Trial Outcomes & Findings for Intravenous Ganaxolone as Adjunctive Therapy to Treat Subjects With Status Epilepticus (NCT NCT03350035)
NCT ID: NCT03350035
Last Updated: 2023-03-09
Results Overview
Number of participants who did not require an intravenous (IV) Anesthetic Drug (a third-line Treatment) for Status Epilepticus (SE) within the first 24 hours after Study Drug Initiation.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
17 participants
Primary outcome timeframe
24 hours post study drug initiation
Results posted on
2023-03-09
Participant Flow
Participant milestones
| Measure |
Low - GNX
500 mg/day
|
Medium - GNX
650 mg/day
|
High - GNX
713 mg/day
|
|---|---|---|---|
|
Overall Study
STARTED
|
5
|
4
|
8
|
|
Overall Study
COMPLETED
|
3
|
4
|
7
|
|
Overall Study
NOT COMPLETED
|
2
|
0
|
1
|
Reasons for withdrawal
| Measure |
Low - GNX
500 mg/day
|
Medium - GNX
650 mg/day
|
High - GNX
713 mg/day
|
|---|---|---|---|
|
Overall Study
Lack of Efficacy
|
2
|
0
|
0
|
|
Overall Study
Subject discharged to hospice
|
0
|
0
|
1
|
Baseline Characteristics
Intravenous Ganaxolone as Adjunctive Therapy to Treat Subjects With Status Epilepticus
Baseline characteristics by cohort
| Measure |
Low - GNX
n=5 Participants
500 mg/day
|
Medium - GNX
n=4 Participants
650 mg/day
|
High - GNX
n=8 Participants
713 mg/day
|
Total
n=17 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Continuous
|
57.8 years
STANDARD_DEVIATION 25.35 • n=5 Participants
|
62.0 years
STANDARD_DEVIATION 18.96 • n=7 Participants
|
53.8 years
STANDARD_DEVIATION 17.04 • n=5 Participants
|
56.9 years
STANDARD_DEVIATION 19.15 • n=4 Participants
|
|
Sex: Female, Male
Female
|
5 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
9 Participants
n=4 Participants
|
|
Sex: Female, Male
Male
|
0 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
6 Participants
n=5 Participants
|
8 Participants
n=4 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
5 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
6 Participants
n=5 Participants
|
15 Participants
n=4 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
2 Participants
n=4 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Black or African American
|
1 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
5 Participants
n=4 Participants
|
|
Race (NIH/OMB)
White
|
3 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
4 Participants
n=5 Participants
|
9 Participants
n=4 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
3 Participants
n=4 Participants
|
PRIMARY outcome
Timeframe: 24 hours post study drug initiationPopulation: Safety Population: included all participants who received at least one dose of IV ganaxolone (GNX).
Number of participants who did not require an intravenous (IV) Anesthetic Drug (a third-line Treatment) for Status Epilepticus (SE) within the first 24 hours after Study Drug Initiation.
Outcome measures
| Measure |
Low - GNX
n=5 Participants
500 mg/day
|
Medium - GNX
n=4 Participants
650 mg/day
|
High - GNX
n=8 Participants
713 mg/day
|
|---|---|---|---|
|
Number of Participants Who Did Not Require an IV Anesthetic Drug for SE Treatment
|
5 Participants
|
4 Participants
|
8 Participants
|
SECONDARY outcome
Timeframe: Time to SE Cessation, assessed up to 24 hoursPopulation: Safety Population. Only participants were included if seizure was confirmed.
