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Open-label Study of Adjunctive GNX Treatment in Children and Adults With TSC-related Epilepsy

Primary Purpose

Tuberous Sclerosis Complex

Status
Enrolling by invitation
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
Ganaxolone
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: Completion of Study 1042-TSC-3001 or participants who continue to meet study requirements in Study 1042-TSC-2001. 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. Parent(s)/caregiver(s) is (are) willing and able to maintain an accurate and complete daily seizure diary for the duration of the study. Willing and able to take Investigational product (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 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 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: Pregnant or breastfeeding. An active Central nervous system (CNS) infection, demyelinating disease, or degenerative neurological disease. History of psychogenic nonepileptic seizures. 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. Unwillingness to avoid excessive alcohol use or cannabis use throughout the study. 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. 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 Hospital
  • Arkansas Children's Research Institute
  • UCLA Mattel Children's Hospital, TSC Center
  • Children's Hospital of Orange County
  • Children's Hospital Colorado
  • Nemours Children's Hospital - Delaware Valley
  • University of Florida Gainsville
  • NW FL Clinical Research Group, LLC
  • Nicklaus Children's Hospital
  • Comprehensive Neurology Clinic
  • Children's Healthcare of Atlanta
  • Ann & Robert H. Lurie Children's Hospital of Chicago
  • Mid-Atlantic Epilepsy & Sleep Center
  • Boston Children's Hospital, Harvard Medical School
  • Children's Hospital of Michigan Central Michigan University
  • Mayo Clinic - Rochester
  • Children's Mercy Hospital
  • TSC Clinic at Northeast Regional Epilepsy Group
  • Institute of Neurology and Neurosurgery at Saint Barnabas
  • University of Rochester Medical Center
  • University of North Carolina at Chapel Hill
  • Atrium Health/Levine Children's Hospital
  • Duke University Medical Center
  • Cincinnati Children's Hospital Medical Center
  • Penn State Children's Hospital
  • Children's Hospital of Philadelphia
  • Medical University of South Carolina
  • Le Bonheur Children's Hospital
  • Child Neurology Consultants of Austin (CNCA)
  • University of Texas Southwestern Medical Center
  • McGovern Medical School at the University of Texas Health Science Center
  • University of Utah Health Care-Pediatric Neurology
  • Seattle Children's Hospital
  • Queensland Health
  • Austin Health
  • Royal Brisbane and Women's Hospital
  • Alfred Health
  • Western Sydney Local health District
  • Royal Melbourne Hospital
  • The Royal Children's Hospital Melbourne
  • Prince of Wales Hospital
  • Antwerp University Hospital (UZA)
  • UZ Brussel
  • Hôtel Dieu de Montréal - CHUM
  • CHU Sainte-Justine
  • The Hospital for Sick Children
  • Toronto Western Hospital
  • BC Children's Hospital
  • Peking University First Hospital
  • The Affiliated Hospital of Guizhou Medical University
  • University Hospital of Lyon
  • University Hospital of Lille
  • Robert-Debré Hospital
  • Hôpital de la Pitié-Salpêtrière
  • University Hospital of Rennes
  • University of Strasbourg
  • Epilepsie-Zentrum Bethel - Krankenhaus Mara
  • University Hospital Bonn
  • ZNN - Epilepsiezentrum Frankfurt am Main
  • Universitäts Krankenhaus Freiburg
  • Gemeinschaftskrankenhaus Herdecke
  • Epilepsiezentrum Kleinwachau gGmbH
  • Soroka University Medical Center
  • Hadassah Medical Center
  • Schneider Children´s Medical Center
  • Tel-Aviv Sourasky Medical Center
  • Sheba Medical Center
  • 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 Pau
  • Hospital Universitari Vall d'Hebron
  • Hospital Sant Joan de Déu
  • Hospital Infantil Universitario Niño Jesús
  • Hospital Ruber International
  • Hospital Regional Universitario de Málaga
  • Hospital Universitario y Politécnico La Fe
  • Royal Aberdeen Children's Hospital, NHS Grampian
  • Bristol Royal Hospital for Children
  • Leeds General Infirmary
  • NHS acute tertiary referral centre, John Radcliffe Hospital
  • Salford Royal Hospital
  • Sheffield Children's Hospital

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Ganaxolone (GNX) oral suspension, 3 times a day (TID)

