Transthyretin Amyloid Cardiomyopathy Clinical Trial

MAGNITUDE: A Phase 3 Study of NTLA-2001 in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR-CM)

Summary

To evaluate the efficacy and safety of a single dose of NTLA-2001 compared to placebo in participants with ATTR-CM.

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Full 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.

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Eligibility Criteria

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 is for people with:

Transthyretin Amyloid Cardiomyopathy

Phase:

Phase 3

Estimated Enrollment:

765

Study ID:

NCT06128629

Recruitment Status:

Recruiting

Sponsor:

Intellia Therapeutics

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There are 3 Locations for this study

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MedStar Washington Hospital Center - MedStar Heart and Vascular Institute
Washington District of Columbia, 20010, United States
University of Kentucky Gill Heart Institute
Lexington Kentucky, 40536, United States
Columbia University Medical Center
New York New York, 10032, United States
The Canberra Hospital
Garran , , Australia
Fiona Stanley Hospital
Perth , , Australia
Westmead Hospital
Westmead , , Australia
New Zealand Clinical Research
Auckland , , New Zealand
New Zealand Clinical Research
Christchurch , , New Zealand
Richmond Pharmacology
London , , United Kingdom

How clear is this clinincal trial information?

Study is for people with:

Transthyretin Amyloid Cardiomyopathy

Phase:

Phase 3

Estimated Enrollment:

765

Study ID:

NCT06128629

Recruitment Status:

Recruiting

Sponsor:


Intellia Therapeutics

How clear is this clinincal trial information?

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