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Tests treatment safety and results for Hereditary Transthyretin-Mediated Amyloidosis With...
Official title TRITON-PN: A Study to Evaluate the Efficacy and Safety of Nucresiran in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy
ClinicalTrials.gov ID: NCT07223203
What this study is testing
What is Nucresiran?
Nucresiran is an investigational medicine, given as an injection under the skin, being studied as a potential treatment for hereditary transthyretin-mediated amyloidosis with polyneuropathy.
Also referred to as ALN-TTRSC04.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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
- Phase 3: a large, late-stage study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 to 85
You may be able to join if
- 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%
You likely can't join if
- 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
See the full eligibility 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%
- 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
The study team makes the final eligibility decision.
Where it's taking place
- Aurora, Colorado, United States
- Chicago, Illinois, United States
- Baltimore, Maryland, United States
- Boston, Massachusetts, United States
- Rochester, Minnesota, United States
- New York, New York, United States
- Chapel Hill, North Carolina, United States
- Dallas, Texas, United States
- Darlinghurst, Australia
- São Paulo, Brazil
- Égkomi, Cyprus
- Créteil, France
- Le Kremlin-Bicêtre, France
- Marseille, France
- Athens, Greece
- Heraklion, Greece
- Florence, Italy
- Milan, Italy
- Pavia, Italy
- Suita, Japan
+ 7 more site(s).
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 years to 85 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Aurora, Colorado, United States; Chicago, Illinois, United States; Baltimore, Maryland, United States; Boston, Massachusetts, United States; Rochester, Minnesota, United States; New York, New York, United States and 21 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 ClinicalTrials.gov; the study team decides eligibility.