Amyotrophic Lateral Sclerosis | ION363
Study to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of ION363 in Amyotrophic Lateral Sclerosis Participants With Fused in Sarcoma Mutations (FUS-ALS)
This is a multi-center, three-part study of ION363 in up to 77 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 60 weeks, followed by Part 2, in which participants will receive ION363 for a period of 80 weeks.Participants who have completed Part 2 are eligible to participate in Part 3 and continue to receive ION363 for an additional 156 weeks.
Treatment extension period was added to allow patients who complete Part 2 to continue to receive ION363 until the drug may become commercially available.
Purpose of Study
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).
Amyotrophic Lateral Sclerosis
Clinical Trials ID
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Start Date / End Date:
06 2021 - 03 2028