Overview

This trial is active, not recruiting.

Condition transthyretin (ttr) amyloid cardiomyopathy
Treatments tafamidis, placebo
Phase phase 3
Sponsor Pfizer
Start date December 2013
End date February 2018
Trial size 441 participants
Trial identifier NCT01994889, 2012-002465-35, ATTR-ACT, B3461028

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.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Allocation randomized
Endpoint classification safety/efficacy study
Intervention model parallel assignment
Masking double blind (subject, caregiver, investigator, outcomes assessor)
Primary purpose treatment
Arm
(Experimental)
Active Treatment-Low dose
tafamidis
Tafamidis 20 mg in soft gel capsules administered once a day for 30 months
(Experimental)
Active Treatment-High Dose
tafamidis
Tafamidis 80 mg in soft gel capsules administered once a day for 30 months
(Placebo Comparator)
Placebo control
placebo
Placebo in soft gel capsules administered once a day for 30 months

Primary Outcomes

Measure
All-cause mortality and frequency of cardiovascular-related hospitalization
time frame: From Baseline to Month 30

Secondary Outcomes

Measure
6-Minute Walk Test (6MWT).
time frame: From Baseline to Month 30
Kansas City Cardiomyopathy Questionnaire (KCCQ)
time frame: From Baseline to Month 30
Cardiovascular-related mortality
time frame: From Baseline to Month 30
Frequency of cardiovascular-related hospitalization
time frame: From Baseline to Month 30
All-cause mortality
time frame: From Baseline to Month 30
TTR stabilization at Month 1
time frame: From Baseline to Month 1

Eligibility Criteria

Male or female participants from 18 years up to 90 years old.

Inclusion Criteria: - Medical history of Heart Failure (HF) with at least 1 prior hospitalization for HF or clinical evidence of HF (without hospitalization) manifested by signs or symptoms of volume overload or elevated intracardiac pressures (e.g., elevated jugular venous pressure, shortness of breath or signs of pulmonary congestion on x-ray or auscultation, peripheral edema) that required/requires treatment with a diuretic for improvement, - Evidence of cardiac involvement by echocardiography with an end-diastolic interventricular septal wall thickness > 12 mm, - Presence of amyloid deposits in biopsy tissue and presence of a variant TTR genotype and/or TTR precursor protein identification by immunohistochemistry, scintigraphy or mass spectrometry Exclusion Criteria: - A New York Heart Association (NYHA) classification of IV. - Presence of primary (light chain) amyloidosis. - Prior liver or heart transplantation or implanted cardiac mechanical assist device.

Additional Information

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) 20 Mg Or 80 Mg In Comparison To Placebo In Subjects Diagnosed With Transthyretin Cardiomyopathy (ttr-cm)
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.
Trial information was received from ClinicalTrials.gov and was last updated in October 2016.
Information provided to ClinicalTrials.gov by Pfizer.