Trial Outcomes & Findings for Ganaxolone Treatment in Children With Fragile X Syndrome (NCT NCT01725152)
NCT ID: NCT01725152
Last Updated: 2023-04-10
Results Overview
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.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
59 participants
Primary outcome timeframe
Week 14 (End of Treatment)
Results posted on
2023-04-10
Participant Flow
A total of 48 participants from the University of California at Davis Medical Center site and 11 participants from a Belgium site between November 1, 2012 and November 1, 2016 were enrolled in the study.
59 participants were randomized in the study.
Participant milestones
| Measure |
Ganaxolone, Then Placebo
Participants received ganaxolone were first titrated up to the 12 milligram per kilogram (mg/kg) three times daily (tid) during a 2-week titration phase, then maintained on that dose for an additional 4 weeks. After a washout period of 2 weeks, they received placebo for a duration of 6 weeks (from week 8 to 14).
|
Placebo, Then Ganaxolone
Participants received placebo were first titrated up to the 12 mg/kg tid during a 2-week titration phase, then maintained on that dose for an additional 4 weeks. After a washout period of 2 weeks, they received ganaxolone for a duration of 6 weeks (from week 8 to 14).
|
|---|---|---|
|
Treatment Period 1 (Week 1 to Week 6)
STARTED
|
30
|
29
|
|
Treatment Period 1 (Week 1 to Week 6)
COMPLETED
|
26
|
29
|
|
Treatment Period 1 (Week 1 to Week 6)
NOT COMPLETED
|
4
|
0
|
|
Washout Period (2 Weeks)
STARTED
|
26
|
29
|
|
Washout Period (2 Weeks)
COMPLETED
|
26
|
29
|
|
Washout Period (2 Weeks)
NOT COMPLETED
|
0
|
0
|
|
Treatment Period 2 (Week 8 to Week 14)
STARTED
|
26
|
29
|
|
Treatment Period 2 (Week 8 to Week 14)
COMPLETED
|
25
|
26
|
|
Treatment Period 2 (Week 8 to Week 14)
NOT COMPLETED
|
1
|
3
|
Reasons for withdrawal
| Measure |
Ganaxolone, Then Placebo
Participants received ganaxolone were first titrated up to the 12 milligram per kilogram (mg/kg) three times daily (tid) during a 2-week titration phase, then maintained on that dose for an additional 4 weeks. After a washout period of 2 weeks, they received placebo for a duration of 6 weeks (from week 8 to 14).
|
Placebo, Then Ganaxolone
Participants received placebo were first titrated up to the 12 mg/kg tid during a 2-week titration phase, then maintained on that dose for an additional 4 weeks. After a washout period of 2 weeks, they received ganaxolone for a duration of 6 weeks (from week 8 to 14).
|
|---|---|---|
|
Treatment Period 1 (Week 1 to Week 6)
Adverse Event
|
3
|
0
|
|
Treatment Period 1 (Week 1 to Week 6)
Consent Withdrawn
|
1
|
0
|
|
Treatment Period 2 (Week 8 to Week 14)
Adverse Event
|
1
|
1
|
|
Treatment Period 2 (Week 8 to Week 14)
Other
|
0
|
2
|
Baseline Characteristics
Ganaxolone Treatment in Children With Fragile X Syndrome
Baseline characteristics by cohort
| Measure |
Ganaxolone, Then Placebo
n=30 Participants
Participants received ganaxolone were first titrated up to the 12 mg/kg tid during a 2-week titration phase, then maintained on that dose for an additional 4 weeks. After a washout period of 2 weeks, they received placebo for a duration of 6 weeks (from week 8 to 14).
|
Placebo, Then Ganaxolone
n=29 Participants
Participants received placebo were first titrated up to the 12 mg/kg tid during a 2-week titration phase, then maintained on that dose for an additional 4 weeks. After a washout period of 2 weeks, they received ganaxolone for a duration of 6 weeks (from week 8 to 14).
|
Total
n=59 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
10.58 Years
STANDARD_DEVIATION 3.245 • n=5 Participants
|
11.33 Years
STANDARD_DEVIATION 3.368 • n=7 Participants
|
10.95 Years
STANDARD_DEVIATION 3.299 • n=5 Participants
|
|
Sex: Female, Male
Female
|
3 Participants
n=5 Participants
|
6 Participants
n=7 Participants
|
9 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
27 Participants
n=5 Participants
|
23 Participants
n=7 Participants
|
50 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
5 Participants
n=5 Participants
|
5 Participants
n=7 Participants
|
10 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
25 Participants
n=5 Participants
|
23 Participants
n=7 Participants
|
48 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
1 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
|
2 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
4 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
|
4 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
4 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
22 Participants
n=5 Participants
|
23 Participants
n=7 Participants
|
45 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
2 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
5 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Week 14 (End of Treatment)Population: Intention-to-Treat (ITT) Population consisted of all participants who received medication and who had at least one post-Baseline assessment. 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.
