Trial Outcomes & Findings for Study of Adjunctive Ganaxolone Treatment in Children and Young Adults With CDKL5 Deficiency Disorder (NCT NCT03572933)
NCT ID: NCT03572933
Last Updated: 2023-04-14
Results Overview
Summary of 28-day seizure frequency for Major Motor Seizure Types during the double-blind treatment period relative to the 6-week prospective baseline period Note: Summaries are based on the sum of the individual seizures, the countable seizures, and the clusters with uncountable seizures (each cluster with uncountable seizures counts as 1 seizure). Within the baseline and post baseline intervals, 28-day seizure frequency was calculated as the total number of seizures in the interval divided by the number of days with available seizure data in the interval, multiplied by 28.
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
101 participants
Primary outcome timeframe
End of the double-blind 17 week treatment period
Results posted on
2023-04-14
Participant Flow
Participant milestones
| Measure |
Placebo
placebo suspension 3x's /day for 17 weeks
Placebo: inactive
|
Ganaxolone
ganaxolone suspension (50 mg/ml) 3x's /day for 17 weeks
ganaxolone: active drug
|
|---|---|---|
|
Overall Study
STARTED
|
51
|
50
|
|
Overall Study
COMPLETED
|
47
|
48
|
|
Overall Study
NOT COMPLETED
|
4
|
2
|
Reasons for withdrawal
| Measure |
Placebo
placebo suspension 3x's /day for 17 weeks
Placebo: inactive
|
Ganaxolone
ganaxolone suspension (50 mg/ml) 3x's /day for 17 weeks
ganaxolone: active drug
|
|---|---|---|
|
Overall Study
Adverse Event
|
4
|
1
|
|
Overall Study
Withdrawal by Subject
|
0
|
1
|
Baseline Characteristics
Study of Adjunctive Ganaxolone Treatment in Children and Young Adults With CDKL5 Deficiency Disorder
Baseline characteristics by cohort
| Measure |
Placebo
n=51 Participants
placebo suspension 3x's /day for 17 weeks
Placebo: inactive
|
Ganaxolone
n=50 Participants
ganaxolone suspension (50 mg/ml) 3x's /day for 17 weeks
ganaxolone: active drug
|
Total
n=101 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
7.73 years
STANDARD_DEVIATION 4.382 • n=5 Participants
|
6.78 years
STANDARD_DEVIATION 4.705 • n=7 Participants
|
7.26 years
STANDARD_DEVIATION 4.547 • n=5 Participants
|
|
Sex: Female, Male
Female
|
41 Participants
n=5 Participants
|
39 Participants
n=7 Participants
|
80 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
10 Participants
n=5 Participants
|
11 Participants
n=7 Participants
|
21 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
6 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
10 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
43 Participants
n=5 Participants
|
44 Participants
n=7 Participants
|
87 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
2 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
4 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
|
3 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
5 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
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
47 Participants
n=5 Participants
|
46 Participants
n=7 Participants
|
93 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
24 participants
n=5 Participants
|
18 participants
n=7 Participants
|
42 participants
n=5 Participants
|
|
Region of Enrollment
Poland
|
5 participants
n=5 Participants
|
5 participants
n=7 Participants
|
10 participants
n=5 Participants
|
|
Region of Enrollment
Italy
|
6 participants
n=5 Participants
|
9 participants
n=7 Participants
|
15 participants
n=5 Participants
|
|
Region of Enrollment
United Kingdom
|
3 participants
n=5 Participants
|
4 participants
n=7 Participants
|
7 participants
n=5 Participants
|
|
Region of Enrollment
Israel
|
1 participants
n=5 Participants
|
0 participants
n=7 Participants
|
1 participants
n=5 Participants
|
|
Region of Enrollment
Australia
|
2 participants
n=5 Participants
|
4 participants
n=7 Participants
|
6 participants
n=5 Participants
|
|
Region of Enrollment
France
|
3 participants
n=5 Participants
|
3 participants
n=7 Participants
|
6 participants
n=5 Participants
|
|
Region of Enrollment
Russia
|
7 participants
n=5 Participants
|
7 participants
n=7 Participants
|
14 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: End of the double-blind 17 week treatment periodPopulation: ITT Population
Summary of 28-day seizure frequency for Major Motor Seizure Types during the double-blind treatment period relative to the 6-week prospective baseline period Note: Summaries are based on the sum of the individual seizures, the countable seizures, and the clusters with uncountable seizures (each cluster with uncountable seizures counts as 1 seizure). Within the baseline and post baseline intervals, 28-day seizure frequency was calculated as the total number of seizures in the interval divided by the number of days with available seizure data in the interval, multiplied by 28.
