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NCT06706388 · ClinicalTrials.gov registry record · Phase 1
Personalized Antisense Oligonucleotide Therapy for a Single Participant with ATN1 Gene Mutation
A Phase 1 study of Dentatorubral-Pallidoluysian Atrophy, sponsored by n-Lorem Foundation.
- Active
- Registry status
- Phase 1
- Development phase
- 1
- Enrollment target
- 1
- Study location
NCT06706388: Active Phase 1 study of Dentatorubral-Pallidoluysian Atrophy, sponsored by n-Lorem Foundation.
NCT06706388 is a Phase 1 study of Dentatorubral-Pallidoluysian Atrophy 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). The trial reports 1 study location across 1 state. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT06706388, a Phase 1 study of Dentatorubral-Pallidoluysian Atrophy, is active but no longer recruiting, sponsored by n-Lorem Foundation.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 1
- Development phase
- 1 participants
- Enrollment target
- 1
- Study location
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 mobility and ataxia from baseline to 6-, 12-, 18- and 24-months post nL-ATN1-002 administration as measured by the Scale for Assessment and Rating of Ataxia (SARA).
Conditions Studied
Interventions
- DRUG nL-ATN1-002
Study Locations (1)
New York
- Columbia University - New York
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 1 participants |
| Start Date | 2024-02-21 |
| Est. Completion | 2026-02 |
| Phase | Phase 1 |
What the registry record for NCT06706388 still lists
NCT06706388 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 1 condition, with Dentatorubral-Pallidoluysian Atrophy appearing as the primary indexed condition, and to 1 intervention - of which nL-ATN1-002 is the first listed.
NCT06706388 reports a single indexed study location in New York.
Frequently Asked Questions
What is clinical trial NCT06706388 about?
NCT06706388 is a clinical study titled "Personalized Antisense Oligonucleotide Therapy for a Single Participant 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 NCT06706388?
This trial is currently active not recruiting. It is a Phase 1 study. The enrollment target is 1 participants. The study started on 2024-02-21. Estimated completion is 2026-02.
What conditions does trial NCT06706388 study?
This clinical trial studies the following conditions: Dentatorubral-Pallidoluysian Atrophy.
What interventions are being tested in trial NCT06706388?
The interventions under investigation include: nL-ATN1-002 (DRUG).
Who is sponsoring clinical trial NCT06706388?
This trial is sponsored by n-Lorem Foundation, which has 12 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT06706388 being conducted?
This trial has 1 study location across New York. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
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Where NCT06706388'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
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Source ClinicalTrials.gov registry export · 2026-08-08
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