Summary of Time to SE Cessation
Outcome measures
| Measure |
Low - GNX
n=5 Participants
500 mg/day
|
Medium - GNX
n=4 Participants
650 mg/day
|
High - GNX
n=7 Participants
713 mg/day
|
|---|---|---|---|
|
Time to Cessation of SE
|
5.000 Minutes
Interval 2.45 to 6.05
|
5.580 Minutes
Interval 4.18 to 22.25
|
10.16 Minutes
Interval 1.34 to 241.0
|
SECONDARY outcome
Timeframe: Drug initiation through follow-up period, up to approximately 4 weeksPopulation: Safety Population
Number of participants who Required No Escalation of Treatment for Ongoing or Recurrent SE
Outcome measures
| Measure |
Low - GNX
n=5 Participants
500 mg/day
|
Medium - GNX
n=4 Participants
650 mg/day
|
High - GNX
n=8 Participants
713 mg/day
|
|---|---|---|---|
|
Number of Participants Who Required No Escalation of Treatment for Ongoing or Recurrent SE
|
3 Participants
|
3 Participants
|
8 Participants
|
SECONDARY outcome
Timeframe: Baseline (within 24hrs of treatment) through follow up period, up to approximately 4 weeks.Population: Safety Population
Number of participants with No SE Recurrence per Principal Investigator within 24hrs of starting treatment, during treatment period (excluding taper), during taper, during 24-hr follow-up period, and during follow-up period.
Outcome measures
| Measure |
Low - GNX
n=5 Participants
500 mg/day
|
Medium - GNX
n=4 Participants
650 mg/day
|
High - GNX
n=8 Participants
713 mg/day
|
|---|---|---|---|
|
Number of Participants With No SE Recurrence Per Principal Investigator
Within 24hrs of starting treatment
|
5 Participants
|
4 Participants
|
7 Participants
|
|
Number of Participants With No SE Recurrence Per Principal Investigator
During treatment period (excluding taper)
|
3 Participants
|
4 Participants
|
7 Participants
|
|
Number of Participants With No SE Recurrence Per Principal Investigator
During taper
|
3 Participants
|
2 Participants
|
7 Participants
|
|
Number of Participants With No SE Recurrence Per Principal Investigator
During 24-hr follow-up period
|
3 Participants
|
3 Participants
|
8 Participants
|
|
Number of Participants With No SE Recurrence Per Principal Investigator
During follow-up period
|
1 Participants
|
3 Participants
|
7 Participants
|
SECONDARY outcome
Timeframe: Baseline (Pre-dose) to <-24hrs (Post Dose)Population: Safety Population
Seizure Burden (%) Baseline and Percentage Change from Baseline by Time Point
Outcome measures
| Measure |
Low - GNX
n=5 Participants
500 mg/day
|
Medium - GNX
n=4 Participants
650 mg/day
|
High - GNX
n=8 Participants
713 mg/day
|
|---|---|---|---|
|
Seizure Burden
Change from Baseline at 0 to <- 1 Hour Post-Dose
|
-94.80 Percentage change from baseline
Standard Deviation 8.458
|
-74.63 Percentage change from baseline
Standard Deviation 25.247
|
-84.01 Percentage change from baseline
Standard Deviation 18.862
|
|
Seizure Burden
Change from Baseline at 0 to <- 4 Hour Post-Dose
|
-98.59 Percentage change from baseline
Standard Deviation 2.092
|
-71.75 Percentage change from baseline
Standard Deviation 33.498
|
-87.99 Percentage change from baseline
Standard Deviation 21.439
|
|
Seizure Burden
Change from Baseline at >4 to <- 8 Hours Post-Dose
|
-77.53 Percentage change from baseline
Standard Deviation 17.950
|
-59.80 Percentage change from baseline
Standard Deviation 48.352
|
-96.58 Percentage change from baseline
Standard Deviation 6.772
|
|
Seizure Burden
Change from Baseline at >8 to <- 16 Hours Post-Dose
|
-47.98 Percentage change from baseline
Standard Deviation 38.063
|
-53.29 Percentage change from baseline
Standard Deviation 44.490
|
-93.75 Percentage change from baseline
Standard Deviation 13.306
|
|
Seizure Burden
Change from Baseline at >16 to <- 24 Hours Post-Dose
|
-99.53 Percentage change from baseline
Standard Deviation 0.935
|
-48.25 Percentage change from baseline
Standard Deviation 45.475
|
-81.16 Percentage change from baseline
Standard Deviation 47.368
|
|
Seizure Burden
Change from Baseline at 0 to <- 24 Hours Post-Dose
|
