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

Primary Purpose

Tuberous Sclerosis Complex

Status
Recruiting
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
Ganaxalone
Placebo
Sponsored by
Marinus Pharmaceuticals
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Tuberous Sclerosis Complex focused on measuring Tuberous Sclerosis Complex-Related Epilepsy, Ganaxolone, Adjunctive

Eligibility Criteria

1 Year - 65 Years (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  1. Clinical or mutational diagnosis of TSC consistent with (Northrup and Krueger, 2013):

    1. 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 PI or designee must review the results of the genetic analysis and confirm that the causal relationship to the epilepsy syndrome is likely. OR
    2. Clinical diagnosis of definite TSC which includes 2 major features or 1 major feature with ≥ 2 minor features.
  2. Male or female participants aged 1 through 65 years, inclusive.
  3. Participant/parent or LAR 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.
  4. Assent for participants over 7 years of age should be obtained if appropriate.
  5. Failure to control seizures despite appropriate trial of 2 or more AEDs at therapeutic doses and for adequate duration of treatment per PI judgment.
  6. Participants should be on a stable regimen of AEDs (including moderate or strong inducer or inhibitor anti-seizure medications 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.)
  7. 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.
  8. Have an average of at least 2 primary endpoint seizures per week in the 28 days following the screening visit.

    The primary endpoint seizure types are defined as the following:

    1. focal motor seizures without impairment of consciousness or awareness
    2. focal seizures with impairment of consciousness or awareness with motor features
    3. focal seizures evolving to bilateral, tonic-clonic seizures
    4. generalized motor seizures including tonic-clonic, bilateral tonic, bilateral clonic, or atonic/drop seizures.

    Seizures that do not count towards the primary endpoint include:

    1. Focal aware seizures without motor features
    2. Focal and generalized nonmotor seizures (eg, absence or focal nonmotor seizures with or without impairment of awareness)
    3. Infantile or epileptic spasms
    4. Myoclonic seizures.
  9. Participants with surgically implanted VNS will be allowed to enter the study provided that all of the following conditions are met:

    1. The VNS has been in place for ≥ 1 year prior to the screening visit.
    2. The settings must have remained constant for 3 months prior to the screening visit and are expected to remain constant throughout the study.
    3. The battery is expected to last for the duration of the study.
  10. Parent/caregiver or the participant, as appropriate, is able and willing to maintain an accurate and complete daily seizure eDiary for the duration of the study.
  11. Able and willing to take IP (suspension) as directed with food TID.
  12. Sexually active WOCBP must be using a medically acceptable method of birth control and have a negative quantitative serum β-HCG test collected at the initial screening visit.

    Childbearing potential is defined as a female who is biologically capable of becoming pregnant. A medically acceptable method of birth control includes intrauterine devices in place for 1 month prior to the screening visit, surgical sterilization, or adequate double barrier methods (eg, diaphragm or condom and foam). An oral contraceptive alone is not considered adequate for the purpose of this study. Use of oral contraceptives in combination with another method (eg, a spermicidal cream) is acceptable. In participants who are not sexually active, abstinence is an acceptable method of birth control.

  13. Male participants must agree to take all necessary measures to avoid causing pregnancy in their sexual partners during the study and for 30 days after the last dose of IP. Medically acceptable contraceptives include surgical sterilization (such as a vasectomy) and a condom used with a spermicidal gel or foam.

Exclusion Criteria:

  1. Previous exposure to GNX.
  2. Pregnant or breastfeeding.
  3. Participants who have been taking felbamate for less than 1 year prior to screening.
  4. Participants taking CBD preparations other than Epidiolex.
  5. A positive result on plasma drug screen for CBD or THC at Visit 1 (screening), with the exception of results that are fully explained by Epidiolex, which is being prescribed and managed by the investigator.
  6. Concurrent use of ACTH, prednisone or other glucocorticoid is not permitted, nor use of the strong inducers of 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.

