Trial Outcomes & Findings for Randomized Therapy In Status Epilepticus (NCT NCT04391569)
NCT ID: NCT04391569
Last Updated: 2025-05-29
Results Overview
SE cessation was determined by the investigator based on clinical and electroencephalography (EEG). Medications for the acute treatment of SE were defined as antiepileptic drugs (AEDs) administered to abort ongoing SE or prevent imminent recurrence of SE based on clinical or EEG evidence.
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
100 participants
Primary outcome timeframe
Up to 30 minutes
Results posted on
2025-05-29
Participant Flow
This was a double-blind, randomized, placebo-controlled study that evaluated the efficacy and safety of ganaxolone intravenous (IV) solution in status epilepticus (SE).
A total of 100 participants were enrolled in the study.
Participant milestones
| Measure |
Ganaxolone
Ganaxolone IV administered with a 30-milligrams (mg) bolus dose with initiation of a 36-hour continuous infusion followed by a 12-hour taper.
|
Placebo
Placebo IV administered with a bolus and infusion equivalent to volume of IV ganaxolone.
|
|---|---|---|
|
Overall Study
STARTED
|
51
|
49
|
|
Overall Study
COMPLETED
|
30
|
35
|
|
Overall Study
NOT COMPLETED
|
21
|
14
|
Reasons for withdrawal
| Measure |
Ganaxolone
Ganaxolone IV administered with a 30-milligrams (mg) bolus dose with initiation of a 36-hour continuous infusion followed by a 12-hour taper.
|
Placebo
Placebo IV administered with a bolus and infusion equivalent to volume of IV ganaxolone.
|
|---|---|---|
|
Overall Study
Death
|
15
|
10
|
|
Overall Study
Withdrawal by Subject
|
1
|
0
|
|
Overall Study
Lost to Follow-up
|
3
|
2
|
|
Overall Study
Lack of Efficacy
|
0
|
1
|
|
Overall Study
Participant discharged to hospice facility
|
0
|
1
|
|
Overall Study
Participant family decision
|
1
|
0
|
|
Overall Study
Participant discharged prematurely
|
1
|
0
|
Baseline Characteristics
Randomized Therapy In Status Epilepticus
Baseline characteristics by cohort
| Measure |
Ganaxolone
n=51 Participants
Ganaxolone IV administered with a 30-milligrams (mg) bolus dose with initiation of a 36-hour continuous infusion followed by a 12-hour taper
|
Placebo
n=49 Participants
Placebo IV administered with a bolus and infusion equivalent to volume of IV ganaxolone
|
Total
n=100 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
57.9 years
STANDARD_DEVIATION 19.40 • n=5 Participants
|
57.0 years
STANDARD_DEVIATION 19.03 • n=7 Participants
|
57.5 years
STANDARD_DEVIATION 19.13 • n=5 Participants
|
|
Sex: Female, Male
Female
|
21 Participants
n=5 Participants
|
19 Participants
n=7 Participants
|
40 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
30 Participants
n=5 Participants
|
30 Participants
n=7 Participants
|
60 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
7 Participants
n=5 Participants
|
6 Participants
n=7 Participants
|
13 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
43 Participants
n=5 Participants
|
43 Participants
n=7 Participants
|
86 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 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
|
|
Race (NIH/OMB)
Black or African American
|
13 Participants
n=5 Participants
|
14 Participants
n=7 Participants
|
27 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
34 Participants
n=5 Participants
|
32 Participants
n=7 Participants
|
66 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
4 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
7 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Up to 30 minutesPopulation: Intent-To-Treat (ITT) population comprised of all randomized participants in the double-blind phase of the study who received IP and had at least one non-missing efficacy assessment.
SE cessation was determined by the investigator based on clinical and electroencephalography (EEG). Medications for the acute treatment of SE were defined as antiepileptic drugs (AEDs) administered to abort ongoing SE or prevent imminent recurrence of SE based on clinical or EEG evidence.
