Epilepsy, Complex Partial
Conditions
Keywords
Epileptic Seizures, Seizure Disorder, Seizures, Convulsions, Anticonvulsant, Anticonvulsive, Antiepileptic, Ganaxolone
Brief summary
This study is designed to provide ganaxolone to those patients deriving significant benefit from current treatment in protocol 1042-0601.
Interventions
DRUGganaxolone
liquid suspension, 50 mg/mL, up to 500 mg/day TID or 1500 mg/day, up to 1 year
Sponsors
Marinus Pharmaceuticals
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 55 Years
Healthy volunteers
No
Inclusion criteria
* subjects who have completed all study visits from previous protocol 1042-0601 and have been deemed eligible by the Investigator as: * having had no major adverse events thought to be drug related * deriving benefit from ganaxolone treatment * be properly informed of the nature and risks of the study and give written informed consent prior to study entry * must be willing to use a medically acceptable method of birth control and if female of child-bearing potential, have a negative qualitative serum pregnancy test
Exclusion criteria
* significant medical or surgical condition at screening or that develops during protocol participation that might compromise hematologic, cardiovascular, pulmonary, renal, gastrointestinal, or hepatic systems, or other conditions that would place the subject under increased risk * unwilling to use a double-barrier method of birth control or submit to a serum pregnancy test * history of chronic non-compliance with drug regimens * females currently breastfeeding * AST or ALT levels greater than 3 times the upper limit of normal at screen * Inability to withhold grapefruit or grapefruit products during the study
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Investigators Global Assessment | Screening through 52 weeks | Investigator's global assessment of the patient's response to ganaxolone treatment was collected during the study to support continued trial participation. The investigator's global assessment of the patient's response to ganaxolone treatment was collected during the study to support continued trial participation. Responses categories were Improved Markedly, Improved Moderately, Improved Slightly and No change from baseline. |
Countries
United States
Participant flow
Recruitment details
The study population was to have completed all scheduled clinical study visits in the previous protocol 1042-0601 and have been deemed eligible by the Investigator including no major adverse events \[AEs\] thought to be drug related and deriving benefit from ganaxolone treatment.
Participants by arm
| Arm | Count |
|---|---|
| Ganaxolone liquid suspension, 50 mg/mL, up to 500 mg/day TID or 1500 mg/day, up to 1 year | 11 |
| Total | 11 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Adverse Event | 1 |
| Overall Study | Lack of Efficacy | 1 |
| Overall Study | Withdrawal by Subject | 2 |
Baseline characteristics
| Characteristic | Ganaxolone |
|---|---|
| Age, Categorical <=18 years | 0 Participants |
| Age, Categorical >=65 years | 0 Participants |
| Age, Categorical Between 18 and 65 years | 11 Participants |
| Age, Continuous | 44.5 years STANDARD_DEVIATION 10.35 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 1 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 10 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 1 Participants |
| Race (NIH/OMB) White | 10 Participants |
| Sex: Female, Male Female | 8 Participants |
| Sex: Female, Male Male | 3 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 11 |
| other Total, other adverse events | 8 / 11 |
| serious Total, serious adverse events | 1 / 11 |
Outcome results
Primary
Investigators Global Assessment
Investigator's global assessment of the patient's response to ganaxolone treatment was collected during the study to support continued trial participation. The investigator's global assessment of the patient's response to ganaxolone treatment was collected during the study to support continued trial participation. Responses categories were Improved Markedly, Improved Moderately, Improved Slightly and No change from baseline.
Time frame: Screening through 52 weeks
Population: ITT Population
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Ganaxolone | Investigators Global Assessment | Improved Markedly - Visit 1 - Screening | 6 Participants |
| Ganaxolone | Investigators Global Assessment | Improved Moderately - Visit 1 - Screening | 4 Participants |
| Ganaxolone | Investigators Global Assessment | Improved Slightly - Visit 1 - Screening | 0 Participants |
| Ganaxolone | Investigators Global Assessment | No change from baseline - Visit 1 - Screening | 1 Participants |
| Ganaxolone | Investigators Global Assessment | Improved Markedly - Visit 2 - Week 26 | 3 Participants |
| Ganaxolone | Investigators Global Assessment | Improved Moderately - Visit 2 - Week 26 | 4 Participants |
| Ganaxolone | Investigators Global Assessment | Improved Slightly - Visit 2 - Week 26 | 0 Participants |
| Ganaxolone | Investigators Global Assessment | No change from baseline - Visit 2 - Week 26 | 0 Participants |
| Ganaxolone | Investigators Global Assessment | Improved Markedly - Visit 3 - Week 52 or Taper Visit | 2 Participants |
| Ganaxolone | Investigators Global Assessment | Improved Moderately - Visit 3 - Week 52 or Taper Visit | 4 Participants |
| Ganaxolone | Investigators Global Assessment | Improved Slightly - Visit 3 - Week 52 or Taper Visit | 1 Participants |
| Ganaxolone | Investigators Global Assessment | No change from baseline - Visit 3 - Week 52 or Taper Visit | 1 Participants |