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

March 23, 2026 updated by: Intellia Therapeutics

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)

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

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Detailed 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.

Study Type

Interventional

Enrollment (Estimated)

1200

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • Argentina
      • Buenos Aires, Argentina
        • Recruiting
        • Hospital Italiano de Buenos Aires
      • Buenos Aires, Argentina
        • Recruiting
        • Fundacion Favalro
      • Buenos Aires, Argentina
        • Recruiting
        • ICBA
      • Ciudad Autonoma de Buenos Aire, Argentina
        • Recruiting
        • Hospital Italiano de Buenos Aires
      • Ciudad Autonoma de Buenos Aire, Argentina
        • Recruiting
        • Fundacion Favaloro
      • Rosario, Argentina
        • Recruiting
        • Hospital Privado de Rosario
  • Australia
      • Adelaide, Australia
        • Recruiting
        • Royal Adelaide Hospital
      • Melbourne, Australia
        • Recruiting
        • The Alfred Hospital
      • Woolloongabba, Australia
        • Recruiting
        • Princess Alexandra Hospital
    • Australian Capital Territory
      • Garran, Australian Capital Territory, Australia
        • Recruiting
        • The Canberra Hospital
    • New South Wales
      • Westmead, New South Wales, Australia
        • Recruiting
        • Westmead Hospital
    • Queensland
      • Cairns, Queensland, Australia
        • Withdrawn
        • Cairns Hospital
    • Victoria
      • Box Hill, Victoria, Australia
        • Recruiting
        • Eastern Health - Box Hill Hospital
    • Western Australia
      • Perth, Western Australia, Australia
        • Recruiting
        • Fiona Stanley Hospital
  • Austria
      • Innsbruck, Austria
        • Recruiting
        • Medizinische Universitaet Innsbruck
      • Sankt Pölten, Austria
        • Recruiting
        • Universitaesklinikum St. Poelten
  • Belgium
      • Bruges, Belgium
        • Recruiting
        • AZ Sint-Jan Brugge
      • Brussels, Belgium
        • Recruiting
        • Universite Libre de Bruxelles (ULB) - Hopital Erasme
      • Edegem, Belgium
        • Recruiting
        • Antwerp University Hospital
      • Leuven, Belgium
        • Recruiting
        • UZ Leuven
      • Liège, Belgium
        • Recruiting
        • Centre Hospitalier Regional de la Citadelle
  • Brazil
      • Belo Horizonte, Brazil
        • Recruiting
        • Centro de Pesquisa NUPEC - Instituto Orizonti em Belo Horizonte
      • Niterói, Brazil
        • Recruiting
        • Complexo Hospitalar de Niterói (CHN-DASA)
      • Ribeirão Preto, Brazil
        • Recruiting
        • HCFMRP - Hospital das Clinicas da Faculdade de Medicina de Ribeirao Preto
      • Ribeirão Preto, Brazil
        • Recruiting
        • CAPED - Centro Avancado de Pesquisa
      • Salvador, Brazil
        • Recruiting
        • Cardiopulmonar da Bahia S. A. (Hospital Cardio Pulmonar)
      • São Paulo, Brazil
        • Recruiting
        • Instituto Dante Pazzanese de Cardiologia
      • São Paulo, Brazil
        • Recruiting
        • Hospital Israelita Albert Einstein
      • São Paulo, Brazil
        • Recruiting
        • PSEG Centro de Pesquisa Clinica S.A.
      • São Paulo, Brazil
        • Recruiting
        • Instituto do Coracao (InCor) do Hospital das Clinicas da FMUSP
  • Canada
    • Alberta
      • Calgary, Alberta, Canada
        • Recruiting
        • Alberta Health Services - University of Calgary
    • British Columbia
      • Vancouver, British Columbia, Canada
        • Recruiting
        • Vancouver Coastal Health Research Institute
    • Ontario
      • Toronto, Ontario, Canada
        • Recruiting
        • University Health Network - Toronto General Hospital
    • Quebec
      • Montreal, Quebec, Canada
        • Recruiting
        • McGill University Health Centre
  • Czechia
      • Brno, Czechia
        • Recruiting
        • Fakultni nemocnice u sv. Anny v Brne
  • Denmark
      • Aarhus, Denmark
        • Recruiting
        • Aarhus University Hospital
      • Copenhagen, Denmark
        • Recruiting
        • Rigshospitalet
  • France
      • Créteil, France
        • Recruiting
        • AP-HP Hopital Henri Mondor
      • Marseille, France
        • Recruiting
        • AP-HM - Hôpital de la Timone
      • Paris, France
        • Recruiting
        • AP-HP Hopital Bichat - Claude Bernard
      • Rennes, France
        • Recruiting
        • CHU Rennes - Hopital Pontchaillou
      • Toulouse, France
        • Recruiting
        • CHU de Toulouse - Hôpital Rangueil
  • Germany
      • Berlin, Germany
        • Recruiting
        • Charité Universitaetsmedizin Berlin
      • Essen, Germany
        • Recruiting
        • Universitaetsklinikum Essen
