Trial Outcomes & Findings for A Clinical Trial of Intravenous (IV) Ganaxolone in Women With Postpartum Depression (NCT NCT03228394)
NCT ID: NCT03228394
Last Updated: 2023-07-19
Results Overview
The HAMD17 was a 17-item clinician-rated instrument used to assess the range of symptoms that were most frequently observed in participants with major depression. All items were scored on an ordinal scale between 0 and 4 (8 items) or 0 and 2 (9 items) of increasing severity. Each of 17 items was rated by clinician with rating of 0: absent, 1: doubtful to mild, 2: mild to moderate, 3: moderate to severe, and 4: very severe. A total score (0 to 52) was calculated by adding scores of all 17 items, where 0 indicated no depression and 52 indicated severe depression. Higher score represented more severe condition. Baseline was defined as the Day 1 assessment before study drug infusion. Change from Baseline was calculated by subtracting Baseline value from the post-dose visit value.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
91 participants
Primary outcome timeframe
Baseline and Day 3 post-infusion (60 hours post start of infusion) for Cohorts 1 to 3 and Baseline and Day 29 for Cohort 6
Results posted on
2023-07-19
Participant Flow
Participant milestones
| Measure |
Placebo Cohort 1
Intravenous (IV) infusion of placebo (PBO) at rate of 16 milliliter per hour (mL/h) for 48 hours, then 8 mL/h for 12 hours
|
Ganaxolone Cohort 1
IV infusion of ganaxolone (GNX) at rate of 4 milligram per hour (mg/h) (16 mL/h) for 48 hours, then 2 mg/h for 12 hours
|
Placebo Cohort 2
IV infusion of PBO at rate of 16 mL/h for 48 hours, then 8 mL/h for 12 hours.
|
Ganaxolone Cohort 2
IV infusion of GNX at rate of 8 mg/h for 48 hours, then 4 mg/h (8 mL/h) for 12 hours
|
Placebo Cohort 3
IV bolus of 24 mL PBO over 2 minutes; then 24 mL for 48 hours and then 12 mL/h for 12 hours.
|
Ganaxolone Cohort 3
IV bolus of 12 mg (24mL) GNX over 2 minutes; then GNX at 12 mg/h (24 mL/h) for 48 hours and then 6 mg/h (12 mL/h) for 12 hours.
|
Placebo Cohort 6
IV infusion of PBO at rate of 40 mL/h for 6 hours; then 900 mg PBO (4 capsules) orally at dinner for 28 days
|
Ganaxolone Cohort 6
IV infusion of GNX at rate of 20 mg/h (40 mL/h) for 6 hours followed by 900 mg (4 capsules) orally at dinner for 28 days
|
|---|---|---|---|---|---|---|---|---|
|
Overall Study
STARTED
|
4
|
5
|
14
|
15
|
10
|
10
|
17
|
16
|
|
Overall Study
COMPLETED
|
4
|
5
|
12
|
13
|
10
|
10
|
12
|
13
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
2
|
2
|
0
|
0
|
5
|
3
|
Reasons for withdrawal
| Measure |
Placebo Cohort 1
Intravenous (IV) infusion of placebo (PBO) at rate of 16 milliliter per hour (mL/h) for 48 hours, then 8 mL/h for 12 hours
|
Ganaxolone Cohort 1
IV infusion of ganaxolone (GNX) at rate of 4 milligram per hour (mg/h) (16 mL/h) for 48 hours, then 2 mg/h for 12 hours
|
Placebo Cohort 2
IV infusion of PBO at rate of 16 mL/h for 48 hours, then 8 mL/h for 12 hours.
|
Ganaxolone Cohort 2
IV infusion of GNX at rate of 8 mg/h for 48 hours, then 4 mg/h (8 mL/h) for 12 hours
|
Placebo Cohort 3
IV bolus of 24 mL PBO over 2 minutes; then 24 mL for 48 hours and then 12 mL/h for 12 hours.
|
Ganaxolone Cohort 3
IV bolus of 12 mg (24mL) GNX over 2 minutes; then GNX at 12 mg/h (24 mL/h) for 48 hours and then 6 mg/h (12 mL/h) for 12 hours.
|
Placebo Cohort 6
IV infusion of PBO at rate of 40 mL/h for 6 hours; then 900 mg PBO (4 capsules) orally at dinner for 28 days
|
Ganaxolone Cohort 6
IV infusion of GNX at rate of 20 mg/h (40 mL/h) for 6 hours followed by 900 mg (4 capsules) orally at dinner for 28 days
|
|---|---|---|---|---|---|---|---|---|
|
Overall Study
Lost to Follow-up
|
0
|
0
|
1
|
1
|
0
|
0
|
2
|
0
|
|
Overall Study
Withdrawal by Subject
|
0
|
0
|
1
|
0
|
0
|
0
|
1
|
3
|
|
Overall Study
Protocol Violation
|
0
|
0
|
0
|
1
|
0
|
0
|
0
|
0
|
|
Overall Study
Physician Decision
|
0
|
0
|
0
|
0
|
0
|
0
|
1
|
0
|
|
Overall Study
Noncompliance
|
0
|
0
|
0
|
0
|
0
|
0
|
1
|
0
|
Baseline Characteristics
A Clinical Trial of Intravenous (IV) Ganaxolone in Women With Postpartum Depression
Baseline characteristics by cohort
| Measure |
Placebo Cohort 1
n=4 Participants
IV infusion of PBO at rate of 16 mL/h for 48 hours, then 8 mL/h for 12 hours
|
Ganaxolone Cohort 1
n=5 Participants
IV infusion of GNX at rate of 4 mg/h (16 mL/h) for 48 hours, then 2 mg/h for 12 hours
|
Placebo Cohort 2
n=14 Participants
IV infusion of PBO at rate of 16 mL/h for 48 hours, then 8 mL/h for 12 hours
|
Ganaxolone Cohort 2
n=15 Participants
IV infusion of GNX at rate of 8 mg/h for 48 hours, then 4 mg/h (8 mL/h) for 12 hours
|
Placebo Cohort 3
n=10 Participants
IV bolus of 24 mL PBO over 2 minutes; then 24 mL/h for 48 hours and then 12 mL/h for 12 hours
|
Ganaxolone Cohort 3
n=10 Participants
IV bolus of 12 mg (24mL) GNX over 2 minutes; then GNX at 12 mg/h (24 mL/h) for 48 hours and then 6 mg/h (12 mL/h) for 12 hours
|
Placebo Cohort 6
n=17 Participants
IV infusion of PBO at rate of 40 mL/h for 6 hours; then 900 mg PBO (4 capsules) orally at dinner for 28 days
|
Ganaxolone Cohort 6
n=16 Participants
IV infusion of GNX at rate of 20 mg/h (40 mL/h) for 6 hours followed by 900 mg (4 capsules) orally at dinner for 28 days
|
Total
n=91 Participants
Total of all reporting groups
|
|---|---|---|---|---|---|---|---|---|---|
|
Age, Continuous
|
25.3 years
STANDARD_DEVIATION 2.22 • n=39 Participants
|
24.4 years
STANDARD_DEVIATION 6.27 • n=41 Participants
|
26.4 years
STANDARD_DEVIATION 6.02 • n=35 Participants
|
27.0 years
STANDARD_DEVIATION 7.11 • n=31 Participants
|
26.4 years
STANDARD_DEVIATION 5.70 • n=146 Participants
|
25.0 years
STANDARD_DEVIATION 4.11 • n=19 Participants
|
24.9 years
STANDARD_DEVIATION 5.10 • n=147 Participants
|
28.8 years
STANDARD_DEVIATION 4.97 • n=193 Participants
|
26.30 years
STANDARD_DEVIATION 3.495
|
|
Sex: Female, Male
Female
|
4 Participants
n=39 Participants
|
5 Participants
n=41 Participants
|
14 Participants
n=35 Participants
|
15 Participants
n=31 Participants
|
10 Participants
n=146 Participants
|
10 Participants
n=19 Participants
|
17 Participants
n=147 Participants
|
16 Participants
n=193 Participants
|
91 Participants
|
|
Sex: Female, Male
Male
|
0 Participants
n=39 Participants
|
0 Participants
n=41 Participants
|
0 Participants
n=35 Participants
|
0 Participants
n=31 Participants
|
0 Participants
n=146 Participants
|
0 Participants
n=19 Participants
|
0 Participants
n=147 Participants
|
0 Participants
n=193 Participants
|
0 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
2 Participants
n=39 Participants
|
1 Participants
n=41 Participants
|
0 Participants
n=35 Participants
|
1 Participants
n=31 Participants
|
0 Participants
n=146 Participants
|
1 Participants
n=19 Participants
|
0 Participants
n=147 Participants
|
2 Participants
n=193 Participants
|
7 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
2 Participants
n=39 Participants
|
4 Participants
n=41 Participants
|
14 Participants
n=35 Participants
|
14 Participants
n=31 Participants
|
10 Participants
n=146 Participants
|
9 Participants
n=19 Participants
|
17 Participants
n=147 Participants
|
14 Participants
n=193 Participants
|
84 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=39 Participants
|
0 Participants
n=41 Participants
|
0 Participants
n=35 Participants
|
0 Participants
n=31 Participants
|
0 Participants
n=146 Participants
|
0 Participants
n=19 Participants
|
0 Participants
n=147 Participants
|
0 Participants
n=193 Participants
|
0 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=39 Participants
|
0 Participants
n=41 Participants
|
0 Participants
n=35 Participants
|
0 Participants
n=31 Participants
|
1 Participants
n=146 Participants
|
0 Participants
n=19 Participants
|
1 Participants
n=147 Participants
|
0 Participants
n=193 Participants
|
2 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=39 Participants
|
0 Participants
n=41 Participants
|
0 Participants
n=35 Participants
|
0 Participants
n=31 Participants
|
0 Participants
n=146 Participants
|
0 Participants
n=19 Participants
|
0 Participants
n=147 Participants
|
0 Participants
n=193 Participants
|
0 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=39 Participants
|
0 Participants
n=41 Participants
|
0 Participants
n=35 Participants
|
1 Participants
n=31 Participants
|
0 Participants
n=146 Participants
|
0 Participants
n=19 Participants
|
0 Participants
n=147 Participants
|
0 Participants
n=193 Participants
|
1 Participants
|
|
Race (NIH/OMB)
Black or African American
|
1 Participants
n=39 Participants
|
2 Participants
n=41 Participants
|
8 Participants
n=35 Participants
|
10 Participants
n=31 Participants
|
7 Participants
n=146 Participants
|
8 Participants
n=19 Participants
|
12 Participants
n=147 Participants
|
8 Participants
n=193 Participants
|
56 Participants
|
|
Race (NIH/OMB)
White
|
3 Participants
n=39 Participants
|
3 Participants
n=41 Participants
|
5 Participants
n=35 Participants
|
4 Participants
n=31 Participants
|
2 Participants
n=146 Participants
|
1 Participants
n=19 Participants
|
4 Participants
n=147 Participants
|
8 Participants
n=193 Participants
|
30 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=39 Participants
|
0 Participants
n=41 Participants
|
0 Participants
n=35 Participants
|
0 Participants
n=31 Participants
|
0 Participants
n=146 Participants
|
0 Participants
n=19 Participants
|
0 Participants
n=147 Participants
|
0 Participants
n=193 Participants
|
0 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=39 Participants
|
0 Participants
n=41 Participants
|
1 Participants
n=35 Participants
|
0 Participants
n=31 Participants
|
0 Participants
n=146 Participants
|
1 Participants
n=19 Participants
|
0 Participants
n=147 Participants
|
0 Participants
n=193 Participants
|
2 Participants
|
PRIMARY outcome
Timeframe: Baseline and Day 3 post-infusion (60 hours post start of infusion) for Cohorts 1 to 3 and Baseline and Day 29 for Cohort 6Population: Modified intent-to-treat (mITT) population: Included all participants in the safety set who had ≥1 post-randomization efficacy assessment. Only those participants with data available at the specified data points were analyzed.
