Evaluating NTLA-2001 for treating heart issues in patients with ATTR amyloidosis

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This Phase 3 clinical trial aims to assess the efficacy and safety of a single dose of NTLA-2001 compared to a placebo in participants diagnosed with transthyretin amyloidosis with cardiomyopathy. The study will involve approximately 765 participants across multiple centers and will be conducted in a double-blind, placebo-controlled manner. Participants will be randomized to receive either the treatment or placebo, with a focus on those who have stable heart failure symptoms. The primary goal is to determine the impact of NTLA-2001 on heart function and overall health outcomes.

Why it matters

Who can join: the full rules

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

Where it's running

• Cedars-Sinai Medical Center
  Los Angeles, California, United States Recruiting
• Western Greater Los Angeles VA Medical Center
  Los Angeles, California, United States Recruiting
• Stanford University
  Palo Alto, California, United States Recruiting
• University of California, San Diego (UCSD) - Medical Center
  San Diego, California, United States Recruiting
• University of Colorado Denver
  Denver, Colorado, United States Recruiting
• Yale University School of Medicine
  New Haven, Connecticut, United States Recruiting

+88 more sites. See ClinicalTrials.gov for the full list.

Study contacts

• Study coordinator: Trial Manager at Intellia
• Email: medicalinformation@intelliatx.com
• Phone: 1-857-285-6200

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Acoramidis for ATTR-CM: Cardiac Imaging Study

  This study tests whether acoramidis can improve heart structure and function in people with transthyretin amyloid cardiomyopathy.

  Recruiting Phase 4 Eidos Therapeutics, a BridgeBio company · St Louis, Missouri · NCT07695701

• Characterizing wild-type transthyretin amyloidosis cardiomyopathy and its impact on quality of life

  This study is trying to understand how wild-type transthyretin amyloidosis cardiomyopathy affects the quality of life for patients at different stages of the disease compared to healthy individuals.

  Recruiting Aarhus University Hospital Skejby · Aarhus, Arrhus N · NCT06291805

• Acoramidis in adults with transthyretin amyloid cardiomyopathy in France

  This is an observational study to see how acoramidis is used in everyday care for adults with ATTR‑CM in France and how it affects their quality of life and daily function.

  Recruiting Bayer · Many Locations · NCT07791160

• Acoramidis for transthyretin amyloid cardiomyopathy in routine care

  See how acoramidis works for adults with transthyretin amyloid cardiomyopathy (wild-type or variant) when given as part of regular clinical care.

  Recruiting at 17 sites Bayer · Multiple Locations and 16 other locations · NCT07235462

• Coramitug (NNC6019-0001) for heart failure from transthyretin amyloid cardiomyopathy

  This study tests whether coramitug (NNC6019-0001) can lower the risk of heart-related death and illness in adults with transthyretin amyloid cardiomyopathy (ATTR-CM) while they continue their usual heart medicines.

  Recruiting at 237 sites Phase 3 Novo Nordisk A/S · Phoenix, Arizona and 298 other locations · NCT07207811

• Registry for studying Transthyretin Amyloid Cardiomyopathy in Thailand

  This study is collecting information from people with Transthyretin Amyloid Cardiomyopathy in Thailand to better understand the disease and improve early detection and treatment options.

  Recruiting Mahidol University · Bangkok and 7 other locations · NCT06338696

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.