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

Summary

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

Eligibility

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

Conditions2

Interventions1

Locations38 sites

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