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Personalized Antisense Oligonucleotide for A Single Participant With ATN1 Gene Mutation

NCT07084311 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 1

Last updated 2025-07-24

No results posted yet for this study

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

Conditions

  • Dentatorubral-Pallidoluysian Atrophy

Interventions

DRUG

nL-ATN1-002

Personalized antisense oligonucleotide

Sponsors & Collaborators

  • Hawaii Pacific Neuroscience

    collaborator UNKNOWN

  • n-Lorem Foundation

    lead OTHER

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
29 Years
Max Age
29 Years
Sex
FEMALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-10-24
Primary Completion
2026-11-30
Completion
2026-11-30
FDA Drug
Yes

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07084311 on ClinicalTrials.gov