Trial Title:
Open-label Study of Adjunctive GNX Treatment in Children and Adults With TSC-related Epilepsy
NCT ID:
NCT05604170
Condition:
Tuberous Sclerosis Complex
Conditions: Official terms:
Tuberous Sclerosis
Epilepsy
Sclerosis
Ganaxolone
Conditions: Keywords:
Tuberous Sclerosis Complex-Related Epilepsy
Ganaxolone
Adjunctive
Study type:
Interventional
Study phase:
Phase 3
Overall status:
Enrolling by invitation
Study design:
Allocation:
N/A
Intervention model:
Single Group Assignment
Primary purpose:
Treatment
Masking:
None (Open Label)
Masking description:
This is an open-label, single arm study with no blinding as all participants will receive
adjunctive GNX
Intervention:
Intervention type:
Drug
Intervention name:
Ganaxolone
Description:
GNX will be administered.
Arm group label:
Ganaxolone (GNX) oral suspension, 3 times a day (TID)
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
Criteria for eligibility:
Criteria:
Inclusion Criteria:
1. Completion of Study 1042-TSC-3001 or participants who continue to meet study
requirements in Study 1042-TSC-2001.
2. Participant/parent(s)/LAR(s) willing and able to give written informed
consent/assent, after being properly informed of the nature and risks of the study
and prior to engaging in any study-related procedures. If the participant is not
qualified or able to provide written informed consent based on age, developmental
stage, intellectual capacity, or other factors, parent(s)/LAR(s) must provide assent
for study participation, if appropriate.
3. Parent(s)/caregiver(s) is (are) willing and able to maintain an accurate and
complete daily seizure diary for the duration of the study.
4. Willing and able to take Investigational product (IP) (suspension) as directed with
food TID.
5. Women of childbearing potential (WOCBP) must be using a medically acceptable method
of birth control and have a negative quantitative serum beta-human chorionic growth
hormone (β-HCG) test collected at the initial visit. Childbearing potential is
defined as a female who is biologically capable of becoming pregnant. Medically
acceptable methods of birth control include intrauterine devices (that have been in
place for at least 1 month prior to the screening visit), hormonal contraceptives
(eg, combined oral contraceptives, patch, vaginal ring, injectables, and implants),
and surgical sterilization (such as oophorectomy or tubal ligation). When used
consistently and correctly, "double-barrier" methods of contraception can be used as
an effective alternative to highly effective contraception methods. Contraceptive
measures such as Plan B™, sold for emergency use after unprotected sex, are not
acceptable methods for routine use
6. Male participants must agree to use highly effective contraceptive methods during
the study and for 30 days after the last dose of IP. Highly effective methods of
contraception include surgical sterilization (such as a vasectomy) and adequate
"double-barrier" methods.
Exclusion Criteria:
1. Pregnant or breastfeeding.
2. An active Central nervous system (CNS) infection, demyelinating disease, or
degenerative neurological disease.
3. History of psychogenic nonepileptic seizures.
4. Any disease or condition (other than TSC) at the initial visit that could compromise
the hematologic, cardiovascular (including any cardiac conduction defect),
pulmonary, renal, gastrointestinal, or hepatic systems; or other conditions that
might interfere with the absorption, distribution, metabolism, or excretion of the
IP, or would place the participant at increased risk or interfere with the
assessment of safety/efficacy. This may include any illness in the past 4 weeks
which in the opinion of the investigator may affect seizure frequency.
5. Unwillingness to avoid excessive alcohol use or cannabis use throughout the study.
6. Have active suicidal plan/intent or have had active suicidal thoughts in the past 6
months or a suicide attempt in the past 6 months.
7. Known sensitivity or allergy to any component in the IP(s), progesterone, or other
related steroid compounds.
8. Exposed to any other investigational drug (except for GNX in Study 1042-TSC-2001 or
Study 1042-TSC-3001) or investigational device within 30 days or fewer than 5
half-lives prior to Visit 1 (first visit of the OLE). For therapies in which
half-life cannot be readily established, the Sponsor's medical monitor should be
consulted.
