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NCT07197294 · ClinicalTrials.gov registry record · Phase 1
Personalized Antisense Oligonucleotide for a Single Participant With MAPK8IP3 Neurodevelopmental Disorder With or Without Variable Brain Abnormalities (NEDBA)
A Phase 1 study of Neurodevelopmental Disorder With or Without Variable Brain Abnormalities, sponsored by n-Lorem Foundation.
- Active
- Registry status
- Phase 1
- Development phase
- 1
- Enrollment target
- 1
- Study location
NCT07197294: Active Phase 1 study of Neurodevelopmental Disorder With or Without Variable Brain Abnormalities, sponsored by n-Lorem Foundation.
NCT07197294 is a Phase 1 study of Neurodevelopmental Disorder With or Without Variable Brain Abnormalities 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
NCT07197294, a Phase 1 study of Neurodevelopmental Disorder With or Without Variable Brain Abnormalities, 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 Neurodevelopmental Disorder with or without Brain Abnormalities (NEDBA) due to a heterozygous pathogenic missense mutation in MAPK8IP3
Primary Outcome
Change in gross motor function from baseline to 12- and 24-months post nL-MAPK8-001 administration as measured by the Gross Motor Function Measure-88 score.
Conditions Studied
Interventions
- DRUG nL-MAPK8-001
Study Locations (1)
New York
- Columbia University - New York
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 1 participants |
| Start Date | 2025-02-24 |
| Est. Completion | 2027-02 |
| Phase | Phase 1 |
What the registry record for NCT07197294 still lists
NCT07197294 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 Neurodevelopmental Disorder With or Without Variable Brain Abnormalities appearing as the primary indexed condition, and to 1 intervention - of which nL-MAPK8-001 is the first listed.
NCT07197294 reports a single indexed study location in New York.
Frequently Asked Questions
What is clinical trial NCT07197294 about?
NCT07197294 is a clinical study titled "Personalized Antisense Oligonucleotide for a Single Participant With MAPK8IP3 Neurodevelopmental Disorder With or Without Variable Brain Abnormalities (NEDBA)". This research project entails delivery of a personalized antisense oligonucleotide (ASO) drug designed for a single participant with Neurodevelopmental Disorder with or without Brain Abnormalities (NEDBA) due to a heterozygous pathogenic missense mutation in MAPK8IP3
What is the current status of trial NCT07197294?
This trial is currently active not recruiting. It is a Phase 1 study. The enrollment target is 1 participants. The study started on 2025-02-24. Estimated completion is 2027-02.
What conditions does trial NCT07197294 study?
This clinical trial studies the following conditions: Neurodevelopmental Disorder With or Without Variable Brain Abnormalities.
What interventions are being tested in trial NCT07197294?
The interventions under investigation include: nL-MAPK8-001 (DRUG).
Who is sponsoring clinical trial NCT07197294?
This trial is sponsored by n-Lorem Foundation, which has 12 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT07197294 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
How this trial's enrollment target compares
Where NCT07197294'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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