NCT04285346

Adjunctive Ganaxolone Treatment (Part A) in TSC Followed by Long-term Treatment (Part B)

A Phase 2 Open-label 12-Week Trial of Adjunctive Ganaxolone Treatment (Part A) in Tuberous Sclerosis Complex-related Epilepsy Followed by Long-term Treatment (Part B)

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 23 (actual)
• Sponsor: Marinus Pharmaceuticals · Industry
• Sex: All
• Age: 2 Years – 65 Years
• Healthy volunteers: Not accepted

Summary

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.

Detailed description

This is an OL proof of concept study of adjunctive GNX treatment in patients with a confirmed clinical diagnosis of TSC and/or a mutation in either the TSC1 or TSC2 gene. The trial consists of two parts: Part A consists of a 4-week baseline period followed by a 12-week treatment period (4-week titration and 8-week maintenance). For patients not continuing in the 24-week OLE period (Part B), a 2-week taper period followed by a 2-week safety period would follow. The main difference between Part A and Part B is the length of treatment, less frequent assessments, and the ability to alter drug doses (both GNX and other antiepileptic drug \[AED\] treatments which includes initiating and stopping other medications) based on investigator evaluation of the patient's clinical course during Part B. Patients with a seizure frequency reduction during the 12-week treatment period in Part A compared to baseline may continue into Part B ("OLE eligible"), to assess long-term safety, efficacy and tolerability in patients with TSC-related Epilepsy.

Conditions

• Tuberous Sclerosis

Interventions

Type	Name	Description
DRUG	Ganaxolone	titration followed by maintenance and extension period

Timeline

• Start date: 2020-04-08
• Primary completion: 2021-06-25
• Completion: 2022-08-30
• First posted: 2020-02-26
• Last updated: 2023-04-04
• Results posted: 2023-04-04

Locations

7 sites across 1 country: United States

Regulatory

• FDA-regulated drug study