An Open-Label Extension Study to Assess the Long-Term Safety and Efficacy of ISIS 420915 in Patients with Familial Amyloid Polyneuropathy (FAP)
ID Number 14-1502Principal Investigator(s)
Peter D Gorevic
Department(s) or Division(s)
The purpose of this study is to determine whether longer treatment with ISIS 420915 (the study drug) is safe and effective in helping people with mild or moderate Familial Amyloid Polyneuropathy (FAP).
This is a multicenter open-label study. Eligible patients who have satisfactorily completed ISIS 420915-CS2 will receive 300 mg ISIS 420915 once weekly for 78 weeks (18 months). Exceptions will be given to patients that had a dose reduction or schedule change in ISIS 420915-CS2 who may continue with the same dose level or schedule in this study. All patients will receive supplemental doses of the recommended daily allowance (RDA) of vitamin A. After completing the treatment period, patients will enter the 3-month post-treatment evaluation period.
All patients will receive 300 mg ISIS 420915 given once weekly. However, patients who had a dose reduction or schedule change in ISIS 420915-CS2 may continue with the same dose or schedule in this study.
Participation in this study will last about 2 years. There are 3 study periods: The screening period, the treatment period, and the post-treatment period.
The number of people expected to take part in this research study at this site is 10. The total number of people expected to take part in this research study is approximately 195 patients, between 19 and 83 years of age, at multiple centers worldwide.
Recruiting Patients: No