Outcome measures
| Measure |
Ganaxolone
n=49 Participants
Ganaxolone IV administered with a 30-milligrams (mg) bolus dose with initiation of a 36-hour continuous infusion followed by a 12-hour taper.
|
Placebo
n=47 Participants
Placebo IV administered with a bolus and infusion equivalent to volume of IV ganaxolone.
|
|---|---|---|
|
Percentage of Participants With SE Cessation Within 30 Minutes of Investigational Product (IP) Initiation Without Medications for the Acute Treatment of SE
|
79.59 Percentage of participants
Interval 65.657 to 89.755
|
12.77 Percentage of participants
Interval 4.832 to 25.741
|
PRIMARY outcome
Timeframe: Up to 36 hours after IP initiationPopulation: ITT population
Percentage of participants with no progression to IV anesthesia for 36 hours following IP initiation SE cessation was based on investigator report with confirmation by assessment of concomitant medication data.
Outcome measures
| Measure |
Ganaxolone
n=49 Participants
Ganaxolone IV administered with a 30-milligrams (mg) bolus dose with initiation of a 36-hour continuous infusion followed by a 12-hour taper.
|
Placebo
n=47 Participants
Placebo IV administered with a bolus and infusion equivalent to volume of IV ganaxolone.
|
|---|---|---|
|
Percentage of Participants With no Progression to Intravenous (IV) Anesthesia for 36 Hours Following Investigational Product (IP) Initiation
|
63.27 Percentage of participants
Interval 48.288 to 76.578
|
51.06 Percentage of participants
Interval 36.064 to 65.924
|
PRIMARY outcome
Timeframe: Up to 4 weeks after IP initiationPopulation: Safety Population
An adverse event (AE) is any untoward medical occurrence in a clinical investigation participant who has been administered a pharmaceutical product; it does not necessarily have a causal relationship with this treatment. Treatment-emergent adverse event (TEAE) is defined as an AE that occurred or worsened at the time of or following IP initiation.
Outcome measures
| Measure |
Ganaxolone
n=51 Participants
Ganaxolone IV administered with a 30-milligrams (mg) bolus dose with initiation of a 36-hour continuous infusion followed by a 12-hour taper.
|
Placebo
n=49 Participants
Placebo IV administered with a bolus and infusion equivalent to volume of IV ganaxolone.
|
|---|---|---|
|
Number of Participants With Treatment Emergent Adverse Events
Any TEAE
|
48 Participants
|
43 Participants
|
|
Number of Participants With Treatment Emergent Adverse Events
Serious TEAEs
|
19 Participants
|
18 Participants
|
|
Number of Participants With Treatment Emergent Adverse Events
TEAE Leading to study drug discontinuation
|
1 Participants
|
2 Participants
|
|
Number of Participants With Treatment Emergent Adverse Events
TEAE leading to withdrawal
|
2 Participants
|
0 Participants
|
|
Number of Participants With Treatment Emergent Adverse Events
TEAE resulting in death
|
11 Participants
|
11 Participants
|
SECONDARY outcome
Timeframe: Up to 72 hours after IP initiationPopulation: ITT population
Percentage of participants with no progression to IV anesthesia for 72 hours following IP initiation SE cessation was assessed by the investigator based on clinical and EEG features.
Outcome measures
| Measure |
Ganaxolone
n=49 Participants
Ganaxolone IV administered with a 30-milligrams (mg) bolus dose with initiation of a 36-hour continuous infusion followed by a 12-hour taper.
|
Placebo
n=47 Participants
Placebo IV administered with a bolus and infusion equivalent to volume of IV ganaxolone.
|
|---|---|---|
|
Percentage of Participants With no Progression to IV Anesthesia for 72 Hours Following IP Initiation
|
51.02 Percentage of participants
Interval 36.338 to 65.575
|
46.81 Percentage of participants
Interval 32.112 to 61.922
|
SECONDARY outcome
Timeframe: Up to 72 hours after IP initiationPopulation: ITT population
Time to SE cessation was assessed for the first 72 hours following IP using the Kaplan-Meier method.
Outcome measures
| Measure |
Ganaxolone
n=49 Participants
Ganaxolone IV administered with a 30-milligrams (mg) bolus dose with initiation of a 36-hour continuous infusion followed by a 12-hour taper.
|
Placebo
n=47 Participants
Placebo IV administered with a bolus and infusion equivalent to volume of IV ganaxolone.
|
|---|---|---|
|
Time to SE Cessation Following IP Initiation
|
0.07 hours
Interval 0.0 to 5.4
|
2.85 hours
Interval 0.0 to 40.8
|
SECONDARY outcome
Timeframe: Up to 24 hours after IP initiationPopulation: Intent-To-Treat (ITT) population comprised of all randomized participants in the double-blind phase of the study who received IP and had at least one non-missing efficacy assessment.
