TrustTSC
Research type
Research Study
Full title
A Phase 3, Double-blind, Randomized, Placebo-controlled Trial of Adjunctive Ganaxolone (GNX) Treatment in Children and Adults with Tuberous Sclerosis Complex (TSC)-related Epilepsy (TrustTSC)
IRAS ID
1005562
Contact name
Regulatory Affairs
Contact email
Sponsor organisation
Marinus Pharmaceuticals Inc.
Eudract number
2021-003441-38
Clinicaltrials.gov Identifier
Research summary
The 1042-TSC-3001 study is a global phase-III-study sponsored by Marinus Pharmaceuticals Inc. It is an double-blind, randomized, placebo-controlled, multi-center study. The purpose of this research study is to evaluate the safety and efficacy of Ganaxolone compared to placebo as adjunctive therapy for seizures associated with Tuberous Sclerosis Complex (TSC) in children and adults. \nTSC is a rare, multi-system genetic disease, that can affect many organs through the overgrowth of benign tumors. Neurologic manifestations such as epilepsy can be seen in up to 90% of patients with TSC. Over 30% of patients develop treatment-refractory epilepsy. As shown by previous clinical studies Ganaxolone (GNX) has anticonvulsant activity with an acceptable safety and tolerability profile in the dose range of 900 to 1800 mg in adults and children, reduces seizures in children with infantile spasm (form of epilepsy).\nThe phase 2 study (protocol 1042-TSC-2001) showed a consistent safety profile, the primary outcome of a percent reduction in TSC-associated seizures showed a median value of 16.6%, and a 50% responder rate of 30.4%.\nThe primary objective of 1042-TSC-3001 study is to assess the preliminary safety and efficacy of GNX as adjunctive therapy for the treatment of seizures in participants aged 2 to 65 years with genetically or clinically confirmed TSC-related epilepsy. The study consists of a 4-week baseline followed by a treatment phase, consisting of a 4-week titration and 8-week maintenance period. \nFor participants not taking concomitant Epidiolex or taking Epidiolex at a dose of 10 mg/kg/day or less, the target dose of GNX (or matching placebo) is\n○ 63 mg/kg/day (weighing 28 kg or less), or\n○ 1800 mg/day (weighing more than 28 kg)\nFor participants taking concomitant Epidiolex at a dose greater than 10 mg/kg/day, the target dose of GNX (or matching volumes of placebo) is:\n○ 42 mg/kg/day (weighing 28 kg or less) or\n○ 1200 mg/day (weighing more than 28 kg)
REC name
South West - Central Bristol Research Ethics Committee
REC reference
22/SW/0079
Date of REC Opinion
4 Jul 2022
REC opinion
Further Information Favourable Opinion