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

Description

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

Conditions

Transthyretin Amyloidosis (ATTR) with Cardiomyopathy

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Study Overview

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Study Details

Study overview

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

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)

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

Condition
Transthyretin Amyloidosis (ATTR) with Cardiomyopathy
Intervention / Treatment

-

Contacts and Locations

Los Angeles

Western Greater Los Angeles VA Medical Center, Los Angeles, California, United States, 90073

Palo Alto

Stanford University, Palo Alto, California, United States, 94305

Denver

University of Colorado Denver, Denver, Colorado, United States, 80045

New Haven

Yale University School of Medicine, New Haven, Connecticut, United States, 06510

Washington

MedStar Washington Hospital Center - MedStar Heart and Vascular Institute, Washington, District of Columbia, United States, 20010

Atlanta

Emory University School of Medicine, Atlanta, Georgia, United States, 30322

Chicago

Northwestern University, Chicago, Illinois, United States, 60611

Lexington

University of Kentucky Gill Heart Institute, Lexington, Kentucky, United States, 40536

Baltimore

Johns Hopkins University, Baltimore, Maryland, United States, 21287

Boston

Brigham and Women's Hospital, Boston, Massachusetts, United States, 02115

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility 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)
  • * 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
  • * Unable or unwilling to take vitamin A supplementation for the duration of the study

Ages Eligible for Study

18 Years to 90 Years

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Intellia Therapeutics,

Study Record Dates

2028-04