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A Phase 3 Study of NTLA 2001 in participants with amyloidosis with cardiac muscle disease.
EU CTIS ID: 2023-507220-23-00
What this study is testing
To evaluate the efficacy of NTLA‑2001, as measured by the composite risk of CV‑related mortality and CV events, compared to placebo.
- Therapeutic confirmatory (Phase III)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- 1. 18 to 90 years of age.
- 2. Male or Female.
- 3. Documented diagnosis of ATTR amyloidosis with cardiomyopathy
- 4. Medical history of heart failure (HF)
- 5. Symptoms of HF are optimally managed and clinically stable within 28 days prior to administration of study intervention
- 6. Screening NT-proBNP, a blood marker of HF severity, greater than or equal to 600 pg/mL and less than 10,000 pg/mL
You likely can't join if
- 1. New York Heart Association (NYHA) Class IV HF
- 10. Unable or unwilling to take vitamin A supplementation for the duration of the study.
- 2. Polyneuropathy Disability score of IV (confined to wheelchair or bed)
- 3. Has hepatitis B, hepatitis C or human immunodeficiency virus (HIV) infection
- 4. History of active malignancy within 3 years prior to screening
- 5. RNA silencer therapy (patisiran, inotersen and/or eplontersen) within 12 months prior to dosing. Any prior vutrisiran use is not allowed
See the full eligibility criteria
- 1. 18 to 90 years of age.
- 2. Male or Female.
- 3. Documented diagnosis of ATTR amyloidosis with cardiomyopathy
- 4. Medical history of heart failure (HF)
- 5. Symptoms of HF are optimally managed and clinically stable within 28 days prior to administration of study intervention
- 6. Screening NT-proBNP, a blood marker of HF severity, greater than or equal to 600 pg/mL and less than 10,000 pg/mL
- 1. New York Heart Association (NYHA) Class IV HF
- 10. Unable or unwilling to take vitamin A supplementation for the duration of the study.
- 2. Polyneuropathy Disability score of IV (confined to wheelchair or bed)
- 3. Has hepatitis B, hepatitis C or human immunodeficiency virus (HIV) infection
- 4. History of active malignancy within 3 years prior to screening
- 5. RNA silencer therapy (patisiran, inotersen and/or eplontersen) within 12 months prior to dosing. Any prior vutrisiran use is not allowed
- 6. Initiation of tafamidis or acoramidis within 56 days prior to study dosing
- 7. Estimated glomerular filtration rate (eGFR) <30 mL/min/1.73m^2
- 8. History of Liver failure
- 9. Uncontrolled blood pressure
The study team makes the final eligibility decision.
Where it's taking place
- Canada
- Colombia
- United Kingdom
- United States
- Japan
- Singapore
- Brazil
- Israel
- Argentina
- Australia
- Taiwan
- New Zealand
- Mexico
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18-64 years, 65+ years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Canada; Colombia; United Kingdom; United States; Japan; Singapore and 7 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.