Arm Description

Outcomes

Primary Outcome Measures

Number of participants with adverse events (AEs), serious adverse events (SAEs) and withdrawals and dose-reductions due to AEs
Number of participants with abnormal vital signs
Number of participants with abnormal physical, neurological and developmental examination
Number of participants with abnormal 12-lead electrocardiogram (ECG) findings
Number of participants with abnormal clinical laboratory tests
Number of participants with abnormal Columbia-Suicide Severity Rating Scale (C-SSRS)
The C-SSRS is a clinician administered assessment tool that evaluates suicidal ideation and behavior. Number of participants that have an affirmative response to the 5 items for suicidal ideation (1. Wish to be dead, 2. Non-specific active suicidal thoughts, 3. Active suicidal ideation with any methods (not plan) without intent to act, 4. Active suicidal ideation with some intent to act, without specific plan, 5. Active suicidal ideation with specific plan and intent) and/or to the 5 items for suicidal behavior (1. Preparatory acts or behavior, 2. Aborted attempt, 3. Interrupted attempt, 4. Actual attempt, 5. Completed suicide) will be reported. Higher scores indicate worse symptoms.

Secondary Outcome Measures

Percent change from Baseline in 28-day seizure frequency during open label extension
Seizure frequency will be calculated as the total number of seizures divided by the number of days with seizure data, multiplied by 28.
Percent change from Baseline in 28-day seizure frequency during the long-term treatment
Number of participants who will be considered as Treatment Responders
Treatment responders are defined as those participants with ≥ 50% reduction from Baseline in seizure frequency during open-label treatment.
Number of Participants with Clinical Global Impression of Improvement (CGI-I)
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.
Change from Baseline in quality-of-life scale Short Form-36 (SF-36)
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.
Change from Baseline in percentage of Seizure-Free Days during treatment, based on seizure type
Change from Baseline in Caregiver Global Impression of Change in Seizure Intensity/Duration (CGI-CSID)
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.

Full Information

First Posted
October 5, 2022
Last Updated
August 30, 2023
Sponsor
Marinus Pharmaceuticals
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1. Study Identification

Unique Protocol Identification Number
NCT05604170
Brief Title
Open-label Study of Adjunctive GNX Treatment in Children and Adults With TSC-related Epilepsy
Official Title
A Phase 3, Open-label Study of Adjunctive Ganaxolone (GNX) Treatment in Children and Adults With Tuberous Sclerosis Complex (TSC)-Related Epilepsy (TrustTSC OLE)
Study Type
Interventional

2. Study Status

Record Verification Date
August 2023
Overall Recruitment Status
Enrolling by invitation
Study Start Date
May 16, 2022 (Actual)
Primary Completion Date
June 2027 (Anticipated)
Study Completion Date
June 2027 (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, open-label extension (OLE) study of adjunctive GNX treatment in children and adults with TSC who previously participated in either Study 1042-TSC-3001 or Study 1042-TSC-2001

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
Single Group Assignment
Masking
None (Open Label)
Masking Description
This is an open-label, single arm study with no blinding as all participants will receive adjunctive GNX
Allocation
N/A
Enrollment
169 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Ganaxolone (GNX) oral suspension, 3 times a day (TID)
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
Ganaxolone
Intervention Description
GNX will be administered.
Primary Outcome Measure Information:
Title
Number of participants with adverse events (AEs), serious adverse events (SAEs) and withdrawals and dose-reductions due to AEs
Time Frame
Week 1 through Week 52
Title
Number of participants with abnormal vital signs
Time Frame
Week 1 through Week 52
Title
Number of participants with abnormal physical, neurological and developmental examination
Time Frame
Week 1 through Week 52
Title
Number of participants with abnormal 12-lead electrocardiogram (ECG) findings
Time Frame
Week 1 through Week 52
Title
Number of participants with abnormal clinical laboratory tests
Time Frame
Week 1 through Week 52
Title
Number of participants with abnormal Columbia-Suicide Severity Rating Scale (C-SSRS)
Description
The C-SSRS is a clinician administered assessment tool that evaluates suicidal ideation and behavior. Number of participants that have an affirmative response to the 5 items for suicidal ideation (1. Wish to be dead, 2. Non-specific active suicidal thoughts, 3. Active suicidal ideation with any methods (not plan) without intent to act, 4. Active suicidal ideation with some intent to act, without specific plan, 5. Active suicidal ideation with specific plan and intent) and/or to the 5 items for suicidal behavior (1. Preparatory acts or behavior, 2. Aborted attempt, 3. Interrupted attempt, 4. Actual attempt, 5. Completed suicide) will be reported. Higher scores indicate worse symptoms.
Time Frame
Week 1 through Week 52
Secondary Outcome Measure Information:
Title
Percent change from Baseline in 28-day seizure frequency during open label extension
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) and Week 1 through Week 52
Title
Percent change from Baseline in 28-day seizure frequency during the long-term treatment
Time Frame
Baseline (Day 1) and Week 1 through Week 52
Title
Number of participants who will be considered as Treatment Responders
Description
Treatment responders are defined as those participants with ≥ 50% reduction from Baseline in seizure frequency during open-label treatment.
Time Frame
Week 1 through Week 52
Title
Number of Participants with Clinical Global Impression of Improvement (CGI-I)
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
Week 1 through Week 52
Title
Change from Baseline in quality-of-life scale Short Form-36 (SF-36)
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) and Week 1 through Week 52
Title
Change from Baseline in percentage of Seizure-Free Days during treatment, based on seizure type
Time Frame
Baseline (Day 1) and Week 1 through Week 52
Title
Change from Baseline in Caregiver Global Impression of Change in Seizure Intensity/Duration (CGI-CSID)
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) and Week 1 through Week 52

