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)
Brief description of study
This trial is recruiting patients with a condition known as Transthyretin (TTR) Amyloidosis with Cardiomyopathy or ATTR-CM. In individuals with this condition, the heart muscle does not pump blood through the body as it should, due to build-up of abnormal protein between the muscle fibers in the heart. The specific protein is called transthyretin amyloid (TTR).
Intellia Therapeutics, Inc. has developed an investigational drug called NTLA-2001. NTLA-2001 consists of a CRISPR/Cas9 gene editing system, which can “knockout” the TTR gene. This means NTLA-2001 permanently changes the DNA so the gene stops making the TTR protein. Because most of the TTR protein in the body is made in the liver, NTLA-2001 is packaged within lipid nanoparticles which are able to deliver NTLA-2001 directly into the liver and thereby avoid most of the other organs and tissues. NTLA-2001 is given intravenously (IV) (through a small flexible tube inserted into a vein in the arm or hand) slowly over about 4 hours. An individual's amyloidosis symptoms may not change, may stop worsening, or perhaps could improve as a result.
By using the CRISPR/Cas 9 technology, NTLA-2001 will disable the TTR gene, but this does not involve the insertion of any permanent genetic material into the body.
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