Clinical Trial

Personalized Antisense Oligonucleotide 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 2025-07-16.
Trial Details
NCT Number NCT07084311
Lead Sponsor n-Lorem Foundation
Collaborators: Hawaii Pacific Neuroscience
Conditions Dentatorubral-Pallidoluysian Atrophy
Enrollment 1 participants
Start Date 2024-10-24
Primary Completion 2026-11 (estimated)
Study Completion 2026-11 (estimated)
Updated on ClinicalTrials.gov 2026-07-13