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Promising seizure drug study halted early

NCT ID NCT05226780

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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 27, 2026 · Last updated Jun 27, 2026

Summary

This study looked at the long-term safety of the drug NBI-921352 for people with a rare genetic seizure disorder called SCN8A-DEE. It was an extension of an earlier study, and participants took the drug alongside their usual seizure medications. The study was stopped early and only included 8 people, so results are limited.

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 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

8 people

The number who actually took part.

Started

Jul 2022

Finished

Nov 2025

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

2 to 22 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.

Key Inclusion Criteria: For Participants Who Participated in NBI-921352-DEE2012: * Written or oral pediatric assent from the participant and written informed consent from the participant's parent(s) or legal guardian(s) for pediatric participants and adult participants who are not capable of providing consent. Adult participant who are ≥18 years of age and capable of providing consent should sign an Informed Consent Form (ICF). * Completed 16 weeks of treatment in Study NBI-921352-DEE2012. * Continue to use a nocturnal alerting system or practice consistent with standards of care for the duration of the study. * Have an adequate rescue medication regimen per the investigator's judgment in place for the duration of the study. For Participants Who did not Participate in NBI-921352-DEE2012: * Written or oral pediatric assent from the participant if deemed capable of providing assent and written informed consent from the participant's parent(s) or legal guardian(s) for pediatric participants and for adult participants who are not capable of providing consent. Adult participants who are capable of providing consent should sign an ICF. * Be a male or female 2 to 21 years of age, inclusive. * Have a diagnosis of SCN8A-DEE supported by both clinical and genetic findings. * Have on average at least 1 countable motor seizure per week and not be seizure-free for more than 20 consecutive days. * Being treated with at least 1 other antiseizure medication (ASM), but no more than 4 ASMs. * Have failed to achieve seizure freedom with at least 2 ASMs. * Have a body weight of at least 10 kilograms. Key Exclusion Criteria For Participants Who Participated in NBI-921352-DEE2012: \- Have developed any other disorder for which the treatment takes priority over treatment of SCN8A-DEE or is likely to interfere with study treatment or impair treatment compliance. For Participants Who did not Participate in NBI-921352-DEE2012: * Have symptoms that would be more consistent with another epilepsy disorder such as Dravet syndrome (for example, fever-induced episodes of status epilepticus, frequent myoclonic seizures, worsening on sodium channel blockers, absence seizures with generalized spike-and-wave electroencephalogram \[EEG\] as the sole seizure type). * Currently receiving cannabinoids or medical marijuana except Epidiolex/Epidyolex, unless approved by the Sponsor. * Currently taking systemic steroids (excluding inhaled medication for asthma treatments and intranasal steroids for allergies) such as adrenocorticotropin hormone, high-dose prednisolone for epileptic spasms. If participant has received these medications in the past, they must be off these medications for at least 3 months prior to the screening visit and these drugs may not be initiated during the duration of the study. Intermittent steroids to treat non-epilepsy-related diseases (such as allergies or dermatological conditions) are not exclusionary. * Have a history of moderate or severe head trauma or other neurological disorders or systemic medical diseases that are, in the investigator's opinion, likely to affect nervous system functioning. * Have a clinically significant medical condition or chronic disease, that in the opinion of the investigator would preclude the participant from participating in and completing the study or that could confound interpretation of study outcome. * Have clinically significant abnormal vital signs at the screening visit, as determined by the investigator. * Have one or more clinical laboratory test values outside the reference range, based on blood samples taken at the screening visit, that are of potential risk to the participants safety as determined by the investigator. * Have, at the screening visit, an electrocardiogram (ECG) finding of a corrected QT interval using Fridericia's formula (QTcF) \> 450 milliseconds (msec) or presence of any significant cardiac abnormality.

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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

  • Children's National Hospital

    Washington D.C., District of Columbia, 20010, United States

  • Cook Children's Medical Center

    Fort Worth, Texas, 76104, United States

  • UCSF Medical Center

    San Francisco, California, 94143, United States

  • University of Rochester

    Rochester, New York, 14642, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.