    Note:

    1. Use of concomitant intranasal or PRN topical steroids for dermatologic reactions and allergic rhinitis are allowed during the study.
    2. This exclusion criterion does not prohibit the use of approved AEDs
  7. 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.
  8. Participants who have epilepsy surgery planned during the study or who have undergone surgery for epilepsy within the 6 months prior to screening.
  9. An active CNS infection, demyelinating disease, degenerative neurological disease, or CNS disease deemed progressive as evaluated by brain imaging (MRI). This includes tumor growth which in the opinion of the investigator could affect primary endpoint seizure control.
  10. 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.
  11. An AST/SGOT or ALT/SGPT > 3 × the ULN at screening or baseline visits and confirmed by a repeat test.
  12. Biliary impairment sufficient to affect patient 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
  13. Renal impairment sufficient to affect patient safety, or eGFR < 30 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 (Levey et al, 2006). Cases of temporary renal insufficiency should be discussed with the medical monitor to determine the participant's study continuation
  14. 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.
  15. Unwillingness to avoid excessive alcohol use throughout the study.
  16. Have active suicidal plan/intent, active suicidal thoughts or a suicide attempt in the past 6 months.
  17. Known sensitivity or allergy to any component in the IP(s), progesterone, or other related steroid compounds.

Sites / Locations

  • Barrow Neurological Institute at Phoenix Children's HospitalRecruiting
  • Arkansas Children's Research InstituteRecruiting
  • UCLA Mattel Children's Hospital, TSC CenterRecruiting
  • Children's Hospital of Orange CountyRecruiting
  • Childrens Hospital Colorado
  • Nemours Children's Hospital - Delaware ValleyRecruiting
  • University of Florida GainesvilleRecruiting
  • NW FL Clinical Research Group, LLCRecruiting
  • Nicklaus Children's HospitalRecruiting
  • Orlando HealthRecruiting
  • Comprehensive Neurology ClinicRecruiting
  • Children's Healthcare of AtlantaRecruiting
  • Ann & Robert H. Lurie Children's Hospital of ChicagoRecruiting
  • Mid Atlantic Epilepsy & Sleep CenterRecruiting
  • Boston Children's Hospital, Harvard Medical SchoolRecruiting
  • Children's Hospital of Michigan Central Michigan UniversityRecruiting
  • Mayo Clinic - RochesterRecruiting
  • University of Missouri Child Health
  • Children's Mercy HosptialRecruiting
  • TSC Clinic at Northeast Regional Epilepsy GroupRecruiting
  • Institute of Neurology and Neurosurgery at Saint BarnabasRecruiting
  • University of Rochester Medical Center
  • University of North Carolina at Chapel HillRecruiting
  • Atrium Health/Levine Children's HospitalRecruiting
  • Duke University Medical CenterRecruiting
  • Cincinnati Children's Hospital Medical CenterRecruiting
  • Penn State Children's HospitalRecruiting
  • Children's Hospital of Philadelphia
  • Medical University of South CarolinaRecruiting
  • Le Bonheur Children's HospitalRecruiting
  • Child Neurology Consultants of Austin (CNCA)Recruiting
  • University of Texas Southwestern Medical CenterRecruiting
  • McGovern Medical School at the University of Texas Health Science CenterRecruiting
  • University of Utah Health Care-Pediatric NeurologyRecruiting
  • Seattle Children's HospitalRecruiting
  • Hôtel Dieu de Montréal - CHUMRecruiting
  • CHU Sainte-JustineRecruiting
  • The Hospital for Sick ChildrenRecruiting
  • Toronto Western HospitalRecruiting
  • BC Children's HospitalRecruiting
  • University Hospital of LyonRecruiting
  • University Hospital of LilleRecruiting
  • Robert-Debré HospitalRecruiting
  • University Hospital of RennesRecruiting
  • University of StrasbourgRecruiting
  • Epilepsie-Zentrum Bethel - Krankenhaus MaraRecruiting
  • University Hospital BonnRecruiting
  • ZNN - Epilepsiezentrum Frankfurt am Main
  • Universitäts Krankenhaus FreiburgRecruiting
  • Gemeinschaftskrankenhaus HerdeckeRecruiting
  • Epilepsiezentrum Kleinwachau gGmbH
  • Soroka University Medical CenterRecruiting
  • Hadassah Medical CenterRecruiting
  • Schneider Children´s Medical CenterRecruiting
  • Sheba Medical CenterRecruiting
  • Tel-Aviv Sourasky Medical CenterRecruiting
  • Department of Neurology and Sense Organs, AOU Policlinico di Bari
  • Azienda Ospedaliero-Universitaria Meyer
  • Pediatric Neurology and Muscular Diseases Unit - University of Genoa
  • Children's Hospital Bambino Gesù
  • Policlinico Umberto I
  • Hospital de la Santa Creu i Sant PauRecruiting
  • Hospital Universitari Vall d'HebronRecruiting
  • Hospital Sant Joan de DéuRecruiting
  • Hospital Infantil Universitario Niño JesúsRecruiting
  • Hospital Ruber InternationalRecruiting
  • Hospital Regional Universitario de MálagaRecruiting
  • Hospital Universitario y Politécnico La FeRecruiting
  • Royal Aberdeen Children's Hospital, NHS GrampianRecruiting
  • Bristol Royal Hospital for ChildrenRecruiting
  • Leeds General InfirmaryRecruiting
  • NHS acute tertiary referral centre, John Radcliffe HospitalRecruiting
  • Salford Royal HospitalRecruiting
  • Sheffield Children's HospitalRecruiting