The HAMD17 was a 17-item clinician-rated instrument used to assess the range of symptoms that were most frequently observed in participants with major depression. All items were scored on an ordinal scale between 0 and 4 (8 items) or 0 and 2 (9 items) of increasing severity. Each of 17 items was rated by clinician with rating of 0: absent, 1: doubtful to mild, 2: mild to moderate, 3: moderate to severe, and 4: very severe. A total score (0 to 52) was calculated by adding scores of all 17 items, where 0 indicated no depression and 52 indicated severe depression. Higher score represented more severe condition. Baseline was defined as the Day 1 assessment before study drug infusion. Change from Baseline was calculated by subtracting Baseline value from the post-dose visit value.
Outcome measures
| Measure |
Placebo Cohort 1
n=4 Participants
IV infusion of PBO at rate of 16 mL/h for 48 hours, then 8 mL/h for 12 hours
|
Ganaxolone Cohort 1
n=5 Participants
IV infusion of GNX at rate of 4 mg/h (16 mL/h) for 48 hours, then 2 mg/h for 12 hours
|
Placebo Cohort 2
n=12 Participants
IV infusion of PBO at rate of 16 mL/h for 48 hours, then 8 mL/h for 12 hours
|
Ganaxolone Cohort 2
n=12 Participants
IV infusion of GNX at rate of 8 mg/h for 48 hours, then 4 mg/h (8 mL/h) for 12 hours
|
Placebo Cohort 3
n=10 Participants
IV bolus of 24 mL PBO over 2 minutes; then 24 mL/h for 48 hours and then 12 mL/h for 12 hours
|
Ganaxolone Cohort 3
n=9 Participants
IV bolus of 12 mg (24mL) GNX over 2 minutes; then GNX at 12 mg/h (24 mL/h) for 48 hours and then 6 mg/h (12 mL/h) for 12 hours
|
Placebo Cohort 6
n=14 Participants
IV infusion of PBO at rate of 40 mL/h for 6 hours; then 900 mg PBO (4 capsules) orally at dinner for 28 days
|
Ganaxolone Cohort 6
n=15 Participants
IV infusion of GNX at rate of 20 mg/h (40 mL/h) for 6 hours followed by 900 mg (4 capsules) orally at dinner for 28 days
|
|---|---|---|---|---|---|---|---|---|
|
Change From Baseline in Hamilton Depression Rating Scale 17-item Version (HAMD17) Total Score
Day 3 post-infusion (60 hours post start of infusion)
|
-13.0 Scores on a scale
Standard Deviation 10.10
|
-12.0 Scores on a scale
Standard Deviation 6.82
|
-14.0 Scores on a scale
Standard Deviation 9.10
|
-14.3 Scores on a scale
Standard Deviation 10.23
|
-11.0 Scores on a scale
Standard Deviation 6.58
|
-11.3 Scores on a scale
Standard Deviation 7.04
|
—
|
—
|
|
Change From Baseline in Hamilton Depression Rating Scale 17-item Version (HAMD17) Total Score
Day 29
|
—
|
—
|
—
|
—
|
—
|
—
|
-13.6 Scores on a scale
Standard Deviation 11.51
|
-11.5 Scores on a scale
Standard Deviation 11.43
|
SECONDARY outcome
Timeframe: Baseline, Day1 post-infusion(6hours post start of infusion), Day1(12hours post start of infusion), Day2(24hours post start of infusion),Day3 post-infusion(48hours post start of infusion),Day4(72hours post start of infusion),Days 8,11,15,22,36,57 and 71Population: mITT population. Only those participants with data available at the specified data points were analyzed.
The HAMD17 was a 17-item clinician-rated instrument used to assess the range of symptoms that were most frequently observed in participants with major depression. All items were scored on an ordinal scale between 0 and 4 (8 items) or 0 and 2 (9 items) of increasing severity. Each of 17 items was rated by clinician with rating of 0: absent, 1: doubtful to mild, 2: mild to moderate, 3: moderate to severe, and 4: very severe. A total score (0 to 52) was calculated by adding scores of all 17 items, where 0 indicated no depression and 52 indicated severe depression. Higher score represented more severe condition. Baseline was defined as the Day 1 assessment before study drug infusion. Change from Baseline was calculated by subtracting Baseline value from the post-dose visit value.
Outcome measures
| Measure |
Placebo Cohort 1
n=4 Participants
IV infusion of PBO at rate of 16 mL/h for 48 hours, then 8 mL/h for 12 hours
|
Ganaxolone Cohort 1
n=5 Participants
IV infusion of GNX at rate of 4 mg/h (16 mL/h) for 48 hours, then 2 mg/h for 12 hours
|
Placebo Cohort 2
n=14 Participants
IV infusion of PBO at rate of 16 mL/h for 48 hours, then 8 mL/h for 12 hours
|
Ganaxolone Cohort 2
n=15 Participants
IV infusion of GNX at rate of 8 mg/h for 48 hours, then 4 mg/h (8 mL/h) for 12 hours
|
Placebo Cohort 3
n=10 Participants
IV bolus of 24 mL PBO over 2 minutes; then 24 mL/h for 48 hours and then 12 mL/h for 12 hours
|
Ganaxolone Cohort 3
n=10 Participants
IV bolus of 12 mg (24mL) GNX over 2 minutes; then GNX at 12 mg/h (24 mL/h) for 48 hours and then 6 mg/h (12 mL/h) for 12 hours
|
Placebo Cohort 6
n=17 Participants
IV infusion of PBO at rate of 40 mL/h for 6 hours; then 900 mg PBO (4 capsules) orally at dinner for 28 days
|
Ganaxolone Cohort 6
n=16 Participants
IV infusion of GNX at rate of 20 mg/h (40 mL/h) for 6 hours followed by 900 mg (4 capsules) orally at dinner for 28 days
|
|---|---|---|---|---|---|---|---|---|
|
Change From Baseline in HAMD17 Total Score at Indicated Time Points
Day 4 (72 hours post start of infusion)
|
-12.8 Scores on a scale
Standard Deviation 14.73
|
-10.2 Scores on a scale
Standard Deviation 10.06
|
-14.5 Scores on a scale
Standard Deviation 9.12
|
-17.3 Scores on a scale
Standard Deviation 9.22
|
-14.0 Scores on a scale
Standard Deviation 6.72
|
-10.5 Scores on a scale
Standard Deviation 8.57
|
-7.8 Scores on a scale
Standard Deviation 6.83
|
-7.0 Scores on a scale
Standard Deviation 6.16
|
|
Change From Baseline in HAMD17 Total Score at Indicated Time Points
Day 8
|
—
|
—
|
—
|
—
|
—
|
—
|
-8.7 Scores on a scale
Standard Deviation 8.99
|
-8.7 Scores on a scale
Standard Deviation 9.17
|
|
Change From Baseline in HAMD17 Total Score at Indicated Time Points
Day 11
|
-13.3 Scores on a scale
Standard Deviation 11.79
|
-6.2 Scores on a scale
Standard Deviation 5.72
|
-9.7 Scores on a scale
Standard Deviation 9.67
|
-14.0 Scores on a scale
Standard Deviation 12.56
|
-8.2 Scores on a scale
Standard Deviation 7.80
|
-8.9 Scores on a scale
Standard Deviation 9.65
|
-13.6 Scores on a scale
Standard Deviation 7.58
|
-13.6 Scores on a scale
Standard Deviation 9.84
|
|
Change From Baseline in HAMD17 Total Score at Indicated Time Points
Day 15
|
—
|
—
|
—
|
—
|
—
|
—
|
-11.6 Scores on a scale
Standard Deviation 8.38
|
-12.7 Scores on a scale
Standard Deviation 9.72
|
|
Change From Baseline in HAMD17 Total Score at Indicated Time Points
Day 22
|
—
|
—
|
—
|
—
|
—
|
—
|
-12.3 Scores on a scale
Standard Deviation 10.50
|
-12.7 Scores on a scale
Standard Deviation 9.55
|
|
Change From Baseline in HAMD17 Total Score at Indicated Time Points
Day 36
|
-17.0 Scores on a scale
Standard Deviation 12.46
|
-11.0 Scores on a scale
Standard Deviation 9.62
|
-13.3 Scores on a scale
Standard Deviation 6.48
|
-16.5 Scores on a scale
Standard Deviation 10.10
|
-9.1 Scores on a scale
Standard Deviation 8.70
|
-11.1 Scores on a scale
Standard Deviation 12.25
|
-13.5 Scores on a scale
Standard Deviation 10.76
|
-13.0 Scores on a scale
Standard Deviation 10.11
|
|
Change From Baseline in HAMD17 Total Score at Indicated Time Points
Day 57
|
—
|
—
|
—
|
—
|
—
|
—
|
-16.0 Scores on a scale
Standard Deviation 9.70
|
-12.0 Scores on a scale
Standard Deviation 9.49
|
|
Change From Baseline in HAMD17 Total Score at Indicated Time Points
Day 71
|
—
|
—
|
—
|
—
|
—
|
—
|
-19.0 Scores on a scale
Standard Deviation 7.87
|
-14.8 Scores on a scale
Standard Deviation 11.52
|
|
Change From Baseline in HAMD17 Total Score at Indicated Time Points
Day 1 post-infusion (6 hours post start of infusion)
|
—
|
—
|
—
|
—
|
—
|
—
|
-3.2 Scores on a scale
Standard Deviation 6.85
|
-6.1 Scores on a scale
Standard Deviation 9.97
|
|
Change From Baseline in HAMD17 Total Score at Indicated Time Points
Day 1 (12 hours post start of infusion)
|
-3.8 Scores on a scale
Standard Deviation 7.18
|
-2.4 Scores on a scale
Standard Deviation 1.52
|
-3.2 Scores on a scale
Standard Deviation 6.44
|
-2.7 Scores on a scale
Standard Deviation 5.59
|
-1.9 Scores on a scale
Standard Deviation 4.20
|
0.3 Scores on a scale
Standard Deviation 2.92
|
—
|
—
|
|
Change From Baseline in HAMD17 Total Score at Indicated Time Points
Day 2 (24 hours post start of infusion)
|
-8.0 Scores on a scale
Standard Deviation 9.80
|
-3.8 Scores on a scale
Standard Deviation 5.31
|
-6.4 Scores on a scale
Standard Deviation 7.33
|
-10.1 Scores on a scale
Standard Deviation 8.90
|
-5.2 Scores on a scale
Standard Deviation 7.89
|
-3.4 Scores on a scale
Standard Deviation 4.13
|
-5.1 Scores on a scale
Standard Deviation 5.07
|
-7.8 Scores on a scale
Standard Deviation 8.07
|
|
Change From Baseline in HAMD17 Total Score at Indicated Time Points
Day 3 post-infusion (48 hours post start of infusion)
|
-10.8 Scores on a scale
Standard Deviation 12.09
|
-11.0 Scores on a scale
Standard Deviation 3.67
|
-10.5 Scores on a scale
Standard Deviation 9.26
|
-13.7 Scores on a scale
Standard Deviation 11.68
|
-8.7 Scores on a scale
Standard Deviation 7.15
|
-11.2 Scores on a scale
Standard Deviation 4.98
|
—
|
—
|
SECONDARY outcome
Timeframe: Day1 post-infusion (6 hours post start of infusion [SOI]), Day 1 (12 hours post SOI), Day 2 (24 hours post SOI),Day 3 post-infusion (48 hours post SOI), Day 3 post-infusion (60 hours post SOI), Day 4 (72 hours post SOI), Days 8,11,15,22,29,36,57 and 71Population: mITT population. Only those participants with data available at the specified data points were analyzed.
