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New study aims to map rare childhood brain disorder

NCT ID NCT07531511

Knowledge-focused Sponsor: UCB Biopharma SRL Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
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
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 24, 2026 · Last updated Jul 31, 2026 · Updated 5 times

Summary

This study will follow 60 children with SLC6A1-NDD, a rare genetic condition causing seizures, developmental delays, and autism. Researchers will track symptoms like seizure frequency and development over time to better understand the disease. The goal is to gather information that could help design future treatment studies.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

What this could lead to
If successful, this study could provide the data needed to design future trials for treatments targeting SLC6A1-NDD.
What could go wrong
This is an observational study, not a treatment trial. It may not lead directly to new therapies, and results depend on participant engagement and data quality.

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.

Participants

About 60 people

The number the study aims to enrol. It can still change while the study runs.

Started

Jul 2026

Expected to finish

May 2029

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

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

Who is studied

The study population comprises pediatric patients with a diagnosis of SLC6A1-NDD.

Ages

Up to 17 years

Sex

Anyone

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: * Patient with a diagnosis of SLC6A1-NDD characterized by epilepsy, global developmental delay, autism spectrum disorder, or intellectual disability, with a documented history of an SLC6A1 mutation, defined as pathogenic or likely pathogenic by the Investigator. * Patients should not be older than 17 years at time of assent/consent. * Patients under the age of 18 years with legal guardians providing informed consent. Assent will be obtained from any patients judged to have sufficient capacity to provide assent at the discretion of the Investigator. * Patient and patient's caregiver are willing and able to comply with study requirements (including diary completion and visit schedule). Exclusion Criteria: * Patients and their caregivers are unable to complete follow-up visits. * Patients with a history of an alternate diagnosis for disease, including a genetic cause, which is known to contribute to epilepsy or NDD. * Patient is currently receiving an investigational product(s) other than 4-phenylbutyrate or has received an investigational product within 30 days or within \<5 times the half-life of the investigational product, whichever is longer, prior to the Enrollment Visit.

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Conditions

The condition(s) this trial relates to.

developmental and epileptic encephalopathy

As listed by the trial registrant

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

Developmental and epileptic encephalopathies Developmental and epileptic encephalopathies (DEE) SLC6A1 neurodevelopmental disorder (NDD)

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    3 sites in 3 countries. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • Gtep01 40131

    RECRUITING

    Strasbourg, France

  • Gtep01 40870

    RECRUITING

    Madrid, Spain

  • Gtep01 50614

    RECRUITING

    New York, New York, 10021, United States

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