At a glance
- What the study gets you
- Access to the study treatment being tested
- Type of study
- Interventional (receives a drug or procedure)
- Time in hospital
- In-person visits at study sites — visit count not specified by the sponsor
- Drug or intervention
- NTLA-2001 (biological), Placebo (drug)
- How long the study runs
- Study runs about 52 months (dates as stated)
- About the drug or intervention
- NTLA-2001 — biological: NTLA-2001 (55mg) by IV infusion · Placebo — drug: Normal saline (0.9% NaCl) by IV infusion
- Patient visit burden
- Not specified by the sponsor
In plain English
This is a Phase 3 study looking at a medicine called NTLA-2001 in people with transthyretin amyloidosis with cardiomyopathy (ATTR-CM), a condition where a faulty protein builds up in the heart. The study is run by Intellia Therapeutics. Phase 3 means the treatment is being tested in larger groups of people to see how well it works.
Who can take part
- A confirmed diagnosis of transthyretin amyloidosis affecting the heart (cardiomyopathy)
- A history of heart failure
- Heart failure symptoms that are well managed and stable for at least 28 days before receiving the study treatment
- A blood test result for NT-proBNP (a marker of heart failure severity) between 600 and 10,000 pg/mL at screening
Who may not be able to
- The most severe stage of heart failure (New York Heart Association Class IV)
- A polyneuropathy disability score of IV (needing a wheelchair or being confined to bed)
- Hepatitis B, hepatitis C or HIV infection
- A history of active cancer within the past 3 years
- Recent RNA silencer treatment (patisiran, inotersen or eplontersen) within the past 12 months, or any past use of vutrisiran
- Starting tafamidis or acoramidis within 56 days before the study dose
- Severely reduced kidney function (eGFR below 30 mL/min/1.73m²)
- A history of liver disease
- Uncontrolled blood pressure
- Being unable or unwilling to take vitamin A supplements for the whole study
What taking part involves
- • Not stated — ask the trial team
Time commitment: Not stated — ask the trial team how many visits there are, how long the study lasts, and what taking part involves.
Plain-English summary (AI-generated) from registry data. Not eligibility advice — only the trial team can confirm whether you can take part. Use the eligibility checker to see how your health profile matches this trial.
- Type of study
- Testing a treatment
- Ages
- 18 Years to 90 Years
- Who
- All
- Number of participants
- 1,200
- Started
- 2023-12-13
- Last checked
- 2026-03
Plain English Summary
What is this study?
- • Testing a new treatment for transthyretin amyloidosis (attr) with cardiomyopathy
- • Phase3 - 1,200 participants
- • To evaluate the efficacy and safety of a single dose of NTLA-2001 compared to placebo in participants with ATTR-CM
Who can take part?
- • Ages 18 Years to 90 Years
- • Diagnosed with transthyretin amyloidosis (attr) with cardiomyopathy
Where?
- • Birmingham - Synexus Midlands Clinical Research Centre
- • Cardiff - Synexus Wales Clinical Research Centre
- • Glasgow - Synexus Scotland Clinical Research Centre
- • Hexham - Synexus Hexham Clinical Research Centre
- • +3 more UK sites
This is a simplified summary. Always discuss with your doctor before making any decisions.
About This Trial
To evaluate the efficacy and safety of a single dose of NTLA-2001 compared to placebo in participants with ATTR-CM.
More detail
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.
How this trial compares with your answers
Answer 2 more questions to improve match
What we know so far
Still need:
- • Tell us your age for better matching
- • Tell us your sex for better matching
Preliminary match based on your answers. Full eligibility requires on-site assessment including medical history, physical exam, and lab tests. This does not guarantee enrolment.
Eligibility at a Glance
Key info
- Age: 18 Years - 90 Years
- Who can join: All genders
Biomarkers mentioned
Treatment history
Treatments you must NOT have had:
- ✗ to dosing. Any prior vutrisiran use
What the study is looking for
- ✓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
Who cannot take part
- ✗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 cancer within 3 years prior to screening
- ✗RNA silencer therapy (patisiran, inotersen and/or eplontersen) within 12 months prior to dosing. Any prior...
See the full criteria
Where Is This Study? (7 UK sites)
Synexus Midlands Clinical Research Centre
Birmingham, United Kingdom
Synexus Wales Clinical Research Centre
Cardiff, United Kingdom
Synexus Scotland Clinical Research Centre
Glasgow, United Kingdom
Synexus Hexham Clinical Research Centre
Hexham, United Kingdom
Synexus Merseyside Clinical Research Centre
Liverpool, United Kingdom
Richmond Pharmacology
London, United Kingdom
Synexus Manchester Clinical Research Centre
Manchester, United Kingdom
How to Get in Touch
Trial Manager at Intellia
Sponsor contactCONTACT