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Personalized Antisense Oligonucleotide for a Single Participant With MAPK8IP3 Neurodevelopmental Disorder With or Without Variable Brain Abnormalities (NEDBA)
NCT07197294 · View on ClinicalTrials.gov ↗
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
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 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT07197294
The ClinicalTrials.gov registry entry for NCT07197294 describes a study currently listed as active not recruiting. It is categorized as Phase 1, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 1 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is n-Lorem Foundation, which has 9 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
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. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT07197294 reports 1 study location spanning 1 distinct geographic area — top geographies include New York. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
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. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT07197294?
The interventions under investigation include: nL-MAPK8-001 (DRUG). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT07197294?
This trial is sponsored by n-Lorem Foundation, which has 9 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
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.
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