Purpose of this Study
Participants in this study will take a medicine called acoramidis by mouth two times each day for about three years. The study includes a screening period, a treatment period, and a follow up period after treatment ends.
During the study, participants will have regular visits so doctors can monitor their heart health and overall well being. Researchers want to learn more about the long term effects of acoramidis in people with transthyretin amyloid cardiomyopathy, a disease caused by abnormal protein buildup in the heart. The goal is to see how well the medicine works over time and how safe it is for patients.
Who Can Participate?
Eligibility
People may be able to join this study if they are 18 to 80 years old and have a confirmed diagnosis of transthyretin amyloid cardiomyopathy, a disease caused by abnormal protein buildup in the heart. They must have signs that the disease is affecting the heart, have mild symptoms of heart failure, and be stable on their current treatment.
Participants may have used certain ATTR medicines before, but only for a limited time and they must stop those medicines before starting the study drug. They must have acceptable kidney function and meet specific heart test requirements. Participants must also agree to use effective birth control during the study if pregnancy is possible. Researchers are studying the long term effects of acoramidis on heart structure and function in people with ATTR cardiomyopathy.
Age Range
18-80
Sex/Genders
Male (cisgender)
Female (cisgender)
Non-binary or gender fluid
Transgender male
Transgender female
Looking for Healthy Participants
No
What is Involved?
Description
This study is looking at the long term effects of a medicine called acoramidis in people who have transthyretin amyloid cardiomyopathy, a disease caused by abnormal protein buildup in the heart. Participants will take acoramidis by mouth two times each day for about three years.
Researchers will use special heart imaging tests to see how the heart changes over time and whether acoramidis can improve heart structure and function or slow the disease. The study will also look at how much amyloid protein is present in the heart. The goal is to better understand the long term safety and benefits of acoramidis for people with this heart condition.
Locations
Duke University Hospital
Visit Timing
Weekdays
Evenings
Compensation
Yes
Spanish Materials Available
Yes
Study Details
Full Title
An Open-Label Study to Investigate the Long-Term Effects of Acoramidis on Cardiac Function, Structure and Cardiac Amyloid Burden in Participants with Transthyretin Amyloid Cardiomyopathy (ATTR-CM)
Principal Investigator
Michel
Khouri
Protocol Number
PRO00120585
Phase
IV
Enrollment Status
Pending Open to Enrollment