DepleTTR-CM (Transthyretin Amyloid Cardiomyopathy) - Clinical Trial
What is the Purpose of this Study?
We are doing this study to find out if an experimental drug called ALXN2220 (the study drug) is a safe and effective option for people with transthyretin amyloid cardiomyopathy (ATTR-CM). The study drug is designed to act as a "depleter," which means its desired function is to target the active removal of pre-existing amyloids and restore the heart's function and elasticity.
Transthyretin (ATTR) Cardiac Amyloidosis
Who Can Participate in the Study?
Adults ages 18-90 who:
- Are diagnosed with wild-type ATTR-CM or variant ATTR-CM
- Are willing to undergo genetic testing for mutations to the TTR gene
- Have never received any previous treatment with an ATTR amyloid depleter
For more information, contact the study team at matthew.radzom@duke.edu.
What is Involved?
If you choose to join this study, you will get a random assignment (like a coin flip) to either get the study drug or a placebo (inactive substance with no drug in it). Regardless of assignment, everyone in the study will:
- Visit our clinic about 56 times over the course of 4 years
- Get an intravenous (IV) infusion of the study drug or placebo once every 4 weeks
- Fill out questionnaires
- Have physical exams and various fitness tests done throughout the study to assess heart health and function
The study infusions will take about 1-to-2 hours each time. You will stay in our clinic for 30 minutes after each infusion is done for monitoring.
Study Details
