NCT06706388Active, not recruitingPhase 1/2
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
Source: ClinicalTrials.gov · Last updated 9 Apr 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
- 21 Nov 2024
- Start date
- 21 Feb 2024
- Primary completion date
- 28 Feb 2027
- Lead sponsor
- n-Lorem Foundation
- NCT number
- NCT06706388
What you commit to
- Study type: Interventional · Phase 1/2
- Participants enrolled: 1
- From 21 Feb 2024 to the primary completion date, 28 Feb 2027
- 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 enrolment
Other illness
- Participant has any condition that in the opinion of the Site Investigator, would ultimately prevent the completion of study procedures.
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.
- Genetically confirmed Dentatorubral-pallidoluysian atrophy (DRPLA) due to ATN1 mutation
Age in the registry: 17 years to 17 years · Sex: Male · Does not accept healthy volunteers
Sites (1)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Columbia University Coordinator of this site? Confirm its status | New York | United States | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.