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A Phase 3, Open-label Study of Adjunctive Ganaxolone (GNX) Treatment in Children and Adults with Tuberous Sclerosis Complex (TSC)-related Epilepsy (TrustTSC OLE)
EU CTIS ID: 2022-503067-15-00
What this study is testing
To assess the long -term safety and tolerability of GNX as adjunctive therapy for seizures associate with TSC in children and adults.
- Therapeutic confirmatory (Phase III)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- 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.
- Parents/caregivers 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 IP (suspension) as directed with food 3 times a day (tid).
- WOCBP must be using a medically acceptable method of birth control and have a negative quantitative serum β-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 BTM, 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.
You likely can't join if
- 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.
See the full eligibility criteria
Who can join
- 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.
- Parents/caregivers 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 IP (suspension) as directed with food 3 times a day (tid).
- WOCBP must be using a medically acceptable method of birth control and have a negative quantitative serum β-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 BTM, 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.
What rules you out
- 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.
- 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.
The study team makes the final eligibility decision.
Where it's taking place
- United States
- Canada
- Israel
- Australia
- China
- United Kingdom
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18-64 years, 65+ years, 0-17 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include United States; Canada; Israel; Australia; China; United Kingdom. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.