Please note that the copy function is not enabled for this field.
If you wish to
modify
existing outcomes, please copy and paste the current outcome text into the Update field.
LOGIN
CREATE ACCOUNT
LOGIN
CREATE ACCOUNT
MY TRIALS
REGISTER TRIAL
FAQs
HINTS AND TIPS
DEFINITIONS
Trial Review
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this
information for consumers
Download to PDF
Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT05604170
Registration number
NCT05604170
Ethics application status
Date submitted
5/10/2022
Date registered
3/11/2022
Date last updated
13/05/2024
Titles & IDs
Public title
Open-label Study of Adjunctive GNX Treatment in Children and Adults With TSC-related Epilepsy
Query!
Scientific 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)
Query!
Secondary ID [1]
0
0
1042-TSC-3002
Query!
Universal Trial Number (UTN)
Query!
Trial acronym
Query!
Linked study record
Query!
Health condition
Health condition(s) or problem(s) studied:
Tuberous Sclerosis Complex
0
0
Query!
Condition category
Condition code
Human Genetics and Inherited Disorders
0
0
0
0
Query!
Other human genetics and inherited disorders
Query!
Neurological
0
0
0
0
Query!
Other neurological disorders
Query!
Neurological
0
0
0
0
Query!
Epilepsy
Query!
Intervention/exposure
Study type
Interventional
Query!
Description of intervention(s) / exposure
Treatment: Drugs - Ganaxolone
Experimental: Ganaxolone (GNX) oral suspension, 3 times a day (TID) -
Treatment: Drugs: Ganaxolone
GNX will be administered.
Query!
Intervention code [1]
0
0
Treatment: Drugs
Query!
Comparator / control treatment
Query!
Control group
Query!
Outcomes
Primary outcome [1]
0
0
Number of participants with adverse events (AEs), serious adverse events (SAEs) and withdrawals and dose-reductions due to AEs
Query!
Assessment method [1]
0
0
Query!
Timepoint [1]
0
0
Week 1 through Week 160
Query!
Primary outcome [2]
0
0
Number of participants with abnormal vital signs
Query!
Assessment method [2]
0
0
Query!
Timepoint [2]
0
0
Week 1 through Week 160
Query!
Primary outcome [3]
0
0
Number of participants with abnormal physical, neurological and developmental examination
Query!
Assessment method [3]
0
0
Query!
Timepoint [3]
0
0
Week 1 through Week 160
Query!
Primary outcome [4]
0
0
Number of participants with abnormal 12-lead electrocardiogram (ECG) findings
Query!
Assessment method [4]
0
0
Query!
Timepoint [4]
0
0
Week 1 through Week 160
Query!
Primary outcome [5]
0
0
Number of participants with abnormal clinical laboratory tests
Query!
Assessment method [5]
0
0
Query!
Timepoint [5]
0
0
Week 1 through Week 156
Query!
Primary outcome [6]
0
0
Number of participants with abnormal Columbia-Suicide Severity Rating Scale (C-SSRS)
Query!
Assessment method [6]
0
0
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.
Query!
Timepoint [6]
0
0
Week 1 through Week 160
Query!
Secondary outcome [1]
0
0
Percent change from Baseline in 28-day seizure frequency during open label extension
Query!
Assessment method [1]
0
0
Seizure frequency will be calculated as the total number of seizures divided by the number of days with seizure data, multiplied by 28.
Query!
Timepoint [1]
0
0
Baseline (Day 1) and Week 1 through Week 52
Query!
Secondary outcome [2]
0
0
Percent change from Baseline in 28-day seizure frequency during the long-term treatment
Query!
Assessment method [2]
0
0
Query!
Timepoint [2]
0
0
Baseline (Day 1) and Week 1 through Week 52
Query!
Secondary outcome [3]
0
0
Number of participants who will be considered as Treatment Responders
Query!
Assessment method [3]
0
0
Treatment responders are defined as those participants with = 50% reduction from Baseline in seizure frequency during open-label treatment.
