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)

Cancer Categories

Karmanos Trial ID

NCT ID

Age Group

Scope

Phase

Principal Investigator

Primary Objective:

To evaluate the efficacy of NTLA-2001, as measured by the composite risk of CV-related mortality and CV events (urgent HF visits and hospitalizations due to HF, arrhythmia, or stroke), compared to placebo

Secondary Objectives:

To evaluate the effect of NTLA-2001 on serum TTR compared to placebo
To evaluate the impact of NTLA-2001 on participant-reported CM-related symptoms and quality of life compared to placebo

Eligibility

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 1000 pg/mL (or greater than or equal to 2000 pg/mL if participant has known atrial fibrillation)

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 within 6 months prior to study dosing
Estimated glomerular filtration rate (eGFR) <30 mL/min/1.73m^2
Liver failure
Uncontrolled blood pressure

Locations

4100 John R
Detroit, MI 48201
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Phone: 1-800-527-6266

31995 Northwestern Hwy
Farmington Hills, MI 48334
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Phone: 1-800-527-6266

Applicable Disease Site

Therapies, Drugs, Devices