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Experimental seizure drug tested in rare genetic disorder

NCT ID NCT05604170

Disease control Sponsor: Marinus Pharmaceuticals 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
The trial has finished. Results may not be published yet.
Stopped early This study
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This status has not been confirmed recently, so it may be out of date.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 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This phase 3 study tested the drug ganaxolone as an add-on treatment for seizures in children and adults with tuberous sclerosis complex (TSC). The trial was open-label, meaning everyone received the drug, and it included 117 people who had previously been in related studies. The main goals were to check safety and see if seizures decreased, but the study was terminated early.

What this could mean

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

Active substance
ganaxolone
What this could lead to
If it works, this could provide a new add-on treatment option to reduce seizures in people with tuberous sclerosis complex.
What could go wrong
The trial was terminated early, so results are limited. It is an open-label extension, meaning everyone knew they were getting the drug, which can bias results. Side effects and lack of effectiveness are possible.

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 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

117 people

The number who actually took part.

Started

May 2022

Finished

Apr 2025

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

1 year to 65 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. Completion of Study 1042-TSC-3001 or participants who continue to meet study requirements in Study 1042-TSC-2001. 2. Participant/parent(s)/LAR(s) willing and able to give written informed consent/assent, after being properly informed of the nature and risks of the study and prior to engaging in any study-related procedures. If the participant is not qualified or able to provide written informed consent based on age, developmental stage, intellectual capacity, or other factors, parent(s)/LAR(s) must provide assent for study participation, if appropriate. 3. Parent(s)/caregiver(s) is (are) willing and able to maintain an accurate and complete daily seizure diary for the duration of the study. 4. Willing and able to take Investigational product (IP) (suspension) as directed with food TID. 5. Women of childbearing potential (WOCBP) must be using a medically acceptable method of birth control and have a negative quantitative serum beta-human chorionic growth hormone (β-HCG) test collected at the initial visit. Childbearing potential is defined as a female who is biologically capable of becoming pregnant. Medically acceptable methods of birth control include intrauterine devices (that have been in place for at least 1 month prior to the screening visit), hormonal contraceptives (eg, combined oral contraceptives, patch, vaginal ring, injectables, and implants), and surgical sterilization (such as oophorectomy or tubal ligation). When used consistently and correctly, "double-barrier" methods of contraception can be used as an effective alternative to highly effective contraception methods. Contraceptive measures such as Plan B™, sold for emergency use after unprotected sex, are not acceptable methods for routine use 6. Male participants must agree to use highly effective contraceptive methods during the study and for 30 days after the last dose of IP. Highly effective methods of contraception include surgical sterilization (such as a vasectomy) and adequate "double-barrier" methods. Exclusion Criteria: 1. Pregnant or breastfeeding. 2. An active Central nervous system (CNS) infection, demyelinating disease, or degenerative neurological disease. 3. History of psychogenic nonepileptic seizures. 4. Any disease or condition (other than TSC) at the initial visit that could compromise the hematologic, cardiovascular (including any cardiac conduction defect), pulmonary, renal, gastrointestinal, or hepatic systems; or other conditions that might interfere with the absorption, distribution, metabolism, or excretion of the IP, or would place the participant at increased risk or interfere with the assessment of safety/efficacy. This may include any illness in the past 4 weeks which in the opinion of the investigator may affect seizure frequency. 5. Unwillingness to avoid excessive alcohol use or cannabis use throughout the study. 6. Have active suicidal plan/intent or have had active suicidal thoughts in the past 6 months or a suicide attempt in the past 6 months. 7. Known sensitivity or allergy to any component in the IP(s), progesterone, or other related steroid compounds. 8. Exposed to any other investigational drug (except for GNX in Study 1042-TSC-2001 or Study 1042-TSC-3001) or investigational device within 30 days or fewer than 5 half-lives prior to Visit 1 (first visit of the OLE). For therapies in which half-life cannot be readily established, the Sponsor's medical monitor should be consulted.