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: Completion of Study 1042-TSC-3001 or participants who continue to meet study requirements in Study 1042-TSC-2001. 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. Parent(s)/caregiver(s) is (are) willing and able to maintain an accurate and complete daily seizure diary for the duration of the study. Willing and able to take Investigational product (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 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 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: Pregnant or breastfeeding. An active Central nervous system (CNS) infection, demyelinating disease, or degenerative neurological disease. History of psychogenic nonepileptic seizures. 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. Unwillingness to avoid excessive alcohol use or cannabis use throughout the study. 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. Known sensitivity or allergy to any component in the IP(s), progesterone, or other related steroid compounds.
Facility Information:
Facility Name
Barrow Neurological Institute at Phoenix Children's Hospital
City
Phoenix
State/Province
Arizona
ZIP/Postal Code
85016
Country
United States
Facility Name
Arkansas Children's Research Institute
City
Little Rock
State/Province
Arkansas
ZIP/Postal Code
72202
Country
United States
Facility Name
UCLA Mattel Children's Hospital, TSC Center
City
Los Angeles
State/Province
California
ZIP/Postal Code
90095
Country
United States
Facility Name
Children's Hospital of Orange County
City
Orange
State/Province
California
ZIP/Postal Code
92868
Country
United States
Facility Name
Children's Hospital Colorado
City
Aurora
State/Province
Colorado
ZIP/Postal Code
80045
Country
United States
Facility Name
Nemours Children's Hospital - Delaware Valley
City
Wilmington
State/Province
Delaware
ZIP/Postal Code
19803
Country
United States
Facility Name
University of Florida Gainsville
City
Gainesville
State/Province
Florida
ZIP/Postal Code
32608
Country
United States
Facility Name
NW FL Clinical Research Group, LLC
City
Gulf Breeze
State/Province
Florida
ZIP/Postal Code
32561
Country
United States
Facility Name
Nicklaus Children's Hospital
City
Miami
State/Province
Florida
ZIP/Postal Code
33155
Country
United States
Facility Name
Comprehensive Neurology Clinic
City
Orlando
State/Province
Florida
ZIP/Postal Code
32825
Country
United States
Facility Name
Children's Healthcare of Atlanta
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30329
Country
United States
Facility Name
Ann & Robert H. Lurie Children's Hospital of Chicago
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60611
Country
United States
Facility Name
Mid-Atlantic Epilepsy & Sleep Center
City
Bethesda
State/Province
Maryland
ZIP/Postal Code
20817
Country
United States
Facility Name
Boston Children's Hospital, Harvard Medical School
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02115
Country
United States
Facility Name
Children's Hospital of Michigan Central Michigan University
City
Detroit
State/Province
Michigan
ZIP/Postal Code
48201
Country
United States
Facility Name
Mayo Clinic - Rochester
City
Rochester
State/Province
Minnesota
ZIP/Postal Code
55905
Country
United States
Facility Name
Children's Mercy Hospital
City
Kansas City
State/Province
Missouri
ZIP/Postal Code
64108
Country
United States
Facility Name
TSC Clinic at Northeast Regional Epilepsy Group
City
Hackensack
State/Province
New Jersey
ZIP/Postal Code
07601
Country
United States
Facility Name
Institute of Neurology and Neurosurgery at Saint Barnabas
City
Livingston
