MAGNITUDE: A Phase 3 Study of NTLA-2001 in Participants With Transthyretin Amyloidosis With Cardiomyopathy (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)
1 other identifier
interventional
1,200
25 countries
130
Brief Summary
To evaluate the efficacy and safety of a single dose of NTLA-2001 compared to placebo in participants with ATTR-CM.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for phase_3
Started Dec 2023
Typical duration for phase_3
130 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
November 8, 2023
CompletedFirst Posted
Study publicly available on registry
November 13, 2023
CompletedStudy Start
First participant enrolled
December 13, 2023
CompletedPrimary Completion
Last participant's last visit for primary outcome
December 1, 2027
ExpectedStudy Completion
Last participant's last visit for all outcomes
April 1, 2028
March 27, 2026
March 1, 2026
4 years
November 8, 2023
March 23, 2026
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Composite outcome of cardiovascular (CV) mortality and CV events
Maximum study duration is dependent on event rates and is estimated to be at least 18 months and up to approximately 5 years
Secondary Outcomes (2)
Change in baseline to month 18 in serum TTR
Baseline, Month 18
Change from baseline to month 18 in KCCQ-OS score
Baseline, Month 18
Study Arms (2)
NTLA-2001
EXPERIMENTALSingle intravenous (IV) infusion of NTLA-2001
Placebo
PLACEBO COMPARATORSingle IV infusion of normal saline
Interventions
Eligibility Criteria
You may qualify if:
- 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
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Intellia Therapeuticslead
- Regeneron Pharmaceuticalscollaborator
Study Sites (132)
University of Arizona
Tucson, Arizona, 85724, United States
Cedars-Sinai Medical Center
Los Angeles, California, 90048, United States
Western Greater Los Angeles VA Medical Center
Los Angeles, California, 90073, United States
Stanford University
Palo Alto, California, 94305, United States
University of California, San Diego (UCSD) - Medical Center
San Diego, California, 92103, United States
University of Colorado Denver
Denver, Colorado, 80045, United States
Yale University School of Medicine
New Haven, Connecticut, 06510, United States
MedStar Washington Hospital Center - MedStar Heart and Vascular Institute
Washington D.C., District of Columbia, 20010, United States
Mayo Clinic - Jacksonville
Jacksonville, Florida, 32224, United States
Cleveland Clinic of Florida
Weston, Florida, 33331, United States
Emory University School of Medicine
Atlanta, Georgia, 30322, United States
Northwestern University
Chicago, Illinois, 60611, United States
Indiana University Health
Indianapolis, Indiana, 46202, United States
University of Kentucky Gill Heart Institute
Lexington, Kentucky, 40536, United States
Johns Hopkins University
Baltimore, Maryland, 21287, United States
Brigham and Women's Hospital
Boston, Massachusetts, 02115, United States
Boston University School of Medicine
Boston, Massachusetts, 02118, United States
University of Michigan
Ann Arbor, Michigan, 48109, United States
Barbara Ann Karmanos Cancer Institute
Detroit, Michigan, 48201, United States
