Tests NTLA-2001 against placebo in transthyretin amyloidosis with heart disease
Tests a single NTLA-2001 dose against placebo in adults with heart disease from transthyretin amyloidosis.
Plain-language title generated from the registry record
Registry title: MAGNITUDE: A Phase 3 Study of NTLA-2001 in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR-CM)
Key facts
- Where
- Ann Arbor, Atlanta, Baltimore, Boston, Charleston, Chicago, Cleveland, Columbus, Dallas, Denver, Detroit, Durham, Falls Church, Houston, Indianapolis, Jacksonville, Kansas City, Lexington, Los Angeles, Madison, New Haven, New York, Palo Alto, Philadelphia, Pittsburgh, Plano, Portland, Richmond, Rochester, San Diego, Tucson, Washington D.C., and Weston, United StatesSee the 39 sites
- Phase
- Phase 3
Could this be for you?
Answer for yourself. This is not a decision: only the study team can say whether you can take part.
- Are you between 18 and 90 years old?
Source: NCT06128629, who can take part
Where in United States
- University of MichiganAnn Arbor, United StatesTaking people
- Emory University School of MedicineAtlanta, United StatesTaking people
- Johns Hopkins UniversityBaltimore, United StatesTaking people
- Boston University School of MedicineBoston, United StatesTaking people
- Brigham and Women's HospitalBoston, United StatesTaking people
- Medical University of South CarolinaCharleston, United StatesTaking people
- Northwestern UniversityChicago, United StatesTaking people
- Cleveland ClinicCleveland, United StatesTaking people
- Ohio Health Research InstituteColumbus, United StatesTaking people
- Baylor Scott & White Research InstituteDallas, United StatesTaking people
- University of Texas Southwestern Medical CenterDallas, United StatesTaking people
- University of Colorado DenverDenver, United StatesTaking people
- Barbara Ann Karmanos Cancer InstituteDetroit, United StatesNo longer taking part
- Duke University Medical CenterDurham, United StatesTaking people
- Inova Fairfax HospitalFalls Church, United StatesTaking people
- Baylor College of MedicineHouston, United StatesTaking people
- Houston Methodist HospitalHouston, United StatesTaking people
- Indiana University HealthIndianapolis, United StatesTaking people
- Mayo Clinic- JacksonvilleJacksonville, United StatesTaking people
- St. Luke's HospitalKansas City, United StatesTaking people
- University of Kentucky Gill Heart InstituteLexington, United StatesTaking people
- Cedars-Sinai Medical CenterLos Angeles, United StatesTaking people
- Western Greater Los Angeles VA Medical CenterLos Angeles, United StatesTaking people
- University of Wisconsin - MadisonMadison, United StatesTaking people
- Yale University School Of MedicineNew Haven, United StatesTaking people
- Columbia University Medical CenterNew York, United StatesTaking people
- New York University (NYU) School of Medicine - Langone Medical CenterNew York, United StatesTaking people
- Stanford UniversityPalo Alto, United StatesTaking people
- Penn Presbyterian Medical CenterPhiladelphia, United StatesTaking people
- Allegheny General HospitalPittsburgh, United StatesTaking people
- University of Pittsburgh Medical CenterPittsburgh, United StatesTaking people
- Baylor Scott and White - The Heart Hospital Baylor PlanoPlano, United StatesTaking people
- Oregon Health and Science university (OHSU)Portland, United StatesTaking people
- Virginia Commonwealth University - Department of Internal MedicineRichmond, United StatesTaking people
- Mayo ClinicRochester, United StatesTaking people
- University of California, San Diego (UCSD) Medical CenterSan Diego, United StatesTaking people
- University of ArizonaTucson, United StatesNo longer taking part
- MedStar Washington Hospital Center - MedStar Heart and Vascular InstituteWashington D.C., United StatesTaking people
- Cleveland Clinic of FloridaWeston, United StatesTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Intellia Therapeutics
- Trial code
- NCT06128629EU 2023-507220-23-00
- Record updated
- 4 November 2026
- Original description
To evaluate the efficacy and safety of a single dose of NTLA-2001 compared to placebo in participants with ATTR-CM.Read full original descriptionShow less
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.
Inclusion: 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: 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; New York Heart Association (NYHA) Class IV HF; Has hepatitis B, hepatitis C or human immunodeficiency virus (HIV) infection; History of liver disease; Estimated glomerular filtration rate (eGFR) <30 mL/min/1.73m\^2; Polyneuropathy Disability score of IV (confined to wheelchair or bed); Uncontrolled blood pressure; Unable or unwilling to take vitamin A supplementation for the duration of the study.