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Tuberous Sclerosis ComplexPhase 3NCT05323734

Adjunctive GNX Treatment Compared With Placebo in Children and Adults With TSC-related Epilepsy

A Phase 3 study testing Ganaxolone in people with Tuberous Sclerosis Complex, run by Marinus Pharmaceuticals at 63 sites in 10 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
Search all trials

Trial summary

This is a Phase 3, global, double-blind, randomized, placebo-controlled study of adjunctive GNX treatment in children and adults with TSC-related epilepsy. The study consists of a 4-week prospective Baseline phase, defined as the first 28 days following screening, followed by a double-blind phase consisting of a… Read more about this study

Who is it for?
Ages 1 year to 65 years
Conditions the study is for
Tuberous Sclerosis Complex
What is being tested?
Ganaxolone
Type of treatment
Pill or oral medicine
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
129 participants
Where
63 sitesin 10 countries
Study dates
1 Apr 2022to 14 Oct 2024
Phase
Phase 3
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 1 year to 65 years
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • Clinical or mutational diagnosis of TSC consistent with:
  • Molecular confirmation of a pathogenic mutation in TSC1 or TSC2. A pathogenic mutation is defined as a mutation that clearly prevents protein synthesis and/or inactivates the function of the TSC1 or TSC2 proteins (eg, nonsense mutation or frameshift mutations, large genomic deletions) or is a missense mutation whose effect on protein function has been established by functional assessment. The Principal investigator (PI) or designee must review the results of the genetic analysis and confirm that the causal relationship to the epilepsy syndrome is likely. OR
  • Clinical diagnosis of definite TSC which includes 2 major features or 1 major feature with ≥ 2 minor features.
  • Male or female participants aged 1 through 65 years, inclusive. For Europe (EU), Middle East and North Africa (MENA), and Oceania (OC) Male or Female participants aged 2 through 65 years, inclusive.
  • Participant/parent(s) or LAR(s) willing 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 nor able to provide written informed consent based on age, developmental stage, intellectual capacity, or other factors, parent(s)/LAR(s) must provide consent for study participation, if appropriate.
  • Failure to control seizures despite appropriate trial of 2 or more Anti-seizure medication (ASMs) at therapeutic doses and for adequate duration of treatment per PI judgment.
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  • Participants should be on a stable regimen of ASMs (including moderate or strong inducer or inhibitor ASM eg, carbamazepine, phenytoin, etc.) at therapeutic doses for ≥ 28 days prior to the screening visit, and without a foreseeable change in dosing for the duration of the study. (Note: Minor dose adjustment to address tolerability and safety events may be allowed on case-by-case basis and it should be discussed with the study medical monitor.)
  • A history of at least 8 countable seizures per month in the 2 months prior to screening with no more than 1 seizure free week in each month. This includes seizures of any kind.
  • Have at least 8 primary endpoint seizures in the first 28 days following the screening visit.
  • The primary endpoint seizure types are defined as the following:
  • focal motor seizures without impairment of consciousness or awareness
  • focal seizures with impairment of consciousness or awareness with motor features
  • focal seizures evolving to bilateral, tonic-clonic seizures
  • generalized motor seizures including tonic-clonic, bilateral tonic, bilateral clonic, or atonic/drop seizures.
  • Seizures that do not count towards the primary endpoint include:
  • Focal or generalized nonmotor seizures (eg, absence seizures or focal nonmotor seizures with or without impairment of awareness)
  • Infantile or epileptic spasms
  • Myoclonic seizures.
  • Participants with surgically implanted vagal nerve stimulator (VNS) will be allowed to enter the study provided that all of the following conditions are met:
  • The VNS has been in place for ≥ 6 months prior to the screening visit.
  • The settings must have remained constant for 3 months prior to the screening visit and are expected to remain constant throughout the study.
  • The battery is expected to last for the duration of the study.
  • Parent(s)/caregiver(s)/LAR(s) or the participant, as appropriate, is (are) willing and able to maintain an accurate and complete daily seizure eDiary for the duration of the study.
  • Willing and able to take IP (suspension) as directed with food (TID).
  • 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 screening and Baseline visits.Childbearing potential is defined as a female who is biologically capable of becoming pregnant. A medically acceptable method of birth control includes 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.
  • 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.