-78.64 Percentage change from baseline
Standard Deviation 15.286
|
-56.10 Percentage change from baseline
Standard Deviation 42.183
|
-89.06 Percentage change from baseline
Standard Deviation 20.039
|
Adverse Events
Low - GNX
Serious events: 2 serious events
Other events: 5 other events
Deaths: 2 deaths
Medium - GNX
Serious events: 2 serious events
Other events: 3 other events
Deaths: 0 deaths
High - GNX
Serious events: 2 serious events
Other events: 7 other events
Deaths: 1 deaths
Serious adverse events
| Measure |
Low - GNX
n=5 participants at risk
500 mg/day
|
Medium - GNX
n=4 participants at risk
650 mg/day
|
High - GNX
n=8 participants at risk
713 mg/day
|
|---|---|---|---|
|
Nervous system disorders
Sedation
|
0.00%
0/5 • Baseline (within 24hrs of treatment) through follow up period, up to approximately 4 weeks
|
25.0%
1/4 • Baseline (within 24hrs of treatment) through follow up period, up to approximately 4 weeks
|
12.5%
1/8 • Baseline (within 24hrs of treatment) through follow up period, up to approximately 4 weeks
|
|
Nervous system disorders
Loss of consciousness
|
0.00%
0/5 • Baseline (within 24hrs of treatment) through follow up period, up to approximately 4 weeks
|
25.0%
1/4 • Baseline (within 24hrs of treatment) through follow up period, up to approximately 4 weeks
|
0.00%
0/8 • Baseline (within 24hrs of treatment) through follow up period, up to approximately 4 weeks
|
|
Gastrointestinal disorders
Intestinal perforation
|
0.00%
0/5 • Baseline (within 24hrs of treatment) through follow up period, up to approximately 4 weeks
|
0.00%
0/4 • Baseline (within 24hrs of treatment) through follow up period, up to approximately 4 weeks
|
12.5%
1/8 • Baseline (within 24hrs of treatment) through follow up period, up to approximately 4 weeks
|
|
Infections and infestations
Sepsis
|
20.0%
1/5 • Baseline (within 24hrs of treatment) through follow up period, up to approximately 4 weeks
|
0.00%
0/4 • Baseline (within 24hrs of treatment) through follow up period, up to approximately 4 weeks
|
0.00%
0/8 • Baseline (within 24hrs of treatment) through follow up period, up to approximately 4 weeks
|
|
Injury, poisoning and procedural complications
Fall
|
0.00%
0/5 • Baseline (within 24hrs of treatment) through follow up period, up to approximately 4 weeks
|
25.0%
1/4 • Baseline (within 24hrs of treatment) through follow up period, up to approximately 4 weeks
|
12.5%
1/8 • Baseline (within 24hrs of treatment) through follow up period, up to approximately 4 weeks
|
|
Injury, poisoning and procedural complications
Multiple fractures
|
0.00%
0/5 • Baseline (within 24hrs of treatment) through follow up period, up to approximately 4 weeks
|
25.0%
1/4 • Baseline (within 24hrs of treatment) through follow up period, up to approximately 4 weeks
|
0.00%
0/8 • Baseline (within 24hrs of treatment) through follow up period, up to approximately 4 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Pneumothorax
|
0.00%
0/5 • Baseline (within 24hrs of treatment) through follow up period, up to approximately 4 weeks
|
25.0%
1/4 • Baseline (within 24hrs of treatment) through follow up period, up to approximately 4 weeks
|
0.00%
0/8 • Baseline (within 24hrs of treatment) through follow up period, up to approximately 4 weeks
|
|
Surgical and medical procedures
Endotracheal intubation
|
20.0%
1/5 • Baseline (within 24hrs of treatment) through follow up period, up to approximately 4 weeks
|
0.00%
0/4 • Baseline (within 24hrs of treatment) through follow up period, up to approximately 4 weeks
|
0.00%
0/8 • Baseline (within 24hrs of treatment) through follow up period, up to approximately 4 weeks
|
|
Surgical and medical procedures
Withdrawal of life support
|
20.0%
1/5 • Baseline (within 24hrs of treatment) through follow up period, up to approximately 4 weeks
|
0.00%
0/4 • Baseline (within 24hrs of treatment) through follow up period, up to approximately 4 weeks
|
0.00%
0/8 • Baseline (within 24hrs of treatment) through follow up period, up to approximately 4 weeks