HAMD17 Response was defined as ≥50% reduction from Baseline in total score. The HAMD17 was a 17-item clinician-rated instrument used to assess the range of symptoms that were most frequently observed in participants with major depression. All items were scored on an ordinal scale between 0 and 4 (8 items) or 0 and 2 (9 items) of increasing severity. Each of 17 items was rated by clinician with rating of 0: absent, 1: doubtful to mild, 2: mild to moderate, 3: moderate to severe, and 4: very severe. A total score (0 to 52) was calculated by adding scores of all 17 items, where 0 indicated no depression and 52 indicated severe depression. Higher score represented more severe condition. Number of participants with HAMD17 response is presented.
Outcome measures
| Measure |
Placebo Cohort 1
n=4 Participants
IV infusion of PBO at rate of 16 mL/h for 48 hours, then 8 mL/h for 12 hours
|
Ganaxolone Cohort 1
n=5 Participants
IV infusion of GNX at rate of 4 mg/h (16 mL/h) for 48 hours, then 2 mg/h for 12 hours
|
Placebo Cohort 2
n=14 Participants
IV infusion of PBO at rate of 16 mL/h for 48 hours, then 8 mL/h for 12 hours
|
Ganaxolone Cohort 2
n=15 Participants
IV infusion of GNX at rate of 8 mg/h for 48 hours, then 4 mg/h (8 mL/h) for 12 hours
|
Placebo Cohort 3
n=10 Participants
IV bolus of 24 mL PBO over 2 minutes; then 24 mL/h for 48 hours and then 12 mL/h for 12 hours
|
Ganaxolone Cohort 3
n=10 Participants
IV bolus of 12 mg (24mL) GNX over 2 minutes; then GNX at 12 mg/h (24 mL/h) for 48 hours and then 6 mg/h (12 mL/h) for 12 hours
|
Placebo Cohort 6
n=17 Participants
IV infusion of PBO at rate of 40 mL/h for 6 hours; then 900 mg PBO (4 capsules) orally at dinner for 28 days
|
Ganaxolone Cohort 6
n=16 Participants
IV infusion of GNX at rate of 20 mg/h (40 mL/h) for 6 hours followed by 900 mg (4 capsules) orally at dinner for 28 days
|
|---|---|---|---|---|---|---|---|---|
|
Number of Participants With HAMD17 Response
Day 1 post-infusion (6 hours post start of infusion): Responder
|
—
|
—
|
—
|
—
|
—
|
—
|
2 Participants
|
5 Participants
|
|
Number of Participants With HAMD17 Response
Day 1 (12 hours post start of infusion): Responder
|
0 Participants
|
0 Participants
|
1 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
—
|
—
|
|
Number of Participants With HAMD17 Response
Day 2 (24 hours post start of infusion): Responder
|
1 Participants
|
0 Participants
|
4 Participants
|
4 Participants
|
2 Participants
|
0 Participants
|
1 Participants
|
6 Participants
|
|
Number of Participants With HAMD17 Response
Day 3 post-infusion (48 hours post start of infusion): Responder
|
2 Participants
|
1 Participants
|
6 Participants
|
7 Participants
|
4 Participants
|
4 Participants
|
—
|
—
|
|
Number of Participants With HAMD17 Response
Day 3 post-infusion (60 hours post start of infusion): Responder
|
2 Participants
|
2 Participants
|
7 Participants
|
7 Participants
|
3 Participants
|
5 Participants
|
—
|
—
|
|
Number of Participants With HAMD17 Response
Day 4 (72 hours post start of infusion): Responder
|
2 Participants
|
3 Participants
|
7 Participants
|
11 Participants
|
6 Participants
|
5 Participants
|
3 Participants
|
3 Participants
|
|
Number of Participants With HAMD17 Response
Day 8: Responder
|
—
|
—
|
—
|
—
|
—
|
—
|
4 Participants
|
5 Participants
|
|
Number of Participants With HAMD17 Response
Day 11: Responder
|
2 Participants
|
1 Participants
|
5 Participants
|
5 Participants
|
3 Participants
|
3 Participants
|
6 Participants
|
6 Participants
|
|
Number of Participants With HAMD17 Response
Day 15: Responder
|
—
|
—
|
—
|
—
|
—
|
—
|
8 Participants
|
6 Participants
|
|
Number of Participants With HAMD17 Response
Day 22: Responder
|
—
|
—
|
—
|
—
|
—
|
—
|
7 Participants
|
8 Participants
|
|
Number of Participants With HAMD17 Response
Day 29: Responder
|
—
|
—
|
—
|
—
|
—
|
—
|
8 Participants
|
7 Participants
|
|
Number of Participants With HAMD17 Response
Day 36: Responder
|
3 Participants
|
3 Participants
|
5 Participants
|
7 Participants
|
4 Participants
|
5 Participants
|
6 Participants
|
8 Participants
|
|
Number of Participants With HAMD17 Response
Day 57: Responder
|
—
|
—
|
—
|
—
|
—
|
—
|
8 Participants
|
6 Participants
|
|
Number of Participants With HAMD17 Response
Day 71: Responder
|
—
|
—
|
—
|
—
|
—
|
—
|
9 Participants
|
9 Participants
|
SECONDARY outcome
Timeframe: Day1 post-infusion (6 hours post start of infusion [SOI]), Day 1 (12 hours post SOI), Day2 (24 hours post SOI), Day 3 post-infusion (48 hours post SOI), Day 3 post-infusion (60 hours post SOI), Day 4 (72 hours post SOI), Days 8,11,15,22,29,34,57 and 71Population: mITT population. Only those participants with data available at the specified data points were analyzed.
HAMD17 Remission was defined as total score ≤7. The HAMD17 was a 17-item clinician-rated instrument used to assess the range of symptoms that were most frequently observed in participants with major depression. All items were scored on an ordinal scale between 0 and 4 (8 items) or 0 and 2 (9 items) of increasing severity. Each of 17 items was rated by clinician with rating of 0: absent, 1: doubtful to mild, 2: mild to moderate, 3: moderate to severe, and 4: very severe. A total score (0 to 52) was calculated by adding scores of all 17 items, where 0 indicated no depression and 52 indicated severe depression. Higher score represented more severe condition. Number of participants with HAMD17 remission is presented
Outcome measures
| Measure |
Placebo Cohort 1
n=4 Participants
IV infusion of PBO at rate of 16 mL/h for 48 hours, then 8 mL/h for 12 hours
|
Ganaxolone Cohort 1
n=5 Participants
IV infusion of GNX at rate of 4 mg/h (16 mL/h) for 48 hours, then 2 mg/h for 12 hours
|
Placebo Cohort 2
n=14 Participants
IV infusion of PBO at rate of 16 mL/h for 48 hours, then 8 mL/h for 12 hours
|
Ganaxolone Cohort 2
n=15 Participants
IV infusion of GNX at rate of 8 mg/h for 48 hours, then 4 mg/h (8 mL/h) for 12 hours
|
Placebo Cohort 3
n=10 Participants
IV bolus of 24 mL PBO over 2 minutes; then 24 mL/h for 48 hours and then 12 mL/h for 12 hours
|
Ganaxolone Cohort 3
n=10 Participants
IV bolus of 12 mg (24mL) GNX over 2 minutes; then GNX at 12 mg/h (24 mL/h) for 48 hours and then 6 mg/h (12 mL/h) for 12 hours
|
Placebo Cohort 6
n=17 Participants
IV infusion of PBO at rate of 40 mL/h for 6 hours; then 900 mg PBO (4 capsules) orally at dinner for 28 days
|
Ganaxolone Cohort 6
n=16 Participants
IV infusion of GNX at rate of 20 mg/h (40 mL/h) for 6 hours followed by 900 mg (4 capsules) orally at dinner for 28 days
|
|---|---|---|---|---|---|---|---|---|
|
Number of Participants With HAMD17 Remission
Day 2 (24 hours post start of infusion): Remission
|
0 Participants
|
0 Participants
|
0 Participants
|
1 Participants
|
1 Participants
|
0 Participants
|
0 Participants
|
1 Participants
|
|
Number of Participants With HAMD17 Remission
Day 3 post-infusion (48 hours post start of infusion): Remission
|
1 Participants
|
0 Participants
|
3 Participants
|
3 Participants
|
1 Participants
|
2 Participants
|
—
|
—
|
|
Number of Participants With HAMD17 Remission
Day 3 post-infusion (60 hours post start of infusion): Remission
|
1 Participants
|
1 Participants
|
4 Participants
|
4 Participants
|
2 Participants
|
2 Participants
|
—
|
—
|
|
Number of Participants With HAMD17 Remission
Day 29: Remission
|
—
|
—
|
—
|
—
|
—
|
—
|
6 Participants
|
4 Participants
|
|
Number of Participants With HAMD17 Remission
Day 34: Remission
|
3 Participants
|
1 Participants
|
4 Participants
|
5 Participants
|
2 Participants
|
4 Participants
|
5 Participants
|
4 Participants
|
|
Number of Participants With HAMD17 Remission
Day 57: Remission
|
—
|
—
|
—
|
—
|
—
|
—
|
6 Participants
|
4 Participants
|
|
Number of Participants With HAMD17 Remission
Day 71: Remission
|
—
|
—
|
—
|
—
|
—
|
—
|
7 Participants
|
6 Participants
|
|
Number of Participants With HAMD17 Remission
Day 4 (72 hours post start of infusion): Remission
|
2 Participants
|
1 Participants
|
4 Participants
|
7 Participants
|
3 Participants
|
3 Participants
|
2 Participants
|
0 Participants
|
|
Number of Participants With HAMD17 Remission
Day 8: Remission
|
—
|
—
|
—
|
—
|
—
|
—
|
2 Participants
|
3 Participants
|
|
Number of Participants With HAMD17 Remission
Day 11: Remission
|
1 Participants
|
0 Participants
|
3 Participants
|
5 Participants
|
2 Participants
|
1 Participants
|
4 Participants
|
3 Participants
|
|
Number of Participants With HAMD17 Remission
Day 15: Remission
|
—
|
—
|
—
|
—
|
—
|
—
|
4 Participants
|
3 Participants
|
|
Number of Participants With HAMD17 Remission
Day 22: Remission
|
—
|
—
|
—
|
—
|
—
|
—
|
4 Participants
|
5 Participants
|
|
Number of Participants With HAMD17 Remission
Day 1 post-infusion (6 hours post start of infusion): Remission
|
—
|
—
|
—
|
—
|
—
|
—
|
0 Participants
|
3 Participants
|
|
Number of Participants With HAMD17 Remission
Day 1 (12 hours post start of infusion): Remission
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
—
|
—
|
SECONDARY outcome
Timeframe: Baseline, Day 1, Day 2, Day 3 post-infusion (60 hours post start of infusion), Day 8, Day 11, Day 15, Day 22, Day 29, Day 34, Day 36, Day 57 and Day 71Population: mITT population. Only those participants with data available at the specified data points were analyzed.
