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NCT07197268 · ClinicalTrials.gov registry record · Phase 1
Personalized Antisense Oligonucleotide Therapy for A Single Participant With ASXL3 Gene Mutation
A Phase 1 study of Bainbridge-Ropers Syndrome, sponsored by n-Lorem Foundation.
- Active
- Registry status
- Phase 1
- Development phase
- 1
- Enrollment target
- 1
- Study location
NCT07197268: Active Phase 1 study of Bainbridge-Ropers Syndrome, sponsored by n-Lorem Foundation.
NCT07197268 is a Phase 1 study of Bainbridge-Ropers Syndrome 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
NCT07197268, a Phase 1 study of Bainbridge-Ropers Syndrome, 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 Bainbridge-Ropers Syndrome (BRPS) due to a pathogenic, de novo nonsense variant in ASXL3
Primary Outcome
Incidence and severity of treatment-emergent adverse events (AEs) from baseline to 12- and 24-months post nL-ASXL3-001 administration
Conditions Studied
Interventions
- DRUG nL-ASXL3-001
Study Locations (1)
North Carolina
- University of North Carolina Chapel Hill - Chapel Hill
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 1 participants |
| Start Date | 2025-05-19 |
| Est. Completion | 2027-05 |
| Phase | Phase 1 |
What the registry record for NCT07197268 still lists
NCT07197268 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 Bainbridge-Ropers Syndrome appearing as the primary indexed condition, and to 1 intervention - of which nL-ASXL3-001 is the first listed.
NCT07197268 reports a single indexed study location in North Carolina.
Frequently Asked Questions
What is clinical trial NCT07197268 about?
NCT07197268 is a clinical study titled "Personalized Antisense Oligonucleotide Therapy for A Single Participant With ASXL3 Gene Mutation". This research project entails delivery of a personalized antisense oligonucleotide (ASO) drug designed for a single participant with Bainbridge-Ropers Syndrome (BRPS) due to a pathogenic, de novo nonsense variant in ASXL3
What is the current status of trial NCT07197268?
This trial is currently active not recruiting. It is a Phase 1 study. The enrollment target is 1 participants. The study started on 2025-05-19. Estimated completion is 2027-05.
What conditions does trial NCT07197268 study?
This clinical trial studies the following conditions: Bainbridge-Ropers Syndrome.
What interventions are being tested in trial NCT07197268?
The interventions under investigation include: nL-ASXL3-001 (DRUG).
Who is sponsoring clinical trial NCT07197268?
This trial is sponsored by n-Lorem Foundation, which has 12 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT07197268 being conducted?
This trial has 1 study location across North Carolina. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
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Where NCT07197268'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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