NCT01994889

Safety and Efficacy of Tafamidis in Patients With Transthyretin Cardiomyopathy

A MULTICENTER, INTERNATIONAL, PHASE 3, DOUBLE-BLIND, PLACEBO-CONTROLLED, RANDOMIZED STUDY TO EVALUATE THE EFFICACY, SAFETY, AND TOLERABILITY OF DAILY ORAL DOSING OF TAFAMIDIS MEGLUMINE (PF-06291826) 20 MG OR 80 MG IN COMPARISON TO PLACEBO IN SUBJECTS DIAGNOSED WITH TRANSTHYRETIN CARDIOMYOPATHY (TTR-CM)

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 441 (actual)
• Sponsor: Pfizer · Industry
• Sex: All
• Age: 18 Years – 90 Years
• Healthy volunteers: Not accepted

Summary

This Phase 3 study will investigate the efficacy, safety and tolerability of an oral daily dose of 20 mg or 80 mg tafamidis meglumine capsules compared to placebo in subjects with either transthyretin genetic variants or wild-type transthyretin resulting in amyloid cardiomyopathy.

Detailed description

Phase 3, multicenter, global, three-arm, parallel design, placebo-controlled, double-blind, randomized study to determine efficacy, safety and tolerability of tafamidis on clinical outcomes (all-cause mortality and frequency of cardiovascular-related hospitalizations) in subjects with either transthyretin genetic variants or wild-type transthyretin resulting in amyloid cardiomyopathy.

Conditions

• Transthyretin (TTR) Amyloid Cardiomyopathy

Interventions

Type	Name	Description
DRUG	Tafamidis	Tafamidis 20 mg in soft gel capsules administered once a day for 30 months
DRUG	Tafamidis	Tafamidis 80 mg in soft gel capsules administered once a day for 30 months
DRUG	Placebo	Placebo in soft gel capsules administered once a day for 30 months

Timeline

• Start date: 2013-12-09
• Primary completion: 2018-02-07
• Completion: 2018-02-07
• First posted: 2013-11-26
• Last updated: 2019-04-24
• Results posted: 2019-04-03

Locations

80 sites across 13 countries: United States, Belgium, Brazil, Canada, Czechia, France, Germany, Italy, Japan, Netherlands, Spain, Sweden, United Kingdom

Regulatory

• FDA-regulated drug study