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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT05604170




Registration number
NCT05604170
Ethics application status
Date submitted
5/10/2022
Date registered
3/11/2022

Titles & IDs
Public title
Open-label Study of Adjunctive GNX Treatment in Children and Adults With TSC-related Epilepsy
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)
Secondary ID [1] 0 0
1042-TSC-3002
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Tuberous Sclerosis Complex 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Neurological 0 0 0 0
Other neurological disorders
Neurological 0 0 0 0
Epilepsy

Intervention/exposure
Study type
Interventional
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.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

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
Timepoint [1] 0 0
Week 1 through Week 160
Primary outcome [2] 0 0
Number of participants with abnormal vital signs
Timepoint [2] 0 0
Week 1 through Week 160
Primary outcome [3] 0 0
Number of participants with abnormal physical, neurological and developmental examination
Timepoint [3] 0 0
Week 1 through Week 160
Primary outcome [4] 0 0
Number of participants with abnormal 12-lead electrocardiogram (ECG) findings
Timepoint [4] 0 0
Week 1 through Week 160
Primary outcome [5] 0 0
Number of participants with abnormal clinical laboratory tests
Timepoint [5] 0 0
Week 1 through Week 156
Primary outcome [6] 0 0
Number of participants with abnormal Columbia-Suicide Severity Rating Scale (C-SSRS)
Timepoint [6] 0 0
Week 1 through Week 160
Secondary outcome [1] 0 0
Percent change from Baseline in 28-day seizure frequency during open label extension
Timepoint [1] 0 0
Baseline (Day 1) and Week 1 through Week 52
Secondary outcome [2] 0 0
Percent change from Baseline in 28-day seizure frequency during the long-term treatment
Timepoint [2] 0 0
Baseline (Day 1) and Week 1 through Week 52
Secondary outcome [3] 0 0
Number of participants who will be considered as Treatment Responders
Timepoint [3] 0 0
Week 1 through Week 52
Secondary outcome [4] 0 0
Number of Participants with Clinical Global Impression of Improvement (CGI-I)
Timepoint [4] 0 0
Week 1 through Week 156
Secondary outcome [5] 0 0
Change from Baseline in quality-of-life scale Short Form-36 (SF-36)
Timepoint [5] 0 0
Baseline (Day 1) and Week 1 through Week 52
Secondary outcome [6] 0 0
Change from Baseline in percentage of Seizure-Free Days during treatment, based on seizure type
Timepoint [6] 0 0
Baseline (Day 1) and Week 1 through Week 52
Secondary outcome [7] 0 0
Change from Baseline in Caregiver Global Impression of Change in Seizure Intensity/Duration (CGI-CSID)
Timepoint [7] 0 0
Baseline (Day 1) and Week 1 through Week 156

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 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.
Minimum age
1 Year
Maximum age
65 Years
Sex
Both males and females
Can healthy volunteers participate?
No
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.

Study design
Purpose of the study
Treatment
Allocation to intervention
NA
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
16/05/2022
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/06/2027
Actual
Sample size
Target
132
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Austin Health - Heidelberg
Recruitment hospital [2] 0 0
Alfred Health - Melbourne
Recruitment hospital [3] 0 0
Royal Melbourne Hospital - Parkville
Recruitment hospital [4] 0 0
The Royal Children's Hospital Melbourne - Parkville
Recruitment postcode(s) [1] 0 0
VIC 3084 - Heidelberg
Recruitment postcode(s) [2] 0 0
VIC 3004 - Melbourne
Recruitment postcode(s) [3] 0 0
VIC 3050 - Parkville
Recruitment postcode(s) [4] 0 0
VIC 3052 - Parkville
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Arkansas
Country [2] 0 0
United States of America
State/province [2] 0 0
California
Country [3] 0 0
United States of America
State/province [3] 0 0
Colorado
Country [4] 0 0
United States of America
State/province [4] 0 0
Maryland
Country [5] 0 0
United States of America
State/province [5] 0 0
Minnesota
Country [6] 0 0
United States of America
State/province [6] 0 0
New York
Country [7] 0 0
United States of America
State/province [7] 0 0
North Carolina
Country [8] 0 0
United States of America
State/province [8] 0 0
Pennsylvania
Country [9] 0 0
United States of America
State/province [9] 0 0
South Carolina
Country [10] 0 0
United States of America
State/province [10] 0 0
Tennessee
Country [11] 0 0
United States of America
State/province [11] 0 0
Texas
Country [12] 0 0
Canada
State/province [12] 0 0
Montréal
Country [13] 0 0
Canada
State/province [13] 0 0
Toronto
Country [14] 0 0
Canada
State/province [14] 0 0
Vancouver
Country [15] 0 0
China
State/province [15] 0 0
Beijing
Country [16] 0 0
China
State/province [16] 0 0
Jilin
Country [17] 0 0
China
State/province [17] 0 0
Qingyang
Country [18] 0 0
France
State/province [18] 0 0
Bron
Country [19] 0 0
France
State/province [19] 0 0
Paris
Country [20] 0 0
France
State/province [20] 0 0
Rennes
Country [21] 0 0
France
State/province [21] 0 0
Strasbourg
Country [22] 0 0
Germany
State/province [22] 0 0
Bielefeld
Country [23] 0 0
Germany
State/province [23] 0 0
Bonn
Country [24] 0 0
Germany
State/province [24] 0 0
Frankfurt
Country [25] 0 0
Germany
State/province [25] 0 0
Freiburg
Country [26] 0 0
Germany
State/province [26] 0 0
Herdecke
Country [27] 0 0
Germany
State/province [27] 0 0
Radeberg
Country [28] 0 0
Israel
State/province [28] 0 0
Be'er Sheva
Country [29] 0 0
Israel
State/province [29] 0 0
Petah Tikva
Country [30] 0 0
Israel
State/province [30] 0 0
Tel Aviv
Country [31] 0 0
Italy
State/province [31] 0 0
Firenze
Country [32] 0 0
Italy
State/province [32] 0 0
Genova
Country [33] 0 0
Italy
State/province [33] 0 0
Rome
Country [34] 0 0
Spain
State/province [34] 0 0
Barcelona
Country [35] 0 0
Spain
State/province [35] 0 0
Madrid
Country [36] 0 0
Spain
State/province [36] 0 0
Málaga
Country [37] 0 0
Spain
State/province [37] 0 0
Valencia
Country [38] 0 0
United Kingdom
State/province [38] 0 0
Bristol
Country [39] 0 0
United Kingdom
State/province [39] 0 0
Oxford
Country [40] 0 0
United Kingdom
State/province [40] 0 0
Salford
Country [41] 0 0
United Kingdom
State/province [41] 0 0
Sheffield

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Marinus Pharmaceuticals
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT05604170