Query!
Timepoint [3]
0
0
Week 1 through Week 52
Query!
Secondary outcome [4]
0
0
Number of Participants with Clinical Global Impression of Improvement (CGI-I)
Query!
Assessment method [4]
0
0
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.
Query!
Timepoint [4]
0
0
Week 1 through Week 156
Query!
Secondary outcome [5]
0
0
Change from Baseline in quality-of-life scale Short Form-36 (SF-36)
Query!
Assessment method [5]
0
0
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.
Query!
Timepoint [5]
0
0
Baseline (Day 1) and Week 1 through Week 52
Query!
Secondary outcome [6]
0
0
Change from Baseline in percentage of Seizure-Free Days during treatment, based on seizure type
Query!
Assessment method [6]
0
0
Query!
Timepoint [6]
0
0
Baseline (Day 1) and Week 1 through Week 52
Query!
Secondary outcome [7]
0
0
Change from Baseline in Caregiver Global Impression of Change in Seizure Intensity/Duration (CGI-CSID)
Query!
Assessment method [7]
0
0
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.
Query!
Timepoint [7]
0
0
Baseline (Day 1) and Week 1 through Week 156
Query!
Eligibility
Key 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 (ß-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 Bâ„¢, 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.
Query!
Minimum age
1
Year
Query!
Query!
Maximum age
65
Years
Query!
Query!
Sex
Both males and females
Query!
Can healthy volunteers participate?
No
Query!
Key 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.
Query!
Study design
Purpose of the study
Treatment
Query!
Allocation to intervention
N/A
Query!
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Query!
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Query!
Masking / blinding
Open (masking not used)
Query!
Who is / are masked / blinded?
Query!
Query!
Query!
Query!
Intervention assignment
Single group
Query!
Other design features
Query!
Phase
Phase 3
Query!
Type of endpoint/s
Query!
Statistical methods / analysis
Query!
Recruitment
Recruitment status
Recruiting
Query!
Data analysis
Query!
Reason for early stopping/withdrawal
Query!
Other reasons
Query!
Date of first participant enrolment
Anticipated
Query!
Actual
16/05/2022
Query!
Date of last participant enrolment
Anticipated
Query!
Actual
Query!
Date of last data collection
Anticipated
1/06/2027
Query!
Actual
Query!
Sample size
Target
132
Query!
Accrual to date
Query!
Final
Query!
Recruitment in Australia
Recruitment state(s)
Query!
Recruitment hospital [1]
0
0
Austin Health - Heidelberg
Query!
Recruitment hospital [2]
0
0
Alfred Health - Melbourne
Query!
Recruitment hospital [3]
0
0
Royal Melbourne Hospital - Parkville
Query!
Recruitment hospital [4]
0
0
The Royal Children's Hospital Melbourne - Parkville
Query!
Recruitment postcode(s) [1]
0
0
VIC 3084 - Heidelberg
Query!
Recruitment postcode(s) [2]
0
0
VIC 3004 - Melbourne
Query!
Recruitment postcode(s) [3]
0
0
VIC 3050 - Parkville
Query!
Recruitment postcode(s) [4]
0
0
VIC 3052 - Parkville
Query!
Recruitment outside Australia
Country [1]
0
0
United States of America
Query!
State/province [1]
0
0
Arkansas
Query!
Country [2]
0
0
United States of America
Query!
State/province [2]
0
0
California
Query!
Country [3]
0
0
United States of America
Query!
State/province [3]
0
0
Colorado
Query!
Country [4]
0
0
United States of America
Query!
State/province [4]
0
0
Maryland
Query!
Country [5]
0
0
United States of America
Query!
State/province [5]
0
0
Minnesota
Query!
Country [6]
0
0
United States of America
Query!
State/province [6]
0
0
New York
Query!
Country [7]
0
0
United States of America
Query!
State/province [7]
0
0
North Carolina
Query!
Country [8]
0
0
United States of America
Query!
State/province [8]
0
0
Pennsylvania
Query!