SE cessation will be determined by the investigator based on clinical and EEG. Percentage of participants with any escalation of treatment in the first 24 hours following IP initiation, i.e. any medication other than IP administered for the acute treatment of SE in the first 24 hours following IP initiation was summarized
Outcome measures
| Measure |
Ganaxolone
n=49 Participants
Ganaxolone IV administered with a 30-milligrams (mg) bolus dose with initiation of a 36-hour continuous infusion followed by a 12-hour taper.
|
Placebo
n=47 Participants
Placebo IV administered with a bolus and infusion equivalent to volume of IV ganaxolone.
|
|---|---|---|
|
Percentage of Participants With Any Escalation of Treatment in the First 24 Hours Following IP Initiation
|
55.10 Percentage of participants
Interval 40.231 to 69.335
|
80.85 Percentage of participants
Interval 66.74 to 90.851
|
SECONDARY outcome
Timeframe: Up to 24 hours after IP initiationPopulation: ITT population. Participants with treatment escalation following IP initiation were analyzed.
For time to treatment escalation (any mediation used for acute treatment of SE), the estimates are based descriptive statistics on participants with treatment escalation in the first 24 hours following IP initiation. For participants without treatment escalation, the time to treatment escalation was censored at IP completion, or discontinuation from the study or death, whichever occurs earlier. The median time to treatment escalation has been presented.
Outcome measures
| Measure |
Ganaxolone
n=27 Participants
Ganaxolone IV administered with a 30-milligrams (mg) bolus dose with initiation of a 36-hour continuous infusion followed by a 12-hour taper.
|
Placebo
n=37 Participants
Placebo IV administered with a bolus and infusion equivalent to volume of IV ganaxolone.
|
|---|---|---|
|
Time to Treatment Escalation in the First 24 Hours Following IP Initiation
|
5.42 hours
Interval 0.5 to 21.6
|
2.32 hours
Interval 0.3 to 20.1
|
SECONDARY outcome
Timeframe: Up to 4 Weeks following IP initiationPopulation: ITT population. Participants who initiated anesthesia for SE treatment were analyzed.
Time to initiation of anesthesia for SE treatment through the final study follow-up visit/contact, were calculated based on descriptive statistics. The estimate was censored at study discontinuation, death, or last follow-up of the participant, whichever occurs first. The median time to initiation of anesthesia has been presented.
Outcome measures
| Measure |
Ganaxolone
n=22 Participants
Ganaxolone IV administered with a 30-milligrams (mg) bolus dose with initiation of a 36-hour continuous infusion followed by a 12-hour taper.
|
Placebo
n=25 Participants
Placebo IV administered with a bolus and infusion equivalent to volume of IV ganaxolone.
|
|---|---|---|
|
Time to Initiation of Anesthesia for SE Treatment Through the Final Study Follow-up Visit/Contact
|
15.00 hours
Interval 1.5 to 417.2
|
3.17 hours
Interval 0.6 to 202.7
|
SECONDARY outcome
Timeframe: Up to 4 Weeks following IP initiationPopulation: ITT population
Percentage of participants who developed SRSE through the final study follow-up visit/contact was provided for each treatment group.
Outcome measures
| Measure |
Ganaxolone
n=49 Participants
Ganaxolone IV administered with a 30-milligrams (mg) bolus dose with initiation of a 36-hour continuous infusion followed by a 12-hour taper.
|
Placebo
n=47 Participants
Placebo IV administered with a bolus and infusion equivalent to volume of IV ganaxolone.
|
|---|---|---|
|
Percentage of Participants Who Develop Super Refractory Status Epilepticus (SRSE) Through the Final Study Follow-up Visit/Contact
|
22.45 Percentage of participants
Interval 11.774 to 36.624
|
25.53 Percentage of participants
Interval 13.945 to 40.35
|
SECONDARY outcome
Timeframe: Up to 72 hours after IP initiationPopulation: ITT population. Participants with percent change from Baseline in seizure burden through 72 hours has been presented.
