Clinical Trial: Safety and Efficacy of Tafamidis in Patients With Transthyretin Cardiomyopathy

Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional

Official Title: 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) 2

Brief 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 Summary: 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.
Sponsor: Pfizer

Current Primary Outcome: All-cause mortality and frequency of cardiovascular-related hospitalization [ Time Frame: From Baseline to Month 30 ]

A hierarchical combination of the endpoints for a pooled analysis of the tafamidis treatment groups in comparison to placebo


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • 6-Minute Walk Test (6MWT). [ Time Frame: From Baseline to Month 30 ]
    Change from Baseline to Month 30 in the total distance walked in 6 minutes.
  • Kansas City Cardiomyopathy Questionnaire (KCCQ) [ Time Frame: From Baseline to Month 30 ]
    Change from Baseline to Month 30 in the Kansas City Cardiomyopathy Questionnaire Overall Score (KCCQ-OS).
  • Cardiovascular-related mortality [ Time Frame: From Baseline to Month 30 ]
    The total number of deaths adjudicated as being related to cardiovascular causes.
  • Frequency of cardiovascular-related hospitalization [ Time Frame: From Baseline to Month 30 ]
    The number of times that a subject is hospitalized for cardiovascular-related causes.
  • All-cause mortality [ Time Frame: From Baseline to Month 30 ]
    The total number of deaths in the study.
  • TTR stabilization at Month 1 [ Time Frame: From Baseline to Month 1 ]
    Tafamidis stabilizes the transthyretin (TTR) tetramer by binding with very high affinity to the two thyroxine binding sites, preventing the tetramer from dissociating along the weak dimer-dimer interface. TTR stabilization is a measure of the degree of stabilization afforded the TTR molecule by tafamidis, expressed as a percentage change from baseline.


Original Secondary Outcome: Same as current

Information By: Pfizer

Dates:
Date Received: November 14, 2013
Date Started: December 9, 2013
Date Completion: February 20, 2018
Last Updated: April 11, 2017
Last Verified: April 2017