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Personalized Antisense Oligonucleotide Therapy for Rare Pediatric Genetic Disease: SCN2A

NCT06314490 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 1

Last updated 2025-04-01

No results posted yet for this study

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

Conditions

Interventions

DRUG

nL-SCN2A-002

Personalized antisense oligonucleotide

Sponsors & Collaborators

Principal Investigators

  • Olivia Kim-McManus, M.D. · UCSD Rady Children's Hospital

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-02-16
Primary Completion
2026-02-16
Completion
2026-02-16
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Entities

Diseases
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06314490 on ClinicalTrials.gov