Outcome measures
| Measure |
Placebo
n=51 Participants
placebo suspension 3x's /day for 17 weeks
Placebo: inactive
|
Ganaxolone
n=49 Participants
ganaxolone suspension (50 mg/ml) 3x's /day for 17 weeks
ganaxolone: active drug
|
|---|---|---|
|
Summary of 28-day Seizure Frequency for Major Motor Seizure Types
Baseline (Median)
|
49.17 Seizures per day
Interval 32.2 to 60.67
|
54.00 Seizures per day
Interval 38.24 to 106.67
|
|
Summary of 28-day Seizure Frequency for Major Motor Seizure Types
17 week-post baseline phase (Median)
|
55.50 Seizures per day
Interval 35.75 to 80.14
|
45.03 Seizures per day
Interval 31.83 to 76.03
|
SECONDARY outcome
Timeframe: End of the double-blind 17 week treatment periodPopulation: Per Protocol Population
Caregiver global impression of change in attention during the double-blind treatment period of ganaxolone compared to placebo. Investigators and caregivers reported improvements in attention, mood, behavior and sleep via investigator narratives
Outcome measures
| Measure |
Placebo
n=51 Participants
placebo suspension 3x's /day for 17 weeks
Placebo: inactive
|
Ganaxolone
n=50 Participants
ganaxolone suspension (50 mg/ml) 3x's /day for 17 weeks
ganaxolone: active drug
|
|---|---|---|
|
Caregiver Global Impression of Change in Attention
Very Much Improved - Visit 5 (End of Week 17)
|
1 Participants
|
1 Participants
|
|
Caregiver Global Impression of Change in Attention
Much Improved - Visit 5 (End of Week 17)
|
7 Participants
|
2 Participants
|
|
Caregiver Global Impression of Change in Attention
Minimally Improved - Visit 5 (End of Week 17)
|
14 Participants
|
21 Participants
|
|
Caregiver Global Impression of Change in Attention
No Change - Visit 5 (End of Week 17)
|
23 Participants
|
18 Participants
|
|
Caregiver Global Impression of Change in Attention
Minimally Worse - Visit 5 (End of Week 17)
|
1 Participants
|
1 Participants
|
|
Caregiver Global Impression of Change in Attention
Much Worse - Visit 5 (End of Week 17)
|
1 Participants
|
1 Participants
|
|
Caregiver Global Impression of Change in Attention
Very Much Worse - Visit 5 (End of Week 17)
|
0 Participants
|
1 Participants
|
SECONDARY outcome
Timeframe: End of the double-blind 17 week treatment periodPopulation: Intent to Treat Population
Caregiver global impression of change in target behavior during the double-blind treatment period of ganaxolone compared to placebo. Investigators and caregivers reported improvements in attention, mood, behavior and sleep via investigator narratives.
