TrustTSC OLE
Research type
Research Study
Full 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)
IRAS ID
1007254
Contact name
Michelle Morgan
Contact email
Sponsor organisation
Marinus Pharmaceuticals, Inc.
Eudract number
2021-003384-10
Research summary
The 1042-TSC-3002 study is a global, phase-III- open-label extension (OLE) study sponsored by Marinus Pharmaceuticals Inc. The purpose of this research study is to evaluate the long-term safety and efficacy of ganaxolone as adjunctive therapy for seizures associated with Tuberous Sclerosis Complex (TSC) in children and adults (2 to 65 years of age). Researchers want to learn if ganaxolone helps lower the number of seizures when seizures are not fully controlled with standard treatment strategies.
TSC is a rare, multi-system genetic disease, that can affect many organs through the overgrowth of benign tumours. Neurologic manifestations such as epilepsy can be seen in up to 90% of patients with TSC. Over 30% of patients develop treatment-refractory epilepsy.
Ganaxolone, also called ZTALMY®, is approved by the FDA in the United States to treat seizures in patients aged 2 years and older with CDKL5 deficiency disorder (CDD). Ganaxolone is not approved by the MHRA to treat seizures caused by other disorders.
This OLE study will include children and adults with TSC‑related epilepsy who completed either Study 1042‑TSC‑3001 or Study 1042-TSC-2001. The study may last up to 56 weeks for each participant.
The primary objective of 1042-TSC-3002 study is to assess the safety and tolerability of long-term ganaxolone treatment in children and adults with genetically or clinically confirmed TSC-related epilepsy. Participants will continue the GNX dose determined in Study 1042-TSC-3001 or Study 1042-TSC-2001 in this OLE unless a dose change is clinically indicated. Participants will take no more than 1800 milligrams per day of ganaxolone. Participants will take ganaxolone for up to 52 weeks. They will spend 2 or more additional weeks taking lower and lower doses of ganaxolone before stopping completely. Finally, they will attend a follow-up visit.REC name
London - West London & GTAC Research Ethics Committee
REC reference
23/LO/0237
Date of REC Opinion
18 Jul 2023
REC opinion
Further Information Favourable Opinion