Queen Square Centre for Neuromuscular Diseases


A Phase II/III Study of ISIS 420915 in Patients with Familial Amyloid Polyneuropathy

Sponsor / Funder ISIS Pharmaceutical
CI Dr Carol Whelan
Prof. Mary Reilly 
UK sites The National Amyloidosis Centre / London Queen Square
More information  

Trial information

This is a multi-centre, randomised, double-blind, placebo-controlled study.

Study drug (ISIS 420915 or placebo) will be given three times on alternate days during Week 1 (Days 1, 3 and 5), and then once weekly during Weeks 2-65 (for a total of 67 doses).

Patients will also receive daily supplements of the recommended dose of vitamin A.

The end of treatment efficacy assessment takes place at Week 66.

Following completion of this study, eligible patients may enrol in an open-label extension study.

Otherwise, patients will enter the 6 month post-treatment evaluation part of the study.


Approximately 195 patients in a 2:1 ratio. 130 patients receiving 300 mg ISIS 420915 and 65 patients receiving placebo.