Outcome measures
| Measure |
Placebo
n=51 Participants
placebo suspension 3x's /day for 17 weeks
Placebo: inactive
|
Ganaxolone
n=50 Participants
ganaxolone suspension (50 mg/ml) 3x's /day for 17 weeks
ganaxolone: active drug
|
|---|---|---|
|
Caregiver Global Impression of Change in Target Behavior
Very Much Improved - Visit 5 (End of Week 17)
|
0 Participants
|
0 Participants
|
|
Caregiver Global Impression of Change in Target Behavior
Much Improved - Visit 5 (End of Week 17)
|
6 Participants
|
4 Participants
|
|
Caregiver Global Impression of Change in Target Behavior
Minimally Improved - Visit 5 (End of Week 17)
|
14 Participants
|
20 Participants
|
|
Caregiver Global Impression of Change in Target Behavior
No Change - Visit 5 (End of Week 17)
|
22 Participants
|
19 Participants
|
|
Caregiver Global Impression of Change in Target Behavior
Minimally Worse - Visit 5 (End of Week 17)
|
1 Participants
|
2 Participants
|
|
Caregiver Global Impression of Change in Target Behavior
Much Worse - Visit 5 (End of Week 17)
|
2 Participants
|
0 Participants
|
|
Caregiver Global Impression of Change in Target Behavior
Very Much Worse - Visit 5 (End of Week 17)
|
1 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: End of the double-blind 17 week treatment periodPopulation: Intent to Treat Population
Clinical global impression of improvement during the double-blind treatment period of ganaxolone compared to placebo. The CGI is rated on a 7-point scale, with the severity of illness scale using a range of responses.
Outcome measures
| Measure |
Placebo
n=48 Participants
placebo suspension 3x's /day for 17 weeks
Placebo: inactive
|
Ganaxolone
n=48 Participants
ganaxolone suspension (50 mg/ml) 3x's /day for 17 weeks
ganaxolone: active drug
|
|---|---|---|
|
Clinical Global Impression of Improvement - Parent/Caregiver
Very Much Improved - Visit 5 (End of Week 17) - Parent/Caregiver
|
1 Participants
|
0 Participants
|
|
Clinical Global Impression of Improvement - Parent/Caregiver
Much Improved - Visit 5 (End of Week 17) - Parent/Caregiver
|
7 Participants
|
13 Participants
|
|
Clinical Global Impression of Improvement - Parent/Caregiver
Minimally Improved - Visit 5 (End of Week 17) - Parent/Caregiver
|
13 Participants
|
17 Participants
|
|
Clinical Global Impression of Improvement - Parent/Caregiver
No Change - Visit 5 (End of Week 17) - Parent/Caregiver
|
22 Participants
|
14 Participants
|
|
Clinical Global Impression of Improvement - Parent/Caregiver
Minimally Worse - Visit 5 (End of Week 17) - Parent/Caregiver
|
4 Participants
|
2 Participants
|
|
Clinical Global Impression of Improvement - Parent/Caregiver
Much Worse - Visit 5 (End of Week 17) - Parent/Caregiver
|
1 Participants
|
2 Participants
|
|
Clinical Global Impression of Improvement - Parent/Caregiver
Very Much Worse - Visit 5 (End of Week 17) - Parent/Caregiver
|
0 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: [Time Frame: End of the double-blind 17 week treatment period]Population: ITT Population
Clinical global impression of improvement during the double-blind treatment period of ganaxolone compared to placebo
Outcome measures
| Measure |
Placebo
n=48 Participants
placebo suspension 3x's /day for 17 weeks
Placebo: inactive
|
Ganaxolone
n=48 Participants
ganaxolone suspension (50 mg/ml) 3x's /day for 17 weeks
ganaxolone: active drug
|
|---|---|---|
|
Clinical Global Impression of Improvement - Clinician
Very Much Improved - Visit 5 (End of Week 17) - Clinician
|
0 Participants
|
0 Participants
|
|
Clinical Global Impression of Improvement - Clinician
Much Improved - Visit 5 (End of Week 17) - Clinician
|
7 Participants
|
7 Participants
|
|
Clinical Global Impression of Improvement - Clinician
Minimally Improved - Visit 5 (End of Week 17) - Clinician
|
13 Participants
|
19 Participants
|
|
Clinical Global Impression of Improvement - Clinician
No Change - Visit 5 (End of Week 17) - Clinician
|
19 Participants
|
16 Participants
|
|
Clinical Global Impression of Improvement - Clinician
Minimally Worse - Visit 5 (End of Week 17) - Clinician
|
9 Participants
|
2 Participants
|
|
Clinical Global Impression of Improvement - Clinician
Much Worse - Visit 5 (End of Week 17) - Clinician
|
0 Participants
|
3 Participants
|
|
Clinical Global Impression of Improvement - Clinician
Very Much Worse - Visit 5 (End of Week 17) - Clinician
|
0 Participants
|
1 Participants
|
SECONDARY outcome
Timeframe: End of the double-blind 17 week treatment periodPopulation: ITT Population
Percentage of Seizure-free Days for Major Motor Seizure types during the double-blind treatment period of ganaxolone compared to placebo. The major motor seizure types include bilateral tonic (sustained motor activity = 3 seconds), generalized tonic-clonic, atonic/drop, bilateral clonic, and focal to bilateral tonic-clonic.
