NCT06706388View on ClinicalTrials.gov

Personalized Antisense Oligonucleotide Therapy for a Single Participant with ATN1 Gene Mutation

PHASE1/PHASE2Active, not recruitingINTERVENTIONAL
Enrollment

1

Participants

Timeline

Start Date

February 21, 2024

Primary Completion Date

February 28, 2026

Study Completion Date

February 28, 2026

Conditions
Dentatorubral-Pallidoluysian Atrophy
Interventions
DRUG

nL-ATN1-002

Personalized antisense oligonucleotide

Trial Locations (1)

10027

Columbia University, New York

All Listed Sponsors
collaborator

Columbia University

OTHER

lead

n-Lorem Foundation

OTHER