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

Launched by INTELLIA THERAPEUTICS · Nov 8, 2023

Keywords

ClinConnect Summary

The MAGNITUDE trial is a Phase 3 study that is looking at a new treatment called NTLA-2001 for people with a condition called transthyretin amyloidosis with cardiomyopathy (ATTR-CM), which affects the heart. The main goal of the trial is to see if a single dose of NTLA-2001 is effective and safe compared to a placebo (a treatment that looks like the real thing but doesn’t contain active medicine). The trial is currently recruiting participants aged between 65 and 74 years, and it is open to all genders.

To be eligible for the study, participants should have a documented diagnosis of ATTR amyloidosis with cardiomyopathy and a history of heart failure that is currently well-managed. They should also be stable for at least 28 days before starting the study. However, those with severe heart failure symptoms, certain infections, or specific kidney and liver issues won’t be able to participate. If you join the study, you can expect to receive either the treatment or a placebo and will be closely monitored throughout the trial. It’s important to note that participants will need to take vitamin A supplements during the study. This trial could provide valuable information about a potential new treatment for this heart condition.

Gender

ALL

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 600 pg/mL and less than 10,000 pg/mL
• 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 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