Who cannot join

  • Previous exposure to GNX.
  • Pregnant or breastfeeding.
  • Participants who have been taking felbamate for less than 1 year prior to screening.
  • Participants taking cannabidiol (CBD) preparations other than Epidiolex.
  • A positive result on plasma drug screen for CBD or tetrahydrocannabinol (THC) at Visit 1 (screening), with the exception of results that are fully explained by Epidiolex, which can be adjusted by the investigator in the event of any Adverse events (AEs).
  • Concurrent use of adrenocorticotropic hormone (ACTH), prednisone or other glucocorticoid is not permitted, nor use of the strong inducers of cytochrome P450 3A4 (CYP3A4), rifampin and St John's Wort. Participants on ACTH, prednisone, or other systemically (non-inhaled or topical) administered steroids should be off the product > 28 days prior to screening. Rifampin and St John's Wort must be discontinued at least 28 days before Visit 2, study drug initiation.
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  • Note:
  • Use of concomitant intranasal or pro re nata (PRN) topical steroids for dermatologic reactions and allergic rhinitis are allowed during the study.
  • This exclusion criterion does not prohibit the use of approved ASMs.
  • Changes in any chronic medications within the 4 weeks prior to the screening visit. All chronic concomitant medications must be relatively stable in dose for at least 4 weeks prior to the screening visit unless otherwise noted. Small dose adjustment to manage tolerability and safety events is permitted and should be discussed with the study medical monitor.
  • Participants who have epilepsy surgery planned during the study or who have undergone surgery for epilepsy within the 6 months prior to screening.
  • An active central nervous system (CNS) infection, demyelinating disease, degenerative neurological disease, or CNS disease deemed progressive as evaluated by brain magnetic resonance imaging (MRI). This includes tumor growth which in the opinion of the investigator could affect primary endpoint seizure control.
  • Any disease or condition (medical or surgical; other than TSC) at the screening visit that might 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.
  • Hepatic impairment sufficient to affect participant safety, or an aspartate aminotransferase (AST)/ serum glutamic oxaloacetic transaminase (SGOT) or alanine aminotransferase (ALT)/ serum glutamic pyruvic transaminase (SGPT) > 3 × the upper limit of normal (ULN) at screening or Baseline visits and confirmed by a repeat test.
  • Biliary impairment sufficient to affect participant safety, or total bilirubin levels > 1.5 × ULN at screening or Baseline visit and confirmed by a repeat test. In cases of Gilbert's Syndrome, resulting in stable levels of total bilirubin greater than ULN, the medical monitor can determine if a protocol exception can be made
  • Renal impairment sufficient to affect participant safety, or estimated glomerular filtration rate (eGFR) < 30 milliliter per minute (mL/min) (calculated using the Cockcroft-Gault formula or Pediatric GFR calculator or Bedside Schwartz), will be excluded from study entry or will be discontinued if the criterion is met post Baseline. Cases of temporary renal insufficiency should be discussed with the medical monitor to determine the participant's study continuation.
  • Exposed to any other investigational drug or investigational device within 30 days or fewer than 5 half-lives prior to the screening visit. For therapies in which half-life cannot be readily established, the Sponsor's Medical Monitor should be consulted.
  • Unwillingness to avoid excessive alcohol use throughout the study.
  • Have active suicidal plan/intent, active suicidal thoughts or a suicide attempt in the past 6 months.
  • Known sensitivity or allergy to any component in the IP(s), progesterone, or other related steroid compounds.
  • Participants deprived of their liberty by a judicial or administrative decision, or for psychiatric treatment, or participants admitted to a health or social services facility for purposes other than research.
  • Participants receiving traditional Chinese medicine therapies within the prior 28 days of the screening.

Trial locations and contacts

63 sites in 10 countries.

63 sites in 10 countries

United States23 sites

  • Childrens Hospital Colorado

    Aurora, Colorado, 80045

  • Child Neurology Consultants of Austin (CNCA)

