Experimental drug zilganersen aims to slow rare brain disease
NCT ID NCT04849741
First seen Jun 27, 2026 · Last updated Aug 21, 2026 · Updated 1 time
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.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 3
Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.
- Participants
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54 people
The number who actually took part.
- Started
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Jun 2021
- Expected to finish
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Jul 2034
An estimate. End dates often move.
- Lead sponsor
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A company
The lead sponsor is a pharmaceutical, biotech, or medical-device company.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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2 to 65 years
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Key Inclusion Criteria: 1. Clinical phenotype and brain imaging consistent with a diagnosis of Alexander disease 2. Documented genetic mutation in the GFAP gene 3. Aged ≥ 2 to 65 years old at the time of informed consent 4. Able and willing to meet all study requirements, including travel to Study Center, procedures, measurements and visits 5. Patients \< 18 years old at Screening must have a trial partner (parent, caregiver or other) Key Exclusion Criteria: 1. Clinically significant abnormalities in medical history or physical examination 2. Any clinically significant laboratory abnormalities that would render a patient unsuitable for inclusion 3. Any contraindication or unwillingness to undergo MRI 4. Treatment with another investigational drug, biological agent, or device within 1 month of Screening, or 5 half-lives of investigational agent, whichever is longer; concurrent participation in any other clinical study (including observational and non-interventional studies) 5. Previous treatment with an oligonucleotide (including small interfering ribonucleic acid \[siRNA\]) within 4 months of Screening if single dose received, or within 12 months of Screening if multiple doses received. This exclusion does not apply to vaccines (both messenger ribonucleic acid \[mRNA\] and viral vector vaccines). 6. History of gene therapy or cell transplantation or any other experimental brain surgery \[ROW\] 7. Obstructive hydrocephalus 8. Presence of a functional ventriculoperitoneal shunt for the drainage of cerebrospinal fluid (CSF) or an implanted central nervous system (CNS) catheter 9. Known brain or spinal disease that would interfere with the lumbar puncture (LP) process, CSF circulation or safety assessment. 10. Hospitalization for any major medical or surgical procedure involving general anesthesia within 12 weeks prior to Screening or planned during the study 11. Have any other conditions, which, in the opinion of the Investigator would make the patient unsuitable for inclusion, or could interfere with the patient participating in or completing the study
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Genom att skicka in godkänner du våra Användarvillkor
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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