The EPDS is a validated 10-question depression assessment for postpartum women, which includes anxiety symptoms and excludes constitutional symptoms associated with depression common in the postpartum period, such as changes in sleeping patterns. It consists of 10 multiple choice questions with scores for each question ranging from 0-3. Questions 1, 2, \& 4 are scored 0, 1, 2 or 3 with top box scored as 0 and the bottom box scored as 3. Questions 3 and 5 to 10 are reverse scored, with the top box scored as a 3 and the bottom box scored as 0. Scores are then summed for a total score, which ranges from 0: no depression to 30: severe depression. Higher score indicates severe depression. Baseline was defined as the Day 1 assessment before study drug infusion. Change from Baseline was calculated by subtracting Baseline value from the post-dose visit value.
Outcome measures
| Measure |
Placebo Cohort 1
n=4 Participants
IV infusion of PBO at rate of 16 mL/h for 48 hours, then 8 mL/h for 12 hours
|
Ganaxolone Cohort 1
n=5 Participants
IV infusion of GNX at rate of 4 mg/h (16 mL/h) for 48 hours, then 2 mg/h for 12 hours
|
Placebo Cohort 2
n=13 Participants
IV infusion of PBO at rate of 16 mL/h for 48 hours, then 8 mL/h for 12 hours
|
Ganaxolone Cohort 2
n=13 Participants
IV infusion of GNX at rate of 8 mg/h for 48 hours, then 4 mg/h (8 mL/h) for 12 hours
|
Placebo Cohort 3
n=10 Participants
IV bolus of 24 mL PBO over 2 minutes; then 24 mL/h for 48 hours and then 12 mL/h for 12 hours
|
Ganaxolone Cohort 3
n=10 Participants
IV bolus of 12 mg (24mL) GNX over 2 minutes; then GNX at 12 mg/h (24 mL/h) for 48 hours and then 6 mg/h (12 mL/h) for 12 hours
|
Placebo Cohort 6
n=17 Participants
IV infusion of PBO at rate of 40 mL/h for 6 hours; then 900 mg PBO (4 capsules) orally at dinner for 28 days
|
Ganaxolone Cohort 6
n=16 Participants
IV infusion of GNX at rate of 20 mg/h (40 mL/h) for 6 hours followed by 900 mg (4 capsules) orally at dinner for 28 days
|
|---|---|---|---|---|---|---|---|---|
|
Change From Baseline in Edinburgh Postnatal Depression Scale (EPDS) Total Score
Day 34
|
-14.5 Scores on a scale
Standard Deviation 10.08
|
-8.6 Scores on a scale
Standard Deviation 5.32
|
-9.8 Scores on a scale
Standard Deviation 5.83
|
-13.4 Scores on a scale
Standard Deviation 5.81
|
-9.1 Scores on a scale
Standard Deviation 5.88
|
-8.8 Scores on a scale
Standard Deviation 8.27
|
—
|
—
|
|
Change From Baseline in Edinburgh Postnatal Depression Scale (EPDS) Total Score
Day 36
|
—
|
—
|
—
|
—
|
—
|
—
|
-11.8 Scores on a scale
Standard Deviation 6.57
|
-12.4 Scores on a scale
Standard Deviation 6.57
|
|
Change From Baseline in Edinburgh Postnatal Depression Scale (EPDS) Total Score
Day 57
|
—
|
—
|
—
|
—
|
—
|
—
|
-11.8 Scores on a scale
Standard Deviation 6.79
|
-9.4 Scores on a scale
Standard Deviation 8.50
|
|
Change From Baseline in Edinburgh Postnatal Depression Scale (EPDS) Total Score
Day 71
|
—
|
—
|
—
|
—
|
—
|
—
|
-14.0 Scores on a scale
Standard Deviation 5.62
|
-11.5 Scores on a scale
Standard Deviation 7.34
|
|
Change From Baseline in Edinburgh Postnatal Depression Scale (EPDS) Total Score
Day 1
|
—
|
—
|
—
|
—
|
—
|
—
|
-1.9 Scores on a scale
Standard Deviation 2.87
|
-5.4 Scores on a scale
Standard Deviation 6.61
|
|
Change From Baseline in Edinburgh Postnatal Depression Scale (EPDS) Total Score
Day 2
|
—
|
—
|
—
|
—
|
—
|
—
|
-4.9 Scores on a scale
Standard Deviation 4.76
|
-7.5 Scores on a scale
Standard Deviation 5.81
|
|
Change From Baseline in Edinburgh Postnatal Depression Scale (EPDS) Total Score
Day 3 post-infusion (60 hours post start of infusion)
|
-8.8 Scores on a scale
Standard Deviation 14.64
|
-8.0 Scores on a scale
Standard Deviation 8.63
|
-10.8 Scores on a scale
Standard Deviation 6.66
|
-10.8 Scores on a scale
Standard Deviation 6.32
|
-12.2 Scores on a scale
Standard Deviation 6.21
|
-10.9 Scores on a scale
Standard Deviation 6.06
|
—
|
—
|
|
Change From Baseline in Edinburgh Postnatal Depression Scale (EPDS) Total Score
Day 8
|
—
|
—
|
—
|
—
|
—
|
—
|
-6.7 Scores on a scale
Standard Deviation 6.01
|
-9.6 Scores on a scale
Standard Deviation 5.62
|
|
Change From Baseline in Edinburgh Postnatal Depression Scale (EPDS) Total Score
Day 11
|
-13.5 Scores on a scale
Standard Deviation 11.47
|
-8.0 Scores on a scale
Standard Deviation 4.12
|
-9.6 Scores on a scale
Standard Deviation 6.99
|
-11.5 Scores on a scale
Standard Deviation 8.02
|
-8.8 Scores on a scale
Standard Deviation 6.63
|
-8.2 Scores on a scale
Standard Deviation 7.35
|
—
|
—
|
|
Change From Baseline in Edinburgh Postnatal Depression Scale (EPDS) Total Score
Day 15
|
—
|
—
|
—
|
—
|
—
|
—
|
-8.0 Scores on a scale
Standard Deviation 6.02
|
-11.3 Scores on a scale
Standard Deviation 5.06
|
|
Change From Baseline in Edinburgh Postnatal Depression Scale (EPDS) Total Score
Day 22
|
—
|
—
|
—
|
—
|
—
|
—
|
-8.2 Scores on a scale
Standard Deviation 7.37
|
-11.2 Scores on a scale
Standard Deviation 6.13
|
|
Change From Baseline in Edinburgh Postnatal Depression Scale (EPDS) Total Score
Day 29
|
—
|
—
|
—
|
—
|
—
|
—
|
-10.9 Scores on a scale
Standard Deviation 8.14
|
-10.0 Scores on a scale
Standard Deviation 7.33
|
SECONDARY outcome
Timeframe: Baseline, Day 1 (12 hours post start of infusion), Day 2, Day 3 post-infusion (60 hours post start of infusion), Day 4, Day 8, Day 11, Day 15, Day 22, Day 29, Day 34, Day 36, Day 57 and Day 71Population: mITT population. Only those participants with data available at the specified data points were analyzed.
The STAI6 is a 6-item self-rated instrument used to assess anxiety state. The STAI6 questions included: 1. "I feel calm"; 2. "I am tense"; 3. "I feel upset"; 4. "I am relaxed"; 5. "I feel content"; 6. "I am worried". Each of these questions were rated as: a) "not at all"; b) "somewhat"; c) "moderately"; d) "very much". For questions 2, 3, and 6, the scoring was a = 1, b = 2, c = 3, and d = 4. For the other 3 questions, the scoring was a = 4, b = 3, c = 2, and d = 1. The STAI6 score was the result of first totaling the 6 individual item scores, and then prorating by a multiplication factor of 20/6 to acquire a score range of 20 (low anxiety) to 80 (high anxiety). Higher score indicated higher level of anxiety. Baseline was defined as the Day 1 assessment before study drug infusion. Change from Baseline was calculated by subtracting Baseline value from the post-dose visit value.