    Austin, Texas, 78757

  • Mid-Atlantic Epilepsy and Sleep Center

    Bethesda, Maryland, 20817

  • University of North Carolina at Chapel Hill

    Chapel Hill, North Carolina, 27599

  • Atrium Health/Levine Children's Hospital

    Charlotte, North Carolina, 28207

  • Cincinnati Children's Hospital Medical Center

    Cincinnati, Ohio, 45229

  • University of Texas Southwestern Medical Center

    Dallas, Texas, 75207

  • Duke University Medical Center

    Durham, North Carolina, 27712

  • University of Florida Gainesville

    Gainesville, Florida, 32608

  • Penn State Children's Hospital

    Hershey, Pennsylvania, 17033

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

    Houston, Texas, 77030

  • Nemours Children's Health

    Jacksonville, Florida, 32207

  • Children's Mercy Hosptial

    Kansas City, Missouri, 64108

  • Arkansas Children's Research Institute

    Little Rock, Arkansas, 72202

  • UCLA Mattel Children's Hospital, TSC Center

    Los Angeles, California, 90095

  • Le Bonheur Children's Hospital

    Memphis, Tennessee, 38103

  • Children's Hospital of Orange County

    Orange, California, 92868

  • Mayo Clinic - Rochester

    Rochester, Minnesota, 55905

  • University of Rochester Medical Center

    Rochester, New York, 14642

  • University of Utah Health Care-Pediatric Neurology

    Salt Lake City, Utah, 84108

  • University of California, San Diego

    San Diego, California, 92123

  • Seattle Children's Hospital

    Seattle, Washington, 98105

  • Nemours Children's Hospital - Delaware Valley

    Wilmington, Delaware, 19803

Other trials in United States

China7 sites

  • Beijing Children Hospital, Capital Medical University

    Beijing, Xicheng District, 100045

  • Peking University First Hospital

    Beijing, 100034

  • Chinese PLA General Hospital

    Beijing, 100080

  • First Hospital of Jilin University

    Changchun, Jilin, 130028

  • Chengdu's Women and Children's Central Hospital

    Chengdu, 610000

  • The Affiliated Hospital of Guizhou Medical University

    Guiyang, Yunyan District, 550004

  • Jiangxi Provincial Children's Hospital

    Jiangxi, Nanchang City, 330000

Other trials in China

Germany6 sites

  • Epilepsie-Zentrum Bethel - Krankenhaus Mara

    Bielefeld, 33617

  • University Hospital Bonn

    Bonn, 53127

  • ZNN - Epilepsiezentrum Frankfurt am Main

    Frankfurt, 60528

  • Universitäts Krankenhaus Freiburg

    Freiburg im Breisgau, 79106

  • Gemeinschaftskrankenhaus Herdecke

    Herdecke, 58313

  • Epilepsiezentrum Kleinwachau gGmbH

    Radeberg, 01454

Other trials in Germany

Spain6 sites

  • Hospital Universitari Vall d'Hebron

    Barcelona, 08035

  • Hospital Sant Joan de Déu

    Barcelona, 08950

  • Hospital Infantil Universitario Niño Jesús

    Madrid, 28009

  • Hospital Ruber International

    Madrid, 28034

  • Hospital Regional Universitario de Málaga

    Málaga, 29010

  • Hospital Universitario y Politécnico La Fe

    Valencia, 46026

Other trials in Spain

Canada5 sites

  • Hôtel Dieu de Montréal - CHUM

    Montreal, H2X 0C2

  • CHU Sainte-Justine

    Montreal, H3T 1C5

  • The Hospital for Sick Children

    Toronto, M5G 1X8

  • Toronto Western Hospital

    Toronto, M5T 2S8

  • BC Children's Hospital

    Vancouver, V6H 3V4

Other trials in Canada

Australia4 sites

  • Austin Health

    Heidelberg, 3084

  • Royal Brisbane and Women's Hospital

    Herston, Queensland, 4029

  • Alfred Health

    Melbourne, 3004

  • Royal Melbourne Hospital

    Parkville, 3050

Other trials in Australia

United Kingdom4 sites

  • Bristol Royal Hospital for Children

    Bristol, BS2 8AE

  • NHS acute tertiary referral centre, John Radcliffe Hospital

    Oxford, OX3 9DU

  • Salford Royal Hospital

    Salford, M6 8HD

  • Sheffield Children's Hospital

    Sheffield, S10 2TH

Other trials in United Kingdom

France3 sites

  • University Hospital of Lyon

    Bron, 69229

  • University Hospital of Rennes

    Rennes, 35033

  • University of Strasbourg

    Strasbourg, 67084

Other trials in France

Italy3 sites

  • Department of Neurology and Sense Organs, AOU Policlinico di Bari

    Bari, 1170124

  • Pediatric Neurology and Muscular Diseases Unit - University of Genoa

    Genova, 16147

  • Policlinico Umberto I

    Rome, 00185

Other trials in Italy

Israel2 sites

  • Schneider Children´s Medical Center

    Petah Tikva, 4920235

  • Sheba Medical Center

    Tel HaShomer, 52621

Other trials in Israel

About this study

Official title: A Phase 3, Double-blind, Randomized, Placebo-controlled Trial of Adjunctive Ganaxolone (GNX) Treatment in Children and Adults With Tuberous Sclerosis Complex (TSC)-Related Epilepsy (TrustTSC)

This is a Phase 3, global, double-blind, randomized, placebo-controlled study of adjunctive GNX treatment in children and adults with TSC-related epilepsy. The study consists of a 4-week prospective Baseline phase, defined as the first 28 days following screening, followed by a double-blind phase consisting of a 4-week titration period (Day 1 to Day 28) and a 12-week maintenance period (Day 29 to Week 16).

