Personalized Antisense Oligonucleotide Therapy for Rare Pediatric Genetic Disease: SCN2A

Plain-language summary

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.

Trial details

Phase

PHASE1/PHASE2

Status

Active, not recruiting

Sponsor

University of California, San Diego

Intervention

nL-SCN2A-002

Start date

2/16/2024

Estimated completion

2/16/2026

Countries

United States

Related genes

Locations

• San Diego, United States

Eligibility criteria

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