Recruiting

Phase 3

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

Sponsor:

Intellia Therapeutics

Code:

NCT06128629

Conditions

Transthyretin Amyloidosis (ATTR) With Cardiomyopathy

Eligibility Criteria

Sex: All

Age: 18 - 70+

Healthy Volunteers: Not accepted

Interventions

NTLA-2001

Placebo

Study Details

Brief summary:

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

Study ID

NCT06128629

Start date

Dec 13, 2023

Status verified date

Mar, 2026

Completion date

Apr, 2028

Anticipated

Primary completion date

Dec, 2027

Anticipated

Eligibility Criteria

Sex: All

Age: 18 - 70+

Healthy Volunteers: Not accepted

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 Design

Enrollment

1200 participants

Anticipated

Allocation

Randomized

Intervention Model

Parallel Assignment

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: NTLA-2001

Single intravenous (IV) infusion of NTLA-2001

placebo comparator: Placebo

Single IV infusion of normal saline

Interventions

NTLA-2001

NTLA-2001 (55mg) by IV infusion

Placebo

Normal saline (0.9% NaCl) by IV infusion

Primary outcome measure

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 ]

Central Contacts and Locations

Central contacts

Trial Manager at Intellia

1-857-285-6200 medicalinformation@intelliatx.com

Locations

University of Arizona

Recruiting

Tucson, Arizona, United States, 85724

Cedars-Sinai Medical Center

Recruiting

Los Angeles, California, United States, 90048

Western Greater Los Angeles VA Medical Center

Recruiting

Los Angeles, California, United States, 90073

Stanford University

Recruiting

Palo Alto, California, United States, 94305

University of California, San Diego (UCSD) - Medical Center

Recruiting

San Diego, California, United States, 92103

University of Colorado Denver

Recruiting

Denver, Colorado, United States, 80045

Yale University School of Medicine

Recruiting

New Haven, Connecticut, United States, 06510

MedStar Washington Hospital Center - MedStar Heart and Vascular Institute

Recruiting

Washington D.C., District of Columbia, United States, 20010

Mayo Clinic - Jacksonville

Recruiting

Jacksonville, Florida, United States, 32224

Cleveland Clinic of Florida

Recruiting

Weston, Florida, United States, 33331

Emory University School of Medicine

Recruiting

Atlanta, Georgia, United States, 30322

Northwestern University

Recruiting

Chicago, Illinois, United States, 60611

Indiana University Health

Recruiting

Indianapolis, Indiana, United States, 46202

University of Kentucky Gill Heart Institute

Recruiting

Lexington, Kentucky, United States, 40536

Johns Hopkins University

Recruiting

Baltimore, Maryland, United States, 21287

Brigham and Women's Hospital

Recruiting

Boston, Massachusetts, United States, 02115

Boston University School of Medicine

Recruiting

Boston, Massachusetts, United States, 02118

University of Michigan

Recruiting

Ann Arbor, Michigan, United States, 48109

Mayo Clinic

Recruiting

Rochester, Minnesota, United States, 55905

St. Luke's Hospital

Recruiting

Kansas City, Missouri, United States, 64111

New York University (NYU) School of Medicine - Langone Medical Center

Recruiting

New York, New York, United States, 10016

Columbia University Medical Center

Recruiting

New York, New York, United States, 10032

Duke University Medical Center

Recruiting

Durham, North Carolina, United States, 27710

Cleveland Clinic

Recruiting

Cleveland, Ohio, United States, 44195

Ohio Health Research Institute

Recruiting

Columbus, Ohio, United States, 43214

Oregon Health and Science University (OHSU)

Recruiting

Portland, Oregon, United States, 97239

Penn Presbyterian Medical Center

Recruiting

Philadelphia, Pennsylvania, United States, 19104

Allegheny General Hospital

Recruiting

Pittsburgh, Pennsylvania, United States, 15212

University of Pittsburgh Medical Center

Recruiting

Pittsburgh, Pennsylvania, United States, 15219

Medical University of South Carolina

Recruiting

Charleston, South Carolina, United States, 29425

Baylor Scott & White Research Institute

Recruiting

Dallas, Texas, United States, 75201

University of Texas Southwestern Medical Center

Recruiting

Dallas, Texas, United States, 75390

Baylor College of Medicine

Recruiting

Houston, Texas, United States, 77030

Houston Methodist Hospital

Recruiting

Houston, Texas, United States, 77030

Baylor Scott and White - The Heart Hospital Baylor Plano

Recruiting

Plano, Texas, United States, 75093

INOVA Fairfax Hospital

Recruiting

Falls Church, Virginia, United States, 22042

Virginia Commonwealth University - Department of Internal Medicine

Recruiting

Richmond, Virginia, United States, 23298

Alberta Health Services - University of Calgary

Recruiting

Calgary, Alberta, Canada

Vancouver Coastal Health Research Institute

Recruiting

Vancouver, British Columbia, Canada

University Health Network - Toronto General Hospital

Recruiting

Toronto, Ontario, Canada

McGill University Health Centre

Recruiting

Montreal, Quebec, Canada

More Information

Sponsor

Intellia Therapeutics

Last update posted

Mar 27, 2026

Last verified

Mar, 2026

Keywords

TTR
Amyloidosis
Cardiomyopathy
NTLA-2001
ATTR-CM
Transthyretin
ATTR
TTR-mediated amyloidosis
Amyloidosis, Hereditary
Amyloidosis, Hereditary, Transthyretin-Related Amyloidosis
Transthyretin amyloid cardiomyopathy
TTR cardiomyopathy
Wild-type TTR
V122I
Amyloidosis, Wild Type
CRISPR/Cas9

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-08. This information was provided to ClinicalTrials.gov by Intellia Therapeutics on 2026-03-27.