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

Biological
NTLA-2001
NTLA-2001 (55mg) by IV infusion
Drug
Placebo
Normal saline (0.9% NaCl) by IV infusion

Locations (132)

University of Arizona
Tucson, Arizona
Cedars-Sinai Medical Center
Los Angeles, California
Western Greater Los Angeles VA Medical Center
Los Angeles, California
Stanford University
Palo Alto, California
University of California, San Diego (UCSD) - Medical Center
San Diego, California
University of Colorado Denver
Denver, Colorado