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NCT07221760 · ClinicalTrials.gov registry record · Phase 1
Personalized Antisense Oligonucleotide for A Single Participant (nL62541) With ATN1 Gene Mutation
A Phase 1 study, sponsored by n-Lorem Foundation.
- Active
- Registry status
- Phase 1
- Development phase
- 1
- Enrollment target
NCT07221760: Active Phase 1 study, sponsored by n-Lorem Foundation.
NCT07221760 is a Phase 1 study that is active but no longer recruiting, run by n-Lorem Foundation. The registered enrollment target is 1 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (98% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT07221760, a Phase 1 study, is active but no longer recruiting, sponsored by n-Lorem Foundation.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 1
- Development phase
- 1 participants
- Enrollment target
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
Primary Outcome
Change in seizure length from baseline to every 3 months post nL-ATN1-002 administration as measured by routine electroencephalography (EEG) (changes in frequency of ictal and interictal discharges, evoked potentials, and changes in EEG background)
Interventions
- DRUG nL-ATN1-001
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 1 participants |
| Start Date | 2025-11-06 |
| Est. Completion | 2027-11 |
| Phase | Phase 1 |
What the registry record for NCT07221760 still lists
NCT07221760 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 1 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (98% lower).
The record links to 0 conditions, and to 1 intervention - of which nL-ATN1-001 is the first listed.
NCT07221760 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT07221760 about?
NCT07221760 is a clinical study titled "Personalized Antisense Oligonucleotide for A Single Participant (nL62541) With ATN1 Gene Mutation". 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
What is the current status of trial NCT07221760?
This trial is currently active not recruiting. It is a Phase 1 study. The enrollment target is 1 participants. The study started on 2025-11-06. Estimated completion is 2027-11.
What interventions are being tested in trial NCT07221760?
The interventions under investigation include: nL-ATN1-001 (DRUG).
Who is sponsoring clinical trial NCT07221760?
This trial is sponsored by n-Lorem Foundation, which has 12 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT07221760's enrollment target sits among peer trials
1 2001st of 2000 the lowest of 2,000 other Phase 1 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 1 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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