Clinical Trial

Personalized Antisense Oligonucleotide Therapy for A Single Participant With ATN1 Gene Mutation

Active, Not Recruiting Phase 1/2
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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
Protocol Amendment History 1 amendment
This ClinicalTrials.gov record has been amended once since 2024-11-22.
Trial Details
NCT Number NCT06706388
Lead Sponsor n-Lorem Foundation
Collaborators: Columbia University
Conditions Dentatorubral-Pallidoluysian Atrophy
Enrollment 1 participants
Start Date 2024-02-21
Primary Completion 2027-02 (estimated)
Study Completion 2027-02 (estimated)
Updated on ClinicalTrials.gov 2026-04-09