NCT06706388

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

An Open-label Single Center, Single Participant Study of an Experimental Antisense Oligonucleotide Treatment for ATN1 Gene Mutation

• Status: Active Not Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 1 (actual)
• Sponsor: n-Lorem Foundation · Academic / Other
• Sex: Male
• Age: 17 Years – 17 Years
• Healthy volunteers: Not accepted

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

Detailed description

This is an interventional study to evaluate the safety and efficacy of treatment with an individualized antisense oligonucleotide (ASO) treatment in a single participant with DRPLA due to a heterozygous pathogenic CAG trinucleotide expansion in ATN1

Conditions

• Dentatorubral-Pallidoluysian Atrophy

Interventions

Type	Name	Description
DRUG	nL-ATN1-002	Personalized antisense oligonucleotide

Timeline

• Start date: 2024-02-21
• Primary completion: 2027-02-01
• Completion: 2027-02-01
• First posted: 2024-11-26
• Last updated: 2026-04-09

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study