This Phase 1/2 study at the University of California, San Diego involves delivering a personalized antisense oligonucleotide (ASO) drug, nL-SCN2A-002, designed for a single pediatric participant with SCN2A-associated developmental epileptic encephalopathy. Eligibility requires a genetically confirmed mutation, informed consent from the participant's parent(s)/guardian(s), and the ability to travel to the study site and comply with follow-up examinations. The trial is active but no longer recruiting.
The summary above is a plain-language rendering of the official record. The original English title is shown for reference.
Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.
Inclusion Criteria: * Informed consent provided by the participant's parent(s)/guardian(s) * Ability to travel to the study site, adhere to study-related follow-up examinations and/or procedures, and provide access to participant's medical records. * Genetically confirmed mutation Exclusion Criteria: • Use of an investigational medication within less than 5 half-lives of the drug at enrollment