A Two-year Open-label Extension Study of Ganaxolone in Patients With Drug-resistant Partial-onset Seizures

NCT02519439 | Terminated Phase 3 Results

• 1 other identifier: 1042-0604
• Study Type: interventional
• Target: 26
• Locations: 1 country
• Sites: 5

Brief Summary

A follow-on, two-year open-label extension study of ganaxolone as add-on therapy in adult patients with drug-resistant partial-onset seizures

Conditions

Drug Resistant Partial Onset Seizure

Keywords

Partial Onset Seizures; complex partial seizures; simple partial seizures; anticonvulsant; ganaxolone; neurosteroid; Marinus; epilepsy

Outcome Measures

Primary Outcomes (1)

• Percent Change From Baseline in 28-day Seizure Frequency

  Baseline 28-day seizure frequency was calculated as the number of seizures in the Baseline period of Study 1042-0603 (less than or equal to 56 days) divided by the number of days with available seizure data in the Baseline period and multiplied by 28. Post-baseline 28-day seizure frequency was calculated as the number of seizures in the entire treatment period divided by the number of days with available seizure data in the treatment period and multiplied by 28. Baseline was defined as the last non-missing value obtained before the first treatment in the preceding Study 1042-0603. The calculation for percent change from Baseline in 28-day seizure frequency was done as follows for each participant: post-Baseline 28-day seizure frequency minus Baseline 28-day seizure frequency whole divided by Baseline 28-day seizure frequency multiplied by 100 percent.

  Baseline and at Day 28

Secondary Outcomes (3)

• Number of Participants Who Showed Greater Than or Equal to 50% Reduction in 28-day Seizure Frequent From Baseline

  Baseline and at Day 28

• Number of Participants With Clinical Global Impression of Improvement (CGI-I) Scores

  At Week 104

• Number of Participants With Patient/Caregiver Global Impression of Improvement (PGI-I) Scores

  At Week 104

Study Arms (1)

ganaxolone

EXPERIMENTAL

Up to a maximum of 1800 mg/day

Drug: ganaxolone

Interventions

ganaxolone DRUG

225 mg capsules 450 mg to 900 mg 2x/day

Also known as: CCD 1042

ganaxolone

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Subjects who have completed all scheduled clinical study visits in the previous protocol 1042-0603 and have shown a minimum 35% improvement in mean 28-day seizure frequency over the last three 28-day periods in study 1042-603 as compared to the baseline of study 1042-603.
• Subjects whose daily study drug compliance in Study 1042-0603 was 90% or greater, and for whom the investigator feels that the subject was compliant with the full dose as prescribed.
• Able to give informed consent in writing, or have a legally authorized representative able to do so, after being properly informed of the nature and risks of the study and prior to engaging in any study-related procedures.
• Currently being treated and maintained with a stable regimen of 1, 2, or 3 anti-epileptic drugs (AED) at a consistent dose for one month prior to study entry.
• Implanted Vagus Nerve Stimulator (VNS) is permitted and will not count towards the number of concomitant AEDs.
• Able and willing to maintain an accurate and complete daily written seizure calendar or has a caregiver who is able and willing to maintain an accurate and complete daily written seizure calendar.
• Able and willing to take drug with food twice daily. Ganaxolone must be administered with food.
• Sexually active women of childbearing potential (WCBP) must be using a medically acceptable method of birth control and have a negative pregnancy test at Visit 1 and at subsequent visits.

You may not qualify if:

• Have any medical condition that, in the investigator's judgment, is considered to be clinically significant and could potentially affect subject safety or study outcome
• Experienced a Serious Adverse Event or a moderate or severe medically important adverse event judged probably or definitely related to open-label ganaxolone in the previous study, 1042-0603
• Have Alanine transferase (ALT; SGPT) or Aspartate transferase (AST; SGOT) levels \> 3 times upper limits of normal (ULN), or total bilirubin \>1.5 time ULN during Study 1042-0603.
• Have a history of malignancy within the past 2 years, with the exception of basal cell carcinoma.
• Seizures secondary to illicit drug or alcohol use, infection, neoplasm, demyelinating disease, degenerative neurological disease, or central nervous system (CNS) disease deemed progressive, metabolic illness, or progressive degenerative disease.
• Have active suicidal plan/intent, or have had active suicidal thoughts in the past 6 months. Have a history of an actual suicide attempt in the last 5 years or more than 1 lifetime actual suicide attempt as classified by the Columbia-Suicide Severity Rating Scale (C-SSRS).
• Have a history of drug or alcohol abuse within the past 5 years. As with other AEDs, the use of alcohol is not advised.
• Are currently following or planning to follow a ketogenic diet.
• Current use of vigabatrin or ezogabine (retigabine; Potiga; Trobalt) is not permitted.
• Females who are pregnant, currently breastfeeding or planning to become pregnant during the study.
• Inability/unwillingness to withhold grapefruit and grapefruit juice from diet during the entire clinical trial.

Sponsors & Collaborators

• Marinus Pharmaceuticals: lead

Study Sites (5)

Neurological Research Institute

Santa Monica, California, 90404, United States

Location

Bluegrass Epilepsy Research, LLC

Lexington, Kentucky, 40504, United States

Location

Minneapolis Clinic of Neurology

Golden Valley, Minnesota, 55422, United States

Location

Northeast Regional Epilepsy Group

Hackensack, New Jersey, 07601, United States

Location

Texas Epilepsy Group

Dallas, Texas, 75251, United States

Location

MeSH Terms

Conditions

Seizures; Epilepsies, Partial; Epilepsy

Interventions

ganaxolone

Condition Hierarchy (Ancestors)

Neurologic Manifestations; Nervous System Diseases; Signs and Symptoms; Pathological Conditions, Signs and Symptoms; Brain Diseases; Central Nervous System Diseases

Results Point of Contact

• Title: Marinus Clinical Trials Submission Manager
• Organization: Marinus Pharmaceuticals, Inc.

clinicaltrials@marinuspharma.com

484-801-4670

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 4, 2015
• First Posted: August 11, 2015
• Study Start: February 1, 2015
• Primary Completion: December 1, 2016
• Study Completion: December 1, 2016
• Last Updated: June 28, 2023
• Results First Posted: February 14, 2023

Record last verified: 2023-06

Locations

US (5)