Experimental drug zilganersen aims to slow rare brain disease
NCT ID NCT04849741
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This Phase 3 trial tests whether zilganersen (ION373) can improve or stabilize motor function in people with Alexander disease, a rare genetic brain disorder. Fifty-four participants aged 2 to 65 will receive either the drug or a placebo injected into the spine. The study measures walking speed and symptom severity over time.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- zilganersen (ION373)
- What this could lead to
- If successful, this could lead to the first treatment that slows or stabilizes the worsening of motor function in Alexander disease, offering patients a way to maintain abilities longer.
- What could go wrong
- This is an early-stage Phase 3 trial with only 54 participants, so results may not apply to all patients. The drug is injected into the spine, which carries risks like infection or nerve damage, and it may not improve symptoms for everyone.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Amsterdam Universitair Medische Centra - Academisch Medisch Centrum
Amsterdam, North Holland, 1105 AZ, Netherlands
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Children's Hospital of Atlanta
Atlanta, Georgia, 30329, United States
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Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
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Great Ormond Street Hospital for Children NHS Foundation Trust
London, WC1N 3JH, United Kingdom
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Lucile Packard Children's Hospital Stanford
Palo Alto, California, 94304, United States
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Massachusetts General Hospital
Boston, Massachusetts, 02114, United States
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McGill University Health Centre
Montreal, Quebec, H3A 0G4, Canada
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Murdoch Children's Research Institute
Parkville, Victoria, 3052, Australia
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National Center of Neurology and Psychiatry
Tokyo, Kodaira-shi, 187-8551, Japan
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Ospedale Pediatrico Bambino Gesù
Roma, 165, Italy
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Ospedale dei Bambini Vittore Buzzi
Milan, 20154, Italy
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Pediatric Neurology Institute, Dana-Dwek Children's Hospital, Tel Aviv Sourasky Medical Center
Tel Aviv, 6423906, Israel
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University College London Hospitals NHS Foundation Trust
London, WC1N 3BG, United Kingdom
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