Country [9]
0
0
United States of America
Query!
State/province [9]
0
0
South Carolina
Query!
Country [10]
0
0
United States of America
Query!
State/province [10]
0
0
Tennessee
Query!
Country [11]
0
0
United States of America
Query!
State/province [11]
0
0
Texas
Query!
Country [12]
0
0
Canada
Query!
State/province [12]
0
0
Montréal
Query!
Country [13]
0
0
Canada
Query!
State/province [13]
0
0
Toronto
Query!
Country [14]
0
0
Canada
Query!
State/province [14]
0
0
Vancouver
Query!
Country [15]
0
0
China
Query!
State/province [15]
0
0
Beijing
Query!
Country [16]
0
0
China
Query!
State/province [16]
0
0
Jilin
Query!
Country [17]
0
0
China
Query!
State/province [17]
0
0
Qingyang
Query!
Country [18]
0
0
France
Query!
State/province [18]
0
0
Bron
Query!
Country [19]
0
0
France
Query!
State/province [19]
0
0
Paris
Query!
Country [20]
0
0
France
Query!
State/province [20]
0
0
Rennes
Query!
Country [21]
0
0
France
Query!
State/province [21]
0
0
Strasbourg
Query!
Country [22]
0
0
Germany
Query!
State/province [22]
0
0
Bielefeld
Query!
Country [23]
0
0
Germany
Query!
State/province [23]
0
0
Bonn
Query!
Country [24]
0
0
Germany
Query!
State/province [24]
0
0
Frankfurt
Query!
Country [25]
0
0
Germany
Query!
State/province [25]
0
0
Freiburg
Query!
Country [26]
0
0
Germany
Query!
State/province [26]
0
0
Herdecke
Query!
Country [27]
0
0
Germany
Query!
State/province [27]
0
0
Radeberg
Query!
Country [28]
0
0
Israel
Query!
State/province [28]
0
0
Be'er Sheva
Query!
Country [29]
0
0
Israel
Query!
State/province [29]
0
0
Petah Tikva
Query!
Country [30]
0
0
Israel
Query!
State/province [30]
0
0
Tel Aviv
Query!
Country [31]
0
0
Italy
Query!
State/province [31]
0
0
Firenze
Query!
Country [32]
0
0
Italy
Query!
State/province [32]
0
0
Genova
Query!
Country [33]
0
0
Italy
Query!
State/province [33]
0
0
Rome
Query!
Country [34]
0
0
Spain
Query!
State/province [34]
0
0
Barcelona
Query!
Country [35]
0
0
Spain
Query!
State/province [35]
0
0
Madrid
Query!
Country [36]
0
0
Spain
Query!
State/province [36]
0
0
Málaga
Query!
Country [37]
0
0
Spain
Query!
State/province [37]
0
0
Valencia
Query!
Country [38]
0
0
United Kingdom
Query!
State/province [38]
0
0
Bristol
Query!
Country [39]
0
0
United Kingdom
Query!
State/province [39]
0
0
Oxford
Query!
Country [40]
0
0
United Kingdom
Query!
State/province [40]
0
0
Salford
Query!
Country [41]
0
0
United Kingdom
Query!
State/province [41]
0
0
Sheffield
Query!
Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Query!
Name
Marinus Pharmaceuticals
Query!
Address
Query!
Country
Query!
Ethics approval
Ethics application status
Query!
Summary
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
Query!
Trial website
https://clinicaltrials.gov/ct2/show/NCT05604170
Query!
Trial related presentations / publications
Query!
Public notes
Query!
Contacts
Principal investigator
Name
0
0
Query!
Address
0
0
Query!
Country
0
0
Query!
Phone
0
0
Query!
Fax
0
0
Query!
Email
0
0
Query!
Contact person for public queries
Name
0
0
Query!
Address
0
0
Query!
Country
0
0
Query!
Phone
0
0
Query!
Fax
0
0
Query!
Email
0
0
Query!
Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT05604170
Download to PDF