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Personalized Antisense Oligonucleotide Therapy for Rare Pediatric Genetic Disease: SCN2A

Genetic Disease Clinical Trial

Official title:
Personalized Antisense Oligonucleotide Therapy for Rare Pediatric Genetic Disease: SCN2A

Clinical Trial Summary

This research project entails delivery of a personalized antisense oligonucleotide (ASO) drug designed for a single pediatric participant with SCN2A associated developmental epileptic encephalopathy

Clinical Trial Description

This is an interventional study to evaluate the safety and efficacy of treatment with an individualized antisense oligonucleotide (ASO) treatment in a single pediatric participant with a de novo pathogenic gain of function SCN2A mutation associated with severe developmental epileptic encephalopathy. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT06314490
Study type Interventional
Source University of California, San Diego
Contact
Status Active, not recruiting
Phase Phase 1/Phase 2
Start date February 16, 2024
Completion date February 16, 2026
View Details
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