Outcome measures
| Measure |
Placebo Cohort 1
n=4 Participants
IV infusion of PBO at rate of 16 mL/h for 48 hours, then 8 mL/h for 12 hours
|
Ganaxolone Cohort 1
n=5 Participants
IV infusion of GNX at rate of 4 mg/h (16 mL/h) for 48 hours, then 2 mg/h for 12 hours
|
Placebo Cohort 2
n=14 Participants
IV infusion of PBO at rate of 16 mL/h for 48 hours, then 8 mL/h for 12 hours
|
Ganaxolone Cohort 2
n=15 Participants
IV infusion of GNX at rate of 8 mg/h for 48 hours, then 4 mg/h (8 mL/h) for 12 hours
|
Placebo Cohort 3
n=10 Participants
IV bolus of 24 mL PBO over 2 minutes; then 24 mL/h for 48 hours and then 12 mL/h for 12 hours
|
Ganaxolone Cohort 3
n=10 Participants
IV bolus of 12 mg (24mL) GNX over 2 minutes; then GNX at 12 mg/h (24 mL/h) for 48 hours and then 6 mg/h (12 mL/h) for 12 hours
|
Placebo Cohort 6
n=17 Participants
IV infusion of PBO at rate of 40 mL/h for 6 hours; then 900 mg PBO (4 capsules) orally at dinner for 28 days
|
Ganaxolone Cohort 6
n=16 Participants
IV infusion of GNX at rate of 20 mg/h (40 mL/h) for 6 hours followed by 900 mg (4 capsules) orally at dinner for 28 days
|
|---|---|---|---|---|---|---|---|---|
|
Change From Baseline in Spielberger State-Trait Anxiety Inventory 6-item Version (STAI6) Total Score
Day 1 (12 hours post start of infusion)
|
-8.9 Scores on a scale
Standard Deviation 20.09
|
-4.2 Scores on a scale
Standard Deviation 13.44
|
-14.5 Scores on a scale
Standard Deviation 14.48
|
-12.01 Scores on a scale
Standard Deviation 11.25
|
-10.7 Scores on a scale
Standard Deviation 10.52
|
-11.3 Scores on a scale
Standard Deviation 19.51
|
-9.4 Scores on a scale
Standard Deviation 14.78
|
-17.7 Scores on a scale
Standard Deviation 20.28
|
|
Change From Baseline in Spielberger State-Trait Anxiety Inventory 6-item Version (STAI6) Total Score
Day 2
|
-9.2 Scores on a scale
Standard Deviation 15.00
|
-8.7 Scores on a scale
Standard Deviation 13.25
|
-15.0 Scores on a scale
Standard Deviation 11.23
|
-14.2 Scores on a scale
Standard Deviation 17.88
|
-12.7 Scores on a scale
Standard Deviation 10.04
|
-12.0 Scores on a scale
Standard Deviation 13.07
|
-12.9 Scores on a scale
Standard Deviation 11.42
|
-19.0 Scores on a scale
Standard Deviation 17.96
|
|
Change From Baseline in Spielberger State-Trait Anxiety Inventory 6-item Version (STAI6) Total Score
Day 3 post-infusion (60 hours post start of infusion)
|
-11.7 Scores on a scale
Standard Deviation 33.39
|
-22.0 Scores on a scale
Standard Deviation 18.65
|
-18.2 Scores on a scale
Standard Deviation 12.95
|
-21.0 Scores on a scale
Standard Deviation 16.46
|
-24.3 Scores on a scale
Standard Deviation 16.11
|
-24.0 Scores on a scale
Standard Deviation 15.85
|
—
|
—
|
|
Change From Baseline in Spielberger State-Trait Anxiety Inventory 6-item Version (STAI6) Total Score
Day 4
|
-20.0 Scores on a scale
Standard Deviation 23.73
|
-20.7 Scores on a scale
Standard Deviation 19.78
|
-21.0 Scores on a scale
Standard Deviation 13.77
|
-22.9 Scores on a scale
Standard Deviation 17.14
|
-25.0 Scores on a scale
Standard Deviation 13.99
|
-22.3 Scores on a scale
Standard Deviation 17.78
|
—
|
—
|
|
Change From Baseline in Spielberger State-Trait Anxiety Inventory 6-item Version (STAI6) Total Score
Day 8
|
—
|
—
|
—
|
—
|
—
|
—
|
-10.2 Scores on a scale
Standard Deviation 18.88
|
-15.6 Scores on a scale
Standard Deviation 15.57
|
|
Change From Baseline in Spielberger State-Trait Anxiety Inventory 6-item Version (STAI6) Total Score
Day 11
|
-21.7 Scores on a scale
Standard Deviation 25.17
|
-18.7 Scores on a scale
Standard Deviation 20.22
|
-16.9 Scores on a scale
Standard Deviation 13.06
|
-20.8 Scores on a scale
Standard Deviation 17.17
|
-17.0 Scores on a scale
Standard Deviation 16.74
|
-16.0 Scores on a scale
Standard Deviation 15.93
|
—
|
—
|
|
Change From Baseline in Spielberger State-Trait Anxiety Inventory 6-item Version (STAI6) Total Score
Day 15
|
—
|
—
|
—
|
—
|
—
|
—
|
-12.3 Scores on a scale
Standard Deviation 20.39
|
-17.4 Scores on a scale
Standard Deviation 19.00
|
|
Change From Baseline in Spielberger State-Trait Anxiety Inventory 6-item Version (STAI6) Total Score
Day 22
|
—
|
—
|
—
|
—
|
—
|
—
|
-15.9 Scores on a scale
Standard Deviation 15.88
|
-21.8 Scores on a scale
Standard Deviation 16.08
|
|
Change From Baseline in Spielberger State-Trait Anxiety Inventory 6-item Version (STAI6) Total Score
Day 29
|
—
|
—
|
—
|
—
|
—
|
—
|
-20.8 Scores on a scale
Standard Deviation 13.89
|
-18.9 Scores on a scale
Standard Deviation 18.72
|
|
Change From Baseline in Spielberger State-Trait Anxiety Inventory 6-item Version (STAI6) Total Score
Day 34
|
-20.8 Scores on a scale
Standard Deviation 27.54
|
-16.0 Scores on a scale
Standard Deviation 21.53
|
-16.1 Scores on a scale
Standard Deviation 13.55
|
-24.4 Scores on a scale
Standard Deviation 13.80
|
-18.7 Scores on a scale
Standard Deviation 13.90
|
-17.7 Scores on a scale
Standard Deviation 20.37
|
—
|
—
|
|
Change From Baseline in Spielberger State-Trait Anxiety Inventory 6-item Version (STAI6) Total Score
Day 36
|
—
|
—
|
—
|
—
|
—
|
—
|
-21.9 Scores on a scale
Standard Deviation 9.69
|
-21.7 Scores on a scale
Standard Deviation 19.07
|
|
Change From Baseline in Spielberger State-Trait Anxiety Inventory 6-item Version (STAI6) Total Score
Day 57
|
—
|
—
|
—
|
—
|
—
|
—
|
-21.7 Scores on a scale
Standard Deviation 12.67
|
-13.3 Scores on a scale
Standard Deviation 25.23
|
|
Change From Baseline in Spielberger State-Trait Anxiety Inventory 6-item Version (STAI6) Total Score
Day 71
|
—
|
—
|
—
|
—
|
—
|
—
|
-31.7 Scores on a scale
Standard Deviation 16.97
|
-21.0 Scores on a scale
Standard Deviation 16.91
|
SECONDARY outcome
Timeframe: Day 1 (12 hours post start of infusion), Day 2, Day 3 post-infusion (60 hours post start of infusion), Day 4, Day 8, Day 11, Day 15, Day 22, Day 29, Day 34, Day 36, Day 57 and Day 71Population: mITT population. Only those participants with data available at the specified data points were analyzed.
The CGI-I scale is a clinician-rated 7-point scale used to assess how much the participant's illness had improved or worsened relative to a Baseline state at the beginning of the intervention. It was rated as: 1. "very much improved"; 2. "much improved"; 3. "minimally improved"; 4. "no change"; 5. "minimally worse"; 6. "much worse"; 7. "very much worse". Higher scores indicated worse condition. Response was defined as \>50% decrease from Baseline in CGI-I total score.
Outcome measures
| Measure |
Placebo Cohort 1
n=4 Participants
IV infusion of PBO at rate of 16 mL/h for 48 hours, then 8 mL/h for 12 hours
|
Ganaxolone Cohort 1
n=5 Participants
IV infusion of GNX at rate of 4 mg/h (16 mL/h) for 48 hours, then 2 mg/h for 12 hours
|
Placebo Cohort 2
n=14 Participants
IV infusion of PBO at rate of 16 mL/h for 48 hours, then 8 mL/h for 12 hours
|
Ganaxolone Cohort 2
n=15 Participants
IV infusion of GNX at rate of 8 mg/h for 48 hours, then 4 mg/h (8 mL/h) for 12 hours
|
Placebo Cohort 3
n=10 Participants
IV bolus of 24 mL PBO over 2 minutes; then 24 mL/h for 48 hours and then 12 mL/h for 12 hours
|
Ganaxolone Cohort 3
n=10 Participants
IV bolus of 12 mg (24mL) GNX over 2 minutes; then GNX at 12 mg/h (24 mL/h) for 48 hours and then 6 mg/h (12 mL/h) for 12 hours
|
Placebo Cohort 6
n=17 Participants
IV infusion of PBO at rate of 40 mL/h for 6 hours; then 900 mg PBO (4 capsules) orally at dinner for 28 days
|
Ganaxolone Cohort 6
n=16 Participants
IV infusion of GNX at rate of 20 mg/h (40 mL/h) for 6 hours followed by 900 mg (4 capsules) orally at dinner for 28 days
|
|---|---|---|---|---|---|---|---|---|
|
Number of Participants With Response to Clinical Global Impression-Improvement (CGI-I) Scale
Day 1 (12 hours post start of infusion): Very much or much improved
|
0 Participants
|
0 Participants
|
2 Participants
|
3 Participants
|
1 Participants
|
0 Participants
|
3 Participants
|
7 Participants
|
|
Number of Participants With Response to Clinical Global Impression-Improvement (CGI-I) Scale
Day 1 (12 hours post start of infusion): Minimally or not improved
|
4 Participants
|
5 Participants
|
12 Participants
|
12 Participants
|
9 Participants
|
10 Participants
|
14 Participants
|
9 Participants
|
|
Number of Participants With Response to Clinical Global Impression-Improvement (CGI-I) Scale
Day 2: Very much or much improved
|
1 Participants
|
1 Participants
|
3 Participants
|
8 Participants
|
1 Participants
|
3 Participants
|
3 Participants
|
9 Participants
|
|
Number of Participants With Response to Clinical Global Impression-Improvement (CGI-I) Scale
Day 2: Minimally or not improved
|
3 Participants
|
4 Participants
|
10 Participants
|
7 Participants
|
9 Participants
|
7 Participants
|
14 Participants
|
7 Participants
|
|
Number of Participants With Response to Clinical Global Impression-Improvement (CGI-I) Scale
