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TRITON-PN: A Study to Evaluate the Efficacy and Safety of Nucresiran in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy

NCT ID: NCT07223203

Last Updated: 2025-10-31

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

NOT_YET_RECRUITING

Clinical Phase

PHASE3

Total Enrollment

125 participants

Study Classification

INTERVENTIONAL

Study Start Date

2025-12-31

Study Completion Date

2031-06-12

Brief Summary

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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

Detailed Description

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Conditions

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Hereditary Transthyretin-Mediated Amyloidosis With Polyneuropathy hATTR-PN

Keywords

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Polyneuropathy Amyloidosis Transthyretin TTR RNAi therapeutic

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Nucresiran 300 mg

Patients will be administered nucresiran 300 mg subcutaneously (SC) once every 6 months (q6M) during the Treatment Period and Treatment Extension Period

Group Type EXPERIMENTAL

Nucresiran

Intervention Type DRUG

Nucresiran 300 mg administered SC q6M

Vutrisiran 25 mg followed by Nucresiran 300 mg

Patients will be administered vutrisiran 25 mg SC every 3 months (q3M) during the Treatment Period followed by nucresiran 300 mg SC q6M during the Treatment Extension Period

Group Type ACTIVE_COMPARATOR

Nucresiran

Intervention Type DRUG

Nucresiran 300 mg administered SC q6M

Vutrisiran

Intervention Type DRUG

Vutrisiran 25 mg administered SC q3M

Interventions

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Nucresiran

Nucresiran 300 mg administered SC q6M

Intervention Type DRUG

Vutrisiran

Vutrisiran 25 mg administered SC q3M

Intervention Type DRUG

Other Intervention Names

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ALN-TTRSC04 AMVUTTRA ALN-TTRSC02

Eligibility Criteria

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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
Minimum Eligible Age

18 Years

Maximum Eligible Age

85 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Alnylam Pharmaceuticals

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Medical Director

Role: STUDY_DIRECTOR

Alnylam Pharmaceuticals Inc

Central Contacts

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Clinical Trial Information Line

Role: CONTACT

Phone: 1-877-ALNYLAM

Email: [email protected]

Clinical Trial Information Line

Role: CONTACT

Phone: 1-877-256-9526

Email: [email protected]

Other Identifiers

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2025-522544-40-00

Identifier Type: CTIS

Identifier Source: secondary_id

ALN-TTRSC04-004

Identifier Type: -

Identifier Source: org_study_id