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

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

Hereditary Transthyretin-Mediated Amyloidosis With Polyneuropathy

hATTR-PN

Vutrisiran

Nucresiran

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)

The mNIS+7 is a composite score that measures neurologic impairment which includes the
following components: physical exam of lower limbs, upper limbs and cranial nerves to
assess motor strength/weakness and deep tendon reflexes, electrophysiologic measurement
of large nerve fiber function, sensory testing and postural blood pressure. The mNIS+7 is
scored from 0 (no impairment) to 304 points (maximum impairment). A higher score
indicates a worse outcome.

The Norfolk QoL-DN is a standardized 35-item patient-rated questionnaire used to assess 5
domains: physical function, large fiber neuropathy, activities of daily living, symptoms,
small fiber neuropathy, and autonomic neuropathy. The minimum and maximum values are -4
and 136, respectively. A higher score indicates a worse outcome.

The mBMI, which is a measure of nutritional status, is calculated as the product of body
mass index (BMI) (weight in kilograms divided by the square of height in meters) and
serum albumin (g/L) to reflect fluid balance, such as fluid accumulation or dehydration.
A negative change from baseline indicates a better outcome.

The R-ODS is a patient-reported measure of level of disability on a scale of 0-48, with 0
being the worst and 48 the best (no limitations); scores are based on activities of daily
living and social participation. An increase in R-ODS from baseline suggests improvement
in disability, and a decrease from baseline suggests worsening of disability.

The timed 10-MWT is a measure of ambulatory ability and measures the time (in seconds)
that it takes a participant to walk 10 meters (gait speed). An increase in gait speed
from baseline represents improvement, and a decrease from baseline represents worsening.

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

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

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

About this trial

Study ID

Status

Type

Phase

Placebo

Accepting

Not accepting

Trial Timing

What is this trial about?

What are the participation requirements?

Locations

Location

Status