Outcome measures
| Measure |
Placebo
n=51 Participants
placebo suspension 3x's /day for 17 weeks
Placebo: inactive
|
Ganaxolone
n=50 Participants
ganaxolone suspension (50 mg/ml) 3x's /day for 17 weeks
ganaxolone: active drug
|
|---|---|---|
|
Percentage of Seizure-free Days for Major Motor Seizure Types
Baseline
|
30.32 percent of seizure-free days
Standard Deviation 27.070
|
22.57 percent of seizure-free days
Standard Deviation 25.761
|
|
Percentage of Seizure-free Days for Major Motor Seizure Types
17-week-Post-Baseline Phase
|
36.17 percent of seizure-free days
Standard Deviation 30.932
|
32.29 percent of seizure-free days
Standard Deviation 30.615
|
|
Percentage of Seizure-free Days for Major Motor Seizure Types
Arithmetic Change from Baseline
|
5.86 percent of seizure-free days
Standard Deviation 15.350
|
9.62 percent of seizure-free days
Standard Deviation 21.364
|
SECONDARY outcome
Timeframe: End of the double-blind 17 week treatment periodPopulation: Intent to Treat Population
Arithmetic change in longest seizure free interval, based on primary seizure types during the double-blind treatment period of ganaxolone compared to placebo
Outcome measures
| Measure |
Placebo
n=51 Participants
placebo suspension 3x's /day for 17 weeks
Placebo: inactive
|
Ganaxolone
n=49 Participants
ganaxolone suspension (50 mg/ml) 3x's /day for 17 weeks
ganaxolone: active drug
|
|---|---|---|
|
Arithmetic Change in Longest Seizure Free Interval, Based on Primary Seizure Types
|
-4.63 days
Standard Deviation 14.867
|
-0.02 days
Standard Deviation 9.376
|
SECONDARY outcome
Timeframe: End of the double-blind 17 week treatment periodPopulation: Intent to Treat Population
Caregiver global impression of change in seizure intensity and duration during the double-blind treatment period of ganaxolone compared to placebo. CGI-C is a 7 point scale that requires the clinician to assess how much the patient's illness has improved or worsened relative to a baseline state at the beginning of the intervention.
