Personalized Antisense Oligonucleotide for A Single Participant With ATN1 Gene Mutation
Public ClinicalTrials.gov record NCT07084311. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
An Open-label, Single Center, Single Participant Study of an Experimental Antisense Oligonucleotide Treatment for ATN1 Mutation Associated Dentatorubral-pallidoluysian Atrophy (DRPLA)
Study identification
- NCT ID
- NCT07084311
- Recruitment status
- Active, not recruiting
- Study type
- Interventional
- Phase
- Phase 1, Phase 2
- Lead sponsor
- n-Lorem Foundation
- Other
- Enrollment
- 1 participant
Conditions and interventions
Conditions
Interventions
- nL-ATN1-002 Drug
Drug
Eligibility (public fields only)
- Age range
- 29 Years to 29 Years
- Sex
- Female
- Healthy volunteers
- Not listed
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Oct 23, 2024
- Primary completion
- Oct 31, 2026
- Completion
- Oct 31, 2026
- Last update posted
- Jul 23, 2025
2024 – 2026
United States locations
- U.S. sites
- 1
- U.S. states
- 1
- U.S. cities
- 1
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Hawaii Pacific Neuroscience | Honolulu | Hawaii | 96817 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT07084311, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Jul 23, 2025 · Synced Apr 22, 2026
Related: full search, browse by condition, browse by drug or therapy, browse by sponsor, browse by U.S. city.
Open the official record
The complete protocol, eligibility criteria, and contact information for NCT07084311 live on ClinicalTrials.gov.