The seizure burden through 72 hours following IP initiation is described as the percent of time during which there is electrographic seizure activity from IP initiation to 72 hours. The change from baseline of seizure burden was summarized using descriptive statistics by treatment group. The baseline seizure burden is defined for 30 minutes prior to IP initiation.
Outcome measures
| Measure |
Ganaxolone
n=39 Participants
Ganaxolone IV administered with a 30-milligrams (mg) bolus dose with initiation of a 36-hour continuous infusion followed by a 12-hour taper.
|
Placebo
n=30 Participants
Placebo IV administered with a bolus and infusion equivalent to volume of IV ganaxolone.
|
|---|---|---|
|
Percent Change From Baseline in Seizure Burden Through 72 Hours Following IP Initiation
|
-59.1 Percent change
Standard Deviation 88.98
|
-93.9 Percent change
Standard Deviation 19.79
|
Adverse Events
Placebo
Serious events: 18 serious events
Other events: 39 other events
Deaths: 11 deaths
Ganaxolone
Serious events: 19 serious events
Other events: 43 other events
Deaths: 15 deaths
Serious adverse events
| Measure |
Placebo
n=49 participants at risk
Participants were randomized 1:1 to receive placebo bolus dose followed by continuous infusion for 36 hours, followed by 12-hour taper
|
Ganaxolone
n=51 participants at risk
Participants were randomized 1:1 to receive Ganaxolone at various intervals during Day 1 and Day 2.
|
|---|---|---|
|
Blood and lymphatic system disorders
Heparin-induced thrombocytopenia
|
2.0%
1/49 • Number of events 1 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
0.00%
0/51 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
|
Cardiac disorders
Cardiac arrest
|
2.0%
1/49 • Number of events 1 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
2.0%
1/51 • Number of events 1 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
|
Cardiac disorders
Cardio-respiratory arrest
|
0.00%
0/49 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
3.9%
2/51 • Number of events 2 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
|
Cardiac disorders
Sinus tachycardia
|
2.0%
1/49 • Number of events 1 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
0.00%
0/51 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
|
Gastrointestinal disorders
Dysphagia
|
0.00%
0/49 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
2.0%
1/51 • Number of events 1 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
|
Infections and infestations
Bacteraemia
|
2.0%
1/49 • Number of events 1 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
0.00%
0/51 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
|
Infections and infestations
Central nervous system infection
|
0.00%
0/49 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
2.0%
1/51 • Number of events 1 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
|
Infections and infestations
Pneumonia
|
4.1%
2/49 • Number of events 6 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
2.0%
1/51 • Number of events 6 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
|
Infections and infestations
Pneumonia klebsiella
|
2.0%
1/49 • Number of events 1 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
0.00%
0/51 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
|
Infections and infestations
Sepsis
|
0.00%
0/49 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
2.0%
1/51 • Number of events 1 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
|
Infections and infestations
Septic shock
|
4.1%
2/49 • Number of events 2 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
2.0%
1/51 • Number of events 1 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
|
Infections and infestations
Systemic candida
|
0.00%
0/49 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
2.0%
1/51 • Number of events 1 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
|
Infections and infestations
Urinary tract infection
|
0.00%
0/49 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
2.0%
1/51 • Number of events 1 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
|
Injury, poisoning and procedural complications
Subdural haematoma
|
6.1%
3/49 • Number of events 3 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
5.9%
3/51 • Number of events 3 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
|
Injury, poisoning and procedural complications
Vasoplegia syndrome
|
2.0%
1/49 • Number of events 1 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
0.00%
0/51 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
|
Metabolism and nutrition disorders
Acidosis
|
0.00%
0/49 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
2.0%
1/51 • Number of events 1 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
|
Metabolism and nutrition disorders
Hyponatraemia
|
2.0%
1/49 • Number of events 1 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
0.00%
0/51 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
|
Metabolism and nutrition disorders
Metabolic acidosis
|
2.0%
1/49 • Number of events 1 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
0.00%
0/51 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
|
Musculoskeletal and connective tissue disorders
Mobility decreased
|
2.0%
1/49 • Number of events 1 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
0.00%
0/51 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
|
Musculoskeletal and connective tissue disorders
Myopathy
|
0.00%
0/49 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
2.0%
1/51 • Number of events 1 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Metastases to meninges
|
2.0%
1/49 • Number of events 1 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
0.00%
0/51 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Metastatic neoplasm
|
2.0%
1/49 • Number of events 1 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
0.00%
0/51 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
