A Phase 3 Study of NTLA 2001 in participants with amyloidosis with cardiac muscle disease.

Phase 1

Conditions: Transthyretin Amyloidosis with Cardiomyopathy
MedDRA version: 20.0Level: SOCClassification code: 10007541Term: Cardiac disorders Class: 11
Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14]

Registration Number: CTIS2023-507220-23-00

Lead Sponsor: Intellia Therapeutics Inc.

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 1086

Inclusion Criteria

1. 18 to 90 years of age., 2. Male or Female., 3. Documented diagnosis of ATTR amyloidosis with cardiomyopathy, 4. Medical history of heart failure (HF), 5. Symptoms of HF are optimally managed and clinically stable within 28 days prior to administration of study intervention, 6. Screening NT-proBNP, a blood marker of HF severity, greater than or equal to 1000 pg/mL (or greater than or equal to 2000 pg/mL if participant has known atrial fibrillation)

Exclusion Criteria

1. New York Heart Association (NYHA) Class IV HF, 10. Unable or unwilling to take vitamin A supplementation for the duration of the study., 2. Polyneuropathy Disability score of IV (confined to wheelchair or bed), 3. Has hepatitis B, hepatitis C or human immunodeficiency virus (HIV) infection, 4. History of active malignancy within 3 years prior to screening, 5. RNA silencer therapy (patisiran, inotersen and/or eplontersen) within 12 months prior to dosing. Any prior vutrisiran use is not allowed, 6. Initiation of tafamidis within 6 months prior to study dosing, 7. Estimated glomerular filtration rate (eGFR) <30 mL/min/1.73m^2, 8. Liver failure, 9. Uncontrolled blood pressure

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: To evaluate the efficacy of NTLA-2001, as measured by the composite risk of CV-related mortality and CV events, compared to placebo.;Secondary Objective: 1. To evaluate the effect of NTLA-2001 on serum TTR compared to placebo, 2. To evaluate the impact of NTLA-2001 on participant reported CM related symptoms and quality of life compared to placebo;Primary end point(s): Composite outcome of cardiovascular (CV) mortality and CV events

Secondary Outcome Measures