Outcome measures
| Measure |
Ganaxolone
n=52 Participants
Participants who received Ganaxolone up to 12 mg/kg tid in the first 6 weeks or last 6 weeks of the study.
|
Placebo
n=54 Participants
Participants who received placebo (matching Ganaxolone) in the first 6 weeks or last 6 weeks of the study.
|
|---|---|---|
|
Clinical Global Impression-Improvement (CGI-I) Scale
|
3.4 Scores on a scale
Standard Error 0.13
|
3.5 Scores on a scale
Standard Error 0.13
|
SECONDARY outcome
Timeframe: Week 14 (End of Treatment)Population: ITT Population. Only those participants with data available at the specified data points were analyzed.
Pediatric Anxiety Rating Scale (PARS) is a clinician administered measure of anxiety in children and adolescents. The PARS is comprised of a 50-item symptom checklist used to determine the presence or absence of specific anxiety symptoms during the prior week and 7 severity/impairment items, each scored from 0 to 5. The score on the 7 items allows the clinician to rate symptom severity and associated impairment on a range from 0 to 35, with higher scores reflecting greater symptom severity and associated impairment.
Outcome measures
| Measure |
Ganaxolone
n=52 Participants
Participants who received Ganaxolone up to 12 mg/kg tid in the first 6 weeks or last 6 weeks of the study.
|
Placebo
n=54 Participants
Participants who received placebo (matching Ganaxolone) in the first 6 weeks or last 6 weeks of the study.
|
|---|---|---|
|
Pediatric Anxiety Rating Scale (PARS) Total Score
|
8.3 Scores on a scale
Standard Error 0.54
|
9.2 Scores on a scale
Standard Error 0.54
|
SECONDARY outcome
Timeframe: Week 14 (End of Treatment)Population: ITT Population. Only those participants with data available at the specified data points were analyzed
A semi-structured VAS design was employed, providing two of the three behaviors to be assessed as anxiety and attention, chosen by caregiver(s) of one additional behavior from a bank of five. This bank included sociability, attention, aggression, language, and hyperactivity/impulsivity. Parents mark on a visual line measuring 10 centimeters (cm) with "worst behavior" at 0 cm and "best behavior" at 10 cm. For each behavior the caregiver is instructed to mark their impression of behavior at Baseline visit and again at each visit. The calculated distance in cm between the baseline and each visit marks thereby demonstrates whether each behavior improved, worsened, or stayed the same during the study, and by how much. Shown here is the least square mean distance and its corresponding standard error in cm from the "worst behavior" side, at baseline. Smaller the value, worser the behavior.
Outcome measures
| Measure |
Ganaxolone
n=51 Participants
Participants who received Ganaxolone up to 12 mg/kg tid in the first 6 weeks or last 6 weeks of the study.
|
Placebo
n=54 Participants
Participants who received placebo (matching Ganaxolone) in the first 6 weeks or last 6 weeks of the study.
|
|---|---|---|
|
Visual Analogue Scale (VAS)
Severity of Anxiety
|
5.6 Centimeters (cm)
Standard Error 0.27
|
5.0 Centimeters (cm)
Standard Error 0.26
|
|
Visual Analogue Scale (VAS)
Severity of Attention
|
4.4 Centimeters (cm)
Standard Error 0.24
|
3.9 Centimeters (cm)
Standard Error 0.24
|
|
Visual Analogue Scale (VAS)
Severity of Target Behavior 3
|
4.4 Centimeters (cm)
Standard Error 0.28
|
3.9 Centimeters (cm)
Standard Error 0.27
|
SECONDARY outcome
Timeframe: Week 14 (End of Treatment)Population: ITT Population. Only those participants with data available at the specified data points were analyzed.
