NCT01737398

Efficacy and Safety of Inotersen in Familial Amyloid Polyneuropathy

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
• Phase: Phase 2 / Phase 3
• Study type: Interventional
• Enrollment: 173 (actual)
• Sponsor: Ionis Pharmaceuticals, Inc. · Industry
• Sex: All
• Age: 18 Years – 82 Years
• Healthy volunteers: Not accepted

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).

Detailed 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.

Conditions

• FAP
• Familial Amyloid Polyneuropathy
• TTR
• Transthyretin
• Amyloidosis

Interventions

Type	Name	Description
DRUG	Inotersen
DRUG	Placebo

Timeline

• Start date: 2013-03-15
• Primary completion: 2017-03-03
• Completion: 2017-11-07
• First posted: 2012-11-29
• Last updated: 2019-07-17
• Results posted: 2019-01-23

Locations

24 sites across 10 countries: United States, Argentina, Brazil, France, Germany, Italy, New Zealand, Portugal, Spain, United Kingdom

Regulatory

• FDA-regulated drug study