Clinical Research Directory
Browse clinical research sites, groups, and studies.
Back to Studies
ACTIVE NOT RECRUITING
NCT06706388
PHASE1/PHASE2
Personalized Antisense Oligonucleotide Therapy for A Single Participant With ATN1 Gene Mutation
Sponsor: n-Lorem Foundation
View on ClinicalTrials.gov
Summary
This research project entails delivery of a personalized antisense oligonucleotide (ASO) drug designed for a single participant with dentatorubral-pallidoluysian atrophy (DRPLA) due to a heterozygous pathogenic CAG trinucleotide expansion in ATN1
Official title: An Open-label Single Center, Single Participant Study of an Experimental Antisense Oligonucleotide Treatment for ATN1 Gene Mutation
Key Details
Gender
MALE
Age Range
17 Years - 17 Years
Study Type
INTERVENTIONAL
Enrollment
1
Start Date
2024-02-21
Completion Date
2027-02
Last Updated
2026-04-09
Healthy Volunteers
No
Conditions
Interventions
DRUG
nL-ATN1-002
Personalized antisense oligonucleotide
Locations (1)
Columbia University
New York, New York, United States