The ADAMS is a 28-item behavior-based informant instrument rated by the parent(s), legal authorized guardian(s), or consistent caregiver(s). The scale is composed of 5 factors, which addresses Manic/Hyperactive Behavior, Depressed Mood, Social Avoidance, General Anxiety, and Obsessive/Compulsive Behavior. Items are scored on a 0-3 Likert scale that combines frequency and severity ratings (where 0=behavior has not occurred or is not a problem, 3=behavior occurs a lot or is a severe problem). Each subscale score is calculated separately; the scale range for Manic/Hyperactive Behavior is 0-15; for Depressed Mood, 0-21; for Social Avoidance, 0-21; for General Anxiety, 0-21; for Obsessive Behavior, 0-9. There is an overlapped item between Manic/Hypertension Behavior and General Anxiety; hence, the subscale ranges appear to reflect scores for 29 items instead of 28 items.
Outcome measures
| Measure |
Ganaxolone
n=50 Participants
Participants who received Ganaxolone up to 12 mg/kg tid in the first 6 weeks or last 6 weeks of the study.
|
Placebo
n=54 Participants
Participants who received placebo (matching Ganaxolone) in the first 6 weeks or last 6 weeks of the study.
|
|---|---|---|
|
Anxiety, Depression, and Mood Scale (ADAMS)
Manic/Hyperactive Behavior Total
|
7.4 Scores on a scale
Standard Error 0.42
|
7.8 Scores on a scale
Standard Error 0.40
|
|
Anxiety, Depression, and Mood Scale (ADAMS)
Depress Mood Total
|
3.5 Scores on a scale
Standard Error 0.32
|
2.6 Scores on a scale
Standard Error 0.32
|
|
Anxiety, Depression, and Mood Scale (ADAMS)
Social Avoidance Total
|
5.8 Scores on a scale
Standard Error 0.42
|
6.3 Scores on a scale
Standard Error 0.41
|
|
Anxiety, Depression, and Mood Scale (ADAMS)
General Anxiety Total
|
6.2 Scores on a scale
Standard Error 0.45
|
7.0 Scores on a scale
Standard Error 0.44
|
|
Anxiety, Depression, and Mood Scale (ADAMS)
Obsessive/Compulsive Behavior Total
|
2.7 Scores on a scale
Standard Error 0.21
|
2.8 Scores on a scale
Standard Error 0.20
|
SECONDARY outcome
Timeframe: Week 14 (End of Treatment)Population: ITT Population. Only those participants with data available at the specified data points were analyzed.
The ABC is a 58-item parent rated from 0 (not at all a problem) to 3 (the problem is severe in degree with 6 subscales: Irritability (includes agitation, aggression, and self-injury, 15 items) with range of scores from 0-45; Social Withdrawal/Lethargy (16 items) with range of scores from 0-48; Stereotypy (7 items) with range of scores from 0-21; Hyperactivity (16 items) with range of scores from 0-48; Inappropriate Speech (4 items) with range of scores from 0-12 and Social avoidance (4 items) with range of scores from 0-12. Higher scores indicated greater severity. Social Withdrawal and Lethargy are reporting the same scale and Social Avoidance is a subscale of Social Withdrawal; hence, the subscale ranges appear to reflect scores for 62 items instead of 58 items.
Outcome measures
| Measure |
Ganaxolone
n=50 Participants
Participants who received Ganaxolone up to 12 mg/kg tid in the first 6 weeks or last 6 weeks of the study.
|
Placebo
n=54 Participants
Participants who received placebo (matching Ganaxolone) in the first 6 weeks or last 6 weeks of the study.
|
|---|---|---|
|
Aberrant Behavior Checklist (ABC)
Total - Subscale II (Social Withdrawal/Lethargy)
|
5.6 Scores on a scale
Standard Error 0.53
|
5.7 Scores on a scale
Standard Error 0.54
|
|
Aberrant Behavior Checklist (ABC)
Total - Subscale I (Irritability)
|
15.7 Scores on a scale
Standard Error 0.87
|
16.1 Scores on a scale
Standard Error 0.86
|
|
Aberrant Behavior Checklist (ABC)
Total - Subscale III - (Stereotypy)
|
5.7 Scores on a scale
Standard Error 0.40
|
6.4 Scores on a scale
Standard Error 0.39
|
|
Aberrant Behavior Checklist (ABC)
Total - Subscale IV (Hyperactivity)
|
11.3 Scores on a scale
Standard Error 0.65
|
12.3 Scores on a scale
Standard Error 0.62
|
|
Aberrant Behavior Checklist (ABC)
Total - Subscale V (Inappropriate Speech)
|
5.1 Scores on a scale
Standard Error 0.32
|
5.3 Scores on a scale
Standard Error 0.31
|
|
Aberrant Behavior Checklist (ABC)
Total - Subscale VI (Social Avoidance)
|
2.4 Scores on a scale
Standard Error 0.22
|
2.8 Scores on a scale
Standard Error 0.21
|
SECONDARY outcome
Timeframe: Week 14 (End of Treatment)Population: ITT Population. Only those participants with data available at the specified data points were analyzed.
