Clinical Trial: Efficacy and Safety of IONIS-TTR Rx in Familial Amyloid Polyneuropathy

Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional

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

Brief Summary: The purpose of this study is to evaluate the efficacy and safety of IONIS-TTR Rx given for 65 weeks in patients with Familial Amyloid Polyneuropathy

Detailed Summary:

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.

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


Sponsor: Ionis Pharmaceuticals, Inc.

Current Primary Outcome:

  • Efficacy of IONIS-TTR Rx as measured by change from baseline in the modified Neuropathy Impairment Score +7 [ Time Frame: 65 weeks ]
  • Efficacy of IONIS-TTR Rx as measured by change from baseline in the Norfolk Quality of Life Diabetic Neuropathy questionnaire [ Time Frame: 65 weeks ]


Original Primary Outcome: Efficacy of ISIS-TTR Rx as measured by change from baseline in the modified Neuropathy Impairment Score +7 [ Time Frame: 65 weeks ]

Current Secondary Outcome:

  • Efficacy of IONIS-TTR Rx based on the change from baseline in the following measures: [ Time Frame: 65 weeks ]
    • Modified Body Mass Index and Body Mass Index
    • Individual components of the mNIS+7
    • NIS+7
  • Pharmacodynamic effect of IONIS-TTR Rx based on the change from baseline in transthyretin and retinol binding protein 4 [ Time Frame: 65 weeks ]


Original Secondary Outcome:

  • Efficacy of ISIS-TTR Rx based on the change from baseline in the following measures: [ Time Frame: 65 weeks ]
    • Norfolk Quality of Life Diabetic Neuropathy questionnaire
    • Modified Body Mass Index and Body Mass Index
    • Individual components of the mNIS+7
    • NIS+7
    • NIS
  • Pharmacodynamic effect of ISIS-TTR Rx based on the change from baseline in transthyretin and retinol binding protein 4 [ Time Frame: 65 weeks ]


Information By: Ionis Pharmaceuticals, Inc.

Dates:
Date Received: November 27, 2012
Date Started: December 2012
Date Completion: September 2017
Last Updated: March 23, 2016
Last Verified: March 2016