MAGNITUDE: A Phase 3, Multinational, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of NTLA-2001 in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR-CM)
N
Noel Dasgupta
Primary Investigator
Enrolling By Invitation
18 years - 100 years
All
Phase
3
5 participants needed
1 Location
Brief description of study
What is the purpose of this study?
To evaluate the efficacy and safety of a single dose of NTLA-2001 compared to placebo in participants with ATTR-CM.
Detailed description of study
What will happen during the study?
- 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.
- NTLA-2001 will be administered as a single intravenous infusion at a fixed dose of 55 milligrams (mg).
- To mitigate potential infusion related reactions (IRRs), participants will receive pre-medication with oral dexamethasone (or equivalent) prior to infusion.
- Clinical samples including blood and urine may be collected throughout the study for various medical- and study-related assessments including measurement of anti-drug antibodies (ADAs) and anti-Cas9 protein antibodies.
- Participants will be followed for a minimum of 18 months (screening through final visit) and will then be asked to participate in a separate long-term follow-up (LTFU) study for an additional 15 years to assess long-term safety and efficacy.
Eligibility of study
You may be eligible for this study if you meet the following criteria:
- Conditions: Transthyretin Amyloidosis (ATTR) With Cardiomyopathy
-
Age: 18 years - 100 years
-
Gender: All
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
Updated on
07 Feb 2025.
Study ID: PATH-IT-NTLA-2001, 21070
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