Gene therapy trial for rare childhood epilepsy halted after just one patient
NCT ID NCT06983158
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This trial tested a single dose of CAP-002 gene therapy in children aged 18 months to 8 years with STXBP1 encephalopathy, a rare genetic brain disorder causing seizures and developmental delays. The study aimed to check safety and whether it could reduce seizures and improve skills. However, the trial was terminated after enrolling only one participant, so results are very limited.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
CAP-002 gene therapy
What this could lead to
If successful, this could point toward a treatment that reduces seizures and improves development in children with STXBP1 encephalopathy.
What could go wrong
The trial was terminated early with only 1 participant enrolled, so we have very little data. Gene therapy carries risks like immune reactions or liver problems, and it is unclear if it will work for this rare condition.
Disclaimer
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This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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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
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Locations
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Buerger Center for Advanced Pediatric Care, Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19146, United States
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Colorado Child Health Research Institute
Aurora, Colorado, 80045, United States
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Texas Children's Hospital
Houston, Texas, 77030, United States
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Weill Cornell Medicine
New York, New York, 10021, United States