|
Other adverse events
| Measure |
Low - GNX
n=5 participants at risk
500 mg/day
|
Medium - GNX
n=4 participants at risk
650 mg/day
|
High - GNX
n=8 participants at risk
713 mg/day
|
|---|---|---|---|
|
Nervous system disorders
Somnolence
|
20.0%
1/5 • Baseline (within 24hrs of treatment) through follow up period, up to approximately 4 weeks
|
25.0%
1/4 • Baseline (within 24hrs of treatment) through follow up period, up to approximately 4 weeks
|
37.5%
3/8 • Baseline (within 24hrs of treatment) through follow up period, up to approximately 4 weeks
|
|
Nervous system disorders
Sedation
|
0.00%
0/5 • Baseline (within 24hrs of treatment) through follow up period, up to approximately 4 weeks
|
25.0%
1/4 • Baseline (within 24hrs of treatment) through follow up period, up to approximately 4 weeks
|
12.5%
1/8 • Baseline (within 24hrs of treatment) through follow up period, up to approximately 4 weeks
|
|
General disorders
Pain
|
20.0%
1/5 • Baseline (within 24hrs of treatment) through follow up period, up to approximately 4 weeks
|
25.0%
1/4 • Baseline (within 24hrs of treatment) through follow up period, up to approximately 4 weeks
|
0.00%
0/8 • Baseline (within 24hrs of treatment) through follow up period, up to approximately 4 weeks
|
|
Vascular disorders
Hypotension
|
40.0%
2/5 • Baseline (within 24hrs of treatment) through follow up period, up to approximately 4 weeks
|
0.00%
0/4 • Baseline (within 24hrs of treatment) through follow up period, up to approximately 4 weeks
|
25.0%
2/8 • Baseline (within 24hrs of treatment) through follow up period, up to approximately 4 weeks
|
|
Blood and lymphatic system disorders
Leukocytosis
|
0.00%
0/5 • Baseline (within 24hrs of treatment) through follow up period, up to approximately 4 weeks
|
0.00%
0/4 • Baseline (within 24hrs of treatment) through follow up period, up to approximately 4 weeks
|
25.0%
2/8 • Baseline (within 24hrs of treatment) through follow up period, up to approximately 4 weeks
|
|
Metabolism and nutrition disorders
Hypokalaemia
|
0.00%
0/5 • Baseline (within 24hrs of treatment) through follow up period, up to approximately 4 weeks
|
25.0%
1/4 • Baseline (within 24hrs of treatment) through follow up period, up to approximately 4 weeks
|
25.0%
2/8 • Baseline (within 24hrs of treatment) through follow up period, up to approximately 4 weeks
|
|
Metabolism and nutrition disorders
Hypocalcaemia
|
0.00%
0/5 • Baseline (within 24hrs of treatment) through follow up period, up to approximately 4 weeks
|
0.00%
0/4 • Baseline (within 24hrs of treatment) through follow up period, up to approximately 4 weeks
|
25.0%
2/8 • Baseline (within 24hrs of treatment) through follow up period, up to approximately 4 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Hypercaprnia
|
0.00%
0/5 • Baseline (within 24hrs of treatment) through follow up period, up to approximately 4 weeks
|
0.00%
0/4 • Baseline (within 24hrs of treatment) through follow up period, up to approximately 4 weeks
|
25.0%
2/8 • Baseline (within 24hrs of treatment) through follow up period, up to approximately 4 weeks
|
|
Infections and infestations
Pneumonia
|
0.00%
0/5 • Baseline (within 24hrs of treatment) through follow up period, up to approximately 4 weeks
|
25.0%
1/4 • Baseline (within 24hrs of treatment) through follow up period, up to approximately 4 weeks
|
12.5%
1/8 • Baseline (within 24hrs of treatment) through follow up period, up to approximately 4 weeks
|
|
Renal and urinary disorders
Haematuria
|
20.0%
1/5 • Baseline (within 24hrs of treatment) through follow up period, up to approximately 4 weeks
|
0.00%
0/4 • Baseline (within 24hrs of treatment) through follow up period, up to approximately 4 weeks
|
12.5%
1/8 • Baseline (within 24hrs of treatment) through follow up period, up to approximately 4 weeks
|
Additional Information
Marinus Clinical Trials Submission Manager
Marinus Pharmaceuticals, Inc.
Phone: 484-801-4670
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place