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Safety and Efficacy of Tafamidis in Patients With Transthyretin Cardiomyopathy
Not Recruiting
Trial ID: NCT01994889
Purpose
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.
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)
Stanford Investigator(s)
Ronald Witteles
Professor of Medicine (Cardiovascular Medicine)
Eligibility
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.
Intervention(s):
drug: Tafamidis
drug: Tafamidis
drug: Placebo
Not Recruiting
Contact Information
Stanford University
School of Medicine
300 Pasteur Drive
Stanford,
CA
94305
Ed Finn
650-724-6167