Tests NTLA-2001 against placebo in transthyretin amyloidosis with heart disease
Tests a single NTLA-2001 dose against placebo in adults with heart disease from transthyretin amyloidosis.
Plain-language title generated from the registry record
Registry title: MAGNITUDE: A Phase 3 Study of NTLA-2001 in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR-CM)
Key facts
- Where
- Aarhus N and Copenhagen Oe, DenmarkSee the 2 sites
- Phase
- Phase 3
AI summary: who the study is looking for
From the trial's eligibility criteria. Machine-read from the registry text on this page, so it can be wrong. The registry text below and the trial team decide who can take part.
The trial is looking for adults with transthyretin amyloidosis that affects the heart (ATTR-CM) and a history of heart failure. It tests a single dose of NTLA-2001 against placebo.
- Age
- 18 to 90 years
- Sex
- All sexes can join.
- Disease stage
- Documented ATTR amyloidosis with cardiomyopathy and a history of heart failure. Heart failure symptoms must be well managed and stable for 28 days before treatment. NYHA Class IV heart failure is not allowed.
- Earlier treatment
- No RNA silencer therapy (patisiran, inotersen, eplontersen) within 12 months before dosing. No prior vutrisiran. No start of tafamidis or acoramidis within 56 days before dosing.
- Biomarker or test result
- NT-proBNP in the blood of 600 pg/mL or more and less than 10,000 pg/mL at screening.
Main reasons for not taking part
- NYHA Class IV heart failure
- Polyneuropathy Disability score of IV (confined to wheelchair or bed)
- Hepatitis B, hepatitis C or HIV infection
- Active cancer within 3 years before screening
- Liver disease, or eGFR below 30 mL/min/1.73m^2
Read by machine on 12 November 2026.
Could this be for you?
Answer for yourself. This is not a decision: only the study team can say whether you can take part.
- Are you between 18 and 90 years old?
Source: NCT06128629, who can take part
Where in Denmark
- Aarhus UniversitetshospitalAarhus N, DenmarkTaking people
- Copenhagen University HospitalCopenhagen Oe, DenmarkTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Contact the study team: see the registry record → Contacts and Locations
Print a page for my doctorTrial details
- Who runs it
- Intellia Therapeutics
- Trial code
- NCT06128629EU 2023-507220-23-00
- Record updated
- 4 November 2026
- Original description
To evaluate the efficacy and safety of a single dose of NTLA-2001 compared to placebo in participants with ATTR-CM.Read full original descriptionShow less
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)
To evaluate the efficacy and safety of a single dose of NTLA-2001 compared to placebo in participants with ATTR-CM.
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: 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; New York Heart Association (NYHA) Class IV HF; Has hepatitis B, hepatitis C or human immunodeficiency virus (HIV) infection; History of liver disease; Estimated glomerular filtration rate (eGFR) <30 mL/min/1.73m\^2; Polyneuropathy Disability score of IV (confined to wheelchair or bed); Uncontrolled blood pressure; Unable or unwilling to take vitamin A supplementation for the duration of the study.