State/Province
New Jersey
ZIP/Postal Code
07039
Country
United States
Facility Name
University of Rochester Medical Center
City
Rochester
State/Province
New York
ZIP/Postal Code
14642
Country
United States
Facility Name
University of North Carolina at Chapel Hill
City
Chapel Hill
State/Province
North Carolina
ZIP/Postal Code
27599
Country
United States
Facility Name
Atrium Health/Levine Children's Hospital
City
Charlotte
State/Province
North Carolina
ZIP/Postal Code
28207
Country
United States
Facility Name
Duke University Medical Center
City
Durham
State/Province
North Carolina
ZIP/Postal Code
27712
Country
United States
Facility Name
Cincinnati Children's Hospital Medical Center
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45229
Country
United States
Facility Name
Penn State Children's Hospital
City
Hershey
State/Province
Pennsylvania
ZIP/Postal Code
17033
Country
United States
Facility Name
Children's Hospital of Philadelphia
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
Facility Name
Medical University of South Carolina
City
Charleston
State/Province
South Carolina
ZIP/Postal Code
29425
Country
United States
Facility Name
Le Bonheur Children's Hospital
City
Memphis
State/Province
Tennessee
ZIP/Postal Code
38103
Country
United States
Facility Name
Child Neurology Consultants of Austin (CNCA)
City
Austin
State/Province
Texas
ZIP/Postal Code
78757
Country
United States
Facility Name
University of Texas Southwestern Medical Center
City
Dallas
State/Province
Texas
ZIP/Postal Code
75207
Country
United States
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
Facility Name
University of Utah Health Care-Pediatric Neurology
City
Salt Lake City
State/Province
Utah
ZIP/Postal Code
84108
Country
United States
Facility Name
Seattle Children's Hospital
City
Seattle
State/Province
Washington
ZIP/Postal Code
98105
Country
United States
Facility Name
Queensland Health
City
Brisbane
ZIP/Postal Code
4001
Country
Australia
Facility Name
Austin Health
City
Heidelberg
ZIP/Postal Code
VIC 3084
Country
Australia
Facility Name
Royal Brisbane and Women's Hospital
City
Herston
ZIP/Postal Code
QLD 4029
Country
Australia
Facility Name
Alfred Health
City
Melbourne
ZIP/Postal Code
VIC 3004
Country
Australia
Facility Name
Western Sydney Local health District
City
North Parramatta
ZIP/Postal Code
NSW 2151
Country
Australia
Facility Name
Royal Melbourne Hospital
City
Parkville
ZIP/Postal Code
VIC 3050
Country
Australia
Facility Name
The Royal Children's Hospital Melbourne
City
Parkville
ZIP/Postal Code
VIC 3052
Country
Australia
Facility Name
Prince of Wales Hospital
City
Randwick
ZIP/Postal Code
NSW 2031
Country
Australia
Facility Name
Antwerp University Hospital (UZA)
City
Edegem
ZIP/Postal Code
02650
Country
Belgium
Facility Name
UZ Brussel
City
Jette
ZIP/Postal Code
01090
Country
Belgium
Facility Name
Hôtel Dieu de Montréal - CHUM
City
Montréal
ZIP/Postal Code
H2X 0C2
Country
Canada
Facility Name
CHU Sainte-Justine
City
Montréal
ZIP/Postal Code
H3T 1C5
Country
Canada
Facility Name
The Hospital for Sick Children
City
Toronto
ZIP/Postal Code
M5G 1X8
Country
Canada
Facility Name
Toronto Western Hospital
City
Toronto
ZIP/Postal Code
M5T 2S8
Country
Canada
Facility Name
BC Children's Hospital
City
Vancouver
ZIP/Postal Code
V6H 3V4
Country
Canada
Facility Name
Peking University First Hospital
City
Beijing
ZIP/Postal Code
100034
Country
China
Facility Name
The Affiliated Hospital of Guizhou Medical University
City
Beijing
ZIP/Postal Code
550004
Country
China
Facility Name
University Hospital of Lyon
City
Bron
ZIP/Postal Code
69229
Country
France
Facility Name
University Hospital of Lille
