Experimental seizure drug tested in rare genetic disorder
NCT ID NCT05604170
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.
Disclaimer
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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.
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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Alfred Health
Melbourne, VIC 3004, Australia
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Arkansas Children's Research Institute
Little Rock, Arkansas, 72202, United States
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Atrium Health/Levine Children's Hospital
Charlotte, North Carolina, 28207, United States
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Austin Health
Heidelberg, VIC 3084, Australia
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BC Children's Hospital
Vancouver, V6H 3V4, Canada
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Beijing Children Hospital, Capital Medical University
Beijing, 100045, China
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Boston Children's Hospital, Harvard Medical School
Boston, Massachusetts, 02115, United States
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Bristol Royal Hospital for Children
Bristol, BS2 8AE, United Kingdom
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CHU Sainte-Justine
Montreal, H3T 1C5, Canada
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Child Neurology Consultants of Austin (CNCA)
Austin, Texas, 78757, United States
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Children's Hospital Colorado
Aurora, Colorado, 80045, United States
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Children's Hospital of Orange County
Orange, California, 92868, United States
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Children's Mercy Hospital
Kansas City, Missouri, 64108, United States
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Chinese PLA General Hospital
Beijing, 100853, China
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Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229, United States
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Duke University Medical Center
Durham, North Carolina, 27712, United States
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Epilepsie-Zentrum Bethel - Krankenhaus Mara
Bielefeld, 33617, Germany
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Epilepsiezentrum Kleinwachau gGmbH
Radeberg, 1454, Germany
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First Hospital of Jilin University
Jilin City, 130021, China
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Gemeinschaftskrankenhaus Herdecke
Herdecke, 58313, Germany
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Hospital Infantil Universitario Niño Jesús
Madrid, 28009, Spain
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Hospital Regional Universitario de Málaga
Málaga, 29010, Spain
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Hospital Ruber International
Madrid, 28034, Spain
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Hospital Sant Joan de Déu
Barcelona, 8950, Spain
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Hospital Universitario y Politécnico La Fe
Valencia, 46026, Spain
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Hospital de la Santa Creu i Sant Pau
Barcelona, 8025, Spain
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Hôpital Sud
Rennes, 35000, France
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Hôtel Dieu de Montréal - CHUM
Montreal, H2X 0C2, Canada
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Le Bonheur Children's Hospital
Memphis, Tennessee, 38103, United States
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Mayo Clinic - Rochester
Rochester, Minnesota, 55905, United States
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McGovern Medical School at the University of Texas Health Science Center
Houston, Texas, 77030, United States
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Nemours Children's Hospital - Delaware Valley
Wilmington, Delaware, 19803, United States
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Pediatric Neurology and Muscular Diseases Unit - University of Genoa
Genova, 16147, Italy
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Peking University First Hospital
Beijing, 100034, China
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Policlinico Umberto I
Rome, 00185, Italy
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Rady Children's Hospital - San Diego
San Diego, California, 92123, United States
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Royal Brisbane and Women's Hospital
Herston, QLD 4029, Australia
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Royal Melbourne Hospital
Parkville, VIC 3050, Australia
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Schneider Children´s Medical Center
Petah Tikva, 4920235, Israel
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Seattle Children's Hospital
Seattle, Washington, 98105, United States
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Sheffield Children's Hospital
Sheffield, S10 2TH, United Kingdom
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The Affiliated Hospital of Guizhou Medical University
Beijing, 550004, China
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The Hospital for Sick Children
Toronto, M5G 1X8, Canada
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Toronto Western Hospital
Toronto, M5T 2S8, Canada
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UCLA Mattel Children's Hospital, TSC Center
Los Angeles, California, 90095, United States
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University Hospital Bonn
Bonn, 53127, Germany
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University Hospital of Lyon
Bron, 69229, France
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University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, 27599, United States
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University of Rochester Medical Center
Rochester, New York, 14642, United States
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University of Strasbourg
Strasbourg, 67084, France
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University of Texas Southwestern Medical Center
Dallas, Texas, 75207, United States
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University of Utah Health Care-Pediatric Neurology
Salt Lake City, Utah, 84108, United States
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Universitäts Krankenhaus Freiburg
Freiburg im Breisgau, 79106, Germany
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ZNN - Epilepsiezentrum Frankfurt am Main
Frankfurt, 60528, Germany