Vyndaqel Drug Use Investigation (Regulatory Post Marketing Commitment Plan)

NCT02146378 | Completed Results FDA Drug

• 2 other identifiers: B3461042NCT02146378
• Study Type: observational
• Target: 500
• Locations: 0 countries
• Sites: N/A

Brief 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.

Conditions

Transthyretin Familial Amyloid Polyneuropathy

Outcome Measures

Primary Outcomes (1)

• Percentage of Participants With ADRs in This Study

  An adverse drug reaction (ADR) was any untoward medical occurrence attributed to Vyndaqel capsules 20mg in a participant who received Vyndaqel capsules 20mg. A serious adverse drug reaction (SADR) was ADR resulting in any of the following outcomes or deemed significant for any other reason: results in death; is life-threatening; requires inpatient hospitalization or prolongation of hospitalization; results in persistent or significant disability/incapacity (substantial disruption of the ability to conduct normal life functions); results in congenital anomaly/birth defect. Relatedness to Vyndaqel capsules 20mg was assessed by the physician.

  3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.)

Secondary Outcomes (7)

• Change From Baseline in the NIS-LL Score

  Week 78, Week 156

• Change From Baseline in the TQOL Score of the Norfolk QOL-DN

  Week 78, Week 156

• Change From Baseline in mBMI

  Week 26, Week 52, Week 78, Week 104, Week 130, Week 156

• Time Course of Ambulatory Status

  Week 156

• Change From Baseline in the Total Neurological Assessment Score

  Week 52, Week 104, Week 156

Study Arms (1)

Vyndaqel

Drug: Vyndaqel

Interventions

Vyndaqel DRUG

20mg/day

Vyndaqel

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Probability Sample

Study Population

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

You may qualify if:

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

You may not qualify if:

• Patients not receive Vyndaqel.

Sponsors & Collaborators

• Pfizer: lead

Related Publications (2)

• 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.

  PMID: 40495907 DERIVED

• 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.

  PMID: 32800381 DERIVED

MeSH Terms

Interventions

tafamidis

Results Point of Contact

• Title: Pfizer ClinicalTrials.gov Call Center
• Organization: Pfizer, Inc.

ClinicalTrials.gov_Inquiries@pfizer.com

1-800-718-1021

Study Officials

• Pfizer CT.gov Call Center

  Pfizer

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: OTHER
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 21, 2014
• First Posted: May 23, 2014
• Study Start: January 10, 2014
• Primary Completion: March 10, 2023
• Study Completion: March 10, 2023
• Last Updated: February 3, 2025
• Results First Posted: February 3, 2025

Record last verified: 2025-01

Data Sharing

• IPD Sharing: Will not share