A Multicenter, Open-label, Extension Study to Evaluate the Long-term Safety and Efficacy ofPatisiran in Patients with Familial Amyloidotic Polyneuropathy Who Have Complete a Prior Clinical Study with Patisiran
ID Number 16-2465Principal Investigator(s)
Peter D Gorevic
Department(s) or Division(s)
The purpose of this study is to evaluate the long-term safety and efficacy of patisiran in patients with FAP who have completed a prior study with patisiran. This includes studying any potential improvements in patients' disease (FAP) and any side effects that they may have from patisiran.
In this study, participants will receive patisiran (at a dose of 0.3mg/kg) once every 3 weeks for as long as they remain on the study. Patisiran will be given through the vein (by infusion) over a 70-minute period. Participants may continue to receive their patisiran infusion for more than 70 minutes, if previously done on a prior study.
The number of people expected to take part in this research study at this site is 1. The total number of people expected to take part in this research study is 200.
Recruiting Patients: Yes