Gene therapy trial for rare childhood epilepsy halted after just one patient

NCT ID NCT06983158

Terminated Disease control Sponsor: Capsida Biotherapeutics, Inc. Source: ClinicalTrials.gov ↗

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.

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.

developmental and epileptic encephalopathy developmental and epileptic encephalopathy, 4

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

DEVELOPMENTAL AND EPILEPTIC ENCEPHALOPATHY

Contacts and locations

Locations

Buerger Center for Advanced Pediatric Care, Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19146, United States
Colorado Child Health Research Institute

Aurora, Colorado, 80045, United States
Texas Children's Hospital

Houston, Texas, 77030, United States
Weill Cornell Medicine

New York, New York, 10021, United States

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