      • Heidelberg, Germany
        • Recruiting
        • Universitaetsklinikum Heidelberg
      • Würzburg, Germany
        • Recruiting
        • Universitaetsklinikum Wuerzburg
  • Hungary
      • Budapest, Hungary
        • Recruiting
        • Semmelweis Egyetem Belgyógyászati és Hematológiai Klinika
  • Israel
      • Haifa, Israel
        • Recruiting
        • Rambam Health Care Campus
      • Jerusalem, Israel
        • Recruiting
        • Hadassah University Hospital, Ein Karem
      • Tel Litwinsky, Israel
        • Recruiting
        • Sheba Medical Center
  • Italy
      • Bologna, Italy
        • Recruiting
        • IRCCS Azienda Ospedaliero-Universitaria di Bologna - Policlinico di Sant'Orsola
      • Cona, Italy
        • Recruiting
        • Azienda Ospedaliero-Universitaria di Ferrara-Arcispedale Sant'Anna
      • Florence, Italy
        • Recruiting
        • Azienda Ospedaliero-Universitaria Careggi
      • Genova, Italy
        • Recruiting
        • Ospedale Policlinico San Martino IRCCS
      • Pavia, Italy
        • Recruiting
        • Fondazione IRCCS Policlinico San Matteo
      • Pisa, Italy
        • Recruiting
        • Fondazione Toscana Gabriele Monasterio per la Ricerca Medica
      • Trieste, Italy
        • Recruiting
        • Azienda sanitaria universitaria Giuliano Isontina (ASU GI) - Ospedale di Cattinara
  • Japan
      • Kumamoto, Japan
        • Recruiting
        • Kumamoto University Hospital
      • Osaka, Japan
        • Recruiting
        • National Cerebral and Cardiovascular Center
      • Tokyo, Japan
        • Recruiting
        • The University of Tokyo Hospital
      • Toyama, Japan
        • Recruiting
        • Toyama University Hospital
  • Mexico
      • Mexico City, Mexico
        • Recruiting
        • Instituto Nacional de Ciencias Medicas y Nutricion Salvador Zubiran
  • Netherlands
      • Groningen, Netherlands
        • Recruiting
        • University Medical Center
      • Rotterdam, Netherlands
        • Recruiting
        • Erasmus MC
      • Utrecht, Netherlands
        • Recruiting
        • Universitair Medisch Centrum Utrecht
  • New Zealand
      • Auckland, New Zealand
        • Recruiting
        • New Zealand Clinical Research
      • Christchurch, New Zealand
        • Recruiting
        • New Zealand Clinical Research
  • Norway
      • Oslo, Norway
        • Recruiting
        • Oslo University Hospital - Rikshospitalet
  • Portugal
      • Lisbon, Portugal
        • Recruiting
        • Centro Hospitalar Universitario de Lisboa Norte
      • Porto, Portugal
        • Recruiting
        • Centro Hospitalar Universitario do Porto
  • Singapore
      • Singapore, Singapore
        • Recruiting
        • National University Hospital
      • Singapore, Singapore
        • Recruiting
        • National Heart Centre
  • South Korea
      • Gwangju, South Korea
        • Withdrawn
        • Chonnam National University Hospital
      • Seoul, South Korea
        • Withdrawn
        • Severance Hospital, Yonsei University Health System
      • Seoul, South Korea
        • Withdrawn
        • Samsung Medical Center
      • Seoul, South Korea
        • Withdrawn
        • Seoul National University Hospital
  • Spain
      • A Coruña, Spain
        • Recruiting
        • Hospital Universitario de A Coruña
      • Barcelona, Spain
        • Recruiting
        • Hospital Universitario Vall d'Hebron
      • Barcelona, Spain
        • Recruiting
        • Hospital Universitari de Bellvitge
      • Majadahonda, Spain
        • Recruiting
        • Hospital Universitario Puerta de Hierro
      • Málaga, Spain
        • Recruiting
        • Hospital Universitario Virgen de la Victoria
      • Palma de Mallorca, Spain
        • Recruiting
        • Hospital Universitario Son Llatzer
  • Sweden
      • Stockholm, Sweden
        • Recruiting
        • Karolinska University Hospital
      • Umeå, Sweden
        • Recruiting
        • University Hospital of Umeå
  • Taiwan
      • New Taipei City, Taiwan
        • Recruiting
        • Far Eastern Memorial Hospital
      • Taipei, Taiwan
        • Recruiting
        • National Taiwan University Hospital
      • Taipei, Taiwan
        • Recruiting
        • Taipai Veterans General Hospital
  • United Kingdom
      • Birmingham, United Kingdom
        • Active, not recruiting
        • Synexus Midlands Clinical Research Centre
      • Cardiff, United Kingdom
        • Active, not recruiting
        • Synexus Wales Clinical Research Centre
      • Glasgow, United Kingdom
        • Active, not recruiting
        • Synexus Scotland Clinical Research Centre
      • Hexham, United Kingdom
        • Active, not recruiting
        • Synexus Hexham Clinical Research Centre
      • Liverpool, United Kingdom
        • Active, not recruiting
        • Synexus Merseyside Clinical Research Centre
      • London, United Kingdom
        • Recruiting
