A Phase 1, Double-Blind, Placebo-Controlled, Dose-Escalation Study of the Safety, Tolerability, & Pharmacokinetics of ISIS333611 Administered Intrathecally to Patients w Familial Amyotrophic Lateral Sclerosis Due to Superoxide Dismutase 1 Gene Mutation
Study Purpose:
This study will test the safety, tolerability, and pharmacokinetics of single doses of ISIS 333611 administered as 12-hour intrathecal infusions.Disease:
Amyotrophic Lateral Sclerosis (ALS), Familial ALSStudy Type:
Interventional TrialStudy Category:
Drug TrialStudy Status:
Not enrollingPhase:
Phase IStudy Chair(s)/Principal Investigator(s):
Merit Cudkowicz MD, MSc, (Massachusetts General Hospital)Timothy Miller MD, PhD, (Washington University)
Clinicaltrials.gov ID (11 digit #):
NCT01041222Neals Affiliated?
YesCoordinating Center Contact Information
Massachusetts General HospitalKaty Mahoney / .(JavaScript must be enabled to view this email address) / 617-643-7434
.(JavaScript must be enabled to view this email address) 149 13th Street
Charlestown, Massachusetts 02129 United States