Custom drug targets rare genetic brain condition in one patient
NCT ID NCT07197294
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tests a custom-made drug called an antisense oligonucleotide (ASO) designed specifically for one person with a rare genetic brain disorder (NEDBA) caused by a MAPK8IP3 mutation. The goal is to see if the drug can improve motor skills and reduce seizures over 12 to 24 months. Only one participant is enrolled, and the study is early-stage (Phase 1/2), so results will be very limited.
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Locations
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Columbia University
New York, New York, 10032, United States