MAGNITUDE: A Phase 3 Study of NTLA-2001 in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR-CM)
- Conditions
- Transthyretin Amyloidosis (ATTR) With Cardiomyopathy
- Interventions
- Drug: Placebo
- Registration Number
- NCT06128629
- Lead Sponsor
- Intellia Therapeutics
- Brief Summary
To evaluate the efficacy and safety of a single dose of NTLA-2001 compared to placebo in participants with ATTR-CM.
- Detailed Description
This is a multinational, multicenter, double-blind, placebo-controlled study in approximately 765 participants, who will be randomized to receive a single infusion of either NTLA-2001 or placebo.
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 765
- 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
- Liver failure
- Uncontrolled blood pressure
- Unable or unwilling to take vitamin A supplementation for the duration of the study
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description NTLA-2001 NTLA-2001 Single intravenous (IV) infusion of NTLA-2001 Placebo Placebo Single IV infusion of normal saline
- Primary Outcome Measures
Name Time Method Composite outcome of cardiovascular (CV) mortality and CV events Maximum study duration is dependent on event rates and is estimated to be at least 18 months and up to 48 months
- Secondary Outcome Measures
Name Time Method Change in baseline to month 18 in serum TTR Baseline, Month 18 Change from baseline to month 18 in KCCQ-OS score Baseline, Month 18
Trial Locations
- Locations (130)
University of Arizona
🇺🇸Tucson, Arizona, United States
Cedars-Sinai Medical Center
🇺🇸Los Angeles, California, United States
Western Greater Los Angeles VA Medical Center
🇺🇸Los Angeles, California, United States
Stanford University
🇺🇸Palo Alto, California, United States
University of California, San Diego (UCSD) - Medical Center
🇺🇸San Diego, California, United States
University of Colorado Denver
🇺🇸Denver, Colorado, United States
Yale University School of Medicine
🇺🇸New Haven, Connecticut, United States
MedStar Washington Hospital Center - MedStar Heart and Vascular Institute
🇺🇸Washington D.C., District of Columbia, United States
Mayo Clinic - Jacksonville
🇺🇸Jacksonville, Florida, United States
Cleveland Clinic of Florida
🇺🇸Weston, Florida, United States
Scroll for more (120 remaining)University of Arizona🇺🇸Tucson, Arizona, United States