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ACTIVE NOT RECRUITING
NCT06314490
PHASE1/PHASE2
Personalized Antisense Oligonucleotide Therapy for Rare Pediatric Genetic Disease: SCN2A
Sponsor: University of California, San Diego
View on ClinicalTrials.gov
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
Key Details
Gender
All
Age Range
Any - Any
Study Type
INTERVENTIONAL
Enrollment
1
Start Date
2024-02-16
Completion Date
2026-02-16
Last Updated
2025-04-01
Healthy Volunteers
No
Conditions
Interventions
DRUG
nL-SCN2A-002
Personalized antisense oligonucleotide
Locations (1)
Rady Children's Hospital
San Diego, California, United States