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Evaluating NTLA-2001 for treating heart issues in patients with ATTR amyloidosis

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)

Phase 3 Interventional Intellia Therapeutics · NCT06128629

This study is testing if a new treatment called NTLA-2001 can improve heart function and health for people with heart issues caused by ATTR amyloidosis.

Quick facts

PhasePhase 3
Study typeInterventional
Enrollment1200 (estimated)
Ages18 Years to 90 Years
SexAll
SponsorIntellia Therapeutics Industry-sponsored
Locations132 sites (Tucson, Arizona and 131 other locations)
Trial IDNCT06128629 on ClinicalTrials.gov

What this trial studies

This Phase 3 clinical trial aims to assess the efficacy and safety of a single dose of NTLA-2001 compared to a placebo in participants diagnosed with transthyretin amyloidosis with cardiomyopathy. The study will involve approximately 765 participants across multiple centers and will be conducted in a double-blind, placebo-controlled manner. Participants will be randomized to receive either the treatment or placebo, with a focus on those who have stable heart failure symptoms. The primary goal is to determine the impact of NTLA-2001 on heart function and overall health outcomes.

Who should consider this trial

Good fit: Ideal candidates include individuals with a documented diagnosis of ATTR amyloidosis with cardiomyopathy who are clinically stable and have managed heart failure symptoms.

Not a fit: Patients with severe heart failure (NYHA Class IV) or those with certain comorbidities such as active malignancy or uncontrolled blood pressure may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly improve heart function and quality of life for patients suffering from ATTR-CM.

How similar studies have performed: Previous studies have shown promise in treating ATTR amyloidosis, but this specific approach with NTLA-2001 is novel and has not been extensively tested.

Eligibility criteria

Show full inclusion / exclusion criteria 
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

Where this trial is running

Tucson, Arizona and 131 other locations

+82 more sites — see ClinicalTrials.gov for the full list.

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Transthyretin Amyloidosis With CardiomyopathyTTRAmyloidosisCardiomyopathyNTLA-2001ATTR-CMTransthyretinATTR
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.