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NCT06314490 · ClinicalTrials.gov registry record · Phase 1
Personalized Antisense Oligonucleotide Therapy for Rare Pediatric Genetic Disease: SCN2A
A Phase 1 study of Genetic Disease, sponsored by University of California, San Diego.
- Active
- Registry status
- Phase 1
- Development phase
- 1
- Enrollment target
- 1
- Study location
NCT06314490: Active Phase 1 study of Genetic Disease, sponsored by University of California, San Diego.
NCT06314490 is a Phase 1 study of Genetic Disease that is active but no longer recruiting, run by University of California, San Diego. The registered enrollment target is 1 participants, below the 2,561-participant average among 30 other Genetic Disease trials with a reported enrollment target (100% lower). The trial reports 1 study location across 1 state. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT06314490, a Phase 1 study of Genetic Disease, is active but no longer recruiting, sponsored by University of California, San Diego.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 1
- Development phase
- 1 participants
- Enrollment target
- 1
- Study location
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
Primary Outcome
As measured by caregiver provided seizure diary
Conditions Studied
Interventions
- DRUG nL-SCN2A-002
Study Locations (1)
California
- Rady Children's Hospital - San Diego
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 1 participants |
| Start Date | 2024-02-16 |
| Est. Completion | 2026-02-16 |
| Phase | Phase 1 |
What the registry record for NCT06314490 still lists
NCT06314490 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 1 participants, a relatively small participant target, below the 2,561-participant average among 30 other Genetic Disease trials with a reported enrollment target (100% lower).
The record links to 1 condition, with Genetic Disease appearing as the primary indexed condition, and to 1 intervention - of which nL-SCN2A-002 is the first listed.
NCT06314490 reports a single indexed study location in California.
Frequently Asked Questions
What is clinical trial NCT06314490 about?
NCT06314490 is a clinical study titled "Personalized Antisense Oligonucleotide Therapy for Rare Pediatric Genetic Disease: SCN2A". This research project entails delivery of a personalized antisense oligonucleotide (ASO) drug designed for a single pediatric participant with SCN2A associated developmental epileptic encephalopathy
What is the current status of trial NCT06314490?
This trial is currently active not recruiting. It is a Phase 1 study. The enrollment target is 1 participants. The study started on 2024-02-16. Estimated completion is 2026-02-16.
What conditions does trial NCT06314490 study?
This clinical trial studies the following conditions: Genetic Disease.
What interventions are being tested in trial NCT06314490?
The interventions under investigation include: nL-SCN2A-002 (DRUG).
Who is sponsoring clinical trial NCT06314490?
This trial is sponsored by University of California, San Diego, which has 775 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT06314490 being conducted?
This trial has 1 study location across California. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
Similar trials for Genetic Disease
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT06314490's enrollment target sits among peer trials
1 30th of 30 the lowest of 30 other Genetic Disease trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Genetic Disease trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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