NTLA-2001 in Transthyretin Amyloidosis With Cardiomyopathy

What is the Purpose of this Study?

The purpose of this study is to understand how an experimental drug called NTLA-2001 affects a heart condition called transthyretin (TTR) amyloidosis with cardiomyopathy (ATTR-CM), a condition in which the heart muscle does not pump blood through the body as it should due to build-up of abnormal protein (TTR) between the muscle fibers in the heart. The study aims to determine whether NTLA-2001 is safe and effective in people with ATTR-CM. NTLA-2001 uses a special gene-editing tool to change an individual’s DNA and turn off the TTR gene in liver cells. This stops the TTR gene from making the TTR protein, which may help improve the symptoms of ATTR-CM. (The process does not add any new permanent genetic material to the body; it just changes the way the patient’s DNA works to stop producing the harmful TTR protein.) Participants will be assigned to receive either NTLA-2001 or placebo (fluids that contain no active ingredients). For every 3 participants in the study, a computer will randomly choose 2 people to receive NTLA-2001 and 1 person to receive placebo. NTLA-2001 is designed to work only in the liver, where the TTR protein is made.


Eligibility

  • * Male and/or female participants 18 to 80 years of age inclusive, at the time of signing the informed consent
  • * Diagnosis of polyneuropathy (PN) due to transthyretin (TTR) amyloidosis (ATTR)
  • * Must have a body weight of at least 45 kilograms (kg) at Screening visit
  • * Lack of access to approved treatments for ATTR and/or progression of hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN) despite use of approved treatment for ATTRv-PN
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Where can I participate?

Beverly

More about this Clinical Trial

What is the full name of this clinical trial?

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

Study Details
Disease Type/Condition

Other

Principal Investigator

Stern, Lily

Age Group

Adult

Phase

III

IRB Number

STUDY00003277

ClinicalTrials.gov ID

NCT04601051

Key Eligibility
ClinicalTrials.gov

How do I learn more about this study?
Email
clinicaltrials@cshs.org
Study Detail
Disease Type/Condition

Other

Principal Investigator

Stern, Lily

Age Group

Adult

Phase

III

IRB Number

ITL-2001-CL-301

ClinicalTrials.gov ID

NCT04601051

Key Eligibility
ClinicalTrials.gov

Contact
Email
clinicaltrials@cshs.org