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

Ganaxalone (GNX)

Placebo matching GNX

Arm Description

oral suspension, 3 times a day (TID)

oral suspension, TID

Outcomes

Primary Outcome Measures

Double-blind phase: Percent change from Baseline in 28-day seizure frequency during titration and maintenance period
Seizure frequency will be calculated as the total number of seizures divided by the number of days with seizure data, multiplied by 28.

Secondary Outcome Measures

Double-blind phase: Percent change from Baseline in 28-day seizure frequency during maintenance period
Double-blind phase: Number of participants who will be considered as treatment responders during titration and maintenance phase
Treatment responders are defined as those participants with ≥ 50% reduction from Baseline in seizure frequency
Double-blind phase: Number of participants who will be considered as treatment responders during maintenance phase
Double-blind phase: Number of Participants with Clinical Global Impression of Improvement (CGI-I) during titration and maintenance phase
The CGI-I is a 7-point Likert scale that the parent(s)/caregiver(s)/legally authorized representative (LAR)(s) and clinician uses to rate the change in overall seizure control, behavior, safety, and tolerability after initiation of the Investigational product (IP) relative to Baseline (prior to treatment with the IP). It was rated as: 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.
Double-blind phase: Change from Baseline in Anxiety, Depression, and Mood Scale (ADAMS) during titration and maintenance phase
The ADAMS is a rating scale designed to screen for anxiety and depression in individuals with intellectual disability. The 28-question scale is filled out by the parent(s)/caregiver(s)/LAR(s) and is based on the participant's behavior. Each question will be rated on 4-point Likert scale as follows: 0- "behavior has not occurred or is not a problem"; 1- "behavior occurs occasionally or is a mild problem"; 2- "behavior occurs quite often or is a moderate problem"; and 3- "behavior occurs a lot or is a severe problem". Higher scores indicated worse condition.
Double-blind phase: Change from Baseline in Short Form-36 (SF-36) during titration and maintenance phase
The SF-36 is a multi-purpose survey designed to capture participant or parent(s)/caregiver(s)/LAR(s) perceptions of own health and well-being. The SF-36 has 36 items grouped in 8 dimensions: physical functioning, physical and emotional limitations, social functioning, bodily pain, general, and mental health, which are the weighted sums of the questions in each section. Each scale is directly transformed into a 0-100 scale on the assumption that each question carries equal weight. Higher scores represent better health-related quality-of-life.
Double-blind phase: Change from Baseline in Pediatric Quality of Life Inventory - Family Impact Module (Peds-QL-FIM) during titration and maintenance phase
Peds-QL-FIM is designed to measure the impact of pediatric chronic health conditions on parents and the family. It composes of 6 scales measuring parent self-reported functioning: 1) Physical functioning (6 items), 2) Emotional functioning (5 items), 3) Social functioning (4 items), 4) Cognitive functioning (5 items), 5) Communication (3 items), 6) Worry (5 items), and in addition two scales measuring parent-reported family functioning: 7) Daily activities (3 items) and 8) Family relationships (5 items). Each of the items have five Likert response options: 0 = never a problem, 1 = almost never, 2 = sometimes, 3 = often, 4= almost always. Items are reverse-scored and linearly transformed to a 0-100 scale (0 = 100, 1 = 75, 2 = 50, 3 = 25, 4 = 0); higher scores indicate better functioning (less negative impact).
Double-blind phase: Change from Baseline in Epilepsy and Learning Disabilities Quality of Life (ELDQOL) Scale during titration and maintenance phase
The ELDQOL is a parent/caregiver/LAR-reported measure that examines seizure severity, seizure related injury, anti-seizure medication (ASM) side effects, behavior, mood, physical status, cognitive and social functioning, communication, overall health and quality of life, and family concerns. There are a total of 70 items, some of which are responded as 1=yes/2=no, some of which range 1-4 and others 1-5. For those items, higher is more severe. For mood-related items, however, the scoring is always/often/sometimes/never which are coded as 1-4 but may be good or bad, depending on the mood. Finally: there are qualitative questions with open-ended answers related to "were any questions difficult to answer" and "are there any other comments you would like to make". Each of the 70 items is characterized as its own measurement, and they are not aggregated or tabulated into an overall score.
Double-blind phase: Change from Baseline in the percentage of seizure-free day during titration and maintenance phase
Double-blind phase: Change from Baseline in Caregiver Global Impression of Change in Seizure Intensity/Duration (CGI-CSID) during titration and maintenance phase
The CGI-CSID is a 7-point Likert scale in which the parent(s)/caregiver(s)/LAR(s) assesses change in seizure intensity and/or duration after initiation of investigational product relative to Baseline (prior to treatment with investigational product). The scale ranges from 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 indicate worse condition
Double-blind phase: Number of participants with a ≥ 25% and ≥ 75% reduction from Baseline during titration and maintenance phase
Double-blind phase: Number of participants with a ≥ 25%, ≥ 50%, and ≥ 75% reduction from Baseline during maintenance phase
Double-blind phase: Number of participants with Responder Analysis using ≤ 0%, > 0% to < 25%, ≥ 25% to < 50%, ≥ 50% to < 75%, and ≥ 75% to 100% during titration and maintenance phase
Double-blind phase: Percent change in 28-day frequency of all seizures during titration and maintenance phase
Double-blind phase: Change from Baseline in percentage of Seizure-Free Days during titration and maintenance phase
Double-blind phase: Change from Baseline in longest Seizure-Free Interval during titration and maintenance phase