Mayo Clinic
Rochester, Minnesota, 55905, United States
St. Luke's Hospital
Kansas City, Missouri, 64111, United States
New York University (NYU) School of Medicine - Langone Medical Center
New York, New York, 10016, United States
Columbia University Medical Center
New York, New York, 10032, United States
Duke University Medical Center
Durham, North Carolina, 27710, United States
Cleveland Clinic
Cleveland, Ohio, 44195, United States
Ohio Health Research Institute
Columbus, Ohio, 43214, United States
Oregon Health and Science University (OHSU)
Portland, Oregon, 97239, United States
Penn Presbyterian Medical Center
Philadelphia, Pennsylvania, 19104, United States
Allegheny General Hospital
Pittsburgh, Pennsylvania, 15212, United States
University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania, 15219, United States
Medical University of South Carolina
Charleston, South Carolina, 29425, United States
Baylor Scott & White Research Institute
Dallas, Texas, 75201, United States
University of Texas Southwestern Medical Center
Dallas, Texas, 75390, United States
Baylor College of Medicine
Houston, Texas, 77030, United States
Houston Methodist Hospital
Houston, Texas, 77030, United States
Baylor Scott and White - The Heart Hospital Baylor Plano
Plano, Texas, 75093, United States
INOVA Fairfax Hospital
Falls Church, Virginia, 22042, United States
Virginia Commonwealth University - Department of Internal Medicine
Richmond, Virginia, 23298, United States
Fundacion Favalro
Buenos Aires, Argentina
Hospital Italiano de Buenos Aires
Buenos Aires, Argentina
ICBA
Buenos Aires, Argentina
Fundacion Favaloro
Ciudad Autonoma de Buenos Aire, Argentina
Hospital Italiano de Buenos Aires
Ciudad Autonoma de Buenos Aire, Argentina
Hospital Privado de Rosario
Rosario, Argentina
The Canberra Hospital
Garran, Australian Capital Territory, Australia
Westmead Hospital
Westmead, New South Wales, Australia
Cairns Hospital
Cairns, Queensland, Australia
Eastern Health - Box Hill Hospital
Box Hill, Victoria, Australia
Fiona Stanley Hospital
Perth, Western Australia, Australia
Royal Adelaide Hospital
Adelaide, Australia
The Alfred Hospital
Melbourne, Australia
Princess Alexandra Hospital
Woolloongabba, Australia
Medizinische Universitaet Innsbruck
Innsbruck, Austria
Universitaesklinikum St. Poelten
Sankt Pölten, Austria
AZ Sint-Jan Brugge
Bruges, Belgium
Universite Libre de Bruxelles (ULB) - Hopital Erasme
Brussels, Belgium
Antwerp University Hospital
Edegem, Belgium
UZ Leuven
Leuven, Belgium
Centre Hospitalier Regional de la Citadelle
Liège, Belgium
Centro de Pesquisa NUPEC - Instituto Orizonti em Belo Horizonte
Belo Horizonte, Brazil
Complexo Hospitalar de Niterói (CHN-DASA)
Niterói, Brazil
CAPED - Centro Avancado de Pesquisa
Ribeirão Preto, Brazil
HCFMRP - Hospital das Clinicas da Faculdade de Medicina de Ribeirao Preto
Ribeirão Preto, Brazil
Cardiopulmonar da Bahia S. A. (Hospital Cardio Pulmonar)
Salvador, Brazil
Hospital Israelita Albert Einstein
São Paulo, Brazil
Instituto Dante Pazzanese de Cardiologia
São Paulo, Brazil
Instituto do Coracao (InCor) do Hospital das Clinicas da FMUSP
São Paulo, Brazil
PSEG Centro de Pesquisa Clinica S.A.