Being tested
Ganaxolone, Placebo
Keywords
Tuberous Sclerosis Complex-Related Epilepsy, Ganaxolone, Adjunctive
Medical subject headings
Tuberous Sclerosis

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • GanaxoloneDrug

    GNX will be administered

  • PlaceboDrug

    Placebo will be administered

Study groups in NCT05323734
Study groupRoleWhat they receive
Ganaxolone (GNX)Experimentaloral suspension, 3 times a day (TID)
Receives: Drug: Ganaxolone
Placebo matching GNXPlacebo comparatororal suspension, TID
Receives: Drug: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Percent Change From Baseline in 28-day Seizure Frequency for Primary Seizure Type During Double Blind Period

    Primary seizures include atonic/drop, bilateral clonic, bilateral tonic, focal motor with altered awareness, focal motor with intact awareness, focal to bilateral tonic-clonic seizures, focal with hypotonia impaired awareness, and generalized tonic-clonic. Seizure frequency was calculated as the total number of seizures divided by the number of days with seizure data in the period, multiplied by 28. Percent change from Baseline in 28-day seizure frequency was calculated as follows for each participant: post-baseline 28-day seizure frequency minus baseline 28-day seizure frequency whole divided by baseline 28-day seizure frequency and multiplied by 100.

    Measured: Baseline (Day 1), Day 28

Secondary outcomes (3)

  1. Number of Participants Who Were Considered as Treatment Responders During Double Blind Period

    Treatment responders are defined as those participants with ≥ 50% reduction from Baseline in primary seizure type frequency during the given period.

    Measured: Up to 16 weeks

  2. Number of Responders to Clinical Global Impression of Improvement (CGI-I) Scale as Assessed by Parent/Caregiver

    The CGI-I is a 7-point Likert scale that the parent(s)/caregiver(s)/ legally authorized representative (LAR) and clinician uses to rate the change in overall seizure control, behavior, safety, and tolerability after initiation of the IP relative to baseline (prior to treatment with the IP). The participant was rated as follows: 1 - very much improved, 2 - much improved, 3 - minimally improved, 4 - no change, 5 - minimally worse, 6 - much worse, and 7 - very much worse. Higher scores indicated worse condition. Number of responders to each score on the scale has been presented.

    Measured: Baseline (Day 1) through 16 weeks

  3. Number of Responders to Clinical Global Impression of Improvement (CGI-I) Scale as Assessed by Clinician

    The CGI-I is a 7-point Likert scale that the parent(s)/caregiver(s)/ LAR and clinician uses to rate the change in overall seizure control, behavior, safety, and tolerability after initiation of the IP relative to baseline (prior to treatment with the IP). The participant was rated as follows: 1 - very much improved, 2 - much improved, 3 - minimally improved, 4 - no change, 5 - minimally worse, 6 - much worse, and 7 - very much worse. Number of responders to each score on the scale has been presented.

    Measured: Baseline (Day 1) through 16 weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted12 Apr 2022
  2. Study start1 Apr 2022
  3. Primary completion9 Sep 2024
  4. Study completion14 Oct 2024

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Triple blind
Who is masked
Participant, Care provider, Investigator
Enrolment
129 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05323734
Sponsor's protocol ID
1042-TSC-3001
Status last verified
Jun 2025
First submitted
21 Mar 2022
Last updated
11 Jul 2025
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Ganaxolone in people with Tuberous Sclerosis Complex, run by Marinus Pharmaceuticals at 63 sites in 10 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 26 inclusion and 22 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 11 Jul 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 63 sites in United States, China, Germany, Spain, Canada and 5 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 11 Jul 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Percent Change From Baseline in 28-day Seizure Frequency for Primary Seizure Type During Double Blind Period (time frame: Baseline (Day 1), Day 28). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05323734.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 11 Jul 2025.

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