Efficacy and Safety of Inotersen in Familial Amyloid Polyneuropathy

Amyloidosis Clinical Trial

Official title: A Phase 2/3 Randomized, Double-Blind, Placebo-Controlled Study to Assess the Efficacy and Safety of ISIS 420915 in Patients With Familial Amyloid Polyneuropathy (NEURO-TTR Study)

Status Completed

Clinical Trial Summary

The purpose of this study is to evaluate the efficacy and safety of inotersen given for 65 weeks in participants with Familial Amyloid Polyneuropathy (FAP).

Clinical Trial Description

FAP is a rare, hereditary disease caused by mutations in the transthyretin (TTR) protein. TTR is made by the liver and secreted into the blood. TTR mutations cause it to misfold and deposit in multiple organs causing FAP.

Inotersen (also known as ISIS 420915) is an antisense drug that was designed to decrease the amount of mutant and normal TTR made by the liver. It is predicted that decreasing the amount of TTR protein would result in a decrease in the formation of TTR deposits, and thus slow or stop disease progression.

The purpose of this study is to determine if inotersen can slow or stop the nerve damage caused by TTR deposits. This study will enroll late Stage 1 and early Stage 2 FAP participants. Participants will receive either inotersen or placebo for 65 weeks. ;

Related Conditions & MeSH terms

• Amyloid Neuropathies
• Amyloid Neuropathies, Familial
• Amyloidosis
• Familial Amyloid Polyneuropathy
• FAP
• Polyneuropathies
• Transthyretin
• TTR

• NCT number: NCT01737398
• Study type: Interventional
• Source: Ionis Pharmaceuticals, Inc.
• Status: Completed
• Phase: Phase 2/Phase 3
• Start date: March 15, 2013
• Completion date: November 7, 2017