São Paulo, Brazil
Alberta Health Services - University of Calgary
Calgary, Alberta, Canada
Vancouver Coastal Health Research Institute
Vancouver, British Columbia, Canada
University Health Network - Toronto General Hospital
Toronto, Ontario, Canada
McGill University Health Centre
Montreal, Quebec, Canada
Fakultni nemocnice u sv. Anny v Brne
Brno, Czechia
Aarhus University Hospital
Aarhus, Denmark
Rigshospitalet
Copenhagen, Denmark
AP-HP Hopital Henri Mondor
Créteil, France
AP-HM - Hopital de la Timone
Marseille, France
AP-HP Hopital Bichat - Claude Bernard
Paris, France
CHU Rennes - Hopital Pontchaillou
Rennes, France
CHU de Toulouse - Hopital Rangueil
Toulouse, France
Charite Universitaetsmedizin Berlin
Berlin, Germany
Universitaetsklinikum Essen
Essen, Germany
Universitaetsklinikum Heidelberg
Heidelberg, Germany
Universitaetsklinikum Wuerzburg
Würzburg, Germany
Semmelweis Egyetem Belgyogyaszati es Hematologiai Klinika
Budapest, Hungary
Rambam Health Care Campus
Haifa, Israel
Hadassah University Hospital, Ein Karem
Jerusalem, Israel
Sheba Medical Center
Tel Litwinsky, Israel
IRCCS Azienda Ospedaliero-Universitaria di Bologna - Policlinico di Sant'Orsola
Bologna, Italy
Azienda Ospedaliero-Universitaria di Ferrara-Arcispedale Sant'Anna
Cona, Italy
Azienda Ospedaliero-Universitaria Careggi
Florence, Italy
Ospedale Policlinico San Martino IRCCS
Genova, Italy
Fondazione IRCCS Policlinico San Matteo
Pavia, Italy
Fondazione Toscana Gabriele Monasterio per la Ricerca Medica
Pisa, Italy
Azienda sanitaria universitaria Giuliano Isontina (ASU GI) - Ospedale di Cattinara
Trieste, Italy
Kumamoto University Hospital
Kumamoto, Japan
National Cerebral and Cardiovascular Center
Osaka, Japan
The University of Tokyo Hospital
Tokyo, Japan
Toyama University Hospital
Toyama, Japan
Instituto Nacional de Ciencias Medicas y Nutricion Salvador Zubiran
Mexico City, Mexico
University Medical Center
Groningen, Netherlands
Erasmus MC
Rotterdam, Netherlands
Universitair Medisch Centrum Utrecht
Utrecht, Netherlands
New Zealand Clinical Research
Auckland, New Zealand
New Zealand Clinical Research
Christchurch, New Zealand
Oslo University Hospital - Rikshospitalet
Oslo, Norway
Centro Hospitalar Universitario de Lisboa Norte
Lisbon, Portugal
Centro Hospitalar Universitario do Porto
Porto, Portugal
National Heart Centre
Singapore, Singapore
National University Hospital
Singapore, Singapore
Chonnam National University Hospital
Gwangju, South Korea
Samsung Medical Center
Seoul, South Korea
Seoul National University Hospital
Seoul, South Korea
Severance Hospital, Yonsei University Health System
Seoul, South Korea
Hospital Universitario de A Coruna
A Coruña, Spain
Hospital Universitari de Bellvitge
Barcelona, Spain
Hospital Universitario Vall d'Hebron
Barcelona, Spain
Hospital Universitario Puerta de Hierro
Majadahonda, Spain
Hospital Universitario Virgen de la Victoria
Málaga, Spain
Hospital Universitario Son Llatzer
Palma de Mallorca, Spain
Karolinska University Hospital
Stockholm, Sweden
University Hospital of Umea
Umeå, Sweden
Far Eastern Memorial Hospital
New Taipei City, Taiwan
National Taiwan University Hospital
Taipei, Taiwan
Taipai Veterans General Hospital
Taipei, Taiwan
Synexus Midlands Clinical Research Centre
Birmingham, United Kingdom
Synexus Wales Clinical Research Centre
Cardiff, United Kingdom
Synexus Scotland Clinical Research Centre
Glasgow, United Kingdom
Synexus Hexham Clinical Research Centre
Hexham, United Kingdom
Synexus Merseyside Clinical Research Centre
Liverpool, United Kingdom
Richmond Pharmacology
London, United Kingdom
Synexus Manchester Clinical Research Centre
Manchester, United Kingdom
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Central Study Contacts
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- RANDOMIZED
- Masking
- QUADRUPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
November 8, 2023
First Posted
November 13, 2023
Study Start
December 13, 2023
Primary Completion (Estimated)
December 1, 2027
Study Completion (Estimated)
April 1, 2028
Last Updated
March 27, 2026
Record last verified: 2026-03
Data Sharing
- IPD Sharing
- Will not share