Study design & results
Available in the Gosset platform where reported.
Trial intelligence / Profile preview
Spectrum of Peripheral and Autonomic Neuropathies in Patients With aTTRwt Amyloidosis and Response to Patisiran Therapy
NCT05023889Phase 1Completed
- Sponsor
- Austin Neuromuscular Center
- Start
- Aug 2022
- Primary completion
- Apr 2026
- Modality
- Gene Editing, Gene Silencing, Lipid-based Nanoparticles
- Countries
- 1
01
Overview
Phase 1, open-label, single-arm trial of patisiran in wild-type transthyretin amyloidosis (ATTRwt) and polyneuropathy, sponsored by Austin Neuromuscular Center. Primary endpoints: Neurological Impairment Score at 24 months, Norfolk Quality of Life-Diabetic Neuropathy at 24 months and 1 more. Started Aug 2022; status: completed.
02
Drugs
patisiran
03
Conditions
wild-type transthyretin amyloidosis (ATTRwt)polyneuropathy
Beyond the preview
Go deeper on NCT05023889.
Study design, results and competitive context are available in the Gosset platform where reported.
Competitive context
Available in the Gosset platform where reported.
Bring the full picture into focus.
See how Gosset can support your research on NCT05023889.