHA Classification Changes |
25.0; 23.8; 3.8; 1.3; 46.3; 5.0 | — |
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.
Eligibility Criteria
Inclusion Criteria
- Patients administered Vyndaqel for the treatment of ATTR-CM
Exclusion Criteria
- N/A
Data sourced from ClinicalTrials.gov (NCT04108091). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.