ClinicalTrials.gov record NCT06128629
MAGNITUDE: A Phase 3 Study of NTLA-2001 in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR-CM)
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)
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
This Phase 3 randomized, placebo-controlled study compares a single intravenous dose of NTLA-2001 (55 mg) with placebo in people who have transthyretin amyloidosis with cardiomyopathy (ATTR-CM). About 1,200 participants will be randomized and followed for clinical outcomes. The main outcome is a composite of cardiovascular death and cardiovascular events; other measures include blood transthyretin levels and a heart-failure quality-of-life score at 18 months.
What it is studying. The study tests the safety and effectiveness of a single 55 mg IV infusion of NTLA-2001 compared with placebo in people with ATTR-CM, using cardiovascular death and events as the main outcome and measuring changes in blood transthyretin and quality-of-life.
What the researchers hope to learn. To evaluate the efficacy and safety of a single IV dose of NTLA-2001 compared with placebo in ATTR-CM. Primary scientific goal: measure impact on a composite of cardiovascular mortality and cardiovascular events. Secondary goals include measuring change in serum transthyretin at 18 months and change in Kansas City Cardiomyopathy Questionnaire overall summary (KCCQ-OS) score.
What this study is about
The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.
To evaluate the efficacy and safety of a single dose of NTLA-2001 compared to placebo in participants with ATTR-CM.
Interventions being studied
Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.
- Drug
- Placebo
- Biological
- NTLA-2001
Eligibility
The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.
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
- History of liver disease
- Uncontrolled blood pressure
- Unable or unwilling to take vitamin A supplementation for the duration of the study
Study description and what participation involves
The study's detailed description, as published on ClinicalTrials.gov.
Detailed description from the registry
This is a multinational, multicenter, double-blind, placebo-controlled study in approximately 1200 participants, who will be randomized to receive a single infusion of either NTLA-2001 or placebo.
AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
You would receive one IV infusion of either NTLA‑2001 (55 mg) or a saline placebo (randomized, double‑blind). After the infusion you would attend regular clinic visits for safety and heart monitoring, blood tests (including NT‑proBNP and serum TTR), and questionnaires such as the Kansas City Cardiomyopathy Questionnaire at baseline and month 18. Follow‑up is planned for at least 18 months and could extend to about 5 years depending on event rates. You would be required to take vitamin A supplements for the duration of the study.
How the study is structured. Phase 3, randomized, double-blind, placebo-controlled, parallel-group study. Participants receive one IV infusion of NTLA-2001 (55 mg) or saline placebo and are followed over time; the planned enrollment is about 1,200 people. Follow-up for the primary outcome is estimated to be at least 18 months and up to about 5 years depending on event rates. Key measurements include a composite of cardiovascular mortality and events, serum transthyretin levels, and a heart-failure quality-of-life questionnaire score.
Questions you could ask
These questions are suggestions for a conversation with the study team or your own clinician, not advice.
- The registry lists this study under Transthyretin Amyloidosis (ATTR) With Cardiomyopathy. Does it apply to my diagnosis?
- Some participants receive a placebo. How is that decided, and what happens for placebo-group participants when the study ends?
- The listed sites are in Argentina, Australia, Austria, Belgium, Brazil, Canada, Czechia, Denmark, France, Germany, Hungary, Israel, Italy, Japan, Mexico, Netherlands, New Zealand, Norway, Portugal, Singapore, South Korea, Spain, Sweden, Taiwan, United Kingdom, United States. Is any of them reachable for me, and does the study help with travel?
- Which of the eligibility criteria would need to be checked with tests before I could join?
More on ATTRv amyloidosis
This trial is also listed on this site under wild-type ATTR amyloidosis.
Important dates
- Study start
- Dec 13, 2023
- Primary completion
- December 2027 (estimated)
- Study completion
- April 2028 (estimated)
- Target enrollment
- 1200 participants (estimated)
- Last updated on registry
- Mar 27, 2026
- Data verified
- Oct 10, 2026
Sponsor and research team
The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.
- Lead sponsor
- Intellia Therapeutics
- Collaborators
- Regeneron Pharmaceuticals
Central study contact
If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.
- Trial Manager at Intellia 📞 1-857-285-6200 ext. 6 ✉ medicalinformation@intelliatx.com
Participating sites (132)
- University of ArizonaRecruiting
Tucson, Arizona, 85724 · United States
- Cedars-Sinai Medical CenterRecruiting
Los Angeles, California, 90048 · United States
- Western Greater Los Angeles VA Medical CenterRecruiting
Los Angeles, California, 90073 · United States
- Stanford UniversityRecruiting
Palo Alto, California, 94305 · United States
- University of California, San Diego (UCSD) - Medical CenterRecruiting
San Diego, California, 92103 · United States
- University of Colorado DenverRecruiting
Denver, Colorado, 80045 · United States
- Yale University School of MedicineRecruiting
New Haven, Connecticut, 06510 · United States
- MedStar Washington Hospital Center - MedStar Heart and Vascular InstituteRecruiting
Washington D.C., District of Columbia, 20010 · United States
- Mayo Clinic - JacksonvilleRecruiting
Jacksonville, Florida, 32224 · United States
- Cleveland Clinic of FloridaRecruiting
Weston, Florida, 33331 · United States
Terms used on this page · explanations by MedicalUpdates.ai
- placebo
- A treatment that looks like the study treatment but contains no active ingredient. Some participants receive it instead of the study treatment, usually without knowing which they got.
- prior to screening
- The checks done before enrolment to see whether a person qualifies, such as tests, scans or a review of medical history.
- Estimated glomerular filtration rate
- A blood-test estimate of how well the kidneys are filtering. Lower numbers mean reduced kidney function; many studies set a minimum value.
- Inclusion Criteria
- The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
- Exclusion Criteria
- The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
- double-blind
- Two types of people involved in the study do not know which intervention a participant is receiving. The ClinicalTrials.gov record specifies who is masked, for example participants, care providers, investigators or outcome assessors.
- placebo-controlled
- A study design in which one group receives a placebo so that the study treatment's effects can be compared with no active treatment.
- randomized
- Participants are assigned to the study groups by chance, like a coin toss, rather than chosen by the doctor or the participant. This is done so the groups are comparable.
The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.
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