Full Information

First Posted
March 21, 2022
Last Updated
October 18, 2023
Sponsor
Marinus Pharmaceuticals
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1. Study Identification

Unique Protocol Identification Number
NCT05323734
Brief Title
Adjunctive GNX Treatment Compared With Placebo in Children and Adults With TSC-related Epilepsy
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)
Study Type
Interventional

2. Study Status

Record Verification Date
October 2023
Overall Recruitment Status
Recruiting
Study Start Date
April 1, 2022 (Actual)
Primary Completion Date
January 1, 2024 (Anticipated)
Study Completion Date
February 1, 2025 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Marinus Pharmaceuticals

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

Brief 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 4-week titration period (Day 1 to Day 28) and a 12-week maintenance period (Day 29 to Week 16).

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Tuberous Sclerosis Complex
Keywords
Tuberous Sclerosis Complex-Related Epilepsy, Ganaxolone, Adjunctive

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigator
Allocation
Randomized
Enrollment
128 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Ganaxalone (GNX)
Arm Type
Experimental
Arm Description
oral suspension, 3 times a day (TID)
Arm Title
Placebo matching GNX
Arm Type
Placebo Comparator
Arm Description
oral suspension, TID
Intervention Type
Drug
Intervention Name(s)
Ganaxalone
Intervention Description
GNX will be administered
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Placebo will be administered
Primary Outcome Measure Information:
Title
Double-blind phase: Percent change from Baseline in 28-day seizure frequency during titration and maintenance period
Description
Seizure frequency will be calculated as the total number of seizures divided by the number of days with seizure data, multiplied by 28.
Time Frame
Baseline (Day 1) through Week 16
Secondary Outcome Measure Information:
Title
Double-blind phase: Percent change from Baseline in 28-day seizure frequency during maintenance period
Time Frame
Baseline (Day 1) and Day 29 to Week 16
Title
Double-blind phase: Number of participants who will be considered as treatment responders during titration and maintenance phase
Description
Treatment responders are defined as those participants with ≥ 50% reduction from Baseline in seizure frequency
Time Frame
Up to 16 weeks
Title
Double-blind phase: Number of participants who will be considered as treatment responders during maintenance phase
Time Frame
Day 29 to Week 16
Title
Double-blind phase: Number of Participants with Clinical Global Impression of Improvement (CGI-I) during titration and maintenance phase
Description
The CGI-I is a 7-point Likert scale that the parent(s)/caregiver(s)/legally authorized representative (LAR)(s) and clinician uses to rate the change in overall seizure control, behavior, safety, and tolerability after initiation of the Investigational product (IP) relative to Baseline (prior to treatment with the IP). It was rated as: 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.
Time Frame
Up to 16 weeks
Title
Double-blind phase: Change from Baseline in Anxiety, Depression, and Mood Scale (ADAMS) during titration and maintenance phase
Description
The ADAMS is a rating scale designed to screen for anxiety and depression in individuals with intellectual disability. The 28-question scale is filled out by the parent(s)/caregiver(s)/LAR(s) and is based on the participant's behavior. Each question will be rated on 4-point Likert scale as follows: 0- "behavior has not occurred or is not a problem"; 1- "behavior occurs occasionally or is a mild problem"; 2- "behavior occurs quite often or is a moderate problem"; and 3- "behavior occurs a lot or is a severe problem". Higher scores indicated worse condition.
Time Frame
Baseline (Day 1) through Week 16
Title
Double-blind phase: Change from Baseline in Short Form-36 (SF-36) during titration and maintenance phase
Description