|
Nervous system disorders
Coma
|
2.0%
1/49 • Number of events 1 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
0.00%
0/51 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
|
Nervous system disorders
Hyperammonaemic encephalopathy
|
2.0%
1/49 • Number of events 1 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
0.00%
0/51 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
|
Nervous system disorders
Sedation
|
0.00%
0/49 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
2.0%
1/51 • Number of events 1 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
|
Nervous system disorders
Status epilepticus
|
2.0%
1/49 • Number of events 1 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
3.9%
2/51 • Number of events 2 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
|
Psychiatric disorders
Delirium
|
2.0%
1/49 • Number of events 1 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
0.00%
0/51 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
|
Respiratory, thoracic and mediastinal disorders
Acute respiratory failure
|
0.00%
0/49 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
2.0%
1/51 • Number of events 1 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
0.00%
0/49 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
2.0%
1/51 • Number of events 1 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
|
Respiratory, thoracic and mediastinal disorders
Laryngeal oedema
|
0.00%
0/49 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
2.0%
1/51 • Number of events 1 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory failure
|
6.1%
3/49 • Number of events 3 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
9.8%
5/51 • Number of events 5 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
|
Skin and subcutaneous tissue disorders
Rash
|
2.0%
1/49 • Number of events 1 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
0.00%
0/51 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
|
Vascular disorders
Deep vein thrombosis
|
2.0%
1/49 • Number of events 1 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
0.00%
0/51 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
|
Vascular disorders
Hypotension
|
2.0%
1/49 • Number of events 1 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
9.8%
5/51 • Number of events 5 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
|
Vascular disorders
Shock
|
0.00%
0/49 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
2.0%
1/51 • Number of events 1 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
Other adverse events
| Measure |
Placebo
n=49 participants at risk
Participants were randomized 1:1 to receive placebo bolus dose followed by continuous infusion for 36 hours, followed by 12-hour taper
|
Ganaxolone
n=51 participants at risk
Participants were randomized 1:1 to receive Ganaxolone at various intervals during Day 1 and Day 2.
|
|---|---|---|
|
Infections and infestations
Urinary tract infection
|
8.2%
4/49 • Number of events 4 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
19.6%
10/51 • Number of events 10 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
|
Infections and infestations
Pneumonia
|
0.00%
0/49 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
9.8%
5/51 • Number of events 6 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
|
Infections and infestations
Pneumonia serratia
|
6.1%
3/49 • Number of events 3 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
0.00%
0/51 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
|
Infections and infestations
Staphylococcal infection
|
6.1%
3/49 • Number of events 3 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
0.00%
0/51 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
|
Infections and infestations
Klebsiella infection
|
4.1%
2/49 • Number of events 2 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
0.00%
0/51 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
|
Vascular disorders
Hypotension
|
16.3%
8/49 • Number of events 9 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
23.5%
12/51 • Number of events 13 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
|
Vascular disorders
Deep vein thrombosis
|
4.1%
2/49 • Number of events 2 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
2.0%
1/51 • Number of events 1 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
|
Vascular disorders
Shock
|
4.1%
2/49 • Number of events 2 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
2.0%
1/51 • Number of events 1 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory failure
|
4.1%
2/49 • Number of events 2 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
3.9%
2/51 • Number of events 2 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
0.00%
0/49 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
9.8%
5/51 • Number of events 5 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonia aspiration
|
2.0%
1/49 • Number of events 1 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
7.8%
4/51 • Number of events 4 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
|
Metabolism and nutrition disorders
Hyperglycaemia
|
8.2%
4/49 • Number of events 5 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
3.9%
2/51 • Number of events 2 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
|
Metabolism and nutrition disorders
Hypokalaemia
|
10.2%
5/49 • Number of events 5 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
2.0%
1/51 • Number of events 1 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
|
Metabolism and nutrition disorders
Hypophosphataemia
|
6.1%
3/49 • Number of events 3 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
5.9%
3/51 • Number of events 3 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
|
Metabolism and nutrition disorders
Hypernatraemia
|
8.2%
4/49 • Number of events 4 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
