Eplontersen for Hereditary Transthyretin Amyloidosis With Polyneuropathy.

ABSTRACT:

Importance

Transthyretin gene silencing is an emerging treatment strategy for hereditary transthyretin (ATTRv) amyloidosis.

Objective

To evaluate eplontersen, an investigational ligand-conjugated antisense oligonucleotide, in ATTRv polyneuropathy.

Design, setting, and participants

NEURO-TTRansform was an open-label, single-group, phase 3 trial conducted at 40 sites across 15 countries (December 2019-April 2023) in 168 adults with Coutinho stage 1 or 2 ATTRv polyneuropathy, Neuropathy Impairment Score 10-130, and a documented TTR variant. Patients treated with placebo from NEURO-TTR (NCT01737398; March 2013-November 2017), an inotersen trial with similar eligibility criteria and end points, served as a historical placebo ("placebo") group.

Interventions

Subcutan

SUBMITTER: Coelho T 

PROVIDER: S-EPMC10540057 | biostudies-literature | 2023 Oct

REPOSITORIES: biostudies-literature