Cure My Disease

Don't miss your cure!

 Log in / Sign up

Custom drug targets rare genetic brain condition in one patient

NCT ID NCT07197294

Disease control Sponsor: n-Lorem Foundation Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 1/2

Runs two stages together: safety and dose first, then whether the treatment works.

Participants

1 person

The number who actually took part.

Started

Feb 2025

Expected to finish

Feb 2027

An estimate. End dates often move.

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

5 to 5 years

Sex

Male participants only

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * 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 Criteria: * 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.

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for Neurodevelopmental disorder with or without variable brain abnormalities are added.

Our safety recommendation!

By submitting, you agree to our Terms of use

Conditions

The condition(s) this trial relates to.

neurodevelopmental disorder with or without variable brain abnormalities; NEDBA

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Neurodevelopmental disorder with or without variable brain abnormalities

Contacts and locations

Locations

  • Columbia University

    New York, New York, 10032, United States