Day 3 post-infusion (60 hours post start of infusion): Very much or much improved
|
3 Participants
|
4 Participants
|
7 Participants
|
9 Participants
|
5 Participants
|
7 Participants
|
—
|
—
|
|
Number of Participants With Response to Clinical Global Impression-Improvement (CGI-I) Scale
Day 3 post-infusion (60 hours post start of infusion): Minimally or not improved
|
1 Participants
|
1 Participants
|
6 Participants
|
4 Participants
|
5 Participants
|
3 Participants
|
—
|
—
|
|
Number of Participants With Response to Clinical Global Impression-Improvement (CGI-I) Scale
Day 4: Very much or much improved
|
3 Participants
|
3 Participants
|
6 Participants
|
12 Participants
|
5 Participants
|
7 Participants
|
—
|
—
|
|
Number of Participants With Response to Clinical Global Impression-Improvement (CGI-I) Scale
Day 4: Minimally or not improved
|
1 Participants
|
2 Participants
|
7 Participants
|
3 Participants
|
5 Participants
|
3 Participants
|
—
|
—
|
|
Number of Participants With Response to Clinical Global Impression-Improvement (CGI-I) Scale
Day 8: Very much or much improved
|
—
|
—
|
—
|
—
|
—
|
—
|
6 Participants
|
9 Participants
|
|
Number of Participants With Response to Clinical Global Impression-Improvement (CGI-I) Scale
Day 8: Minimally or not improved
|
—
|
—
|
—
|
—
|
—
|
—
|
9 Participants
|
6 Participants
|
|
Number of Participants With Response to Clinical Global Impression-Improvement (CGI-I) Scale
Day 11: Very much or much improved
|
2 Participants
|
0 Participants
|
7 Participants
|
9 Participants
|
4 Participants
|
5 Participants
|
—
|
—
|
|
Number of Participants With Response to Clinical Global Impression-Improvement (CGI-I) Scale
Day 11: Minimally or not improved
|
2 Participants
|
5 Participants
|
5 Participants
|
4 Participants
|
6 Participants
|
5 Participants
|
—
|
—
|
|
Number of Participants With Response to Clinical Global Impression-Improvement (CGI-I) Scale
Day 15: Very much or much improved
|
—
|
—
|
—
|
—
|
—
|
—
|
6 Participants
|
13 Participants
|
|
Number of Participants With Response to Clinical Global Impression-Improvement (CGI-I) Scale
Day 15: Minimally or not improved
|
—
|
—
|
—
|
—
|
—
|
—
|
10 Participants
|
2 Participants
|
|
Number of Participants With Response to Clinical Global Impression-Improvement (CGI-I) Scale
Day 22: Very much or much improved
|
—
|
—
|
—
|
—
|
—
|
—
|
8 Participants
|
10 Participants
|
|
Number of Participants With Response to Clinical Global Impression-Improvement (CGI-I) Scale
Day 22: Minimally or not improved
|
—
|
—
|
—
|
—
|
—
|
—
|
7 Participants
|
5 Participants
|
|
Number of Participants With Response to Clinical Global Impression-Improvement (CGI-I) Scale
Day 29: Very much or much improved
|
—
|
—
|
—
|
—
|
—
|
—
|
6 Participants
|
12 Participants
|
|
Number of Participants With Response to Clinical Global Impression-Improvement (CGI-I) Scale
Day 29: Minimally or not improved
|
—
|
—
|
—
|
—
|
—
|
—
|
10 Participants
|
4 Participants
|
|
Number of Participants With Response to Clinical Global Impression-Improvement (CGI-I) Scale
Day 34: Very much or much improved
|
3 Participants
|
3 Participants
|
5 Participants
|
8 Participants
|
5 Participants
|
5 Participants
|
—
|
—
|
|
Number of Participants With Response to Clinical Global Impression-Improvement (CGI-I) Scale
Day 34: Minimally or not improved
|
1 Participants
|
2 Participants
|
7 Participants
|
4 Participants
|
5 Participants
|
5 Participants
|
—
|
—
|
|
Number of Participants With Response to Clinical Global Impression-Improvement (CGI-I) Scale
Day 36: Very much or much improved
|
—
|
—
|
—
|
—
|
—
|
—
|
5 Participants
|
11 Participants
|
|
Number of Participants With Response to Clinical Global Impression-Improvement (CGI-I) Scale
Day 36: Minimally or not improved
|
—
|
—
|
—
|
—
|
—
|
—
|
7 Participants
|
3 Participants
|
|
Number of Participants With Response to Clinical Global Impression-Improvement (CGI-I) Scale
Day 57: Very much or much improved
|
—
|
—
|
—
|
—
|
—
|
—
|
6 Participants
|
8 Participants
|
|
Number of Participants With Response to Clinical Global Impression-Improvement (CGI-I) Scale
Day 57: Minimally or not improved
|
—
|
—
|
—
|
—
|
—
|
—
|
6 Participants
|
4 Participants
|
|
Number of Participants With Response to Clinical Global Impression-Improvement (CGI-I) Scale
Day 71: Very much or much improved
|
—
|
—
|
—
|
—
|
—
|
—
|
7 Participants
|
10 Participants
|
|
Number of Participants With Response to Clinical Global Impression-Improvement (CGI-I) Scale
Day 71: Minimally or not improved
|
—
|
—
|
—
|
—
|
—
|
—
|
5 Participants
|
3 Participants
|
Adverse Events
Placebo Cohort 1
Serious events: 0 serious events
Other events: 1 other events
Deaths: 0 deaths
Ganaxolone Cohort 1
Serious events: 0 serious events
Other events: 2 other events
Deaths: 0 deaths
Placebo Cohort 2
Serious events: 0 serious events
Other events: 3 other events
Deaths: 0 deaths
Ganaxolone Cohort 2
Serious events: 0 serious events
Other events: 5 other events
Deaths: 0 deaths
Placebo Cohort 3
Serious events: 0 serious events
Other events: 3 other events
Deaths: 0 deaths
Ganaxolone Cohort 3
Serious events: 0 serious events
Other events: 8 other events
Deaths: 0 deaths
Placebo Cohort 6
Serious events: 1 serious events
Other events: 7 other events
Deaths: 0 deaths
Ganaxolone Cohort 6
Serious events: 0 serious events
Other events: 11 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Placebo Cohort 1
n=4 participants at risk
IV infusion of PBO at rate of 16 mL/h for 48 hours, then 8 mL/h for 12 hours
|
Ganaxolone Cohort 1
n=5 participants at risk
IV infusion of GNX at rate of 4 mg/h (16 mL/h) for 48 hours, then 2 mg/h for 12 hours
|
Placebo Cohort 2
n=14 participants at risk
IV infusion of PBO at rate of 16 mL/h for 48 hours, then 8 mL/h for 12 hours
|
Ganaxolone Cohort 2
n=15 participants at risk
IV infusion of GNX at rate of 8 mg/h for 48 hours, then 4 mg/h (8 mL/h) for 12 hours
|
Placebo Cohort 3
n=10 participants at risk
IV bolus of 24 mL PBO over 2 minutes; then 24 mL/h for 48 hours and then 12 mL/h for 12 hours
|
Ganaxolone Cohort 3
n=10 participants at risk
IV bolus of 12 mg (24mL) GNX over 2 minutes; then GNX at 12 mg/h (24 mL/h) for 48 hours and then 6 mg/h (12 mL/h) for 12 hours
|
Placebo Cohort 6
n=17 participants at risk
IV infusion of PBO at rate of 40 mL/h for 6 hours; then 900 mg PBO (4 capsules) orally at dinner for 28 days
|
Ganaxolone Cohort 6
n=16 participants at risk
IV infusion of GNX at rate of 20 mg/h (40 mL/h) for 6 hours followed by 900 mg (4 capsules) orally at dinner for 28 days
|
|---|---|---|---|---|---|---|---|---|
|
Psychiatric disorders
Suicide Attempt
|
0.00%
0/4 • Baseline up to 71 days
|
0.00%
0/5 • Baseline up to 71 days
|
0.00%
0/14 • Baseline up to 71 days
|
0.00%
0/15 • Baseline up to 71 days
|
0.00%
0/10 • Baseline up to 71 days
|
0.00%
0/10 • Baseline up to 71 days
|
5.9%
1/17 • Baseline up to 71 days
|
0.00%
0/16 • Baseline up to 71 days
|
Other adverse events
| Measure |
Placebo Cohort 1
n=4 participants at risk
IV infusion of PBO at rate of 16 mL/h for 48 hours, then 8 mL/h for 12 hours
|
Ganaxolone Cohort 1
n=5 participants at risk
IV infusion of GNX at rate of 4 mg/h (16 mL/h) for 48 hours, then 2 mg/h for 12 hours
|
Placebo Cohort 2
n=14 participants at risk
IV infusion of PBO at rate of 16 mL/h for 48 hours, then 8 mL/h for 12 hours
|
Ganaxolone Cohort 2
n=15 participants at risk
IV infusion of GNX at rate of 8 mg/h for 48 hours, then 4 mg/h (8 mL/h) for 12 hours
|
Placebo Cohort 3
n=10 participants at risk
IV bolus of 24 mL PBO over 2 minutes; then 24 mL/h for 48 hours and then 12 mL/h for 12 hours
|
Ganaxolone Cohort 3
n=10 participants at risk
IV bolus of 12 mg (24mL) GNX over 2 minutes; then GNX at 12 mg/h (24 mL/h) for 48 hours and then 6 mg/h (12 mL/h) for 12 hours
|
Placebo Cohort 6
n=17 participants at risk
IV infusion of PBO at rate of 40 mL/h for 6 hours; then 900 mg PBO (4 capsules) orally at dinner for 28 days
|
Ganaxolone Cohort 6
n=16 participants at risk
IV infusion of GNX at rate of 20 mg/h (40 mL/h) for 6 hours followed by 900 mg (4 capsules) orally at dinner for 28 days
|
|---|---|---|---|---|---|---|---|---|
|
Ear and labyrinth disorders
Ear Pain
|
0.00%
0/4 • Baseline up to 71 days
|
0.00%
0/5 • Baseline up to 71 days
|
7.1%
1/14 • Number of events 1 • Baseline up to 71 days
|
0.00%
0/15 • Baseline up to 71 days
|
0.00%
0/10 • Baseline up to 71 days
|
0.00%
0/10 • Baseline up to 71 days
|
0.00%
0/17 • Baseline up to 71 days
|
0.00%
0/16 • Baseline up to 71 days
|
|
Endocrine disorders
Hypothyroidism
|
0.00%
0/4 • Baseline up to 71 days
|
0.00%
0/5 • Baseline up to 71 days
|
0.00%
0/14 • Baseline up to 71 days
|
0.00%
0/15 • Baseline up to 71 days
|
10.0%
1/10 • Number of events 1 • Baseline up to 71 days
|
0.00%
0/10 • Baseline up to 71 days
|
0.00%
0/17 • Baseline up to 71 days
|
0.00%
0/16 • Baseline up to 71 days
|
|
Eye disorders
