Nucresiran versus vutrisiran for hereditary transthyretin polyneuropathy

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, head-to-head interventional trial compares the RNAi therapeutic nucresiran to vutrisiran in adults with genetically confirmed hATTR-PN. Eligible participants have a documented TTR gene variant, a neuropathy impairment score (NIS) between 5 and 130, and a Karnofsky Performance Status of at least 60%. Participants receive assigned treatment and are followed for changes in neurologic impairment, quality of life, nutritional status, disability, and gait speed as well as serum transthyretin levels. The study excludes people with prior or planned liver transplant, non-hATTR forms of amyloidosis, significant heart failure, marked liver enzyme or bilirubin elevation, severe renal impairment, or other known causes of neuropathy.

Why it matters

Who can join: the full rules

Inclusion Criteria:

* Has documented diagnosis of hATTR-PN
* Has a diagnosis of hATTR amyloidosis with polyneuropathy with a documented TTR gene variant
* Has a neuropathy impairment score (NIS) of 5 to 130 (inclusive)
* Has a Karnofsky Performance Status (KPS) of ≥60%

Exclusion Criteria:

* Has had a liver transplant or is likely, in the opinion of the Investigator, to undergo liver transplantation during the Treatment Period of the study
* Has known other (non-hATTR) forms of amyloidosis or clinical evidence of leptomeningeal amyloidosis
* Has a New York Heart Association (NYHA) heart failure classification \>2
* Has alanine aminotransferase (ALT) or aspartate aminotransferase (AST) \>2.5 upper limit of normal (ULN)
* Has total bilirubin \>1.5 ULN
* Has estimated glomerular filtration rate (eGFR) ≤30 mL/min/1.73m\^2
* Has other known causes of sensorimotor or autonomic neuropathy

Where it's running

• Clinical Trial Site
  Aurora, Colorado, United States Recruiting
• Clinical Trial Site
  Chicago, Illinois, United States Recruiting
• Clinical Trial Site
  Baltimore, Maryland, United States Recruiting
• Clinical Trial Site
  Boston, Massachusetts, United States Recruiting
• Clinical Trial Site
  Rochester, Minnesota, United States Recruiting
• Clinical Trial Site
  New York, New York, United States Recruiting

+11 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.

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  Recruiting at 7 sites Alnylam Pharmaceuticals · Iowa City, Iowa and 6 other locations · NCT05040373

• 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

• Observational study of patients with ATTR amyloidosis

  This study looks at the characteristics of people with ATTR amyloidosis and tests how well existing treatments work for them in real life.

  Recruiting at 38 sites Alnylam Pharmaceuticals · La Jolla, California and 39 other locations · NCT04561518

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.