The SNAP-IV is a revision of the Swanson, Nolan, and Pelham (SNAP) Questionnaire. The SNAP-IV: ADHD Inattention Subscale (items 1-9) scores the intensity of each item during the last seven days on a 0 to 3 scale (0=not at all, 1=just a little, 2=pretty much, 3=very much). Possible scores ranged from 0-27; higher scores indicated a greater intensity. The SNAP-IV ADHD Hyperactivity/Impulsivity Subscale (items 10-18) scores the intensity of each item in the last seven days on a 0 to 3 scale (0=not at all, 1=just a little, 2=pretty much, 3=very much). Possible scores ranged from 0-27; higher scores indicated a greater intensity. SNAP-IV ADHD Combined Scale score (inattention + hyperactivity/impulsivity) ranged from 0-54. A low score of 0 indicates less inattention + hyperactivity/impulsivity. A high score of 54 indicates more inattention + hyperactivity.
Outcome measures
| Measure |
Ganaxolone
n=50 Participants
Participants who received Ganaxolone up to 12 mg/kg tid in the first 6 weeks or last 6 weeks of the study.
|
Placebo
n=53 Participants
Participants who received placebo (matching Ganaxolone) in the first 6 weeks or last 6 weeks of the study.
|
|---|---|---|
|
Swanson, Nolan, and Pelham-IV Questionnaire (SNAP-IV)
ADHD Inattention
|
15.5 Scores on a scale
Standard Error 0.66
|
14.6 Scores on a scale
Standard Error 0.62
|
|
Swanson, Nolan, and Pelham-IV Questionnaire (SNAP-IV)
ADHD Hyperactivity/Impulsive
|
13.9 Scores on a scale
Standard Error 0.66
|
12.6 Scores on a scale
Standard Error 0.64
|
|
Swanson, Nolan, and Pelham-IV Questionnaire (SNAP-IV)
ADHD Combined
|
29.3 Scores on a scale
Standard Error 1.19
|
27.1 Scores on a scale
Standard Error 1.14
|
Adverse Events
Ganaxolone
Serious events: 0 serious events
Other events: 49 other events
Deaths: 0 deaths
Placebo
Serious events: 0 serious events
Other events: 46 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Ganaxolone
n=59 participants at risk
Participants who received Ganaxolone up to 12 mg/kg tid in the first 6 weeks or last 6 weeks of the study.
|
Placebo
n=54 participants at risk
Participants who received placebo (matching Ganaxolone) in the first 6 weeks or last 6 weeks of the study.