The SF-36 is a multi-purpose survey designed to capture participant or parent(s)/caregiver(s)/LAR(s) perceptions of own health and well-being. The SF-36 has 36 items grouped in 8 dimensions: physical functioning, physical and emotional limitations, social functioning, bodily pain, general, and mental health, which are the weighted sums of the questions in each section. Each scale is directly transformed into a 0-100 scale on the assumption that each question carries equal weight. Higher scores represent better health-related quality-of-life.
Time Frame
Baseline (Day 1) through 16 weeks
Title
Double-blind phase: Change from Baseline in Pediatric Quality of Life Inventory - Family Impact Module (Peds-QL-FIM) during titration and maintenance phase
Description
Peds-QL-FIM is designed to measure the impact of pediatric chronic health conditions on parents and the family. It composes of 6 scales measuring parent self-reported functioning: 1) Physical functioning (6 items), 2) Emotional functioning (5 items), 3) Social functioning (4 items), 4) Cognitive functioning (5 items), 5) Communication (3 items), 6) Worry (5 items), and in addition two scales measuring parent-reported family functioning: 7) Daily activities (3 items) and 8) Family relationships (5 items). Each of the items have five Likert response options: 0 = never a problem, 1 = almost never, 2 = sometimes, 3 = often, 4= almost always. Items are reverse-scored and linearly transformed to a 0-100 scale (0 = 100, 1 = 75, 2 = 50, 3 = 25, 4 = 0); higher scores indicate better functioning (less negative impact).
Time Frame
Baseline (Day 1) through 16 weeks
Title
Double-blind phase: Change from Baseline in Epilepsy and Learning Disabilities Quality of Life (ELDQOL) Scale during titration and maintenance phase
Description
The ELDQOL is a parent/caregiver/LAR-reported measure that examines seizure severity, seizure related injury, anti-seizure medication (ASM) side effects, behavior, mood, physical status, cognitive and social functioning, communication, overall health and quality of life, and family concerns. There are a total of 70 items, some of which are responded as 1=yes/2=no, some of which range 1-4 and others 1-5. For those items, higher is more severe. For mood-related items, however, the scoring is always/often/sometimes/never which are coded as 1-4 but may be good or bad, depending on the mood. Finally: there are qualitative questions with open-ended answers related to "were any questions difficult to answer" and "are there any other comments you would like to make". Each of the 70 items is characterized as its own measurement, and they are not aggregated or tabulated into an overall score.
Time Frame
Baseline (Day 1) through 16 weeks
Title
Double-blind phase: Change from Baseline in the percentage of seizure-free day during titration and maintenance phase
Time Frame
Baseline (Day 1) through 16 weeks
Title
Double-blind phase: Change from Baseline in Caregiver Global Impression of Change in Seizure Intensity/Duration (CGI-CSID) during titration and maintenance phase
Description
The CGI-CSID is a 7-point Likert scale in which the parent(s)/caregiver(s)/LAR(s) assesses change in seizure intensity and/or duration after initiation of investigational product relative to Baseline (prior to treatment with investigational product). The scale ranges from 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 indicate worse condition
Time Frame
Baseline (Day 1) through 16 weeks
Title
Double-blind phase: Number of participants with a ≥ 25% and ≥ 75% reduction from Baseline during titration and maintenance phase
Time Frame
Up to 16 weeks
Title
Double-blind phase: Number of participants with a ≥ 25%, ≥ 50%, and ≥ 75% reduction from Baseline during maintenance phase
Time Frame
Day 29 to Week 16
Title
Double-blind phase: Number of participants with Responder Analysis using ≤ 0%, > 0% to < 25%, ≥ 25% to < 50%, ≥ 50% to < 75%, and ≥ 75% to 100% during titration and maintenance phase
Time Frame
Up to 16 weeks
Title
Double-blind phase: Percent change in 28-day frequency of all seizures during titration and maintenance phase
Time Frame
Up to 16 weeks
Title
Double-blind phase: Change from Baseline in percentage of Seizure-Free Days during titration and maintenance phase
Time Frame
Baseline (Day 1) and through 16 weeks
Title
Double-blind phase: Change from Baseline in longest Seizure-Free Interval during titration and maintenance phase
Time Frame
Baseline (Day 1) through 16 weeks