        • Richmond Pharmacology
      • Manchester, United Kingdom
        • Active, not recruiting
        • Synexus Manchester Clinical Research Centre
  • United States
    • Arizona
      • Tucson, Arizona, United States, 85724
        • Recruiting
        • University of Arizona
    • California
      • Los Angeles, California, United States, 90048
        • Recruiting
        • Cedars-Sinai Medical Center
      • Los Angeles, California, United States, 90073
        • Recruiting
        • Western Greater Los Angeles VA Medical Center
      • Palo Alto, California, United States, 94305
        • Recruiting
        • Stanford University
      • San Diego, California, United States, 92103
        • Recruiting
        • University of California, San Diego (UCSD) - Medical Center
    • Colorado
      • Denver, Colorado, United States, 80045
        • Recruiting
        • University of Colorado Denver
    • Connecticut
      • New Haven, Connecticut, United States, 06510
        • Recruiting
        • Yale University School of Medicine
    • District of Columbia
      • Washington D.C., District of Columbia, United States, 20010
        • Recruiting
        • MedStar Washington Hospital Center - MedStar Heart and Vascular Institute
    • Florida
      • Jacksonville, Florida, United States, 32224
        • Recruiting
        • Mayo Clinic - Jacksonville
      • Weston, Florida, United States, 33331
        • Recruiting
        • Cleveland Clinic of Florida
    • Georgia
      • Atlanta, Georgia, United States, 30322
        • Recruiting
        • Emory University School of Medicine
    • Illinois
      • Chicago, Illinois, United States, 60611
        • Recruiting
        • Northwestern University
    • Indiana
      • Indianapolis, Indiana, United States, 46202
        • Recruiting
        • Indiana University Health
    • Kentucky
      • Lexington, Kentucky, United States, 40536
        • Recruiting
        • University of Kentucky Gill Heart Institute
    • Maryland
      • Baltimore, Maryland, United States, 21287
        • Recruiting
        • Johns Hopkins University
    • Massachusetts
      • Boston, Massachusetts, United States, 02115
        • Recruiting
        • Brigham and Women's Hospital
      • Boston, Massachusetts, United States, 02118
        • Recruiting
        • Boston University School of Medicine
    • Michigan
      • Ann Arbor, Michigan, United States, 48109
        • Recruiting
        • University of Michigan
      • Detroit, Michigan, United States, 48201
        • Withdrawn
        • Barbara Ann Karmanos Cancer Institute
    • Minnesota
      • Rochester, Minnesota, United States, 55905
        • Recruiting
        • Mayo Clinic
    • Missouri
      • Kansas City, Missouri, United States, 64111
        • Recruiting
        • St. Luke's Hospital
    • New York
      • New York, New York, United States, 10032
        • Recruiting
        • Columbia University Medical Center
      • New York, New York, United States, 10016
        • Recruiting
        • New York University (NYU) School of Medicine - Langone Medical Center
    • North Carolina
      • Durham, North Carolina, United States, 27710
        • Recruiting
        • Duke University Medical Center
    • Ohio
      • Cleveland, Ohio, United States, 44195
        • Recruiting
        • Cleveland Clinic
      • Columbus, Ohio, United States, 43214
        • Recruiting
        • Ohio Health Research Institute
    • Oregon
      • Portland, Oregon, United States, 97239
        • Recruiting
        • Oregon Health and Science University (OHSU)
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • Recruiting
        • Penn Presbyterian Medical Center
      • Pittsburgh, Pennsylvania, United States, 15212
        • Recruiting
        • Allegheny General Hospital
      • Pittsburgh, Pennsylvania, United States, 15219
        • Recruiting
        • University of Pittsburgh Medical Center
    • South Carolina
      • Charleston, South Carolina, United States, 29425
        • Recruiting
        • Medical University of South Carolina
    • Texas
      • Dallas, Texas, United States, 75390
        • Recruiting
        • University of Texas Southwestern Medical Center
      • Dallas, Texas, United States, 75201
        • Recruiting
        • Baylor Scott & White Research Institute
      • Houston, Texas, United States, 77030
        • Recruiting
        • Baylor College of Medicine
      • Houston, Texas, United States, 77030
        • Recruiting
        • Houston Methodist Hospital
      • Plano, Texas, United States, 75093
        • Recruiting
        • Baylor Scott and White - The Heart Hospital Baylor Plano
    • Virginia
      • Falls Church, Virginia, United States, 22042
        • Recruiting
        • Inova Fairfax Hospital
      • Richmond, Virginia, United States, 23298
        • Recruiting
        • Virginia Commonwealth University - Department of Internal Medicine