Eye Pruritus
|
0.00%
0/4 • Baseline up to 71 days
|
0.00%
0/5 • Baseline up to 71 days
|
7.1%
1/14 • Number of events 1 • Baseline up to 71 days
|
0.00%
0/15 • Baseline up to 71 days
|
0.00%
0/10 • Baseline up to 71 days
|
0.00%
0/10 • Baseline up to 71 days
|
0.00%
0/17 • Baseline up to 71 days
|
0.00%
0/16 • Baseline up to 71 days
|
|
Gastrointestinal disorders
Abdominal Discomfort
|
0.00%
0/4 • Baseline up to 71 days
|
0.00%
0/5 • Baseline up to 71 days
|
0.00%
0/14 • Baseline up to 71 days
|
0.00%
0/15 • Baseline up to 71 days
|
0.00%
0/10 • Baseline up to 71 days
|
10.0%
1/10 • Number of events 2 • Baseline up to 71 days
|
0.00%
0/17 • Baseline up to 71 days
|
0.00%
0/16 • Baseline up to 71 days
|
|
Gastrointestinal disorders
Abdominal Pain
|
0.00%
0/4 • Baseline up to 71 days
|
0.00%
0/5 • Baseline up to 71 days
|
7.1%
1/14 • Number of events 1 • Baseline up to 71 days
|
0.00%
0/15 • Baseline up to 71 days
|
0.00%
0/10 • Baseline up to 71 days
|
0.00%
0/10 • Baseline up to 71 days
|
0.00%
0/17 • Baseline up to 71 days
|
0.00%
0/16 • Baseline up to 71 days
|
|
Gastrointestinal disorders
Abdominal Pain Upper
|
0.00%
0/4 • Baseline up to 71 days
|
0.00%
0/5 • Baseline up to 71 days
|
0.00%
0/14 • Baseline up to 71 days
|
0.00%
0/15 • Baseline up to 71 days
|
0.00%
0/10 • Baseline up to 71 days
|
0.00%
0/10 • Baseline up to 71 days
|
0.00%
0/17 • Baseline up to 71 days
|
6.2%
1/16 • Number of events 1 • Baseline up to 71 days
|
|
Gastrointestinal disorders
Diarrhoea
|
0.00%
0/4 • Baseline up to 71 days
|
0.00%
0/5 • Baseline up to 71 days
|
0.00%
0/14 • Baseline up to 71 days
|
0.00%
0/15 • Baseline up to 71 days
|
0.00%
0/10 • Baseline up to 71 days
|
0.00%
0/10 • Baseline up to 71 days
|
5.9%
1/17 • Number of events 1 • Baseline up to 71 days
|
0.00%
0/16 • Baseline up to 71 days
|
|
Gastrointestinal disorders
Dry Mouth
|
0.00%
0/4 • Baseline up to 71 days
|
20.0%
1/5 • Number of events 1 • Baseline up to 71 days
|
0.00%
0/14 • Baseline up to 71 days
|
0.00%
0/15 • Baseline up to 71 days
|
0.00%
0/10 • Baseline up to 71 days
|
0.00%
0/10 • Baseline up to 71 days
|
0.00%
0/17 • Baseline up to 71 days
|
6.2%
1/16 • Number of events 1 • Baseline up to 71 days
|
|
Gastrointestinal disorders
Dyspepsia
|
0.00%
0/4 • Baseline up to 71 days
|
0.00%
0/5 • Baseline up to 71 days
|
7.1%
1/14 • Number of events 1 • Baseline up to 71 days
|
0.00%
0/15 • Baseline up to 71 days
|
0.00%
0/10 • Baseline up to 71 days
|
0.00%
0/10 • Baseline up to 71 days
|
0.00%
0/17 • Baseline up to 71 days
|
0.00%
0/16 • Baseline up to 71 days
|
|
Gastrointestinal disorders
Nausea
|
0.00%
0/4 • Baseline up to 71 days
|
0.00%
0/5 • Baseline up to 71 days
|
0.00%
0/14 • Baseline up to 71 days
|
0.00%
0/15 • Baseline up to 71 days
|
10.0%
1/10 • Number of events 1 • Baseline up to 71 days
|
0.00%
0/10 • Baseline up to 71 days
|
5.9%
1/17 • Number of events 1 • Baseline up to 71 days
|
0.00%
0/16 • Baseline up to 71 days
|
|
Gastrointestinal disorders
Vomiting
|
0.00%
0/4 • Baseline up to 71 days
|
0.00%
0/5 • Baseline up to 71 days
|
0.00%
0/14 • Baseline up to 71 days
|
0.00%
0/15 • Baseline up to 71 days
|
0.00%
0/10 • Baseline up to 71 days
|
10.0%
1/10 • Number of events 1 • Baseline up to 71 days
|
0.00%
0/17 • Baseline up to 71 days
|
12.5%
2/16 • Number of events 3 • Baseline up to 71 days
|
|
General disorders
Fatigue
|
0.00%
0/4 • Baseline up to 71 days
|
0.00%
0/5 • Baseline up to 71 days
|
0.00%
0/14 • Baseline up to 71 days
|
0.00%
0/15 • Baseline up to 71 days
|
0.00%
0/10 • Baseline up to 71 days
|
0.00%
0/10 • Baseline up to 71 days
|
0.00%
0/17 • Baseline up to 71 days
|
6.2%
1/16 • Number of events 1 • Baseline up to 71 days
|
|
General disorders
Feeling Hot
|
0.00%
0/4 • Baseline up to 71 days
|
0.00%
0/5 • Baseline up to 71 days
|
0.00%
0/14 • Baseline up to 71 days
|
0.00%
0/15 • Baseline up to 71 days
|
0.00%
0/10 • Baseline up to 71 days
|
10.0%
1/10 • Number of events 1 • Baseline up to 71 days
|
0.00%
0/17 • Baseline up to 71 days
|
0.00%
0/16 • Baseline up to 71 days
|
|
General disorders
Feeling of Relaxation
|
0.00%
0/4 • Baseline up to 71 days
|
0.00%
0/5 • Baseline up to 71 days
|
0.00%
0/14 • Baseline up to 71 days
|
0.00%
0/15 • Baseline up to 71 days
|
0.00%
0/10 • Baseline up to 71 days
|
10.0%
1/10 • Number of events 1 • Baseline up to 71 days
|
0.00%
0/17 • Baseline up to 71 days
|
0.00%
0/16 • Baseline up to 71 days
|
|
General disorders
Infusion Site Erythema
|
0.00%
0/4 • Baseline up to 71 days
|
0.00%
0/5 • Baseline up to 71 days
|
0.00%
0/14 • Baseline up to 71 days
|
6.7%
1/15 • Number of events 1 • Baseline up to 71 days
|
0.00%
0/10 • Baseline up to 71 days
|
0.00%
0/10 • Baseline up to 71 days
|
0.00%
0/17 • Baseline up to 71 days
|
0.00%
0/16 • Baseline up to 71 days
|
|
General disorders
Infusion Site Irritation
|
0.00%
0/4 • Baseline up to 71 days
|
0.00%
0/5 • Baseline up to 71 days
|
0.00%
0/14 • Baseline up to 71 days
|
6.7%
1/15 • Number of events 2 • Baseline up to 71 days
|
0.00%
0/10 • Baseline up to 71 days
|
0.00%
0/10 • Baseline up to 71 days
|
0.00%
0/17 • Baseline up to 71 days
|
0.00%
0/16 • Baseline up to 71 days
|
|
General disorders
Infusion Site Pain
|
0.00%
0/4 • Baseline up to 71 days
|
0.00%
0/5 • Baseline up to 71 days
|
0.00%
0/14 • Baseline up to 71 days
|
6.7%
1/15 • Number of events 1 • Baseline up to 71 days
|
0.00%
0/10 • Baseline up to 71 days
|
0.00%
0/10 • Baseline up to 71 days
|
0.00%
0/17 • Baseline up to 71 days
|
0.00%
0/16 • Baseline up to 71 days
|
|
General disorders
Infusion Site Phlebitis
|
0.00%
0/4 • Baseline up to 71 days
|
0.00%
0/5 • Baseline up to 71 days
|
0.00%
0/14 • Baseline up to 71 days
|
0.00%
0/15 • Baseline up to 71 days
|
0.00%
0/10 • Baseline up to 71 days
|
10.0%
1/10 • Number of events 1 • Baseline up to 71 days
|
0.00%
0/17 • Baseline up to 71 days
|
0.00%
0/16 • Baseline up to 71 days
|
|
General disorders
Pyrexia
|
0.00%
0/4 • Baseline up to 71 days
|
0.00%
0/5 • Baseline up to 71 days
|
7.1%
1/14 • Number of events 1 • Baseline up to 71 days
|
0.00%
0/15 • Baseline up to 71 days
|
0.00%
0/10 • Baseline up to 71 days
|
0.00%
0/10 • Baseline up to 71 days
|
0.00%
0/17 • Baseline up to 71 days
|
0.00%
0/16 • Baseline up to 71 days
|
|
Infections and infestations
Mastitis
|
0.00%
0/4 • Baseline up to 71 days
|
0.00%
0/5 • Baseline up to 71 days
|
7.1%
1/14 • Number of events 1 • Baseline up to 71 days
|
0.00%
0/15 • Baseline up to 71 days
|
0.00%
0/10 • Baseline up to 71 days
|
0.00%
0/10 • Baseline up to 71 days
|
0.00%
0/17 • Baseline up to 71 days
|
0.00%
0/16 • Baseline up to 71 days
|
|
Infections and infestations
Urinary Tract Infection
|
0.00%
0/4 • Baseline up to 71 days
|
0.00%
0/5 • Baseline up to 71 days
|
0.00%
0/14 • Baseline up to 71 days
|
0.00%
0/15 • Baseline up to 71 days
|
0.00%
0/10 • Baseline up to 71 days
|
0.00%
0/10 • Baseline up to 71 days
|
0.00%
0/17 • Baseline up to 71 days
|
6.2%
1/16 • Number of events 1 • Baseline up to 71 days
|
|
Infections and infestations
Viral Upper Respiratory Tract Infection
|
0.00%
0/4 • Baseline up to 71 days
|
0.00%
0/5 • Baseline up to 71 days
|
0.00%
0/14 • Baseline up to 71 days
|
0.00%
0/15 • Baseline up to 71 days
|
0.00%
0/10 • Baseline up to 71 days
|
0.00%
0/10 • Baseline up to 71 days
|
5.9%
1/17 • Number of events 1 • Baseline up to 71 days
|
0.00%
0/16 • Baseline up to 71 days
|
|
Injury, poisoning and procedural complications
Procedural Pain
|
25.0%
1/4 • Number of events 1 • Baseline up to 71 days
|
0.00%
0/5 • Baseline up to 71 days
|
0.00%
0/14 • Baseline up to 71 days
|
0.00%
0/15 • Baseline up to 71 days
|
0.00%
0/10 • Baseline up to 71 days
|
0.00%
0/10 • Baseline up to 71 days
|
0.00%
0/17 • Baseline up to 71 days
|
0.00%
0/16 • Baseline up to 71 days
|
|
Investigations
Alanine Aminotransferase Increased
|
0.00%
0/4 • Baseline up to 71 days
|
0.00%
0/5 • Baseline up to 71 days
|
0.00%
0/14 • Baseline up to 71 days
|
0.00%
0/15 • Baseline up to 71 days
|
0.00%
0/10 • Baseline up to 71 days
|
0.00%
0/10 • Baseline up to 71 days
|
0.00%
0/17 • Baseline up to 71 days
|
6.2%
1/16 • Number of events 1 • Baseline up to 71 days
|
|
Investigations
Blood Thyroid Stimulating Hormone Increased
|
0.00%
0/4 • Baseline up to 71 days
|
0.00%
0/5 • Baseline up to 71 days
|
0.00%
0/14 • Baseline up to 71 days
|
13.3%
2/15 • Number of events 2 • Baseline up to 71 days
|
0.00%
0/10 • Baseline up to 71 days
|
0.00%
0/10 • Baseline up to 71 days
|
0.00%
0/17 • Baseline up to 71 days
|
0.00%
0/16 • Baseline up to 71 days
|
|
Investigations
Weight Increased
|
0.00%
0/4 • Baseline up to 71 days
|
0.00%
0/5 • Baseline up to 71 days
|
0.00%
0/14 • Baseline up to 71 days
|
0.00%
0/15 • Baseline up to 71 days
|
10.0%
1/10 • Number of events 1 • Baseline up to 71 days
|
0.00%
0/10 • Baseline up to 71 days
|
5.9%
1/17 • Number of events 1 • Baseline up to 71 days
