NCT06314490 is a Phase 1, PHASE2 clinical trial of nL-SCN2A-002 in Genetic Disease, sponsored by University of California, San Diego.

Who is sponsoring NCT06314490?

NCT06314490 is sponsored by University of California, San Diego.

What drugs are being tested in NCT06314490?

Interventions in NCT06314490: nL-SCN2A-002.

What condition does NCT06314490 study?

NCT06314490 studies Genetic Disease.

Is NCT06314490 still recruiting?

NCT06314490 is currently active, enrolled. Last updated 1 April 2025.

Last reviewed 2025-04-01 · How we verify

NCT06314490

Name: Drug Landscape Pharmaceutical Database
Creator: Drug Landscape
Published: 2026-01-01
License: https://creativecommons.org/licenses/by/4.0/

Personalized Antisense Oligonucleotide Therapy for Rare Pediatric Genetic Disease: SCN2A

Active, enrolled Phase 1, PHASE2 Last updated 1 April 2025

What this trial tests

Phase 1, PHASE2 trial testing nL-SCN2A-002 in Genetic Disease in 1 participant. Participants enrolled and being followed up; not accepting new ones.

Timeline

16 February 2024

Primary endpoint
16 February 2026

16 February 2026

Quick facts

Lead sponsor	University of California, San Diego
Phase	Phase 1, PHASE2
Status	Active, enrolled
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	1
Start date	16 February 2024
Primary completion	16 February 2026
Estimated completion	16 February 2026
Sites	1 location across United States

Drugs / interventions tested

nL-SCN2A-002 — full drug profile →

Conditions studied

Genetic Disease — all drugs for Genetic Disease →

Sponsor

University of California, San Diego

Who can join

Eligibility, any sex, with Genetic Disease. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

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

Publications & conference data

2 peer-reviewed publications reference this trial (live from Europe PMC):

Remote monitoring of social attention in neurogenetic syndromes and idiopathic neurodevelopmental disability.
Frazier TW, Busch RM, Klaas P, Lachlan K, et al · · 2025 · cited 3× · PMID 39643599 · DOI 10.1002/aur.3290
Antisense Oligonucleotides: Technological Advances, Clinical Progress, and Expanding Therapeutic Frontiers.
Xu L, Zhang H, Jiang B, Jiang Y, et al · · 2026 · PMID 42076098 · DOI 10.3390/pharmaceutics18040446

Verify or expand the search:

Other recruiting trials for Genetic Disease

Currently open trials in the same condition.

NCT06744543 — Clinical Decision Support to Identify Pediatric Patients With Undiagnosed Genetic Disease · NA · active not recruiting
NCT06435468 — Biocollection of Rare Pediatric-onset of Autoimmune and Autoinflammatory Diseases · NA · recruiting
NCT06821386 — N-Care Project: Enhancing Asian-Pacific Collaboration · recruiting
NCT07365254 — Accurate Assessment and Intervention Research on Newborn Whole Genome Sequencing and Genetic Disease Risk · recruiting
NCT06725901 — Pediatric Neurogenetic Diagnosis Support Platform · NA · recruiting

Other University of California, San Diego trials

Trials by the same sponsor.

NCT07342543 — Guided Physical Activity Counseling for Hypertension in Primary Care · NA · not yet recruiting
NCT06505317 — Artificial Intelligence for Early Detection of Peripheral Artery Disease · NA · not yet recruiting
NCT07211802 — CKM For Safe Use of SGLT2i in Type 1 Diabetes · Phase 3 · not yet recruiting
NCT07524751 — Culturally Adapted Pain Management for Indigenous Peoples · NA · not yet recruiting
NCT07518784 — Accurate Point of Care Liver Disease Diagnostics (Phase 2) · recruiting

Verify against primary sources

ClinicalTrials.gov — authoritative US registry record
WHO ICTRP — international registry index
EU Clinical Trials Register
Sponsor press releases (Google)

Data sources for this page

Trial protocol + status: ClinicalTrials.gov NCT06314490 (US National Library of Medicine, public domain)
Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by University of California, San Diego
Last refreshed: 1 April 2025

Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT06314490.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing