Actively Recruiting
Phase 3 Study Evaluating Single Dose NTLA-2001 Versus Placebo for Transthyretin Amyloidosis With Cardiomyopathy ATTR-CM
Led by Intellia Therapeutics · Updated on 2026-03-27
1200
Participants Needed
132
Research Sites
17 weeks
Total Duration
AI-Summary
What this Trial Is About
Researchers are studying the safety and effectiveness of a single dose of NTLA-2001 compared to placebo in adults with transthyretin amyloidosis with cardiomyopathy ATTR-CM and a history of heart failure. This multinational, phase 3 trial involves about 1200 participants and aims to better understand how this treatment impacts heart-related events and survival over time. Participants will be randomly assigned to receive either one intravenous infusion of NTLA-2001 or a placebo infusion of normal saline. The study is double-blind, meaning neither the participants nor the researchers know who receives which infusion. The main treatment phase will last at least 18 months and could extend up to about 5 years depending on cardiovascular event rates. During the study, participants will be monitored for cardiovascular deaths and events as primary outcomes. Secondary measures include changes in blood levels of transthyretin protein and heart failure symptoms assessed at baseline and month 18. Participants will undergo regular medical evaluations, blood tests, and questionnaires to assess their heart function and overall health. Safety will be continuously monitored throughout the trial period.
CONDITIONS
Brief Title
MAGNITUDE: A Phase 3 Study of NTLA-2001 in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR-CM)
Research Team
T
Trial Manager at Intellia
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