Gender:
All
Minimum age:
1 Year
Maximum age:
65 Years
Healthy volunteers:
No
Locations:
Facility:
Name:
Arkansas Children's Research Institute
Address:
City:
Little Rock
Zip:
72202
Country:
United States
Facility:
Name:
UCLA Mattel Children's Hospital, TSC Center
Address:
City:
Los Angeles
Zip:
90095
Country:
United States
Facility:
Name:
Children's Hospital of Orange County
Address:
City:
Orange
Zip:
92868
Country:
United States
Facility:
Name:
Children's Hospital Colorado
Address:
City:
Aurora
Zip:
80045
Country:
United States
Facility:
Name:
Mid-Atlantic Epilepsy and Sleep Center
Address:
City:
Bethesda
Zip:
20817
Country:
United States
Facility:
Name:
Mayo Clinic - Rochester
Address:
City:
Rochester
Zip:
55905
Country:
United States
Facility:
Name:
University of Rochester Medical Center
Address:
City:
Rochester
Zip:
14642
Country:
United States
Facility:
Name:
University of North Carolina at Chapel Hill
Address:
City:
Chapel Hill
Zip:
27599
Country:
United States
Facility:
Name:
Atrium Health/Levine Children's Hospital
Address:
City:
Charlotte
Zip:
28207
Country:
United States
Facility:
Name:
Duke University Medical Center
Address:
City:
Durham
Zip:
27712
Country:
United States
Facility:
Name:
Penn State Children's Hospital
Address:
City:
Hershey
Zip:
17033
Country:
United States
Facility:
Name:
Children's Hospital of Philadelphia
Address:
City:
Philadelphia
Zip:
19104
Country:
United States
Facility:
Name:
Medical University of South Carolina
Address:
City:
Charleston
Zip:
29425
Country:
United States
Facility:
Name:
Le Bonheur Children's Hospital
Address:
City:
Memphis
Zip:
38103
Country:
United States
Facility:
Name:
Child Neurology Consultants of Austin (CNCA)
Address:
City:
Austin
Zip:
78757
Country:
United States
Facility:
Name:
McGovern Medical School at the University of Texas Health Science Center
Address:
City:
Houston
Zip:
77030
Country:
United States
Facility:
Name:
Austin Health
Address:
City:
Heidelberg
Zip:
VIC 3084
Country:
Australia
Facility:
Name:
Alfred Health
Address:
City:
Melbourne
Zip:
VIC 3004
Country:
Australia
Facility:
Name:
Royal Melbourne Hospital
Address:
City:
Parkville
Zip:
VIC 3050
Country:
Australia
Facility:
Name:
The Royal Children's Hospital Melbourne
Address:
City:
Parkville
Zip:
VIC 3052
Country:
Australia
Facility:
Name:
Hôtel Dieu de Montréal - CHUM
Address:
City:
Montréal
Zip:
H2X 0C2
Country:
Canada
Facility:
Name:
CHU Sainte-Justine
Address:
City:
Montréal
Zip:
H3T 1C5
Country:
Canada
Facility:
Name:
The Hospital for Sick Children
Address:
City:
Toronto
Zip:
M5G 1X8
Country:
Canada
Facility:
Name:
Toronto Western Hospital
Address:
City:
Toronto
Zip:
M5T 2S8
Country:
Canada
Facility:
Name:
BC Children's Hospital
Address:
City:
Vancouver
Zip:
V6H 3V4
Country:
Canada
Facility:
Name:
Peking University First Hospital
Address:
City:
Beijing
Zip:
100034
Country:
China
Facility:
Name:
Beijing Children Hospital, Capital Medical University
Address:
City:
Beijing
Zip:
100045
Country:
China
Facility:
Name:
Chinese PLA General Hospital
Address:
City:
Beijing
Zip:
100080
Country:
China
Facility:
Name:
The Affiliated Hospital of Guizhou Medical University
Address:
City:
Beijing
Zip:
550004
Country:
China
Facility:
Name:
First Hospital of Jilin University
Address:
City:
Jilin
Zip:
130028
Country:
China
Facility:
