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NCT07588581 · ClinicalTrials.gov registry record · Phase 1
Personalized Antisense Oligonucleotide for A Single Participant With UBTF Gene Mutation
A Phase 1 study, sponsored by n-Lorem Foundation.
- Active
- Registry status
- Phase 1
- Development phase
- 1
- Enrollment target
NCT07588581: Active Phase 1 study, sponsored by n-Lorem Foundation.
NCT07588581 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
NCT07588581, 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 Childhood-Onset Neurodegeneration with Brain Atrophy (CONDBA) due to a heterozygous missense gain-of-function mutation in UBTF
Primary Outcome
Change in gross motor function from baseline to 6-, 12-, 18-, and 24-months post nL-UBTF-001 administration as measured by Brief Ataxia Rating Scale (BARS)
Interventions
- DRUG nL-UBTF-001
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 1 participants |
| Start Date | 2025-02-05 |
| Est. Completion | 2027-02 |
| Phase | Phase 1 |
What the registry record for NCT07588581 still lists
NCT07588581 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-UBTF-001 is the first listed.
NCT07588581 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT07588581 about?
NCT07588581 is a clinical study titled "Personalized Antisense Oligonucleotide for A Single Participant With UBTF Gene Mutation". This research project entails delivery of a personalized antisense oligonucleotide (ASO) drug designed for a single participant with Childhood-Onset Neurodegeneration with Brain Atrophy (CONDBA) due to a heterozygous missense gain-of-function mutation in UBTF
What is the current status of trial NCT07588581?
This trial is currently active not recruiting. It is a Phase 1 study. The enrollment target is 1 participants. The study started on 2025-02-05. Estimated completion is 2027-02.
What interventions are being tested in trial NCT07588581?
The interventions under investigation include: nL-UBTF-001 (DRUG).
Who is sponsoring clinical trial NCT07588581?
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 NCT07588581'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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