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

Phase 2

Completed

• Conditions: Tuberous Sclerosis
• Interventions: Drug: Ganaxolone

• Registration Number: NCT04285346
• Lead Sponsor: Marinus Pharmaceuticals

Brief 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.

Study Timeline

• Study First Submitted: 2020-02-18
• Study First Submitted QC: 2020-02-25
• Study First Posted: 2020-02-26
• Study Start: 2020-04-08
• Primary Completion: 2021-06-25
• Study Completion: 2022-08-30
• Status Verified: 2023-03
• Results First Submitted: 2023-03-08
• Results First Submitted QC: 2023-03-08
• Last Update Submitted: 2023-03-08
• Results First Posted: 2023-04-04
• Last Update Posted: 2023-04-04

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 23

Inclusion Criteria

Not provided

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Exclusion Criteria

Not provided

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Open-label	Ganaxolone	ganaxolone suspension (50 mg/ml) TID for 12 weeks with 24 week extension

Primary Outcome Measures

Name	Time	Method
Percent Change From Baseline in 28-day Seizure Frequency Through the End of 12-Week Treatment Period	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 the difference in post-Baseline 28-day seizure frequency and Baseline 28-day seizure frequency, divided by Baseline 28-day seizure frequency, multiplied by 100.

Secondary Outcome Measures

Name	Time	Method
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 Period	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.

Trial Locations

Locations (1)

Marinus Research Site; 🇺🇸 Houston, Texas, United States