Adults 2 to 65, any sex, with Tuberous Sclerosis. Patients with the condition only — healthy volunteers not accepted.
Results — posted to ClinicalTrials.gov
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Percent Change From Baseline in 28-day Seizure Frequency Through the End of 12-Week Treatment PeriodPrimary· Baseline and Up to Week 12
Primary seizures include atonic/drop, bilateral clonic, bilateral tonic, focal motor without impairment of consciousness or awareness, focal (motor or non-motor) with impairment of consciousness or awareness, focal to bilateral tonic-clonic, generalized tonic-clonic. Baseline 28-day seizure frequency was calculated as the total number of primary seizures in the Baseline period divided by the number of days with non-missing seizure data in the Baseline period, multiplied by 28. The Baseline Visit was defined as Week 0. Percent change from Baseline in 28-day seizure frequent was calculated as th
Group
Value
95% CI
Treatment Period: Ganaxolone
-16.61
-56.40 – 14.86
Percentage of Participants Experiencing a >=50 Percent Reduction in 28-day Primary Seizure Frequency Through the End of the 12-week Treatment Period Compared to the Baseline PeriodSecondary· Baseline and up to 12 Weeks
Primary seizures include atonic/drop, bilateral clonic, bilateral tonic, focal motor without impairment of consciousness or awareness, focal (motor or non-motor) with impairment of consciousness or awareness, focal to bilateral tonic-clonic, generalized tonic-clonic. Percentage of participants reporting \>=50 percent reduction in seizure frequency has been presented.
Group
Value
95% CI
Treatment Period: Ganaxolone
30.4
Adverse events — posted to ClinicalTrials.gov
Time frame: Up to Week 12 in Treatment period and From Week 12 to Week 38 in Open label period..
Reporting threshold: 0%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
To assess preliminary safety and efficacy of ganaxolone as adjunctive therapy for the treatment of primary seizure types in patients with genetically- or clinically-confirmed TSC-related epilepsy through the end of the 12 week treatment period.
Publications & conference data
3 peer-reviewed publications reference this trial (live from Europe PMC):
NCT05604170 — Open-label Study of Adjunctive GNX Treatment in Children and Adults With TSC-related Epilepsy
· Phase 3
· terminated
NCT05323734 — Adjunctive GNX Treatment Compared With Placebo in Children and Adults With TSC-related Epilepsy
· Phase 3
· completed
NCT04391569 — Randomized Therapy In Status Epilepticus
· Phase 3
· completed
NCT03865732 — Study of Adjunctive Ganaxolone Treatment in Female Children With Protocadherin 19 (PCDH19)-Related Epilepsy (Violet Stud
· Phase 2
· completed
NCT03460756 — A Clinical Trial of Oral Ganaxolone in Women With Postpartum Depression
· Phase 2
· completed
Other recruiting trials for Tuberous Sclerosis
Currently open trials in the same condition.
NCT06330350 — Qualitative Study in Patients With Genodermatoses and Healthcare Professionals on Reproductive Counselling
· recruiting
NCT06523582 — Genetic Bases of Neuroendocrine Neoplasms in Mexican Patients
· recruiting
NCT05161494 — Gait in Rare Diseases
· active not recruiting
NCT04463316 — GROWing Up With Rare GENEtic Syndromes
· recruiting
NCT03050268 — Familial Investigations of Childhood Cancer Predisposition
· recruiting
Other Marinus Pharmaceuticals trials
Trials by the same sponsor.
NCT05604170 — Open-label Study of Adjunctive GNX Treatment in Children and Adults With TSC-related Epilepsy
· Phase 3
· terminated
NCT05323734 — Adjunctive GNX Treatment Compared With Placebo in Children and Adults With TSC-related Epilepsy
· Phase 3
· completed
NCT04391569 — Randomized Therapy In Status Epilepticus
· Phase 3
· completed
NCT03865732 — Study of Adjunctive Ganaxolone Treatment in Female Children With Protocadherin 19 (PCDH19)-Related Epilepsy (Violet Stud
· Phase 2
· completed
NCT03572933 — Study of Adjunctive Ganaxolone Treatment in Children and Young Adults With CDKL5 Deficiency Disorder
· Phase 3
· completed
Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Marinus Pharmaceuticals
Last refreshed: 4 April 2023
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT04285346.