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
- Anderlecht, Brugge, Edegem, Leuven, and Liège, BelgiumSee the 5 sites
- Phase
- Phase 3
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 Belgium
- Hopital ErasmeAnderlecht, BelgiumTaking people
- Az St-Jan Brugge-Oostende A.V.Brugge, BelgiumTaking people
- Antwerp University HospitalEdegem, BelgiumTaking people
- UZ LeuvenLeuven, BelgiumTaking people
- Centre Hospitalier Regional de la CitadelleLiège, BelgiumTaking 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?
Trial 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.