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

NCT ID NCT06983158

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

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early This study
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 1/2

Runs two stages together: safety and dose first, then whether the treatment works.

Participants

1 person

The number who actually took part.

Started

Jul 2025

Finished

May 2026

Lead sponsor

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

18 months to 7 years

Sex

Anyone

Healthy volunteers

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

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: Male or female, ≥18 months to \<8 years of age; Has diagnosis of developmental encephalopathy due to an STXBP1 mutation with confirmation of a pathogenic or likely pathogenic STXBP1 gene mutation. Has a legally authorized representative (LAR) willing and able to complete the informed consent process, willing to comply with trial procedures, and able to travel for repeat visits. Is stable on any medication regimens (if being administered to control the signs and symptoms of underlying disease) for at least 6 weeks prior to trial entry and expected to be stable for at least 12 weeks post-CAP-002 administration. Exclusion Criteria: History of prior gene therapy; Treatment with antisense oligonucleotide therapy within 6 months; Presence of a confirmed mutation in a gene other than STXBP1 that is known to contribute to a neurodevelopmental disability or epilepsy; Has presence of a significant non-STXBP1-related central nervous impairment/behavioral disturbance that would confound the scientific rigor or interpretation of results of the trial; History of prematurity (defined as gestational age \<35 weeks), history of low birth weight (\<2.5 kg) and/or intra-uterine growth restriction, significant interventricular hemorrhage, structural brain deficit, or congenital heart disease; Known contraindication to immunosuppression or other protocol-defined medications, including but not limited to corticosteroids or PPIs; Clinically significant abnormalities in safety lab tests, vital signs; Other illnesses or medications that may affect the interpretation of the study results; Positive anti-capsid antibody test result.

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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

More trials for these conditions

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