|
0.00%
0/16 • Baseline up to 71 days
|
|
Nervous system disorders
Coordination Abnormal
|
0.00%
0/4 • Baseline up to 71 days
|
0.00%
0/5 • Baseline up to 71 days
|
0.00%
0/14 • Baseline up to 71 days
|
0.00%
0/15 • Baseline up to 71 days
|
0.00%
0/10 • Baseline up to 71 days
|
10.0%
1/10 • Number of events 1 • Baseline up to 71 days
|
0.00%
0/17 • Baseline up to 71 days
|
0.00%
0/16 • Baseline up to 71 days
|
|
Nervous system disorders
Dizziness
|
0.00%
0/4 • Baseline up to 71 days
|
0.00%
0/5 • Baseline up to 71 days
|
7.1%
1/14 • Number of events 1 • Baseline up to 71 days
|
6.7%
1/15 • Number of events 1 • Baseline up to 71 days
|
0.00%
0/10 • Baseline up to 71 days
|
40.0%
4/10 • Number of events 4 • Baseline up to 71 days
|
5.9%
1/17 • Number of events 1 • Baseline up to 71 days
|
25.0%
4/16 • Number of events 8 • Baseline up to 71 days
|
|
Nervous system disorders
Dysgeusia
|
0.00%
0/4 • Baseline up to 71 days
|
0.00%
0/5 • Baseline up to 71 days
|
7.1%
1/14 • Number of events 1 • Baseline up to 71 days
|
0.00%
0/15 • Baseline up to 71 days
|
0.00%
0/10 • Baseline up to 71 days
|
0.00%
0/10 • Baseline up to 71 days
|
0.00%
0/17 • Baseline up to 71 days
|
0.00%
0/16 • Baseline up to 71 days
|
|
Nervous system disorders
Headache
|
0.00%
0/4 • Baseline up to 71 days
|
20.0%
1/5 • Number of events 1 • Baseline up to 71 days
|
7.1%
1/14 • Number of events 1 • Baseline up to 71 days
|
0.00%
0/15 • Baseline up to 71 days
|
10.0%
1/10 • Number of events 1 • Baseline up to 71 days
|
10.0%
1/10 • Number of events 1 • Baseline up to 71 days
|
5.9%
1/17 • Number of events 1 • Baseline up to 71 days
|
6.2%
1/16 • Number of events 1 • Baseline up to 71 days
|
|
Nervous system disorders
Migraine
|
0.00%
0/4 • Baseline up to 71 days
|
0.00%
0/5 • Baseline up to 71 days
|
0.00%
0/14 • Baseline up to 71 days
|
0.00%
0/15 • Baseline up to 71 days
|
0.00%
0/10 • Baseline up to 71 days
|
10.0%
1/10 • Number of events 1 • Baseline up to 71 days
|
0.00%
0/17 • Baseline up to 71 days
|
0.00%
0/16 • Baseline up to 71 days
|
|
Nervous system disorders
Presyncope
|
0.00%
0/4 • Baseline up to 71 days
|
20.0%
1/5 • Number of events 1 • Baseline up to 71 days
|
0.00%
0/14 • Baseline up to 71 days
|
0.00%
0/15 • Baseline up to 71 days
|
0.00%
0/10 • Baseline up to 71 days
|
0.00%
0/10 • Baseline up to 71 days
|
0.00%
0/17 • Baseline up to 71 days
|
0.00%
0/16 • Baseline up to 71 days
|
|
Nervous system disorders
Psychomotor Hyperactivity
|
0.00%
0/4 • Baseline up to 71 days
|
0.00%
0/5 • Baseline up to 71 days
|
7.1%
1/14 • Number of events 1 • Baseline up to 71 days
|
0.00%
0/15 • Baseline up to 71 days
|
0.00%
0/10 • Baseline up to 71 days
|
0.00%
0/10 • Baseline up to 71 days
|
0.00%
0/17 • Baseline up to 71 days
|
0.00%
0/16 • Baseline up to 71 days
|
|
Nervous system disorders
Sedation
|
0.00%
0/4 • Baseline up to 71 days
|
0.00%
0/5 • Baseline up to 71 days
|
0.00%
0/14 • Baseline up to 71 days
|
0.00%
0/15 • Baseline up to 71 days
|
0.00%
0/10 • Baseline up to 71 days
|
60.0%
6/10 • Number of events 6 • Baseline up to 71 days
|
5.9%
1/17 • Number of events 1 • Baseline up to 71 days
|
6.2%
1/16 • Number of events 1 • Baseline up to 71 days
|
|
Nervous system disorders
Somnolence
|
0.00%
0/4 • Baseline up to 71 days
|
0.00%
0/5 • Baseline up to 71 days
|
0.00%
0/14 • Baseline up to 71 days
|
0.00%
0/15 • Baseline up to 71 days
|
0.00%
0/10 • Baseline up to 71 days
|
10.0%
1/10 • Number of events 1 • Baseline up to 71 days
|
5.9%
1/17 • Number of events 1 • Baseline up to 71 days
|
25.0%
4/16 • Number of events 4 • Baseline up to 71 days
|
|
Psychiatric disorders
Anxiety
|
0.00%
0/4 • Baseline up to 71 days
|
0.00%
0/5 • Baseline up to 71 days
|
0.00%
0/14 • Baseline up to 71 days
|
0.00%
0/15 • Baseline up to 71 days
|
0.00%
0/10 • Baseline up to 71 days
|
10.0%
1/10 • Number of events 2 • Baseline up to 71 days
|
0.00%
0/17 • Baseline up to 71 days
|
0.00%
0/16 • Baseline up to 71 days
|
|
Psychiatric disorders
Euphoric Mood
|
0.00%
0/4 • Baseline up to 71 days
|
0.00%
0/5 • Baseline up to 71 days
|
0.00%
0/14 • Baseline up to 71 days
|
0.00%
0/15 • Baseline up to 71 days
|
0.00%
0/10 • Baseline up to 71 days
|
10.0%
1/10 • Number of events 1 • Baseline up to 71 days
|
0.00%
0/17 • Baseline up to 71 days
|
0.00%
0/16 • Baseline up to 71 days
|
|
Psychiatric disorders
Irritability
|
0.00%
0/4 • Baseline up to 71 days
|
0.00%
0/5 • Baseline up to 71 days
|
0.00%
0/14 • Baseline up to 71 days
|
0.00%
0/15 • Baseline up to 71 days
|
0.00%
0/10 • Baseline up to 71 days
|
0.00%
0/10 • Baseline up to 71 days
|
0.00%
0/17 • Baseline up to 71 days
|
6.2%
1/16 • Number of events 1 • Baseline up to 71 days
|
|
Psychiatric disorders
Thinking Abnormal
|
0.00%
0/4 • Baseline up to 71 days
|
0.00%
0/5 • Baseline up to 71 days
|
0.00%
0/14 • Baseline up to 71 days
|
0.00%
0/15 • Baseline up to 71 days
|
0.00%
0/10 • Baseline up to 71 days
|
0.00%
0/10 • Baseline up to 71 days
|
5.9%
1/17 • Number of events 1 • Baseline up to 71 days
|
0.00%
0/16 • Baseline up to 71 days
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal Pain
|
0.00%
0/4 • Baseline up to 71 days
|
0.00%
0/5 • Baseline up to 71 days
|
7.1%
1/14 • Number of events 1 • Baseline up to 71 days
|
0.00%
0/15 • Baseline up to 71 days
|
0.00%
0/10 • Baseline up to 71 days
|
0.00%
0/10 • Baseline up to 71 days
|
0.00%
0/17 • Baseline up to 71 days
|
0.00%
0/16 • Baseline up to 71 days
|
|
Skin and subcutaneous tissue disorders
Dermatitis
|
0.00%
0/4 • Baseline up to 71 days
|
0.00%
0/5 • Baseline up to 71 days
|
0.00%
0/14 • Baseline up to 71 days
|
6.7%
1/15 • Number of events 1 • Baseline up to 71 days
|
0.00%
0/10 • Baseline up to 71 days
|
0.00%
0/10 • Baseline up to 71 days
|
0.00%
0/17 • Baseline up to 71 days
|
0.00%
0/16 • Baseline up to 71 days
|
|
Skin and subcutaneous tissue disorders
Eccymosis
|
0.00%
0/4 • Baseline up to 71 days
|
0.00%
0/5 • Baseline up to 71 days
|
0.00%
0/14 • Baseline up to 71 days
|
6.7%
1/15 • Number of events 1 • Baseline up to 71 days
|
0.00%
0/10 • Baseline up to 71 days
|
0.00%
0/10 • Baseline up to 71 days
|
0.00%
0/17 • Baseline up to 71 days
|
0.00%
0/16 • Baseline up to 71 days
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
0.00%
0/4 • Baseline up to 71 days
|
0.00%
0/5 • Baseline up to 71 days
|
0.00%
0/14 • Baseline up to 71 days
|
0.00%
0/15 • Baseline up to 71 days
|
0.00%
0/10 • Baseline up to 71 days
|
0.00%
0/10 • Baseline up to 71 days
|
0.00%
0/17 • Baseline up to 71 days
|
6.2%
1/16 • Number of events 1 • Baseline up to 71 days
|
|
Skin and subcutaneous tissue disorders
Pruritus Generalised
|
0.00%
0/4 • Baseline up to 71 days
|
0.00%
0/5 • Baseline up to 71 days
|
0.00%
0/14 • Baseline up to 71 days
|
0.00%
0/15 • Baseline up to 71 days
|
0.00%
0/10 • Baseline up to 71 days
|
0.00%
0/10 • Baseline up to 71 days
|
0.00%
0/17 • Baseline up to 71 days
|
6.2%
1/16 • Number of events 1 • Baseline up to 71 days
|
|
Skin and subcutaneous tissue disorders
Rash
|
0.00%
0/4 • Baseline up to 71 days
|
0.00%
0/5 • Baseline up to 71 days
|
7.1%
1/14 • Number of events 1 • Baseline up to 71 days
|
0.00%
0/15 • Baseline up to 71 days
|
0.00%
0/10 • Baseline up to 71 days
|
0.00%
0/10 • Baseline up to 71 days
|
0.00%
0/17 • Baseline up to 71 days
|
0.00%
0/16 • Baseline up to 71 days
|
|
Psychiatric disorders
Rash Erythematous
|
0.00%
0/4 • Baseline up to 71 days
|
0.00%
0/5 • Baseline up to 71 days
|
7.1%
1/14 • Number of events 1 • Baseline up to 71 days
|
0.00%
0/15 • Baseline up to 71 days
|
0.00%
0/10 • Baseline up to 71 days
|
0.00%
0/10 • Baseline up to 71 days
|
0.00%
0/17 • Baseline up to 71 days
|
0.00%
0/16 • Baseline up to 71 days
|
|
Skin and subcutaneous tissue disorders
Rash Papular
|
0.00%
0/4 • Baseline up to 71 days
|
0.00%
0/5 • Baseline up to 71 days
|
0.00%
0/14 • Baseline up to 71 days
|
6.7%
1/15 • Number of events 1 • Baseline up to 71 days
|
0.00%
0/10 • Baseline up to 71 days
|
0.00%
0/10 • Baseline up to 71 days
|
0.00%
0/17 • Baseline up to 71 days
|
0.00%
0/16 • Baseline up to 71 days
|
|
Skin and subcutaneous tissue disorders
Rash Pruritic
|
0.00%
0/4 • Baseline up to 71 days
|
0.00%
0/5 • Baseline up to 71 days
|
0.00%
0/14 • Baseline up to 71 days
|
13.3%
2/15 • Number of events 2 • Baseline up to 71 days
|
0.00%
0/10 • Baseline up to 71 days
|
0.00%
0/10 • Baseline up to 71 days
|
0.00%
0/17 • Baseline up to 71 days
|
0.00%
0/16 • Baseline up to 71 days
|
|
Skin and subcutaneous tissue disorders
Urticaria
|
0.00%
0/4 • Baseline up to 71 days
|
0.00%
0/5 • Baseline up to 71 days
|
0.00%
0/14 • Baseline up to 71 days
|
0.00%
0/15 • Baseline up to 71 days
|
0.00%
0/10 • Baseline up to 71 days
|
0.00%
0/10 • Baseline up to 71 days
|
0.00%
0/17 • Baseline up to 71 days
|
6.2%
1/16 • Number of events 1 • Baseline up to 71 days
|
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