Vyndaqel Drug Use Investigation (Regulatory Post Marketing Commitment Plan)

NCT ID: NCT02146378

Last Updated: 2025-02-03

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Total Enrollment

500 participants

Study Classification

OBSERVATIONAL

Study Start Date

2014-01-10

Study Completion Date

2023-03-10

Brief Summary

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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.

Detailed Description

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Both prospective and retrospective is acceptable All the patients whom an investigator prescribes Vyndaqel should be registered

Conditions

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Transthyretin Familial Amyloid Polyneuropathy

Study Design

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Observational Model Type

CASE_ONLY

Study Time Perspective

OTHER

Study Groups

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Vyndaqel

Vyndaqel

Intervention Type DRUG

20mg/day

Interventions

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Vyndaqel

20mg/day

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

The subjects of this surveillance are all patients who received Vyndaqel.

Exclusion Criteria

Patients not receive Vyndaqel.
Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Pfizer

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Pfizer CT.gov Call Center

Role: STUDY_DIRECTOR

Pfizer

References

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Konishi H, Abe H, Matsumoto N, Endo Y, Sekijima Y, Ueda M, Ando Y. Real-World Utilization Patterns, Safety, and Efficacy of Tafamidis in Patients With Hereditary Transthyretin Amyloidosis in Japan. Curr Ther Res Clin Exp. 2025 Apr 14;102:100793. doi: 10.1016/j.curtheres.2025.100793. eCollection 2025.

Reference Type DERIVED
PMID: 40495907 (View on PubMed)

Ishii T, Hirano Y, Matsumoto N, Takata A, Sekijima Y, Ueda M, Ando Y. Characteristics of Patients with Hereditary Transthyretin Amyloidosis and an Evaluation of the Safety of Tafamidis Meglumine in Japan: An Interim Analysis of an All-case Postmarketing Surveillance. Clin Ther. 2020 Sep;42(9):1728-1737.e6. doi: 10.1016/j.clinthera.2020.07.001. Epub 2020 Aug 12.

Reference Type DERIVED
PMID: 32800381 (View on PubMed)

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

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NCT02146378

Identifier Type: REGISTRY

Identifier Source: secondary_id

B3461042

Identifier Type: -

Identifier Source: org_study_id

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