Magnitude (Transthyretin Amyloidosis with Cardiomyopathy [ATTR-CM]) - Clinical Trial
What is the Purpose of this Study?
We are doing this study to find out if an experimental drug called NTLA-2001 (the study drug) is a safe and effective option for people with transthyretin amyloidosis with cardiomyopathy (ATTR-CM). The study drug is made using the CRISPR-CaS9 gene editing system. The intent of the study drug that is being tested is to target the TTR gene and make it inactive.
Transthyretin Amyloidosis with Cardiomyopathy (ATTR-CM)
Who Can Participate in the Study?
Adults ages 18-90 who:
- Are diagnosed with ATTR-CM diagnosis based on heart biopsy or a positive Scintigraphy scan
- Have not been treated with any RNA silencer therapies (e.g., patisiran, inotersen, or eplonterson) within the past 12 months
For more information, contact the study team at taylor.brenia@duke.edu.
What is Involved?
There are 3 stages to this study:
- Screening (lasts for 56 days)
- Study drug period (1 day infusion)
- Post-study drug period (minimum of 18 months)
If you choose to join the study, you will find out during the screening period if you are eligible to get the study drug. This period will consist of several tests and procedures to ensure eligibility. These tests and procedures will involve reviewing your medical history, taking blood samples, doing heart function tests, an eye exam, and a 6-minute walk test.
If you are eligible, you will get a random assignment (by chance) to either get a single dose of the study drug or a placebo (inactive substance with no drug in it). The study drug/placebo are given as an intravenous (IV) infusion that takes about 4 hours. You will be in our clinic for the whole day when you get the infusion since it involves a 2-hour period of monitoring before and after you get the study drug/placebo. You have a 2-in-3 chance of being assigned to get the study drug.
The post-study drug period will consist of multiple visits for blood samples, echocardiograms, and functional tests. These visits will occur for at least 18 months after you get the study drug/placebo.
Study Details
