Status:
ACTIVE_NOT_RECRUITING
Personalized Antisense Oligonucleotide for a Single Participant With MAPK8IP3 Neurodevelopmental Disorder With or Without Variable Brain Abnormalities (NEDBA)
Lead Sponsor:
n-Lorem Foundation
Collaborating Sponsors:
Columbia University
Conditions:
Neurodevelopmental Disorder With or Without Variable Brain Abnormalities
Eligibility:
MALE
5-5 years
Phase:
PHASE1
PHASE2
Brief 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 (NE...
Detailed Description
This is an interventional study to evaluate the safety and efficacy of treatment with an individualized antisense oligonucleotide (ASO) treatment in a single participant with NEDBA due to a heterozygo...
Eligibility Criteria
Inclusion
- Informed consent/assent provided by the participant (when appropriate), and/or participant's parent(s) or legally authorized representative(s)
- Ability to travel to the study site and adhere to study-related follow-up examinations and/or procedures and provide access to participant's medical records
- Genetically confirmed neurodevelopmental disorder due to MAPK8IP3 mutation
Exclusion
- Use of investigational medication within 5 half-lives of the drug at enrolment
- Participant has any condition that in the opinion of the Site Investigator, would ultimately prevent the completion of study procedures.
Key Trial Info
Start Date :
February 24 2025
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
February 1 2027
Estimated Enrollment :
1 Patients enrolled
Trial Details
Trial ID
NCT07197294
Start Date
February 24 2025
End Date
February 1 2027
Last Update
September 29 2025
Active Locations (1)
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1
Columbia University
New York, New York, United States, 10032