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.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

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 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
  • History of liver disease
  • Uncontrolled blood pressure
  • Unable or unwilling to take vitamin A supplementation for the duration of the study

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: NTLA-2001
Single intravenous (IV) infusion of NTLA-2001
Biological: NTLA-2001
NTLA-2001 (55mg) by IV infusion
Placebo Comparator: Placebo
Single IV infusion of normal saline
Drug: Placebo
Normal saline (0.9% NaCl) by IV infusion

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Composite outcome of cardiovascular (CV) mortality and CV events
Time Frame: Maximum study duration is dependent on event rates and is estimated to be at least 18 months and up to approximately 5 years
Maximum study duration is dependent on event rates and is estimated to be at least 18 months and up to approximately 5 years

Secondary Outcome Measures

Outcome Measure
Time Frame
Change in baseline to month 18 in serum TTR
Time Frame: Baseline, Month 18
Baseline, Month 18
Change from baseline to month 18 in KCCQ-OS score
Time Frame: Baseline, Month 18
Baseline, Month 18

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Intellia Therapeutics

Collaborators

Regeneron Pharmaceuticals

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

December 13, 2023

Primary Completion (Estimated)

December 1, 2027

Study Completion (Estimated)

April 1, 2028

Study Registration Dates

First Submitted

November 8, 2023

First Submitted That Met QC Criteria

November 8, 2023

First Posted (Actual)

November 13, 2023

Study Record Updates

Last Update Posted (Actual)

March 27, 2026

Last Update Submitted That Met QC Criteria

March 23, 2026

Last Verified

March 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ITL-2001-CL-301

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Trials on Transthyretin Amyloidosis (ATTR) With Cardiomyopathy

Clinical Trials on NTLA-2001

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in United Arab Emirates

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

Subscribe