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Conditions

The condition(s) this trial relates to.

tuberous sclerosis tuberous sclerosis 1

As listed by the trial registrant

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

Tuberous sclerosis complex

Contacts and locations

Locations

  • Alfred Health

    Melbourne, VIC 3004, Australia

  • Arkansas Children's Research Institute

    Little Rock, Arkansas, 72202, United States

  • Atrium Health/Levine Children's Hospital

    Charlotte, North Carolina, 28207, United States

  • Austin Health

    Heidelberg, VIC 3084, Australia

  • BC Children's Hospital

    Vancouver, V6H 3V4, Canada

  • Beijing Children Hospital, Capital Medical University

    Beijing, 100045, China

  • Boston Children's Hospital, Harvard Medical School

    Boston, Massachusetts, 02115, United States

  • Bristol Royal Hospital for Children

    Bristol, BS2 8AE, United Kingdom

  • CHU Sainte-Justine

    Montreal, H3T 1C5, Canada

  • Child Neurology Consultants of Austin (CNCA)

    Austin, Texas, 78757, United States

  • Children's Hospital Colorado

    Aurora, Colorado, 80045, United States

  • Children's Hospital of Orange County

    Orange, California, 92868, United States

  • Children's Mercy Hospital

    Kansas City, Missouri, 64108, United States

  • Chinese PLA General Hospital

    Beijing, 100853, China

  • Cincinnati Children's Hospital Medical Center

    Cincinnati, Ohio, 45229, United States

  • Duke University Medical Center

    Durham, North Carolina, 27712, United States

  • Epilepsie-Zentrum Bethel - Krankenhaus Mara

    Bielefeld, 33617, Germany

  • Epilepsiezentrum Kleinwachau gGmbH

    Radeberg, 1454, Germany

  • First Hospital of Jilin University

    Jilin City, 130021, China

  • Gemeinschaftskrankenhaus Herdecke

    Herdecke, 58313, Germany

  • Hospital Infantil Universitario Niño Jesús

    Madrid, 28009, Spain

  • Hospital Regional Universitario de Málaga

    Málaga, 29010, Spain

  • Hospital Ruber International

    Madrid, 28034, Spain

  • Hospital Sant Joan de Déu

    Barcelona, 8950, Spain

  • Hospital Universitario y Politécnico La Fe

    Valencia, 46026, Spain

  • Hospital de la Santa Creu i Sant Pau

    Barcelona, 8025, Spain

  • Hôpital Sud

    Rennes, 35000, France

  • Hôtel Dieu de Montréal - CHUM

    Montreal, H2X 0C2, Canada

  • Le Bonheur Children's Hospital

    Memphis, Tennessee, 38103, United States

  • Mayo Clinic - Rochester

    Rochester, Minnesota, 55905, United States

  • McGovern Medical School at the University of Texas Health Science Center

    Houston, Texas, 77030, United States

  • Nemours Children's Hospital - Delaware Valley

    Wilmington, Delaware, 19803, United States

  • Pediatric Neurology and Muscular Diseases Unit - University of Genoa

    Genova, 16147, Italy

  • Peking University First Hospital

    Beijing, 100034, China

  • Policlinico Umberto I

    Rome, 00185, Italy

  • Rady Children's Hospital - San Diego

    San Diego, California, 92123, United States

  • Royal Brisbane and Women's Hospital

    Herston, QLD 4029, Australia

  • Royal Melbourne Hospital

    Parkville, VIC 3050, Australia

  • Schneider Children´s Medical Center

    Petah Tikva, 4920235, Israel

  • Seattle Children's Hospital

    Seattle, Washington, 98105, United States

  • Sheffield Children's Hospital

    Sheffield, S10 2TH, United Kingdom

  • The Affiliated Hospital of Guizhou Medical University

    Beijing, 550004, China

  • The Hospital for Sick Children

    Toronto, M5G 1X8, Canada

  • Toronto Western Hospital

    Toronto, M5T 2S8, Canada

  • UCLA Mattel Children's Hospital, TSC Center

    Los Angeles, California, 90095, United States

  • University Hospital Bonn

    Bonn, 53127, Germany

  • University Hospital of Lyon

    Bron, 69229, France

  • University of North Carolina at Chapel Hill

    Chapel Hill, North Carolina, 27599, United States

  • University of Rochester Medical Center

    Rochester, New York, 14642, United States

  • University of Strasbourg

    Strasbourg, 67084, France

  • University of Texas Southwestern Medical Center

    Dallas, Texas, 75207, United States

  • University of Utah Health Care-Pediatric Neurology

    Salt Lake City, Utah, 84108, United States

  • Universitäts Krankenhaus Freiburg

    Freiburg im Breisgau, 79106, Germany

  • ZNN - Epilepsiezentrum Frankfurt am Main

    Frankfurt, 60528, Germany

More trials for these conditions

Other studies related to the condition(s) this trial covers.