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

Conditions

• Hereditary Transthyretin-Mediated Amyloidosis With Polyneuropathy
• hATTR-PN

Eligibility Criteria

Sex

ALL

Age

18 Years - 85 Years

Healthy Volunteers

Not accepted

Interventions

• Nucresiran — DRUG
  Nucresiran 300 mg administered SC q6M
• Vutrisiran — DRUG
  Vutrisiran 25 mg administered SC q3M

Study Details

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

Key Dates

Start date

Dec 12, 2025

Status verified

May 2026

Primary completion

Dec 27, 2027

Completion

Jun 12, 2031

Study Design

Enrollment

125 participants (estimated)

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Arms

• Experimental: 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
• Active Comparator: 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

Primary Outcome Measure

Change from Baseline in the Modified Neuropathy Impairment Score +7 (mNIS+7) Compared to the External Placebo Group from the APOLLO Study (NCT01960348) at Month 9 [ Time Frame: Baseline and Month 9 ]

Central Contacts

• Clinical Trial Information Line
  1-877-ALNYLAM
  [email protected]
• Clinical Trial Information Line
  1-877-256-9526
  [email protected]

Locations (3)

Find similar trials in Baltimore, MD