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Clinical Trials in the USA / NCT06128629
Recruiting Phase 3

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

NCT06128629 · tracked via the Priya Life Science USA tracker
Sponsor
Intellia Therapeutics
Phase
Phase 3
Started
2023-12-13
Last updated
2026-03-27

Condition(s) studied

Transthyretin Amyloidosis (ATTR) With Cardiomyopathy

Investigational drug(s) / intervention(s)

NTLA-2001Placebo

NTLA-2001: NTLA-2001 (55mg) by IV infusion

Placebo: Normal saline (0.9% NaCl) by IV infusion

Study summary

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

Eligibility

Sex
ALL
Min age
18 Years
Max age
90 Years
Healthy volunteers
No
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

Primary outcome measure(s)

Trial sites (132)

FacilityCityRegionStatus
University of Arizona Tucson Arizona Recruiting
Cedars-Sinai Medical Center Los Angeles California Recruiting
Western Greater Los Angeles VA Medical Center Los Angeles California Recruiting
Stanford University Palo Alto California Recruiting
University of California, San Diego (UCSD) - Medical Center San Diego California Recruiting
University of Colorado Denver Denver Colorado Recruiting
Yale University School of Medicine New Haven Connecticut Recruiting
MedStar Washington Hospital Center - MedStar Heart and Vascular Institute Washington D.C. District of Columbia Recruiting
Mayo Clinic - Jacksonville Jacksonville Florida Recruiting
Cleveland Clinic of Florida Weston Florida Recruiting
Emory University School of Medicine Atlanta Georgia Recruiting
Northwestern University Chicago Illinois Recruiting
Indiana University Health Indianapolis Indiana Recruiting
University of Kentucky Gill Heart Institute Lexington Kentucky Recruiting
Johns Hopkins University Baltimore Maryland Recruiting
Brigham and Women's Hospital Boston Massachusetts Recruiting
Boston University School of Medicine Boston Massachusetts Recruiting
University of Michigan Ann Arbor Michigan Recruiting
Barbara Ann Karmanos Cancer Institute Detroit Michigan Withdrawn
Mayo Clinic Rochester Minnesota Recruiting
St. Luke's Hospital Kansas City Missouri Recruiting
New York University (NYU) School of Medicine - Langone Medical Center New York New York Recruiting
Columbia University Medical Center New York New York Recruiting
Duke University Medical Center Durham North Carolina Recruiting
Cleveland Clinic Cleveland Ohio Recruiting
Ohio Health Research Institute Columbus Ohio Recruiting
Oregon Health and Science University (OHSU) Portland Oregon Recruiting
Penn Presbyterian Medical Center Philadelphia Pennsylvania Recruiting
Allegheny General Hospital Pittsburgh Pennsylvania Recruiting
University of Pittsburgh Medical Center Pittsburgh Pennsylvania Recruiting
Medical University of South Carolina Charleston South Carolina Recruiting
Baylor Scott & White Research Institute Dallas Texas Recruiting
University of Texas Southwestern Medical Center Dallas Texas Recruiting
Baylor College of Medicine Houston Texas Recruiting
Houston Methodist Hospital Houston Texas Recruiting
Baylor Scott and White - The Heart Hospital Baylor Plano Plano Texas Recruiting
INOVA Fairfax Hospital Falls Church Virginia Recruiting
Virginia Commonwealth University - Department of Internal Medicine Richmond Virginia Recruiting
Fundacion Favalro Buenos Aires Argentina Recruiting
Hospital Italiano de Buenos Aires Buenos Aires Argentina Recruiting

+ 92 more sites — see the full list on the official registry below.

More Intellia Therapeutics trials in the USA

Other trials for the same condition

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06128629 on ClinicalTrials.gov ↗ ← All trials in the USA