Magnitude (Transthyretin Amyloidosis with Cardiomyopathy [ATTR-CM])

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.

Who Can Participate?

Eligibility

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.

Age Range

18-90

Sex/Genders

Male (cisgender)

Female (cisgender)

Non-binary or gender fluid

Transgender male

Transgender female

Looking for Healthy Participants

What is Involved?

Description

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.

Locations

Duke University Hospital

Visit Timing

Weekdays

Compensation

Yes

Spanish Materials Available

Study Details

Full Title

MAGNITUDE: A Phase 3, Multinational, Multicenter, Randomized, Double-Blind,
Placebo-Controlled Study to Evaluate the Efficacy and Safety of NTLA-2001 in
Participants with Transthyretin Amyloidosis with Cardiomyopathy (ATTR-CM)

Study Website

Principal Investigator

Ravi

Karra

Protocol Number

PRO00114260

Phase

III

Enrollment Status

Open to Enrollment