Yükleniyor... / Loading...
This study is investigating the safety and effectiveness of a treatment called NTLA-2001 compared with placebo in patients with transthyretin amyloidosis with cardiomyopathy (ATTR-CM), a condition where abnormal protein buildup affects the heart muscle. It enrolls patients with a history of heart failure whose symptoms are stable and managed. The study is being conducted in Phase 3 and is currently recruiting participants.
The summary above is a plain-language rendering of the official record. The original English title is shown for reference.
+92
Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine 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