Outcome measures
| Measure |
Placebo
n=51 Participants
placebo suspension 3x's /day for 17 weeks
Placebo: inactive
|
Ganaxolone
n=50 Participants
ganaxolone suspension (50 mg/ml) 3x's /day for 17 weeks
ganaxolone: active drug
|
|---|---|---|
|
Caregiver Global Impression of Change in Seizure Intensity and Duration
Very Much Improved - Visit 5 (End of Week 17)
|
1 Participants
|
2 Participants
|
|
Caregiver Global Impression of Change in Seizure Intensity and Duration
Much Improved - Visit 5 (End of Week 17)
|
5 Participants
|
15 Participants
|
|
Caregiver Global Impression of Change in Seizure Intensity and Duration
Minimally Improved - Visit 5 (End of Week 17)
|
11 Participants
|
11 Participants
|
|
Caregiver Global Impression of Change in Seizure Intensity and Duration
No Change - Visit 5 (End of Week 17)
|
21 Participants
|
10 Participants
|
|
Caregiver Global Impression of Change in Seizure Intensity and Duration
4Minimally Worse - Visit 5 (End of Week 17)
|
5 Participants
|
3 Participants
|
|
Caregiver Global Impression of Change in Seizure Intensity and Duration
Much Worse - Visit 5 (End of Week 17)
|
4 Participants
|
2 Participants
|
|
Caregiver Global Impression of Change in Seizure Intensity and Duration
Very Much Worse - Visit 5 (End of Week 17)
|
0 Participants
|
2 Participants
|
Adverse Events
Placebo
Serious events: 5 serious events
Other events: 45 other events
Deaths: 0 deaths
Ganaxolone
Serious events: 6 serious events
Other events: 43 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Placebo
n=51 participants at risk
placebo suspension 3x's /day for 17 weeks
Placebo: inactive
|
Ganaxolone
n=50 participants at risk
ganaxolone suspension (50 mg/ml) 3x's /day for 17 weeks
ganaxolone: active drug
|
|---|---|---|
|
Infections and infestations
Bronchitis
|
0.00%
0/51 • Screening through 17-week Double-blind Phase
|
2.0%
1/50 • Number of events 1 • Screening through 17-week Double-blind Phase
|
|
Infections and infestations
Rhinovirus Infection
|
0.00%
0/51 • Screening through 17-week Double-blind Phase
|
2.0%
1/50 • Number of events 1 • Screening through 17-week Double-blind Phase
|
|
Infections and infestations
Urinary Tract Infection
|
0.00%
0/51 • Screening through 17-week Double-blind Phase
|
2.0%
1/50 • Number of events 1 • Screening through 17-week Double-blind Phase
|
|
Infections and infestations
Pneumonia Mycoplasmal
|
2.0%
1/51 • Number of events 1 • Screening through 17-week Double-blind Phase
|
0.00%
0/50 • Screening through 17-week Double-blind Phase
|
|
Infections and infestations
Pneumonia Viral
|
2.0%
1/51 • Number of events 1 • Screening through 17-week Double-blind Phase
|
0.00%
0/50 • Screening through 17-week Double-blind Phase
|
|
Infections and infestations
Respiratory Syncytial Virus Bronchiolitis
|
2.0%
1/51 • Number of events 1 • Screening through 17-week Double-blind Phase
|
0.00%
0/50 • Screening through 17-week Double-blind Phase
|
|
Investigations
Oxygen Saturation Decreased
|
0.00%
0/51 • Screening through 17-week Double-blind Phase
|
2.0%
1/50 • Number of events 1 • Screening through 17-week Double-blind Phase
|
|
Metabolism and nutrition disorders
Food Refusal
|
0.00%
0/51 • Screening through 17-week Double-blind Phase
|
2.0%
1/50 • Number of events 1 • Screening through 17-week Double-blind Phase
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonia Aspiration
|
0.00%
0/51 • Screening through 17-week Double-blind Phase
|
2.0%
1/50 • Number of events 1 • Screening through 17-week Double-blind Phase
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
2.0%
1/51 • Number of events 2 • Screening through 17-week Double-blind Phase
|
0.00%
0/50 • Screening through 17-week Double-blind Phase
|
|
Gastrointestinal disorders
Faecaloma
|
2.0%
1/51 • Number of events 1 • Screening through 17-week Double-blind Phase
|
0.00%
0/50 • Screening through 17-week Double-blind Phase
|
|