|
|---|---|---|
|
Nervous system disorders
Headache
|
3.4%
2/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
11.1%
6/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Gastrointestinal disorders
Diarrhoea
|
13.6%
8/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
13.0%
7/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Gastrointestinal disorders
Vomiting
|
13.6%
8/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
9.3%
5/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Psychiatric disorders
Aggression
|
10.2%
6/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
3.7%
2/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Skin and subcutaneous tissue disorders
Rash
|
8.5%
5/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
3.7%
2/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Infections and infestations
Upper respiratory tract infection
|
15.3%
9/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
20.4%
11/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Psychiatric disorders
Agitation
|
5.1%
3/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
14.8%
8/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
General disorders
Fatigue
|
49.2%
29/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
20.4%
11/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
General disorders
Condition aggravated
|
16.9%
10/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
11.1%
6/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Nervous system disorders
Somnolence
|
33.9%
20/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
5.6%
3/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Metabolism and nutrition disorders
Decreased appetite
|
13.6%
8/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
3.7%
2/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
General disorders
Pyrexia
|
1.7%
1/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
5.6%
3/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
General disorders
Irritability
|
1.7%
1/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
3.7%
2/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
General disorders
Thirst
|
1.7%
1/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
1.9%
1/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
General disorders
Hyperpyrexia
|
0.00%
0/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
1.9%
1/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Nervous system disorders
Hypersomnia
|
3.4%
2/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
3.7%
2/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Nervous system disorders
Dizziness
|
3.4%
2/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
1.9%
1/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Nervous system disorders
Coordination abnormal
|
1.7%
1/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
1.9%
1/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Nervous system disorders
Poor quality sleep
|
3.4%
2/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
0.00%
0/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Nervous system disorders
Psychomotor hyperactivity
|
1.7%
1/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
1.9%
1/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Nervous system disorders
Repetitive speech
|
3.4%
2/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
0.00%
0/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Nervous system disorders
Balance disorder
|
1.7%
1/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
0.00%
0/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Nervous system disorders
Drooling
|
1.7%
1/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
0.00%
0/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Nervous system disorders
Dysarthria
|
1.7%
1/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
0.00%
0/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Nervous system disorders
Lethargy
|
0.00%
0/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
1.9%
1/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Nervous system disorders
Petit mal epilepsy
|
1.7%
1/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
1.9%
1/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Nervous system disorders
Speech disorder
|
1.7%
1/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
0.00%
0/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Nervous system disorders
Tremor
|
0.00%
0/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
1.9%
1/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Gastrointestinal disorders
Abdominal discomfort
|
5.1%
3/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
0.00%
0/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Gastrointestinal disorders
Abdominal pain upper
|
3.4%
2/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
1.9%
1/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Gastrointestinal disorders
Constipation
|
1.7%
1/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
3.7%
2/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Gastrointestinal disorders
Nausea
|
1.7%
1/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
1.9%
1/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Gastrointestinal disorders
Abdominal pain
|
0.00%
0/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
1.9%
1/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Gastrointestinal disorders
Abdominal pain lower
|
1.7%
1/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
0.00%
0/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Gastrointestinal disorders
Faecal incontinence
|
1.7%
1/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
0.00%
0/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Gastrointestinal disorders
Salivary hypersecretion
|
0.00%
0/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
1.9%
1/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Infections and infestations
Nasopharyngitis
|
1.7%
1/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
7.4%
4/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Infections and infestations
Rhinitis
|
3.4%
2/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
3.7%
2/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Infections and infestations
Otitis media
|
1.7%
1/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
1.9%
1/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Infections and infestations
Candida nappy rash
|
1.7%
1/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
0.00%
0/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Infections and infestations
Cellulitis
|
1.7%
1/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
0.00%
0/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Infections and infestations
Ear infection
|
1.7%
1/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
1.9%
1/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Infections and infestations
Gastroenteritis
|
1.7%
1/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
0.00%
0/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Infections and infestations
Influenza
|
1.7%
1/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
0.00%
0/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Infections and infestations
Otitis externa
|
0.00%
0/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
1.9%
1/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Infections and infestations
Pharyngitis
|
1.7%
1/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
0.00%
0/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Psychiatric disorders
Anxiety
|
5.1%
3/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
0.00%
0/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Psychiatric disorders
Emotional disorder
|
1.7%
1/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
3.7%
2/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Psychiatric disorders
Self injurious behaviour
|
1.7%
1/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
3.7%
2/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Psychiatric disorders
Stereotypy
|
5.1%
3/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
0.00%
0/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Psychiatric disorders
Abnormal behaviour
|
0.00%
0/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
3.7%
2/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Psychiatric disorders
Sleep disorder
|
1.7%
1/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
1.9%
1/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Psychiatric disorders
Terminal insomnia
|
0.00%
0/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
3.7%
2/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Psychiatric disorders
Tic
|
3.4%
2/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
0.00%
