FUSION: A Study to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of ION363 in Amyotrophic Lateral Sclerosis Participants With Fused in Sarcoma Mutations (FUS-ALS)
Trial ID or NCT#
NCT04768972
Status
RECRUITING
Purpose
The primary purpose of this study is to evaluate the efficacy of ION363 on clinical function and survival in carriers of fused in sarcoma mutations with amyotrophic lateral sclerosis (FUS-ALS).
Official Title
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)
Eligibility Criteria
Ages Eligible for Study: Older than 10 Years
Sexes Eligible for Study: ALL
Accepts Healthy Volunteers: No
Exclusion Criteria:
Investigator(s)
John W. Day, MD, PhD
Neuromuscular neurologist,
Neurophysiologist
Professor of Neurology and Neurological Sciences (Adult Neurology), of Pediatrics (Genetics) and, by courtesy, of Pathology
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