A randomized controlled, double blind Phase 3 multicenter trial of IONIS-TTRRx was initiated in December 2012. The purpose of the trial is to determine whether IONIS-TTRRx can slow or stop the nerve damage caused by TTR deposits in patients with familial amyloid polyneuropathy (FAP). This study aims to enroll about 200 patients with early to mid stage neuropathy. Patients will receive either IONIS-TTRRx or placebo for 65 weeks. The official study title is: A Phase 2/3 Randomized, Double-Blind, Placebo-Controlled Study to Assess the Efficacy and Safety of IONIS-TTRRx in Patients with Familial Amyloid Polyneuropathy. For more information, go to ClinicalTrials.gov.
Each patient taking part in the trial will be randomly assigned to one of 2 groups:
This is a double blind trial. This means that neither the doctors running the trial nor the patients participating will know which patients are receiving IONIS-TTRRx and which patients are receiving placebo. There is 2:1 randomization, meaning that 2/3 of the patients will receive IONIS-TTRRx and 1/3 will receive placebo. After the Phase 3 study is completed, all the patients can participate in the open label extension study, where all subjects will receive IONIS-TTRRx.
The main (primary) outcome measures will be change in baseline score in two standardized measures for assessing the severity of neuropathy:
In addition, the level of TTR in blood will also be measured throughout the study to determine if IONIS-TTRRx lowers levels of TTR as a secondary outcome measure.