MAGNITUDE: A Phase 3 Study of NTLA-2001 in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR-CM)

To evaluate the efficacy and safety of a single dose of NTLA-2001 compared to placebo in participants with ATTR-CM.

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

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
Liver failure
Uncontrolled blood pressure
Unable or unwilling to take vitamin A supplementation for the duration of the study

Additional locations may be listed on ClinicalTrials.gov for NCT06128629.

See trial information on ClinicalTrials.gov for a list of participating sites.

This is a multinational, multicenter, double-blind, placebo-controlled study in

approximately 765 participants, who will be randomized to receive a single infusion of

either NTLA-2001 or placebo.

Trial PhasePhase III

Trial Typetreatment

Lead OrganizationIntellia Therapeutics

Description