PHASE 3 STUDY OF TAFAMIDIS MEGLUMINE IN PATIENTS WITH TTR-CM
Research type
Research Study
Full title
A PHASE 3 MULTICENTER, RANDOMIZED, DOUBLE-BLIND, EXTENSION STUDY TO EVALUATE THE SAFETY OF DAILY ORAL DOSING OF TAFAMIDIS MEGLUMINE (PF-06291826) 20 MG OR 80 MG IN SUBJECTS DIAGNOSED WITH TRANSTHYRETIN CARDIOMYOPATHY (TTR-CM)
IRAS ID
214032
Contact name
Perry Elliott
Contact email
Sponsor organisation
Pfizer Inc.
Eudract number
2016-000868-42
Clinicaltrials.gov Identifier
Duration of Study in the UK
5 years, 0 months, 0 days
Research summary
Transthyretin amyloid disease is a rare condition characterised by the build-up of a plasma protein in organs and tissues. When these proteins deposit in the cells of the heart muscle it is known as Transthyretin cardiomyopathy (TTR-CM). These deposits create a thickening and stiffening of the heart tissue which can progress to restrictive cardiomyopathy, congestive heart failure, and ultimately death.
This study is a long-term extension of a prior study. The purpose is to evaluate the safety of daily oral dosing of 20 mg or 80 mg tafamidis meglumine in participants diagnosed with TTR-CM. Suitable participants must complete 30 months of the prior study (B3461028) before enrollment. participants will take part in the study for up to 60 months or until tafamidis is available by prescription for TTR-CM, whichever occurs earlier.
Participants will be assigned to the same blinded tafamidis meglumine dose they received in the prior study B3461028. Participants who received placebo (a dummy drug that contains no active ingredients) in the prior study will be randomized (chosen by chance) to blinded 20 mg or 80 mg tafamidis meglumine.
Participants will be expected to attend clinic visits every 6 months. During these visits the following study procedures may be performed to measure health and body function: blood tests, urine tests, electrocardiograms, blood pressure, pulse rate, temperature, breathing rate and questionnaires to monitor patient health.
This study is being sponsored by Pfizer. Approximately 330 participants will take part in the study, with approximately 2 participants in the UK.
REC name
South Central - Oxford B Research Ethics Committee
REC reference
16/SC/0604
Date of REC Opinion
9 Feb 2017
REC opinion
Further Information Favourable Opinion