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)
Intellia Therapeutics
Summary
To evaluate the efficacy and safety of a single dose of NTLA-2001 compared to placebo in participants with ATTR-CM.
Description
This is a multinational, multicenter, double-blind, placebo-controlled study in approximately 1200 participants, who will be randomized to receive a single infusion of either NTLA-2001 or placebo.
Eligibility
- Age range
- 18–90 years
- Sex
- All
- Healthy volunteers
- No
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 acti…
Interventions
- BiologicalNTLA-2001
NTLA-2001 (55mg) by IV infusion
- DrugPlacebo
Normal saline (0.9% NaCl) by IV infusion
Locations (132)
- University of ArizonaTucson, Arizona
- Cedars-Sinai Medical CenterLos Angeles, California
- Western Greater Los Angeles VA Medical CenterLos Angeles, California
- Stanford UniversityPalo Alto, California
- University of California, San Diego (UCSD) - Medical CenterSan Diego, California
- University of Colorado DenverDenver, Colorado