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)
Running, not enrolling · Phase 3 · Has a placebo group
Conditions studied: Amyotrophic Lateral Sclerosis
In brief
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).
Key facts
- Study ID
- NCT04768972
- Run by
- Ionis Pharmaceuticals, Inc.
- People needed
- 89
- Starts
- 2021-06-14
- Expected to finish
- 2028-03-01
- Last updated by the study team
- 2026-07-21
Who can join
Age: 10 and older. Sex: any. Healthy volunteers: not accepted.
Where it is running
- University of California San Diego — La Jolla, California, United States
- Stanford University Medical Center — Palo Alto, California, United States
- Johns Hopkins University — Baltimore, Maryland, United States
- Massachusetts General Hospital — Boston, Massachusetts, United States
- Washington University School of Medicine — St Louis, Missouri, United States
- Columbia University Medical Center — New York, New York, United States
- The Ohio State University Wexner Medical Center — Columbus, Ohio, United States
- University of Utah — Salt Lake City, Utah, United States
- UZ Leuven — Leuven, VL-Brabant, Belgium
- PSEG Centro de Pesquisa Clinica S.A. — São Paulo, Brazil
- Montreal Neurological Institute — Montreal, Quebec, Canada
- Universitaetsmedizin Rostock — Rostock, Germany
- Universitätsklinikum Ulm — Ulm, Germany
- St. James Hospital — Dublin, Ireland
- Citta della Salute e della Scienza di Torino - Ospedale le Molinette — Torino, Italy
- Toho University Omori Medical Center — Tokyo, Japan
- Universitair Medisch Centrum Utrecht — Utrecht, Netherlands
- Linden spólka z ograniczona odpowiedzialnoscia spólka komandytow — Krakow, Poland
- Seoul National University Hospital — Seoul, South Korea
- Hanyang University Seoul Hospital — Seoul, South Korea
- Hospital Universitari de Bellvitge — Barcelona, Spain
- University Hospital of Umea — Umeå, Sweden
- Kantonsspital St. Gallen — Sankt Gallen, Switzerland
- Taipei Veterans General Hospital (VGHTP) — Taipei, Taiwan
- King's College Hospital — London, United Kingdom
Full record on ClinicalTrials.gov
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