N/A
Completed N=402
Vyndaqel Drug Use Investigation (Regulatory Post Marketing Commitment Plan)
Transthyretin Familial Amyloid Polyneuropathy
Source: ClinicalTrials.gov NCT02146378 ↗
Enrolled (actual)
402
Serious AEs
31.0%
Results posted
Feb 2025
Primary outcomePrimary: Percentage of Participants With ADRs in This Study — 58; 12 Participants
Summary
The purpose of this study is to understand safety (e.g., occurrence of adverse drug reactions [ADRs]) and efficacy data on the long-term use of Vyndaqel Capsules (hereinafter referred to as Vyndaqel) in all patients who received this drug under actual use conditions after its marketing.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percentage of Participants With ADRs in This Study |
58; 12 | — |
| SECONDARY Change From Baseline in the NIS-LL Score |
1.45; 1.88 | — |
| SECONDARY Change From Baseline in the TQOL Score of the Norfolk QOL-DN |
3.44; 6.75 | — |
| SECONDARY Change From Baseline in mBMI |
-6.17; -5.63; -28.57; -47.26; 11.59; -32.81 | — |
| SECONDARY Time Course of Ambulatory Status |
15.0; 3.3; 2.8; 0.5; 0.5; 1.9 | — |
| SECONDARY Change From Baseline in the Total Neurological Assessment Score |
1.94; 8.36; 4.70 | — |
| SECONDARY Assessment of Survival Time for Death From Any Cause |
95.57; 87.98 | — |
| SECONDARY Assessment of Survival Time for Death Associated With ATTR-PN |
97.41; 94.46 | — |
Eligibility Criteria
Inclusion Criteria
The subjects of this surveillance are all patients who received Vyndaqel.
Exclusion Criteria
Patients not receive Vyndaqel.
Data sourced from ClinicalTrials.gov (NCT02146378). 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. Informational only — not medical advice.