Gene therapy breakthrough offers hope for rare brain disease
NCT ID NCT04833907
First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study tests a single dose of AVASPA gene therapy given directly into the brain of children with Canavan disease, a rare and severe genetic disorder that damages white matter. The therapy aims to restore a missing enzyme to help the brain produce myelin and slow disease progression. Up to 24 children will be enrolled across three age groups to evaluate safety and early signs of benefit.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- AVASPA gene therapy (MYR-101)
- What this could lead to
- If successful, this could provide a treatment that slows or stops the progression of Canavan disease, improving quality of life and extending survival for affected children.
- What could go wrong
- This is an early-phase trial with only 24 participants, so results may not apply to all. Gene therapy carries risks like immune reactions or surgical complications, and long-term effects are unknown.
This is an AI summary of the original study and may miss details. Read our disclaimer.
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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
-
Dayton Children's Hospital
Dayton, Ohio, 45404, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Newborn screening study aims to catch rare diseases at birth
- Experimental gene therapy hopes to slow rare brain disease in toddlers
- Scientists track canavan disease in 67 children to map its progression
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