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NCT06816498 · ClinicalTrials.gov registry record · Phase 1
Personalized Antisense Oligonucleotide Therapy for A Single Participant With LMNB1 Mutation Associated Autosomal Dominant Leukodystrophy (ADLD)
A Phase 1 study of Autosomal Dominant Leukodystrophy, sponsored by n-Lorem Foundation.
- Active
- Registry status
- Phase 1
- Development phase
- 1
- Enrollment target
- 1
- Study location
NCT06816498: Active Phase 1 study of Autosomal Dominant Leukodystrophy, sponsored by n-Lorem Foundation.
NCT06816498 is a Phase 1 study of Autosomal Dominant Leukodystrophy 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
NCT06816498, a Phase 1 study of Autosomal Dominant Leukodystrophy, 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 Autosomal Dominant Leukodystrophy (ADLD) due to LMNB1 mutation
Primary Outcome
Change in gait from baseline at 6-, 12-, 18- and 24-months post nL-LMNB1-001 administration as measured by gait motion analysis
Conditions Studied
Interventions
- DRUG nL-LMNB1-001
Study Locations (1)
Minnesota
- Mayo Clinic - Rochester
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 1 participants |
| Start Date | 2025-03-17 |
| Est. Completion | 2027-03 |
| Phase | Phase 1 |
What the registry record for NCT06816498 still lists
NCT06816498 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 Autosomal Dominant Leukodystrophy appearing as the primary indexed condition, and to 1 intervention - of which nL-LMNB1-001 is the first listed.
NCT06816498 reports a single indexed study location in Minnesota.
Frequently Asked Questions
What is clinical trial NCT06816498 about?
NCT06816498 is a clinical study titled "Personalized Antisense Oligonucleotide Therapy for A Single Participant With LMNB1 Mutation Associated Autosomal Dominant Leukodystrophy (ADLD)". This research project entails delivery of a personalized antisense oligonucleotide (ASO) drug designed for a single participant with Autosomal Dominant Leukodystrophy (ADLD) due to LMNB1 mutation
What is the current status of trial NCT06816498?
This trial is currently active not recruiting. It is a Phase 1 study. The enrollment target is 1 participants. The study started on 2025-03-17. Estimated completion is 2027-03.
What conditions does trial NCT06816498 study?
This clinical trial studies the following conditions: Autosomal Dominant Leukodystrophy.
What interventions are being tested in trial NCT06816498?
The interventions under investigation include: nL-LMNB1-001 (DRUG).
Who is sponsoring clinical trial NCT06816498?
This trial is sponsored by n-Lorem Foundation, which has 12 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT06816498 being conducted?
This trial has 1 study location across Minnesota. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT06816498'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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