0/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Psychiatric disorders
Affect lability
|
0.00%
0/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
1.9%
1/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Psychiatric disorders
Binge eating
|
0.00%
0/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
1.9%
1/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Psychiatric disorders
Dysphemia
|
0.00%
0/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
1.9%
1/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Psychiatric disorders
Insomnia
|
0.00%
0/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
1.9%
1/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Psychiatric disorders
Negativism
|
1.7%
1/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
0.00%
0/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Psychiatric disorders
Nervousness
|
1.7%
1/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
0.00%
0/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Psychiatric disorders
Obsessive thoughts
|
0.00%
0/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
1.9%
1/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Psychiatric disorders
Sleep talking
|
1.7%
1/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
1.9%
1/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Psychiatric disorders
Staring
|
0.00%
0/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
1.9%
1/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
8.5%
5/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
0.00%
0/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Skin and subcutaneous tissue disorders
Acne
|
1.7%
1/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
0.00%
0/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Skin and subcutaneous tissue disorders
Dermal cyst
|
1.7%
1/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
0.00%
0/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Skin and subcutaneous tissue disorders
Dermatitis diaper
|
0.00%
0/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
1.9%
1/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Skin and subcutaneous tissue disorders
Eczema
|
0.00%
0/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
1.9%
1/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Skin and subcutaneous tissue disorders
Night sweats
|
0.00%
0/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
1.9%
1/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Injury, poisoning and procedural complications
Fall
|
3.4%
2/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
1.9%
1/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Injury, poisoning and procedural complications
Arthropod bite
|
3.4%
2/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
0.00%
0/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Injury, poisoning and procedural complications
Scratch
|
1.7%
1/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
1.9%
1/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Injury, poisoning and procedural complications
Burns first degree
|
1.7%
1/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
0.00%
0/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Injury, poisoning and procedural complications
Contusion
|
1.7%
1/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
0.00%
0/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Injury, poisoning and procedural complications
Excoriation
|
1.7%
1/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
0.00%
0/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Injury, poisoning and procedural complications
Ligament sprain
|
1.7%
1/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
0.00%
0/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Injury, poisoning and procedural complications
Lip injury
|
1.7%
1/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
0.00%
0/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Injury, poisoning and procedural complications
Sunburn
|
1.7%
1/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
0.00%
0/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Injury, poisoning and procedural complications
Thermal burn
|
0.00%
0/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
1.9%
1/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Injury, poisoning and procedural complications
Tooth avulsion
|
0.00%
0/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
1.9%
1/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Injury, poisoning and procedural complications
Wound
|
0.00%
0/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
1.9%
1/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Metabolism and nutrition disorders
Increased appetite
|
1.7%
1/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
1.9%
1/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Metabolism and nutrition disorders
Hypoglycaemia
|
0.00%
0/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
1.9%
1/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
1.7%
1/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
5.6%
3/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
1.7%
1/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
5.6%
3/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrhoea
|
1.7%
1/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
3.7%
2/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
1.7%
1/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
0.00%
0/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory tract congestion
|
0.00%
0/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
1.9%
1/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Respiratory, thoracic and mediastinal disorders
Upper respiratory tract congestion
|
1.7%
1/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
0.00%
0/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Renal and urinary disorders
Urinary incontinence
|
3.4%
2/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
0.00%
0/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Renal and urinary disorders
Incontinence
|
0.00%
0/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
1.9%
1/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Renal and urinary disorders
Ketonuria
|
0.00%
0/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
1.9%
1/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Vascular disorders
Aortic dilatation
|
0.00%
0/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
1.9%
1/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Vascular disorders
Hot flush
|
0.00%
0/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
1.9%
1/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Vascular disorders
Pallor
|
1.7%
1/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
0.00%
0/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Investigations
Alanine aminotransferase increased
|
0.00%
0/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
1.9%
1/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Investigations
Transaminases increased
|
1.7%
1/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
0.00%
0/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Reproductive system and breast disorders
Dysmenorrhoea
|
0.00%
0/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
1.9%
1/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Reproductive system and breast disorders
Nipple swelling
|
0.00%
0/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
1.9%
1/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Ear and labyrinth disorders
Cerumen impaction
|
1.7%
1/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
0.00%
0/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Ear and labyrinth disorders
Tympanic membrane disorder
|
0.00%
0/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
1.9%
1/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Eye disorders
Blepharitis
|
1.7%
1/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
0.00%
0/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Immune system disorders
Seasonal allergy
|
0.00%
0/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
1.9%
1/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Social circumstances
Treatment noncompliance
|
0.00%
0/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
1.9%
1/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
|
Surgical and medical procedures
Dental operation
|
1.7%
1/59 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
0.00%
0/54 • Up to 14 Weeks
Safety Population included all participants who received at least one dose of intervention. One participant mistakenly did not cross over to receive placebo and remained on Ganaxolone during Period 2 as a result of a pharmacy error.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place