Vyndaqel Drug Use Investigation (Regulatory Post Marketing Commitment Plan)

Vyndaqel Capsules Special Investigation -Investigation on Long-term Use-

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 Polyneuropathy
• 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

• 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	Measure Description	Time Frame
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 Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change From Baseline in the NIS-LL Score	The amounts of change from baseline in the lower limbs (NIS-LL) was investigated for assessment of neurological function.The NIS-LL (Lower Limb) assessed muscle weakness, reflexes, and sensation (possible range: 0 to 88). Summary statistics at each evaluation time point and their amounts of change from the start of treatment with Vyndaqel capsules 20mg were calculated.Higher scores indicate more impaired neurological function.The mean changes from baseline in the NIS-LL at Week 78 and Week 156 were presented with standard deviation.	Week 78, Week 156
Change From Baseline in the TQOL Score of the Norfolk QOL-DN	The amounts of change from baseline in the TQOL score of the QOL-DN were used to assess quality of life .The Norfolk Quality of Life-Diabetic Neuropathy (QOL-DN) questionnaire which consists of symptoms items (1 to 7) and activities of daily living items (8 to 35) . Total Quality of Life (TQOL) (possible range: -4 to 136) in the QOL-DN was evaluated. Summary statistics at each evaluation time point and their amounts of change from the start of treatment with Vyndaqel capsules 20mg were calculated. Higher scores indicate decreased QOL.The mean changes from baseline in the TQOL score at Week 78 and Week 156 were presented with standard deviation.	Week 78, Week 156
Change From Baseline in mBMI	Summary statistics at each evaluation time point and their amounts of change from the start of treatment with Vyndaqel capsules 20mg were calculated. Higher mBMI scores indicate better nutrition status.The mean changes from baseline in modified Body Mass Index (mBMI) at Week 26, Week 52, Week 78, Week 104, Week 130, and Week 156 were presented with standard deviation.mBMI can be calculated by multiplying BMI by the serum albumin level.In Transthyretin Familial Amyloid Polyneuropathy patients, oedema and subsequent weight gain may occur, but weight changes can be corrected with a modified BMI.	Week 26, Week 52, Week 78, Week 104, Week 130, Week 156
Time Course of Ambulatory Status	The percentage of participants were calculated in each group of ambulatory status change from the start of treatment with Vyndaqel capsules 20mg to the final evaluation time point (week 156). Ambulatory status (0, 1, 2, 3a, 3b, 4) was assessed using the ambulatory ability scale. The ambulatory ability scale in the polyneuropathy dysfunction score was defined as follows. Ambulatory status 0：Walking ability is Good. Ambulatory status 1：Walking ability is able to walk without difficulty despite of sensory disorder in the lower extremities. Ambulatory status. Ambulatory status 2：Walking ability is able to walk without assistance despite of some difficulties. Ambulatory status 3a：Walking ability is able to walk with one stick or crutch. Ambulatory status 3b：Walking ability is able to walk with two sticks or crutches. Ambulatory status 4: Walking ability is unable to walk, restricted to wheelchair or bedridden.	Week 156
Change From Baseline in the Total Neurological Assessment Score	The total score of neurological assessment (possible range: 0 to 294), scores of Motor, Reflexes and sensation were evaluated and summary statistics at each evaluation time point and their amounts of change from the start of treatment with Vyndaqel capsules 20mg were calculated. Higher scores indicate more decreased function.The mean changes from baseline in the total neurological assessment at Week 52 , Week 104, and Week 156 were presented with standard deviation.	Week 52, Week 104, Week 156
Assessment of Survival Time for Death From Any Cause	By setting death from any cause as events, the survival probability was estimated using the Kaplan-Meier method that treats cases who didn't have events within the observation period as censoring.	3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.)
Assessment of Survival Time for Death Associated With ATTR-PN	By setting death associated with transthyretin familial amyloid polyneuropathy (ATTR-PN) as events, the survival probability was estimated using the Kaplan-Meier method that treats cases who didn't have events within the observation period as censoring.	3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.)

Collaborators and Investigators

• Sponsor: Pfizer
• Investigators: Study Director: Pfizer CT.gov Call Center, 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 (Estimated): May 23, 2014

Study Record Updates

• Last Update Posted (Actual): March 25, 2025
• Last Update Submitted That Met QC Criteria: January 28, 2025
• Last Verified: January 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Nervous System Diseases; Neuromuscular Diseases; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Metabolic Diseases; Peripheral Nervous System Diseases; Neurodegenerative Diseases; Heredodegenerative Disorders, Nervous System; Proteostasis Deficiencies; Amyloidosis, Familial; Amyloidosis; Polyneuropathies; Amyloid Neuropathies; Amyloid Neuropathies, Familial
• Other Study ID Numbers: B3461042; NCT02146378 (Registry Identifier: ClinicalTrials.gov)

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