2.0%
1/51 • Number of events 1 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
|
Metabolism and nutrition disorders
Hyponatraemia
|
4.1%
2/49 • Number of events 2 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
3.9%
2/51 • Number of events 2 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
|
Metabolism and nutrition disorders
Hypoalbuminaemia
|
4.1%
2/49 • Number of events 2 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
3.9%
2/51 • Number of events 2 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
|
Metabolism and nutrition disorders
Hypocalcaemia
|
6.1%
3/49 • Number of events 3 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
2.0%
1/51 • Number of events 1 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
|
Metabolism and nutrition disorders
Hyperammonaemia
|
4.1%
2/49 • Number of events 2 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
2.0%
1/51 • Number of events 1 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
|
Metabolism and nutrition disorders
Hyperchloraemia
|
4.1%
2/49 • Number of events 2 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
2.0%
1/51 • Number of events 1 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
|
Metabolism and nutrition disorders
Hypoglycaemia
|
6.1%
3/49 • Number of events 3 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
0.00%
0/51 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
|
Metabolism and nutrition disorders
Metabolic alkalosis
|
4.1%
2/49 • Number of events 2 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
0.00%
0/51 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
|
General disorders
Pyrexia
|
16.3%
8/49 • Number of events 9 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
7.8%
4/51 • Number of events 4 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
|
General disorders
Hypothermia
|
4.1%
2/49 • Number of events 3 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
9.8%
5/51 • Number of events 5 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
|
General disorders
Generalised oedema
|
2.0%
1/49 • Number of events 1 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
3.9%
2/51 • Number of events 2 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
|
Injury, poisoning and procedural complications
Skin wound
|
0.00%
0/49 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
3.9%
2/51 • Number of events 4 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
|
Blood and lymphatic system disorders
Leukocytosis
|
6.1%
3/49 • Number of events 3 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
7.8%
4/51 • Number of events 4 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
|
Blood and lymphatic system disorders
Anaemia
|
6.1%
3/49 • Number of events 3 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
5.9%
3/51 • Number of events 3 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
|
Blood and lymphatic system disorders
Thrombocytopenia
|
8.2%
4/49 • Number of events 4 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
0.00%
0/51 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
|
Cardiac disorders
Bradycardia
|
6.1%
3/49 • Number of events 3 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
7.8%
4/51 • Number of events 4 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
|
Cardiac disorders
Tachycardia
|
6.1%
3/49 • Number of events 6 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
5.9%
3/51 • Number of events 4 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
|
Gastrointestinal disorders
Dysphagia
|
6.1%
3/49 • Number of events 3 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
3.9%
2/51 • Number of events 2 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
|
Gastrointestinal disorders
Vomiting
|
4.1%
2/49 • Number of events 2 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
5.9%
3/51 • Number of events 3 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
|
Investigations
Transaminases increased
|
8.2%
4/49 • Number of events 4 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
3.9%
2/51 • Number of events 2 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
|
Renal and urinary disorders
Acute kidney injury
|
6.1%
3/49 • Number of events 3 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
3.9%
2/51 • Number of events 2 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
|
Renal and urinary disorders
Urinary retention
|
2.0%
1/49 • Number of events 1 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
5.9%
3/51 • Number of events 3 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
|
Renal and urinary disorders
Haematuria
|
2.0%
1/49 • Number of events 1 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
3.9%
2/51 • Number of events 2 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
|
Psychiatric disorders
Agitation
|
2.0%
1/49 • Number of events 1 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
3.9%
2/51 • Number of events 2 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
|
Psychiatric disorders
Delirium
|
4.1%
2/49 • Number of events 2 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
0.00%
0/51 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
|
Psychiatric disorders
Insomnia
|
4.1%
2/49 • Number of events 2 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
0.00%
0/51 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
|
Skin and subcutaneous tissue disorders
Decubitus ulcer
|
4.1%
2/49 • Number of events 2 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
0.00%
0/51 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
|
Cardiac disorders
Atrial fibrillation
|
0.00%
0/49 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
3.9%
2/51 • Number of events 2 • Up to 4 Weeks
Serious adverse events and non-serious adverse events were collected in Safety Population.
|
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
Restriction type: OTHER