Name:
Chengdu's Women and Children's Central Hospital
Address:
City:
Qingyang
Zip:
610000
Country:
China
Facility:
Name:
University Hospital of Lyon
Address:
City:
Bron
Zip:
69229
Country:
France
Facility:
Name:
Robert-Debré Hospital
Address:
City:
Paris
Zip:
75019
Country:
France
Facility:
Name:
Hôpital de la Pitié-Salpêtrière
Address:
City:
Paris
Zip:
75651
Country:
France
Facility:
Name:
University Hospital of Rennes
Address:
City:
Rennes
Zip:
35700
Country:
France
Facility:
Name:
University of Strasbourg
Address:
City:
Strasbourg
Zip:
67084
Country:
France
Facility:
Name:
Epilepsie-Zentrum Bethel - Krankenhaus Mara
Address:
City:
Bielefeld
Zip:
33617
Country:
Germany
Facility:
Name:
University Hospital Bonn
Address:
City:
Bonn
Zip:
53127
Country:
Germany
Facility:
Name:
ZNN - Epilepsiezentrum Frankfurt am Main
Address:
City:
Frankfurt
Zip:
60528
Country:
Germany
Facility:
Name:
Universitäts Krankenhaus Freiburg
Address:
City:
Freiburg
Zip:
79106
Country:
Germany
Facility:
Name:
Gemeinschaftskrankenhaus Herdecke
Address:
City:
Herdecke
Zip:
58313
Country:
Germany
Facility:
Name:
Epilepsiezentrum Kleinwachau gGmbH
Address:
City:
Radeberg
Zip:
1454
Country:
Germany
Facility:
Name:
Soroka University Medical Center
Address:
City:
Be'er Sheva
Zip:
8410100
Country:
Israel
Facility:
Name:
Schneider Children´s Medical Center
Address:
City:
Petah Tikva
Zip:
4920235
Country:
Israel
Facility:
Name:
Tel-Aviv Sourasky Medical Center
Address:
City:
Tel Aviv
Zip:
64239
Country:
Israel
Facility:
Name:
Azienda Ospedaliero-Universitaria Meyer
Address:
City:
Firenze
Zip:
50139
Country:
Italy
Facility:
Name:
Pediatric Neurology and Muscular Diseases Unit - University of Genoa
Address:
City:
Genova
Zip:
16147
Country:
Italy
Facility:
Name:
Policlinico Umberto I
Address:
City:
Rome
Zip:
00185
Country:
Italy
Facility:
Name:
Hospital de la Santa Creu i Sant Pau
Address:
City:
Barcelona
Zip:
8025
Country:
Spain
Facility:
Name:
Hospital Universitari Vall d'Hebron
Address:
City:
Barcelona
Zip:
8035
Country:
Spain
Facility:
Name:
Hospital Sant Joan de Déu
Address:
City:
Barcelona
Zip:
8950
Country:
Spain
Facility:
Name:
Hospital Infantil Universitario Niño Jesús
Address:
City:
Madrid
Zip:
28009
Country:
Spain
Facility:
Name:
Hospital Ruber International
Address:
City:
Madrid
Zip:
28034
Country:
Spain
Facility:
Name:
Hospital Regional Universitario de Málaga
Address:
City:
Málaga
Zip:
29010
Country:
Spain
Facility:
Name:
Hospital Universitario y Politécnico La Fe
Address:
City:
Valencia
Zip:
46026
Country:
Spain
Facility:
Name:
Bristol Royal Hospital for Children
Address:
City:
Bristol
Zip:
BS2 8AE
Country:
United Kingdom
Facility:
Name:
NHS acute tertiary referral centre, John Radcliffe Hospital
Address:
City:
Oxford
Zip:
OX3 9DU
Country:
United Kingdom
Facility:
Name:
Salford Royal Hospital
Address:
City:
Salford
Zip:
M6 8HD
Country:
United Kingdom
Facility:
Name:
Sheffield Children's Hospital
Address:
City:
Sheffield
Zip:
S10 2TH
Country:
United Kingdom
Start date:
May 16, 2022
Completion date:
June 2027
Lead sponsor:
Agency:
Marinus Pharmaceuticals
Agency class:
Industry
Source:
Marinus Pharmaceuticals
Record processing date:
ClinicalTrials.gov processed this data on November 12, 2024
Source: ClinicalTrials.gov page:
https://clinicaltrials.gov/ct2/show/NCT05604170