10. Eligibility

Sex
All
Minimum Age & Unit of Time
1 Year
Maximum Age & Unit of Time
65 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: 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. 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. Exclusion Criteria: 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. 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.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Marinus
Email
clinicaltrials@marinuspharma.com
Facility Information:
Facility Name
Barrow Neurological Institute at Phoenix Children's Hospital
City
Phoenix
State/Province
Arizona
ZIP/Postal Code
85106
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Angus Wilfong
Facility Name
Arkansas Children's Research Institute
City
Little Rock
State/Province
Arkansas
ZIP/Postal Code
72202
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Debopam Samanta
Facility Name
UCLA Mattel Children's Hospital, TSC Center
City
Los Angeles
State/Province
California
ZIP/Postal Code
90095
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Rajeskar Rajaraman
Facility Name
Children's Hospital of Orange County
City
Orange
State/Province
California
ZIP/Postal Code
92868
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Donald Phillips
Facility Name
Childrens Hospital Colorado
City
Aurora
State/Province
Colorado
ZIP/Postal Code
80045
Country
United States
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Susan Koh
Facility Name
Nemours Children's Hospital - Delaware Valley
City
Wilmington
State/Province
Delaware
ZIP/Postal Code
19803
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Lily Tran
Facility Name
University of Florida Gainesville
City
Gainesville
State/Province
Florida
ZIP/Postal Code
32608
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Sotiris Mitropanopoulos
Facility Name
NW FL Clinical Research Group, LLC
City
Gulf Breeze
State/Province
Florida
ZIP/Postal Code
32561
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Ben Renfroe
Facility Name
Nicklaus Children's Hospital
City
Miami
State/Province
Florida
ZIP/Postal Code
33155
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Matt Lallas
Facility Name
Orlando Health
City
Orlando
State/Province
Florida
ZIP/Postal Code
32806
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Vikram Prakash
Facility Name
Comprehensive Neurology Clinic
City
Orlando
State/Province
Florida
ZIP/Postal Code
32825
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Refaat El-Said
Facility Name
Children's Healthcare of Atlanta
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30329
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
David Wolf
Facility Name
Ann & Robert H. Lurie Children's Hospital of Chicago
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60611
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Priyamvada Tatachar
Facility Name
Mid Atlantic Epilepsy & Sleep Center
City
Bethesda
State/Province
Maryland
ZIP/Postal Code
20817
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Pavel Klein
Facility Name
Boston Children's Hospital, Harvard Medical School
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02115
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Jurriaan Peters
Facility Name
Children's Hospital of Michigan Central Michigan University
City
Detroit
State/Province
Michigan
ZIP/Postal Code
48201
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Aimee Luat
Facility Name
Mayo Clinic - Rochester
City
Rochester
State/Province
Minnesota
ZIP/Postal Code
55905
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Elaine Wirrell
Facility Name
University of Missouri Child Health
City
Columbia
State/Province
Missouri
ZIP/Postal Code
65201
Country
United States
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Paul Carney
Facility Name
Children's Mercy Hosptial
City
Kansas City
State/Province
Missouri
ZIP/Postal Code
64108
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Mohammed Ilyas
Facility Name
TSC Clinic at Northeast Regional Epilepsy Group
City
Hackensack
State/Province
New Jersey
ZIP/Postal Code
07601
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Asfi Rafiuddin
Facility Name
Institute of Neurology and Neurosurgery at Saint Barnabas
City
Livingston
State/Province
New Jersey
ZIP/Postal Code
07039
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Aliza Alter
Facility Name
University of Rochester Medical Center
City
Rochester
State/Province
New York
ZIP/Postal Code
14642
Country
United States
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Inna Hughes
