Nucresiran for transthyretin amyloidosis with cardiomyopathy

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, randomized, placebo-controlled study compares nucresiran versus placebo (sterile saline) in adults with hereditary or wild-type ATTR cardiomyopathy. The primary focus is on reducing all-cause mortality and cardiovascular events, with additional measures including other CV outcomes and patient-reported health status and quality of life. Eligible patients have symptomatic heart failure (prior HF hospitalization or need for diuretics) and NT-proBNP within protocol-specified ranges; some background therapies such as TTR stabilizers are permitted. Key exclusions include very advanced heart failure (NYHA IV or NYHA III with stage 3 ATTR), advanced neuropathy, or severe renal impairment (eGFR <30). The sponsor is Alnylam Pharmaceuticals and sites are located in the United States.

Why it matters

Who can join: the full rules

Inclusion Criteria

* Has documented diagnosis of ATTR amyloidosis with cardiomyopathy including those with hereditary ATTR (hATTR) or wild-type ATTR (wATTR) amyloidosis.
* Has medical history of heart failure (HF) with at least 1 prior hospitalization for HF or signs and symptoms that require treatment with a diuretic.
* Has screening N-terminal prohormone B-type natriuretic peptide (NT-proBNP) \>300 ng/L and \<8500 ng/L; In patients with permanent or persistent atrial fibrillation, screening NT-proBNP \>600 ng/L and \<8500 ng/L.
* Patients may be receiving approved TTR stabilizers for ATTR amyloidosis (eg, tafamidis, acoramidis) and may be receiving background therapy for HF at the discretion of the Investigator.

Exclusion Criteria

* Has New York Heart Association (NYHA) Class IV HF; or NYHA Class III heart failure AND ATTR Amyloidosis Disease Stage 3.
* Has a polyneuropathy disability (PND) Score IIIa, IIIb, or IV.
* Has an estimated glomerular filtration rate (eGFR) of \<30 mL/min/1.73m\^2 at screening.
* Has received prior or currently receiving TTR-lowering therapy

Where it's running

• Clinical Trial Site
  La Jolla, California, United States Recruiting
• Clinical Trial Site
  Laguna Hills, California, United States Recruiting
• Clinical Trial Site
  Stanford, California, United States Recruiting
• Clinical Trial Site
  Aurora, Colorado, United States Recruiting
• Clinical Trial Site
  Norwich, Connecticut, United States Recruiting
• Clinical Trial Site
  Washington D.C., District of Columbia, United States Recruiting

+216 more sites. See ClinicalTrials.gov for the full list.

Study contacts

• Study coordinator: Clinical Trial Information Line
• Email: clinicaltrials@alnylam.com
• Phone: 1-877-ALNYLAM

Similar studies

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

• Long-term safety of eplontersen in adults with transthyretin amyloidosis, including those with prior liver transplant or severe liver impairment

  This study will see if eplontersen remains safe over time for adults with transthyretin amyloidosis, including people who have had a liver transplant or have severe liver problems.

  Recruiting at 6 sites AstraZeneca · Los Angeles, California and 5 other locations · NCT07654855

• Long-term global follow-up of adults with ATTR-CM treated with vutrisiran or other approved therapies

  This project will follow adults with ATTR-CM who start vutrisiran or another approved treatment to see how they do over up to five years in routine clinical care.

  Recruiting at 27 sites Alnylam Pharmaceuticals · Tucson, Arizona and 26 other locations · NCT07358078

• 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

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

  This study is testing if a new treatment called NTLA-2001 can improve heart function and health for people with heart issues caused by ATTR amyloidosis.

  Recruiting at 128 sites Phase 3 Intellia Therapeutics · Tucson, Arizona and 137 other locations · NCT06128629

• Annular ligament biopsy during endoscopic carpal tunnel surgery to screen for transthyretin amyloidosis

  This study tests whether a biopsy of the annular ligament during carpal tunnel surgery can reliably indicate transthyretin amyloidosis and match the results of the standard synovial biopsy.

  Recruiting Clinique Saint Jean, France · Montpellier · NCT07644325

• Eplontersen exposure during pregnancy and breastfeeding: outcomes

  This project collects reports to see if exposure to eplontersen during pregnancy or breastfeeding affects mothers, fetuses, or infants with transthyretin amyloidosis.

  Recruiting AstraZeneca · Frankfurt · NCT07205666

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.