NCT02175004

Extension Study Assessing Long Term Safety and Efficacy of IONIS-TTR Rx in Familial Amyloid Polyneuropathy (FAP)

An Open-Label Extension Study to Assess the Long-Term Safety and Efficacy of ISIS 420915 in Patients With Familial Amyloid Polyneuropathy (FAP)

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 135 (actual)
• Sponsor: Ionis Pharmaceuticals, Inc. · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This study evaluates the safety and tolerability of extended dosing with IONIS-TTR Rx in patients with Familial Amyloid Polyneuropathy.

Detailed description

Familial Amyloid Polyneuropathy (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. IONIS-TTR Rx is an antisense drug that is designed to decrease the amount of mutant and normal TTR made by the liver. It is predicted that decreasing the amount of TTR protein will result in a decrease in the formation of TTR deposits, and thus slow or stop disease progression. This study evaluates the safety and tolerability of extended dosing with IONIS-TTR Rx in patients with Familial Amyloid Polyneuropathy.

Conditions

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

Interventions

Type	Name	Description
DRUG	Inotersen	Inotersen SC

Timeline

• Start date: 2014-06-26
• Primary completion: 2020-09-11
• Completion: 2021-01-07
• First posted: 2014-06-26
• Last updated: 2023-11-18
• Results posted: 2023-02-09

Locations

22 sites across 9 countries: United States, Argentina, Brazil, France, Germany, Italy, Portugal, Spain, United Kingdom

Regulatory

• FDA-regulated drug study