ATTRIBUTE-CM Study

STUDY BASICS

Have you been diagnosed with symptomatic transthyretin amyloid cardiomyopathy (also called familial amyloid cardiomyopathy or ATTR-CM)? If so, you may be eligible to participate in a research study to help find out if an investigational drug called AG10 is a safe and effective treatment for people who have symptomatic ATTR-CM. Compensation provided.

STUDY PURPOSE

Transthyretin amyloid cardiomyopathy, also called familial amyloid cardiomyopathy or ATTR-CM, is an inherited disease that causes an abnormal protein called amyloid to build up in the heart. Over time, ATTR-CM damages the structure and function of the heart, often leading to heart failure and symptoms such as irregular heartbeat, fatigue, shortness of breath, and leg swelling. Although medications can help control some of the symptoms associated with ATTR-CM, treatment options are limited.

The purpose of this study is to help researchers find out if an investigational drug called AG10 is a safe and effective treatment for people who have symptomatic ATTR-CM. AG10 binds to transthyretin in the blood and may prevent it from forming amyloid. AG10 is considered to be an investigational drug because it has not been approved by the U.S. Food and Drug Administration (FDA).

COULD THIS STUDY BE RIGHT FOR YOU?

• Ages 18-90
• Diagnosed with ATTR-CM (also known as transthyretin amyloid cardiomyopathy or familial amyloid cardiomyopathy)
• Have not had a heart attack or stroke within the past 90 days
• Not likely to undergo heart transplantation within the next year

WHAT PARTICIPANTS CAN EXPECT

Participation involves an initial screening period of up to 35 days to find out if you are eligible for the study. Eligible participants will be randomly assigned (like the flip of a coin) to receive either the study drug, AG10, or a placebo. A placebo looks like the study drug, but contains no medication. Participants will take the study drug by mouth twice per day. During this part of the study, participants will have about 13 visits, about one every three months. Some visits will last up to 2 hours.

During the next part of the study, all participants may choose to receive AG10.

Tests and assessments during the study include blood draws, urine collections, walking tests, electrocardiograph, physical exams, questionnaires, and monthly phone calls.

Participants will receive $50 per visit and a parking voucher at the end of each visit.