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MAGNITUDE: A Phase 3 Study of NTLA-2001 in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR-CM)
RecruitingPhase 3
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)
Who can join
Ages 18 to 90 · All sexes
Full eligibility criteria
Inclusion Criteria: * Documented diagnosis of ATTR amyloidosis with cardiomyopathy * Medical history of heart failure (HF) * Symptoms of HF are optimally managed and clinically stable within 28 days prior to administration of study intervention * Screening NT-proBNP, a blood marker of HF severity, greater than or equal to 600 pg/mL and less than 10,000 pg/mL Exclusion Criteria: * New York Heart Association (NYHA) Class IV HF * Polyneuropathy Disability score of IV (confined to wheelchair or bed) * Has hepatitis B, hepatitis C or human immunodeficiency virus (HIV) infection * History of active malignancy within 3 years prior to screening * RNA silencer therapy (patisiran, inotersen and/or eplontersen) within 12 months prior to dosing. Any prior vutrisiran use is not allowed * Initiation of tafamidis or acoramidis within 56 days prior to study dosing * Estimated glomerular filtration rate (eGFR) \<30 mL/min/1.73m\^2 * History of liver disease * Uncontrolled blood pressure * Unable or unwilling to take vitamin A supplementation for the duration of the study
About the study
To evaluate the efficacy and safety of a single dose of NTLA-2001 compared to placebo in participants with ATTR-CM.
What is being tested
- NTLA-2001 (biological)
- Placebo (drug)
Sponsor: Intellia Therapeutics · Participants: 1,200 · Started: Dec 13, 2023
Contact the study team
- Trial Manager at Intellia · Phone: 1-857-285-6200
Official record on ClinicalTrials.gov — NCT06128629
Locations in the U.S.
Conditions
From ClinicalTrials.gov, data retrieved Sep 29, 2026. Each study sets its own eligibility; the study team decides who can join.