A Study to Evaluate the Safety and Efficacy of Zilganersen (ION373) in Patients With Alexander Disease (AxD)
Running, not enrolling · Phase 3 · Has a placebo group
Conditions studied: Alexander Disease
In brief
The purpose of this study is to evaluate the safety and efficacy of zilganersen (ION373) in improving or stabilizing gross motor function across the full range of affected domains in patients with AxD.
Key facts
- Study ID
- NCT04849741
- Run by
- Ionis Pharmaceuticals, Inc.
- People needed
- 54
- Starts
- 2021-06-01
- Expected to finish
- 2029-09-01
- Last updated by the study team
- 2026-04-03
Who can join
Age: 2 and older, up to 65. Sex: any. Healthy volunteers: not accepted.
Where it is running
- Lucile Packard Children's Hospital Stanford — Palo Alto, California, United States
- Children's Hospital of Atlanta — Atlanta, Georgia, United States
- Massachusetts General Hospital — Boston, Massachusetts, United States
- Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States
- Murdoch Children's Research Institute — Parkville, Victoria, Australia
- McGill University Health Centre — Montreal, Quebec, Canada
- Pediatric Neurology Institute, Dana-Dwek Children's Hospital, Tel Aviv Sourasky Medical Center — Tel Aviv, Israel
- Ospedale dei Bambini Vittore Buzzi — Milan, Italy
- Ospedale Pediatrico Bambino Gesù — Roma, Italy
- National Center of Neurology and Psychiatry — Tokyo, Kodaira-shi, Japan
- Amsterdam Universitair Medische Centra - Academisch Medisch Centrum — Amsterdam, North Holland, Netherlands
- University College London Hospitals NHS Foundation Trust — London, United Kingdom
- Great Ormond Street Hospital for Children NHS Foundation Trust — London, United Kingdom
Full record on ClinicalTrials.gov
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