Summary

for people ages 18 years and up (full criteria)
at Orange, California and other locations
study started
estimated completion

Description

Summary

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

Official Title

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

Details

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.

Keywords

FAPFamilial Amyloid PolyneuropathyTTRTransthyretinAmyloidosisPolyneuropathiesAmyloid NeuropathiesAmyloid Neuropathies, FamilialIONIS-TTR Rx

Eligibility

You can join if…

Open to people ages 18 years and up

  • Satisfactory completion of dosing & efficacy assessments in ISIS 420915-CS2

You CAN'T join if...

  • Any new condition or worsening of existing condition that could make the patient unsuitable for participation, or interfere with the patient participating in and/or completing the study

Locations

  • University of California, Irvine
    OrangeCalifornia92868United States
  • Oregon Health & Science University
    PortlandOregon97239United States

Details

Status
in progress, not accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Ionis Pharmaceuticals, Inc.
ID
NCT02175004
Phase
Phase 3
Study Type
Interventional
Last Updated