Vyndaqel Drug Use Investigation (Regulatory Post Marketing Commitment Plan)

VYNDAQEL DRUG USE INVESTIGATION (REGULATORY POST MARKETING COMMITMENT PLAN)

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.

Study Overview

• Status: Completed
• Conditions: Transthyretin Familial Amyloid Poluneuropathy
• Intervention / Treatment: Drug: Vyndaqel

Detailed Description

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

• Study Type: Observational
• Enrollment (Actual): 500

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Probability Sample

Study Population

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

Description

Inclusion Criteria:

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

Exclusion Criteria:

Patients not receive Vyndaqel.

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Vyndaqel	Drug: Vyndaqel; 20mg/day

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
The incidence of adverse drug reaction in this study	3 years

Secondary Outcome Measures

Outcome Measure	Time Frame
Change from baseline on the following scale; NIS, QOL-DN, mBMI	3 years

Collaborators and Investigators

• Sponsor: Pfizer

Publications and helpful links

General Publications

• 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.

Helpful Links

• To obtain contact information for a study center near you, click here.

Study record dates

Study Major Dates

• Study Start (Actual): January 10, 2014
• Primary Completion (Actual): March 10, 2023
• Study Completion (Actual): March 10, 2023

Study Registration Dates

• First Submitted: May 21, 2014
• First Submitted That Met QC Criteria: May 21, 2014
• First Posted (Estimate): May 23, 2014

Study Record Updates

• Last Update Posted (Actual): March 23, 2023
• Last Update Submitted That Met QC Criteria: March 22, 2023
• Last Verified: March 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Metabolic Diseases; Proteostasis Deficiencies; Amyloidosis
• Other Study ID Numbers: B3461042

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO
• IPD Plan Description: Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No