Azienda Ospedaliero Universitaria Careggi
Florence, Italy
RecruitingRare disease
Study of NTLA-2001 for Patients with Transthyretin Amyloidosis and Heart Disease
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What is this trial about?
A plain-language summary of the goals, design and what participants do
This clinical trial is focused on studying a condition known as Transthyretin Amyloidosis with Cardiomyopathy (ATTR-CM). This is a disease where abnormal proteins build up in the heart, leading to heart problems. The study will test a new treatment called NTLA-2001, which is a type of gene therapy. Gene therapy is a treatment that aims to fix or replace faulty genes in the body. NTLA-2001 contains two special RNA substances that work together to target and modify the gene responsible for the disease.
The purpose of the study is to evaluate how effective and safe NTLA-2001 is for people with ATTR-CM. Participants in the study will receive either NTLA-2001 or a placebo through an intravenous infusion, which means the treatment is given directly into a vein. The study will monitor participants over time to see how the treatment affects their heart health, specifically looking at the risk of heart-related deaths and events.
Throughout the study, researchers will also track changes in certain health markers, such as the level of a protein called transthyretin in the blood, and assess participants' quality of life. The study is designed to provide valuable information about whether NTLA-2001 can help improve the condition of people with ATTR-CM and potentially offer a new treatment option for this challenging disease.
The research process
The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.
Who can join the trial?
6 criteria
- Must be between 18 to 90 years old.
- Can be male or female.
- Must have a documented diagnosis of ATTR amyloidosis with cardiomyopathy. This is a condition where abnormal proteins build up in the heart, affecting its function.
- Must have a medical history of heart failure (HF). Heart failure is when the heart doesn't pump blood as well as it should.
- Symptoms of heart failure must be well-managed and stable for at least 28 days before starting the study treatment.
- Must have a screening level of NT-proBNP, a blood marker that shows how severe heart failure is, of at least 1000 pg/mL. If the person has a known condition called atrial fibrillation (an irregular heart rhythm), the level must be at least 2000 pg/mL.
Who cannot join the trial?
10 criteria
- Patients who have other serious health conditions that could interfere with the study.
- Patients who are currently participating in another clinical trial.
- Patients who have had a recent heart attack or stroke.
- Patients who have a history of severe allergic reactions.
- Patients who are pregnant or breastfeeding.
- Patients who have a history of drug or alcohol abuse.
- Patients who have an infection that requires treatment with antibiotics.
- Patients who have a history of cancer within the last five years.
- Patients who have a condition that affects their immune system.
- Patients who have had a major surgery within the last three months.
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Responsive Responsive Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC)
Rotterdam, The NetherlandsResponsive Katholieke Universiteit te Leuven
Leuven, Belgium
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Investigated drugs
NTLA-2001 is an investigational medication being studied for its potential to treat transthyretin amyloidosis with cardiomyopathy (ATTR-CM). This condition involves the buildup of abnormal proteins in the heart, which can lead to heart problems. The trial aims to assess how well NTLA-2001 works in reducing the risk of heart-related deaths and heart events in patients with this condition.
What is already known about the treatment
NTLA-2001 – This medication is administered intravenously and is currently being studied in clinical trials for its potential to treat transthyretin amyloidosis with cardiomyopathy. It is in the experimental phase, with ongoing research to determine its safety and effectiveness. The main therapeutic indication is to reduce cardiovascular-related mortality and events in affected patients. NTLA-2001 works by using CRISPR technology to edit genes at the molecular level, aiming to decrease the production of abnormal transthyretin protein. It is classified as a gene-editing therapy.
Investigated diseases
Transthyretin Amyloidosis with Cardiomyopathy – This is a rare disease where abnormal protein deposits, known as amyloid, accumulate in the heart tissue. These deposits can cause the heart to become stiff, leading to difficulties in pumping blood effectively. Over time, this can result in symptoms such as fatigue, shortness of breath, and swelling in the legs and abdomen. The condition progresses as the amyloid deposits increase, further impairing heart function. It is a chronic condition that can significantly impact daily activities and quality of life. The disease is often hereditary, meaning it can be passed down through families.
Trial detailsLast updated 4 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2023-507220-23-00Protocol codeITL-2001-CL-301Estimated enrolment1 200 patientsSponsorIntellia Therapeutics Inc.
sourced from the EU Clinical Trials Register and site verification
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