Status:
ACTIVE_NOT_RECRUITING
MAGNITUDE: A Phase 3 Study of NTLA-2001 in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR-CM)
Lead Sponsor:
Intellia Therapeutics
Collaborating Sponsors:
Regeneron Pharmaceuticals
Conditions:
Transthyretin Amyloidosis (ATTR) With Cardiomyopathy
Eligibility:
All Genders
18-90 years
Phase:
PHASE3
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 1200 participants, who will be randomized to receive a single infusion of either NTLA-2001 or placebo.
Eligibility Criteria
Inclusion
- 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
- 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
Key Trial Info
Start Date :
December 13 2023
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
April 1 2028
Estimated Enrollment :
1200 Patients enrolled
Trial Details
Trial ID
NCT06128629
Start Date
December 13 2023
End Date
April 1 2028
Last Update
December 2 2025
Active Locations (126)
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1
University of Arizona
Tucson, Arizona, United States, 85724
2
Cedars-Sinai Medical Center
Los Angeles, California, United States, 90048
3
Western Greater Los Angeles VA Medical Center
Los Angeles, California, United States, 90073
4
Stanford University
Palo Alto, California, United States, 94305