Facility Name
University of North Carolina at Chapel Hill
City
Chapel Hill
State/Province
North Carolina
ZIP/Postal Code
27599
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Chon Lee
Facility Name
Atrium Health/Levine Children's Hospital
City
Charlotte
State/Province
North Carolina
ZIP/Postal Code
28207
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Rani Singh
Facility Name
Duke University Medical Center
City
Durham
State/Province
North Carolina
ZIP/Postal Code
27712
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Muhammad Zafar
Facility Name
Cincinnati Children's Hospital Medical Center
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45229
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
David Ritter
Facility Name
Penn State Children's Hospital
City
Hershey
State/Province
Pennsylvania
ZIP/Postal Code
17033
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Sita Paudel
Facility Name
Children's Hospital of Philadelphia
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Katherine Taub
Facility Name
Medical University of South Carolina
City
Charleston
State/Province
South Carolina
ZIP/Postal Code
29425
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Sonal Bhatia
Facility Name
Le Bonheur Children's Hospital
City
Memphis
State/Province
Tennessee
ZIP/Postal Code
38103
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Sarah Weatherspoon
Facility Name
Child Neurology Consultants of Austin (CNCA)
City
Austin
State/Province
Texas
ZIP/Postal Code
78757
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Karen Keough
Facility Name
University of Texas Southwestern Medical Center
City
Dallas
State/Province
Texas
ZIP/Postal Code
75207
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Charuta Joshi
Facility Name
McGovern Medical School at the University of Texas Health Science Center
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Mary Kay Koenig
Facility Name
University of Utah Health Care-Pediatric Neurology
City
Salt Lake City
State/Province
Utah
ZIP/Postal Code
84108
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Matthew Sweeney
Facility Name
Seattle Children's Hospital
City
Seattle
State/Province
Washington
ZIP/Postal Code
98105
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Russell Saneto
Facility Name
Hôtel Dieu de Montréal - CHUM
City
Montreal
ZIP/Postal Code
H2X 0C2
Country
Canada
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Mark Keezer
Facility Name
CHU Sainte-Justine
City
Montréal
ZIP/Postal Code
H3T 1C5
Country
Canada
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Philippe Major
Facility Name
The Hospital for Sick Children
City
Toronto
ZIP/Postal Code
M5G 1X8
Country
Canada
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Lauren Leigh Sham
Facility Name
Toronto Western Hospital
City
Toronto
ZIP/Postal Code
M5T 2S8
Country
Canada
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Danielle M Andrade
Facility Name
BC Children's Hospital
City
Vancouver
ZIP/Postal Code
V6H 3V4
Country
Canada
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Anita Datta
Facility Name
University Hospital of Lyon
City
Bron
ZIP/Postal Code
69229
Country
France
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Sylvain Rheims
Facility Name
University Hospital of Lille
City
Lille
ZIP/Postal Code
59037
Country
France
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Philippe Derambure
Facility Name
Robert-Debré Hospital
City
Paris
ZIP/Postal Code
75019
Country
France
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Stéphane Auvin
Facility Name
University Hospital of Rennes
City
Rennes
ZIP/Postal Code
35700
Country
France
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Arnaud Biraben
Facility Name
University of Strasbourg
City
Strasbourg
ZIP/Postal Code
67084
Country
France
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Edouard Hirsch
Facility Name
Epilepsie-Zentrum Bethel - Krankenhaus Mara
City
Bielefeld
ZIP/Postal Code
33617
Country
Germany
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Christian Brandt
Facility Name
University Hospital Bonn
City
Bonn
ZIP/Postal Code
53127
Country
Germany
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Rainer Surges
Facility Name
ZNN - Epilepsiezentrum Frankfurt am Main
City
Frankfurt
ZIP/Postal Code
60528
Country
Germany
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Felix Rosenow
Facility Name
Universitäts Krankenhaus Freiburg
City
Freiburg
ZIP/Postal Code
79106
Country
Germany
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Kerstin Alexandra Klotz
