Clinical Trial: The Effect of an Antisense Oligonucleotide to Lower Transthyretin (TTR) Levels on the Progression of -Wild-type TTR Involving the Heart

Study Status: Withdrawn
Recruit Status: Withdrawn
Study Type: Interventional

Official Title: An 18 Month Open Label Study Of The Tolerability And Efficacy Of An Antisense Oligonucleotide In Patients With Wild-Type Transthyretin Amyloid Cardiomyopathy (Senile Systemic Amyloidosis)

Brief Summary: ATTRwt (also known as senile systemic, or senile cardiac amyloidosis) is a progressive heart disease, causing congestive heart failure. It is caused by amyloid protein deposits in the heart, that are derived from a normal protein, TTR, made in the liver. The aim of the study is to determine whether lowering the blood levels of TTR, by a weekly injection of a compound designed specifically to do this, will slow the progression of the disease when treated patients are compared to previously-followed patients who were not receiving this drug. The study also aims to determine how well this drug is tolerated and the existence and severity of any drug side-effects.

Detailed Summary:
Sponsor: Brigham and Women's Hospital

Current Primary Outcome: Systolic strain imaging by echocardiographic speckle tracking [ Time Frame: Month 12 ]

The primary echocardiographic parameter to be measured will be longitudinal left ventricular (LV) strain (units = % LV longitudinal shortening) as compared to baseline.


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Systolic strain evaluation by echocardiography [ Time Frame: Secondary analysis will occur at 18 months ]
    The primary echocardiographic parameter measured will be longitudinal left ventricular (LV) strain (units = %).
  • Echocardiographic determination of Mean thickness of LV septum and posterior wall (units = mm) [ Time Frame: 12 months ]
  • Echocardiographic determination of Mean thickness of LV septum and posterior wall (units = mm) [ Time Frame: 18 months ]
  • Echocardiographic determination of LV ejection fraction (units = %) [ Time Frame: 12 months ]
  • Echocardiographic determination of LV ejection fraction (units = %) [ Time Frame: 18 months ]
  • LV mass measurement by Cardiac MRI (cMRI) (units = grams) [ Time Frame: 18 months ]
  • LV cellular component as determined by cMRI (units = % of total LV mass) [ Time Frame: 12 months ]
  • LV cellular component as determined by cMRI (units = % of total LV mass) [ Time Frame: 18 months ]
  • LV extracellular component as determined by cMRI (units = % of total LV mass) [ Time Frame: 12 months ]
  • LV extracellular component as determined by cMRI (units = % of total LV mass) [ Time Frame: 18 months ]
  • Extent of cMRI late gadolinium enhancement of the LV (unites = % of area) [ Time Frame: 12 months ]
  • Extent of cMRI late gadolinium enhancement of the LV (unites = % of area) [ Time Frame: 18 months ]
  • LV mass measurement by Cardiac MRI (cMRI) (units = grams) [ Time Frame: Month 12 ]


Original Secondary Outcome: Same as current

Information By: Brigham and Women's Hospital

Dates:
Date Received: November 19, 2015
Date Started: January 2016
Date Completion: December 2018
Last Updated: August 9, 2016
Last Verified: January 2016