Nervous system disorders
Hypotonia
|
2.0%
1/51 • Number of events 1 • Screening through 17-week Double-blind Phase
|
0.00%
0/50 • Screening through 17-week Double-blind Phase
|
|
Nervous system disorders
Seizure
|
2.0%
1/51 • Number of events 1 • Screening through 17-week Double-blind Phase
|
0.00%
0/50 • Screening through 17-week Double-blind Phase
|
|
Nervous system disorders
Unresponsive to Stimuli
|
2.0%
1/51 • Number of events 2 • Screening through 17-week Double-blind Phase
|
0.00%
0/50 • Screening through 17-week Double-blind Phase
|
Other adverse events
| Measure |
Placebo
n=51 participants at risk
placebo suspension 3x's /day for 17 weeks
Placebo: inactive
|
Ganaxolone
n=50 participants at risk
ganaxolone suspension (50 mg/ml) 3x's /day for 17 weeks
ganaxolone: active drug
|
|---|---|---|
|
Nervous system disorders
Somnolence
|
15.7%
8/51 • Number of events 8 • Screening through 17-week Double-blind Phase
|
36.0%
18/50 • Number of events 20 • Screening through 17-week Double-blind Phase
|
|
Nervous system disorders
Seizure
|
17.6%
9/51 • Number of events 12 • Screening through 17-week Double-blind Phase
|
14.0%
7/50 • Number of events 8 • Screening through 17-week Double-blind Phase
|
|
Nervous system disorders
Sedation
|
3.9%
2/51 • Number of events 2 • Screening through 17-week Double-blind Phase
|
6.0%
3/50 • Number of events 3 • Screening through 17-week Double-blind Phase
|
|
Nervous system disorders
Hypersomnia
|
0.00%
0/51 • Screening through 17-week Double-blind Phase
|
4.0%
2/50 • Number of events 2 • Screening through 17-week Double-blind Phase
|
|
Nervous system disorders
Lethargy
|
3.9%
2/51 • Number of events 2 • Screening through 17-week Double-blind Phase
|
4.0%
2/50 • Number of events 2 • Screening through 17-week Double-blind Phase
|
|
Nervous system disorders
Hyperaesthesia
|
3.9%
2/51 • Number of events 2 • Screening through 17-week Double-blind Phase
|
0.00%
0/50 • Screening through 17-week Double-blind Phase
|
|
Infections and infestations
Upper Respiratory Tract Infection
|
5.9%
3/51 • Number of events 3 • Screening through 17-week Double-blind Phase
|
10.0%
5/50 • Number of events 6 • Screening through 17-week Double-blind Phase
|
|
Infections and infestations
Bronchitis
|
0.00%
0/51 • Screening through 17-week Double-blind Phase
|
4.0%
2/50 • Number of events 2 • Screening through 17-week Double-blind Phase
|
|
Infections and infestations
Ear Infection
|
5.9%
3/51 • Number of events 3 • Screening through 17-week Double-blind Phase
|
4.0%
2/50 • Number of events 2 • Screening through 17-week Double-blind Phase
|
|
Infections and infestations
Influenza
|
2.0%
1/51 • Number of events 1 • Screening through 17-week Double-blind Phase
|
4.0%
2/50 • Number of events 2 • Screening through 17-week Double-blind Phase
|
|
Infections and infestations
Rhinitis
|
7.8%
4/51 • Number of events 5 • Screening through 17-week Double-blind Phase
|
4.0%
2/50 • Number of events 4 • Screening through 17-week Double-blind Phase
|
|
Infections and infestations
Respiratory Tract Infection Viral
|
5.9%
3/51 • Number of events 3 • Screening through 17-week Double-blind Phase
|
2.0%
1/50 • Number of events 1 • Screening through 17-week Double-blind Phase
|
|
Infections and infestations
Urinary Tract Infection
|
5.9%
3/51 • Number of events 3 • Screening through 17-week Double-blind Phase
|
2.0%
1/50 • Number of events 2 • Screening through 17-week Double-blind Phase
|
|
Infections and infestations
Nasopharyngitis
|
9.8%
5/51 • Number of events 5 • Screening through 17-week Double-blind Phase
|
0.00%
0/50 • Screening through 17-week Double-blind Phase
|
|
Infections and infestations
Sinusitis
|
3.9%
2/51 • Number of events 2 • Screening through 17-week Double-blind Phase
|
0.00%
0/50 • Screening through 17-week Double-blind Phase
|
|
Infections and infestations
Varicella
|
3.9%
2/51 • Number of events 3 • Screening through 17-week Double-blind Phase