Facility Name
Gemeinschaftskrankenhaus Herdecke
City
Herdecke
ZIP/Postal Code
58313
Country
Germany
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Jan-Ulrich Schlump
Facility Name
Epilepsiezentrum Kleinwachau gGmbH
City
Radeberg
ZIP/Postal Code
01454
Country
Germany
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Thomas Mayer
Facility Name
Soroka University Medical Center
City
Beer-Sheva
ZIP/Postal Code
8410100
Country
Israel
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Iris Noyman
Facility Name
Hadassah Medical Center
City
Jerusalem
ZIP/Postal Code
9112991
Country
Israel
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Tal Gilboa
Facility Name
Schneider Children´s Medical Center
City
Petah Tikva
ZIP/Postal Code
4920235
Country
Israel
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Hadassa Goldberg-Stern
Facility Name
Sheba Medical Center
City
Tel Hashomer
ZIP/Postal Code
52621
Country
Israel
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Michal Tzadok
Facility Name
Tel-Aviv Sourasky Medical Center
City
Tel-Aviv
ZIP/Postal Code
64239
Country
Israel
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Shimrit Uliel-Sibony
Facility Name
Department of Neurology and Sense Organs, AOU Policlinico di Bari
City
Bari
ZIP/Postal Code
1170124
Country
Italy
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Angela La Neve
Facility Name
Azienda Ospedaliero-Universitaria Meyer
City
Firenze
ZIP/Postal Code
50139
Country
Italy
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Renzo Guerrini
Facility Name
Pediatric Neurology and Muscular Diseases Unit - University of Genoa
City
Genova
ZIP/Postal Code
16147
Country
Italy
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Margherita Mancardi
Facility Name
Children's Hospital Bambino Gesù
City
Rome
ZIP/Postal Code
00165
Country
Italy
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Nicola Specchio
Facility Name
Policlinico Umberto I
City
Rome
ZIP/Postal Code
00185
Country
Italy
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Carlo Di Bonaventura
Facility Name
Hospital de la Santa Creu i Sant Pau
City
Barcelona
ZIP/Postal Code
08025
Country
Spain
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Susana Boronat
Facility Name
Hospital Universitari Vall d'Hebron
City
Barcelona
ZIP/Postal Code
08035
Country
Spain
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Laura Abraira del Fresno
Facility Name
Hospital Sant Joan de Déu
City
Barcelona
ZIP/Postal Code
08950
Country
Spain
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Federico Ramos
Facility Name
Hospital Infantil Universitario Niño Jesús
City
Madrid
ZIP/Postal Code
28009
Country
Spain
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Beatriz Bernardino Cuesta
Facility Name
Hospital Ruber International
City
Madrid
ZIP/Postal Code
28034
Country
Spain
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Angel Aledo Serrano
Facility Name
Hospital Regional Universitario de Málaga
City
Málaga
ZIP/Postal Code
29010
Country
Spain
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Pedro Serrano Castro
Facility Name
Hospital Universitario y Politécnico La Fe
City
Valencia
ZIP/Postal Code
46026
Country
Spain
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Vicente Villanueva
Facility Name
Royal Aberdeen Children's Hospital, NHS Grampian
City
Aberdeen
ZIP/Postal Code
AB25 2ZG
Country
United Kingdom
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Elma Stephen
Facility Name
Bristol Royal Hospital for Children
City
Bristol
ZIP/Postal Code
BS2 8AE
Country
United Kingdom
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Sam Amin
Facility Name
Leeds General Infirmary
City
Leeds
ZIP/Postal Code
LS1 3EX
Country
United Kingdom
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Melissa Maguire
Facility Name
NHS acute tertiary referral centre, John Radcliffe Hospital
City
Oxford
ZIP/Postal Code
OX3 9DU
Country
United Kingdom
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Mario Sa
Facility Name
Salford Royal Hospital
City
Salford
ZIP/Postal Code
M6 8HD
Country
United Kingdom
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Rajiv Mohanraj
Facility Name
Sheffield Children's Hospital
City
Sheffield
ZIP/Postal Code
S10 2TH
Country
United Kingdom
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Archana Desurkar

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

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

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