Clinical Trial: Open-Label Extension Assessing Long Term Safety and Efficacy of IONIS-TTR Rx in Familial Amyloid Polyneuropathy (FAP)

Study Status: Enrolling by invitation
Recruit Status: Enrolling by invitation
Study Type: Interventional

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)

Brief Summary: This study evaluates the safety and tolerability of extended dosing with IONIS-TTR Rx 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.

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


Sponsor: Ionis Pharmaceuticals, Inc.

Current Primary Outcome:

  • Types of adverse events that occur during treatment [ Time Frame: 78 Weeks ]
  • Change from baseline in blood pressure (systolic and diastolic), heart rate, and body weight [ Time Frame: 78 Weeks ]
  • Change from baseline in results of routine laboratory test panel (routine serum chemistry, hematology, and urinalysis) [ Time Frame: 78 Weeks ]
  • Change from baseline in number of concomitant medications used [ Time Frame: 78 Weeks ]
  • Change from baseline in QTcF determined from electrocardiogram measurements [ Time Frame: 78 Weeks ]
  • Change from baseline in visual acuity measured during ophthalmic exam [ Time Frame: 78 Weeks ]
  • Change from baseline in light detection ability measured by electroretinography [ Time Frame: 78 Weeks ]


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Change from baseline in the modified Neuropathy Impairment Score +7 [ Time Frame: Baseline, Week 78 ]
  • Change from baseline in the Norfolk Quality of Life Diabetic Neuropathy Questionnaire [ Time Frame: Baseline, Week 78 ]
  • Change from baseline in the Polyneuropathy disability score (PND) [ Time Frame: Baseline, Week 78 ]
  • Change from baseline in the Modified body mass index (mBMI) and body mass index (BMI) [ Time Frame: Baseline, Week 78 ]
  • Change in baseline in Transthyretin (TTR) and Retinol Binding Protein 4 (RBP4) [ Time Frame: Baseline, Week 78 ]


Original Secondary Outcome: Same as current

Information By: Ionis Pharmaceuticals, Inc.

Dates:
Date Received: June 12, 2014
Date Started: June 2014
Date Completion: August 2020
Last Updated: March 23, 2016
Last Verified: March 2016