A Phase 1-3 Study to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION363 in Amyotrophic Lateral Sclerosis Patients With Fused in Sarcoma Mutations (FUS-ALS)
Study Purpose:
The primary purpose of this study is to evaluate the clinical efficacy of ION363 on clinical function and survival in carriers of fused in sarcoma mutations with amyotrophic lateral sclerosis (FUS-ALS).
Disease:
Amyotrophic Lateral Sclerosis (ALS), Familial ALSStudy Type:
Interventional TrialStudy Category:
Drug TrialStudy Status:
EnrollingPhase:
Phase IIIStudy Chair(s)/Principal Investigator(s):
Clinicaltrials.gov ID (11 digit #):
NCT04768972Neals Affiliated?
YesCoordinating Center Contact Information
Ionis Pharmaceuticals / .(JavaScript must be enabled to view this email address) / 844-421-0104
.(JavaScript must be enabled to view this email address) United States
Full Study Summary:
This is a multi-center, two-part study of ION363 in up to 64 participants. Part 1 will consist of participants that will be randomized in a 2:1 ratio to receive a multi-dose regimen of ION363 or placebo for a period of 29 weeks, followed by Part 2, which will be an open-label period where all participants will receive ION363 for a period of 73 weeks.