|
0.00%
0/50 • Screening through 17-week Double-blind Phase
|
|
Gastrointestinal disorders
Vomiting
|
19.6%
10/51 • Number of events 12 • Screening through 17-week Double-blind Phase
|
10.0%
5/50 • Number of events 6 • Screening through 17-week Double-blind Phase
|
|
Gastrointestinal disorders
Constipation
|
5.9%
3/51 • Number of events 3 • Screening through 17-week Double-blind Phase
|
6.0%
3/50 • Number of events 3 • Screening through 17-week Double-blind Phase
|
|
Gastrointestinal disorders
Salivary Hypersecretion
|
2.0%
1/51 • Number of events 1 • Screening through 17-week Double-blind Phase
|
6.0%
3/50 • Number of events 3 • Screening through 17-week Double-blind Phase
|
|
Gastrointestinal disorders
Diarrhoea
|
7.8%
4/51 • Number of events 5 • Screening through 17-week Double-blind Phase
|
2.0%
1/50 • Number of events 1 • Screening through 17-week Double-blind Phase
|
|
Gastrointestinal disorders
Abdominal Pain
|
3.9%
2/51 • Number of events 2 • Screening through 17-week Double-blind Phase
|
0.00%
0/50 • Screening through 17-week Double-blind Phase
|
|
Gastrointestinal disorders
Gastrooesophageal Reflux Disease
|
5.9%
3/51 • Number of events 3 • Screening through 17-week Double-blind Phase
|
0.00%
0/50 • Screening through 17-week Double-blind Phase
|
|
General disorders
Pyrexia
|
7.8%
4/51 • Number of events 5 • Screening through 17-week Double-blind Phase
|
18.0%
9/50 • Number of events 10 • Screening through 17-week Double-blind Phase
|
|
General disorders
Gait Disturbance
|
2.0%
1/51 • Number of events 1 • Screening through 17-week Double-blind Phase
|
4.0%
2/50 • Number of events 2 • Screening through 17-week Double-blind Phase
|
|
Respiratory, thoracic and mediastinal disorders
Nasal Congestion
|
2.0%
1/51 • Number of events 1 • Screening through 17-week Double-blind Phase
|
4.0%
2/50 • Number of events 2 • Screening through 17-week Double-blind Phase
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrhoea
|
3.9%
2/51 • Number of events 2 • Screening through 17-week Double-blind Phase
|
2.0%
1/50 • Number of events 1 • Screening through 17-week Double-blind Phase
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
5.9%
3/51 • Number of events 3 • Screening through 17-week Double-blind Phase
|
0.00%
0/50 • Screening through 17-week Double-blind Phase
|
|
Respiratory, thoracic and mediastinal disorders
Insomnia
|
3.9%
2/51 • Number of events 3 • Screening through 17-week Double-blind Phase
|
4.0%
2/50 • Number of events 2 • Screening through 17-week Double-blind Phase
|
|
Psychiatric disorders
Irritability
|
3.9%
2/51 • Number of events 2 • Screening through 17-week Double-blind Phase
|
4.0%
2/50 • Number of events 2 • Screening through 17-week Double-blind Phase
|
|
Investigations
Body Temperature Increased
|
3.9%
2/51 • Number of events 3 • Screening through 17-week Double-blind Phase
|
0.00%
0/50 • Screening through 17-week Double-blind Phase
|
|
Skin and subcutaneous tissue disorders
Rash
|
7.8%
4/51 • Number of events 4 • Screening through 17-week Double-blind Phase
|
6.0%
3/50 • Number of events 3 • Screening through 17-week Double-blind Phase
|
|
Skin and subcutaneous tissue disorders
Alopecia
|
3.9%
2/51 • Number of events 2 • Screening through 17-week Double-blind Phase
|
0.00%
0/50 • Screening through 17-week Double-blind Phase
|
|
Immune system disorders
Seasonal Allergy
|
0.00%
0/51 • Screening through 17-week Double-blind Phase
|
6.0%
3/50 • Number of events 3 • Screening through 17-week Double-blind Phase
|
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 If the study is part of a multicenter study, the first publication of the study results shall be made by the sponsor in conjunction with the sponsor's presentation of a joint, multicenter publication of the compiled and analyzed study results.
- Publication restrictions are in place
Restriction type: OTHER