City
Lille
ZIP/Postal Code
59037
Country
France
Facility Name
Robert-Debré Hospital
City
Paris
ZIP/Postal Code
75019
Country
France
Facility Name
Hôpital de la Pitié-Salpêtrière
City
Paris
ZIP/Postal Code
75651
Country
France
Facility Name
University Hospital of Rennes
City
Rennes
ZIP/Postal Code
35700
Country
France
Facility Name
University of Strasbourg
City
Strasbourg
ZIP/Postal Code
67084
Country
France
Facility Name
Epilepsie-Zentrum Bethel - Krankenhaus Mara
City
Bielefeld
ZIP/Postal Code
33617
Country
Germany
Facility Name
University Hospital Bonn
City
Bonn
ZIP/Postal Code
53127
Country
Germany
Facility Name
ZNN - Epilepsiezentrum Frankfurt am Main
City
Frankfurt
ZIP/Postal Code
60528
Country
Germany
Facility Name
Universitäts Krankenhaus Freiburg
City
Freiburg
ZIP/Postal Code
79106
Country
Germany
Facility Name
Gemeinschaftskrankenhaus Herdecke
City
Herdecke
ZIP/Postal Code
58313
Country
Germany
Facility Name
Epilepsiezentrum Kleinwachau gGmbH
City
Radeberg
ZIP/Postal Code
1454
Country
Germany
Facility Name
Soroka University Medical Center
City
Be'er Sheva
ZIP/Postal Code
8410100
Country
Israel
Facility Name
Hadassah Medical Center
City
Jerusalem
ZIP/Postal Code
9112991
Country
Israel
Facility Name
Schneider Children´s Medical Center
City
Petach Tikva
ZIP/Postal Code
4920235
Country
Israel
Facility Name
Tel-Aviv Sourasky Medical Center
City
Tel Aviv
ZIP/Postal Code
64239
Country
Israel
Facility Name
Sheba Medical Center
City
Tel HaShomer
ZIP/Postal Code
52621
Country
Israel
Facility Name
Department of Neurology and Sense Organs, AOU Policlinico di Bari
City
Bari
ZIP/Postal Code
1170124
Country
Italy
Facility Name
Azienda Ospedaliero-Universitaria Meyer
City
Firenze
ZIP/Postal Code
50139
Country
Italy
Facility Name
Pediatric Neurology and Muscular Diseases Unit - University of Genoa
City
Genova
ZIP/Postal Code
16147
Country
Italy
Facility Name
Children's Hospital Bambino Gesù
City
Rome
ZIP/Postal Code
00165
Country
Italy
Facility Name
Policlinico Umberto I
City
Rome
ZIP/Postal Code
00185
Country
Italy
Facility Name
Hospital de la Santa Creu i Sant Pau
City
Barcelona
ZIP/Postal Code
8025
Country
Spain
Facility Name
Hospital Universitari Vall d'Hebron
City
Barcelona
ZIP/Postal Code
8035
Country
Spain
Facility Name
Hospital Sant Joan de Déu
City
Barcelona
ZIP/Postal Code
8950
Country
Spain
Facility Name
Hospital Infantil Universitario Niño Jesús
City
Madrid
ZIP/Postal Code
28009
Country
Spain
Facility Name
Hospital Ruber International
City
Madrid
ZIP/Postal Code
28034
Country
Spain
Facility Name
Hospital Regional Universitario de Málaga
City
Málaga
ZIP/Postal Code
29010
Country
Spain
Facility Name
Hospital Universitario y Politécnico La Fe
City
Valencia
ZIP/Postal Code
46026
Country
Spain
Facility Name
Royal Aberdeen Children's Hospital, NHS Grampian
City
Aberdeen
ZIP/Postal Code
AB25 2ZG
Country
United Kingdom
Facility Name
Bristol Royal Hospital for Children
City
Bristol
ZIP/Postal Code
BS2 8AE
Country
United Kingdom
Facility Name
Leeds General Infirmary
City
Leeds
ZIP/Postal Code
LS1 3EX
Country
United Kingdom
Facility Name
NHS acute tertiary referral centre, John Radcliffe Hospital
City
Oxford
ZIP/Postal Code
OX3 9DU
Country
United Kingdom
Facility Name
Salford Royal Hospital
City
Salford
ZIP/Postal Code
M6 8HD
Country
United Kingdom
Facility Name
Sheffield Children's Hospital
City
Sheffield
ZIP/Postal Code
S10 2TH
Country
United Kingdom

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

Open-label Study of Adjunctive GNX Treatment in Children and Adults With TSC-related Epilepsy

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