Promising seizure drug study halted early
NCT ID NCT05226780
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.
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Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
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8 people
The number who actually took part.
- Started
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Jul 2022
- Finished
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Nov 2025
- Lead sponsor
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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
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2 to 22 years
- Sex
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Anyone
- Healthy volunteers
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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 Hide 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
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Children's National Hospital
Washington D.C., District of Columbia, 20010, United States
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Cook Children's Medical Center
Fort Worth, Texas, 76104, United States
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UCSF Medical Center
San Francisco, California, 94143, United States
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University of Rochester
Rochester, New York, 14642, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.