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Can a daily tablet quiet the seizures of two devastating genetic disorders?

NCT ID NCT03694275

Symptom relief Sponsor: Takeda 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
Running, but no longer taking on new participants.
Completed This study
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Sep 10, 2026 · Last updated Sep 11, 2026 · Updated 1 time

Summary

Researchers are testing an experimental drug called soticlestat in people with two rare genetic conditions: Dup15q syndrome and CDKL5 deficiency disorder. Both conditions cause frequent motor seizures that are hard to control. The trial enrolls about 20 participants who take one to six standard anti-seizure medications. Everyone receives soticlestat tablets, and researchers track whether the number of motor seizures per month changes during the maintenance phase of treatment.

What this could mean

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

Active substance
soticlestat, an experimental tablet
What this could lead to
If it works, soticlestat could become a new add-on option for reducing seizures in these rare genetic conditions, which currently have few targeted treatments.
What could go wrong
This is a small, open-label pilot study with no placebo group, so any seizure reduction could be partly due to chance or expectation. Larger controlled trials would be needed to confirm a real effect.

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 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

20 people

The number who actually took part.

Started

Sep 2018

Finished

Jul 2020

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.

Ages

2 to 55 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: 1. Clinical diagnosis of Dup 15q or CDKL5 deficiency disorder. 2. Currently taking 1 to 6 antiepileptic drugs (AEDs) at a stable dose. Exclusion Criteria: 1. Two or more episodes of convulsive status epilepticus per 3 months requiring hospitalization and intubation. 2. Currently receiving a study drug or participated in a clinical study involving another investigational product in the previous month.

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Conditions

The condition(s) this trial relates to.

15q11q13 microduplication syndrome autism brain disorder CDKL5 disorder central nervous system disorder developmental and epileptic encephalopathy, 2 epilepsy hereditary mixed polyposis syndrome partial duplication of the long arm of chromosome 15

As listed by the trial registrant

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

15q duplication syndrome CDKL5 deficiency disease

Contacts and locations

Locations

  • Boston Children's Hospital Translational Neuroscience Center

    Boston, Massachusetts, 02115, United States

  • Center for Rare Neurological Diseases

    Norcross, Georgia, 30093, United States

  • Center for Rare Neurological Diseases (CRND)--Massachusetts General Hospital

    Boston, Massachusetts, 02114, United States

  • Columbia University Medical Center

    New York, New York, 10032, United States

  • Minnesota Epilepsy Group, P.A.

    Saint Paul, Minnesota, 55102, United States

  • New York University (NYU)

    New York, New York, 10017, United States

  • Research Institute Children's Hospital Colorado

    Aurora, Colorado, 80045, United States

  • UCLA

    Los Angeles, California, 90095, United States