RecruitingTransthyretin Amyloidosis (ATTR) With Cardiomyopathy

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

Eligible age

18–90 yrs

Accepts

All genders

Locations

23 states

Healthy volunteers

See if you qualify for this study

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

Sponsor: Intellia Therapeutics

✓ 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

✕ 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