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

Recruiting now · Phase 3 · Has a placebo group

Conditions studied: Transthyretin Amyloidosis (ATTR) With Cardiomyopathy

In brief

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

Key facts

Study ID

NCT06128629

Run by

Intellia Therapeutics

People needed

1200

Starts

2023-12-13

Expected to finish

2028-04-01

Last updated by the study team

2026-03-27

Who can join

Age: 18 and older, up to 90. Sex: any. Healthy volunteers: not accepted.

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

Where it is running

• Royal Adelaide Hospital — Adelaide, Australia (enrolling)
• Medizinische Universitaet Innsbruck — Innsbruck, Austria (enrolling)
• Eastern Health - Box Hill Hospital — Box Hill, Victoria, Australia (enrolling)
• Fiona Stanley Hospital — Perth, Western Australia, Australia (enrolling)
• The Alfred Hospital — Melbourne, Australia (enrolling)
• Princess Alexandra Hospital — Woolloongabba, Australia (enrolling)
• University of Pittsburgh Medical Center — Pittsburgh, Pennsylvania, United States (enrolling)
• Medical University of South Carolina — Charleston, South Carolina, United States (enrolling)
• Baylor Scott & White Research Institute — Dallas, Texas, United States (enrolling)
• University of Texas Southwestern Medical Center — Dallas, Texas, United States (enrolling)
• Richmond Pharmacology — London, United Kingdom (enrolling)
• Houston Methodist Hospital — Houston, Texas, United States (enrolling)
• Baylor Scott and White - The Heart Hospital Baylor Plano — Plano, Texas, United States (enrolling)
• INOVA Fairfax Hospital — Falls Church, Virginia, United States (enrolling)
• Virginia Commonwealth University - Department of Internal Medicine — Richmond, Virginia, United States (enrolling)
• Fundacion Favalro — Buenos Aires, Argentina (enrolling)
• Hospital Italiano de Buenos Aires — Buenos Aires, Argentina (enrolling)
• ICBA — Buenos Aires, Argentina (enrolling)
• Fundacion Favaloro — Ciudad Autonoma de Buenos Aire, Argentina (enrolling)
• Hospital Italiano de Buenos Aires — Ciudad Autonoma de Buenos Aire, Argentina (enrolling)
• Hospital Privado de Rosario — Rosario, Argentina (enrolling)
• The Canberra Hospital — Garran, Australian Capital Territory, Australia (enrolling)
• Westmead Hospital — Westmead, New South Wales, Australia (enrolling)
• Baylor College of Medicine — Houston, Texas, United States (enrolling)
• Universitaesklinikum St. Poelten — Sankt Pölten, Austria (enrolling)

Full record on ClinicalTrials.gov

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