N/A
N=402
Vyndaqel Drug Use Investigation (Regulatory Post Marketing Commitment Plan)
Transthyretin Familial Amyloid Polyneuropathy
Bottom Line
View on ClinicalTrials.gov: NCT02146378 ↗Enrolled (actual)
402
Serious AEs
31.0%
Results posted
Feb 2025
Primary outcome: Primary: Percentage of Participants With ADRs in This Study — 58; 12 Participants
Study Design & Population
- Study type
- Observational
- Phase
- N/A
- Interventions
- Vyndaqel (Drug)
- Age
- Pediatric, Adult, Older Adult
- Sex
- All
- Sponsor
- Pfizer
- Primary completion
- Mar 2023
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 | — |
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.
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.