ClinicalTrials.gov record NCT07223203
TRITON-PN: A Study to Evaluate the Efficacy and Safety of Nucresiran in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy
TRITON-PN: A Phase 3, Global, Randomized, Open-Label Study to Evaluate the Efficacy and Safety of Nucresiran in Patients With Hereditary Transthyretin-Mediated Amyloidosis With Polyneuropathy (hATTR-PN)
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
This is a Phase 3, randomized, open-label study testing nucresiran in adults with hereditary transthyretin-mediated amyloidosis with polyneuropathy (hATTR‑PN). It compares regular subcutaneous nucresiran injections to in-study vutrisiran injections and assesses neurologic impairment, quality of life, nutritional status, disability, gait speed, and serum TTR levels. Some outcomes are compared to an external placebo group from the APOLLO study at months 9 and 18.
What it is studying. The study is measuring whether nucresiran, given by subcutaneous injection, changes neurologic impairment and other clinical measures in people with hATTR‑PN, and whether nucresiran lowers blood transthyretin (TTR) more than vutrisiran. The main clinical measure is change in the modified Neuropathy Impairment Score +7 (mNIS+7); the study also collects patient-reported quality-of-life scores, nutritional measures, disability scales, gait speed, and serum TTR levels. Safety of nucresiran is being evaluated alongside these measures.
What the researchers hope to learn. To evaluate the efficacy and safety of nucresiran in people with hATTR‑PN; to assess effects on neurologic impairment, quality of life, nutrition, disability, and gait speed; and to compare serum TTR reduction with in-study vutrisiran.
What this study is about
The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.
The purpose of this study is to:
- Determine the efficacy of nucresiran in patients with hATTR-PN by evaluating the effect on neurologic impairment, quality of life, nutritional status, disability, and gait speed
- Demonstrate superiority of nucresiran compared to in-study vutrisiran with respect to serum transthyretin (TTR) levels
Interventions being studied
Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.
- Drug
- Nucresiran
- Vutrisiran
Eligibility
The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.
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
Study description and what participation involves
The registry record has no detailed description beyond the summary above.
AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
Participants are randomized to receive subcutaneous injections of nucresiran every 6 months or vutrisiran every 3 months during the treatment period (with vutrisiran-treated participants receiving nucresiran during the extension period). They will attend clinic visits for neurologic exams (mNIS+7), nerve testing/electrophysiology, timed walking tests, and patient questionnaires about symptoms and quality of life at baseline and key time points such as Month 9 and Month 18. Blood draws will be done for serum TTR measurements and safety labs (including liver and kidney tests) and safety monitoring during treatment. The registry record does not provide a complete visit schedule or total study duration beyond the listed assessment time points.
How the study is structured. Phase 3, randomized, open-label, parallel-group drug trial. Participants are assigned to either nucresiran 300 mg given subcutaneously every 6 months or to vutrisiran 25 mg given subcutaneously every 3 months (with nucresiran given in a later extension period for the vutrisiran arm). There is no masking (open-label). Key outcomes are measured at around Month 9 and Month 18, and some analyses compare results to an external placebo group from the APOLLO study.
Questions you could ask
These questions are suggestions for a conversation with the study team or your own clinician, not advice.
- The registry lists this study under Hereditary Transthyretin-Mediated Amyloidosis With Polyneuropathy; hATTR-PN. Does it apply to my diagnosis?
- The listed sites are in Argentina, Australia, Brazil, Canada, Cyprus, France, Germany, Greece, Ireland, Italy, Japan, Malaysia, Mexico, Portugal, South Korea, Spain, Sweden, Switzerland, Taiwan, Turkey, United Kingdom, United States. Is any of them reachable for me, and does the study help with travel?
- Which of the eligibility criteria would need to be checked with tests before I could join?
More on wild-type ATTR amyloidosis
This trial is also listed on this site under ATTRv amyloidosis.
Important dates
- Study start
- Feb 3, 2026
- Primary completion
- Dec 27, 2027 (estimated)
- Study completion
- Jun 12, 2031 (estimated)
- Target enrollment
- 125 participants (estimated)
- Last updated on registry
- Oct 8, 2026
- Data verified
- Oct 10, 2026
Sponsor and research team
The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.
- Lead sponsor
- Alnylam Pharmaceuticals
Central study contact
If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.
- Clinical Trial Information Line 📞 1-877-ALNYLAM ✉ clinicaltrials@alnylam.com
- Clinical Trial Information Line 📞 1-877-256-9526 ✉ clinicaltrials@alnylam.com
Participating sites (61)
- Clinical Trial SiteRecruiting
Aurora, Colorado, 80045 · United States
- Clinical Trial SiteRecruiting
Chicago, Illinois, 60611 · United States
- Clinical Trial SiteRecruiting
Baltimore, Maryland, 21287 · United States
- Clinical Trial SiteRecruiting
Boston, Massachusetts, 02118 · United States
- Clinical Trial SiteRecruiting
Rochester, Minnesota, 55905 · United States
- Clinical Trial SiteRecruiting
New York, New York, 10016 · United States
- Clinical Trial SiteRecruiting
New York, New York, 10032 · United States
- Clinical Trial SiteRecruiting
Chapel Hill, North Carolina, 27514 · United States
- Clinical Trial SiteRecruiting
Nashville, Tennessee, 37232 · United States
- Clinical Trial SiteRecruiting
Dallas, Texas, 75246 · United States
Terms used on this page · explanations by MedicalUpdates.ai
- superiority
- What a comparison is designed to show. A superiority study asks whether the new treatment is better than the comparator; a non-inferiority study asks whether it is not meaningfully worse.
- Karnofsky Performance Status
- A scale from 100 (no complaints) down to 0 that describes how well a person can carry out daily activities. Studies often require a minimum score.
- upper limit of normal
- The highest value of a blood test that a laboratory considers normal. Criteria such as '≤ 3 × ULN' mean the result must be no more than three times that value.
- estimated glomerular filtration rate
- A blood-test estimate of how well the kidneys are filtering. Lower numbers mean reduced kidney function; many studies set a minimum value.
- Inclusion Criteria
- The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
- Exclusion Criteria
- The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.
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