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

• Status: Enrolling by invitation
• Trial end date: 2024-12-31
• Target enrollment: 0
• Participant gender: All

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

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Marinus Pharmaceuticals
• Treatments: Ganaxolone

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/caregiver or LAR willing 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.

3. Parent/caregiver is able and willing to maintain an accurate and complete daily seizure diary for the duration of the study.

4. Able and willing to take IP (suspension) as directed with food TID.

5. Sexually active females of childbearing potential must be using a medically acceptable method of birth control and have a negative quantitative serum β-HCG test collected at the initial visit.

6. Male participants must agree to take all necessary measures to avoid causing pregnancy in their sexual partners during the study and for 30 days after the last dose of IP. Medically acceptable contraceptives include surgical sterilization (such as a vasectomy) and a condom used with a spermicidal gel or foam. In male participants who are not sexually active, abstinence is an acceptable method of birth control.

Exclusion Criteria:

1. Pregnant or breastfeeding.

2. An active 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.