MAGNITUDE: a Phase 3 Study of NTLA-2001 in Participants with Transthyretin Amyloidosis with Cardiomyopathy (ATTR-CM)
Purpose
To evaluate the efficacy and safety of a single dose of NTLA-2001 compared to placebo in participants with ATTR-CM.
Condition
- Transthyretin Amyloidosis (ATTR) with Cardiomyopathy
Eligibility
- Eligible Ages
- Between 18 Years and 90 Years
- Eligible Genders
- All
- Accepts Healthy Volunteers
- No
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 1000 pg/mL (or greater than or equal to 2000 pg/mL if participant has known atrial fibrillation)
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 within 6 months 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
- Phase
- Phase 3
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel Assignment
- Intervention Model Description
- Participants will be randomized in a 2:1 ratio to NTLA-2001 or placebo
- Primary Purpose
- Treatment
- Masking
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Arm Groups
| Arm | Description | Assigned Intervention |
|---|---|---|
|
Experimental NTLA-2001 |
Single intravenous (IV) infusion of NTLA-2001 |
|
|
Placebo Comparator Placebo |
Single IV infusion of normal saline |
|
Recruiting Locations
Boston University School of Medicine
Boston, Massachusetts 02118
Boston, Massachusetts 02118
More Details
- Status
- Recruiting
- Sponsor
- Intellia Therapeutics
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.