NCT06128629 · Intellia Therapeutics
MAGNITUDE: A Phase 3 Study of NTLA-2001 in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR-CM)
What this study is about
To evaluate the effectiveness and safety of a single dose of NTLA-2001 compared to placebo in participants with ATTR-CM.
View original scientific description
To evaluate the efficacy and safety of a single dose of NTLA-2001 compared to placebo in participants with ATTR-CM.
Interventions
BIOLOGICAL
NTLA-2001
NTLA-2001 (55mg) by IV infusion
DRUG
Placebo
Normal saline (0.9% NaCl) by IV infusion
Primary outcome measures
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
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
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
- Liver failure
- Uncontrolled blood pressure
- Unable or unwilling t
Where
- Tucson, Arizona
- Los Angeles, California
- Palo Alto, California
- San Diego, California
- Denver, Colorado
- New Haven, Connecticut
- Washington D.C., District of Columbia
- Jacksonville, Florida
- Weston, Florida
- Atlanta, Georgia
- Chicago, Illinois
- Indianapolis, Indiana
And 20 more locations — see the full list below.
Collaborators
Regeneron Pharmaceuticals
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Mar 27, 2026 · Source of record for eligibility and locations