Personalized Antisense Oligonucleotide for a Single Participant With MAPK8IP3 Neurodevelopmental Disorder With or Without Variable Brain Abnormalities (NEDBA)
Public ClinicalTrials.gov record NCT07197294. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
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Official title
An Open-label Single Center, Single Participant Study of an Experimental Antisense Oligonucleotide Treatment for Neurodevelopmental Disorder With or Without Variable Brain Abnormalities (NEDBA) Due to MAPK8IP3 Mutation
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
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
Study identification
- NCT ID
- NCT07197294
- Recruitment status
- Active, not recruiting
- Study type
- Interventional
- Phase
- Phase 1, Phase 2
- Enrollment
- 1 participant
Conditions and interventions
Eligibility (public fields only)
- Age range
- 5 Years to 5 Years
- Sex
- Male
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Feb 24, 2025 (Actual)
- Primary completion
- Feb 2027 (Estimated)
- Completion
- Feb 2027 (Estimated)
- Last update posted
- Sep 29, 2025
2025 – 2027
United States locations
- U.S. sites
- 1
- U.S. states
- 1
- U.S. cities
- 1
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Columbia University | New York | New York | 10032 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT07197294, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Sep 29, 2025 · Synced Nov 6, 2026
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Open the official record
The complete public registry record, listed eligibility criteria, and available contact information for NCT07197294 live on ClinicalTrials.gov.