NCT07084311Active, not recruitingPhase 1/2
Personalized Antisense Oligonucleotide for A Single Participant With ATN1 Gene Mutation
An Open-label, Single Center, Single Participant Study of an Experimental Antisense Oligonucleotide Treatment for ATN1 Mutation Associated Dentatorubral-pallidoluysian Atrophy (DRPLA)
Source: ClinicalTrials.gov · Last updated 13 Jul 2026
Brief summary (registry, in English)
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
- Status (registry)
- Active, not recruiting
- Phase (registry)
- Phase 1/2
- Study type
- Interventional
- Enrollment
- 1 (actual)
- First submitted
- 8 May 2025
- Start date
- 24 Oct 2024
- Primary completion date
- Nov 2026
- Lead sponsor
- n-Lorem Foundation
- NCT number
- NCT07084311
What you commit to
- Study type: Interventional · Phase 1/2
- Participants enrolled: 1
- From 24 Oct 2024 to the primary completion date, Nov 2026
- 1 site in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- Use of investigational medication within 5 half-lives of the drug at enrollment.
Other illness
- Participant has any condition that in the opinion of the Site Investigator, would ultimately prevent the completion of study procedures.
Other
- Participant has any known contraindication to or unwillingness to undergo lumbar puncture.
Who the study is looking for
- Informed consent/assent provided by the participant (when appropriate), and/or participant's parent(s) or legally authorized representative(s).
- Ability to travel to the study site and adhere to study-related follow-up examinations and/or procedures and provide access to participant's medical records.
- Clinical phenotype and neuroimaging consistent with a diagnosis of ATN1 mutation associated Dentatorubral-pallidoluysian atrophy (DRPLA).
- Documented genetic mutation in ATN1.
Age in the registry: 29 years to 29 years · Sex: Female · Does not accept healthy volunteers
Sites (1)
Sites in registry order. Save your city in your profile to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Hawaii Pacific Neuroscience Coordinator of this site? Confirm its status | Honolulu | United States | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.