Trial Outcomes & Findings for A Randomized, Controlled Trial of Ganaxolone in Patients With Infantile Spasms (NCT NCT00441896)
NCT ID: NCT00441896
Last Updated: 2023-06-02
Results Overview
Spasm clusters were determined by a 24-hour video-electroencephalography (vEEG) at Day 10. Change from Baseline was calculated as the post-Baseline value minus the Baseline value. Baseline was defined as the Day 0 assessment before study drug infusion.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
57 participants
Primary outcome timeframe
Baseline (Day 0) and Day 10
Results posted on
2023-06-02
Participant Flow
This was a randomized, placebo-controlled, double-blind, dose-ranging study in which participants were randomized to ganaxolone:placebo in a 2:1 ratio into one of 5 cohorts. The primary focus of the study was to explore the dosing and titration schedule to help determine how rapidly infants can be titrated to a Maximum tolerated dose (MTD) to control spasms.
Participant milestones
| Measure |
Cohort 1: Sequence A: Ganaxolone/Placebo/Ganaxolone
Participants were administered oral suspension of ganaxolone followed by placebo and then ganaxolone using an oral dosing syringe by hospital staff/parents/guardians 3 times a day (tid). Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 1 received starting doses of 3 milligrams per kilogram (mg/kg) tid with maximum allowable doses of 36 mg/kg/day (12 mg/kg tid).
|
Cohort 1: Sequence B: Placebo/Ganaxolone/Placebo
Participants were administered placebo followed by oral suspension of ganaxolone and then placebo using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 1 received starting doses of 3 mg/kg tid with maximum allowable doses of 36 mg/kg/day (12 mg/kg tid).
|
Cohort 2: Sequence A: Ganaxolone/Placebo/Ganaxolone
Participants were administered oral suspension of ganaxolone followed by placebo and then ganaxolone using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 2 received starting doses of 6 mg/kg tid with maximum allowable doses of 45 mg/kg/day (15 mg/kg tid).
|
Cohort 2: Sequence B: Placebo/Ganaxolone/Placebo
Participants were administered placebo followed by oral suspension of ganaxolone and then placebo using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 2 received starting doses of 6 mg/kg tid with maximum allowable doses of 45 mg/kg/day (15 mg/kg tid).
|
Cohort 3: Sequence A: Ganaxolone/Placebo/Ganaxolone
Participants were administered oral suspension of ganaxolone followed by placebo and then ganaxolone using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 3 received starting doses of 9 mg/kg tid with maximum allowable doses of 54 mg/kg/day (18 mg/kg tid).
|
Cohort 3: Sequence B: Placebo/Ganaxolone/Placebo
Participants were administered placebo followed by oral suspension of ganaxolone and then placebo using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 3 received starting doses of 9 mg/kg tid with maximum allowable doses of 54 mg/kg/day (18 mg/kg tid).
|
Cohort 4: Sequence A: Ganaxolone/Placebo/Ganaxolone
Participants were administered oral suspension of ganaxolone followed by placebo and then ganaxolone using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 4 received starting doses of 12 mg/kg tid with maximum allowable doses of 54 mg/kg/day (18 mg/kg tid).
|
Cohort 4: Sequence B: Placebo/Ganaxolone/Placebo
Participants were administered placebo followed by oral suspension of ganaxolone and then placebo using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 4 received starting doses of 12 mg/kg tid with maximum allowable doses of 54 mg/kg/day (18 mg/kg tid).
|
Cohort 5: Sequence A: Ganaxolone/Placebo/Ganaxolone
Participants were administered oral suspension of ganaxolone followed by placebo and then ganaxolone using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 5 received starting doses of 18 mg/kg tid with maximum allowable doses of 54 mg/kg/day (18 mg/kg tid).
|
Cohort 5: Sequence B: Placebo/Ganaxolone/Placebo
Participants were administered placebo followed by oral suspension of ganaxolone and then placebo using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 5 received starting doses of 18 mg/kg tid with maximum allowable doses of 54 mg/kg/day (18 mg/kg tid).
|
|---|---|---|---|---|---|---|---|---|---|---|
|
Overall Study
STARTED
|
6
|
3
|
7
|
4
|
12
|
3
|
7
|
5
|
6
|
4
|
|
Overall Study
COMPLETED
|
6
|
3
|
7
|
3
|
11
|
3
|
7
|
5
|
6
|
4
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
0
|
1
|
1
|
0
|
0
|
0
|
0
|
0
|
Reasons for withdrawal
| Measure |
Cohort 1: Sequence A: Ganaxolone/Placebo/Ganaxolone
Participants were administered oral suspension of ganaxolone followed by placebo and then ganaxolone using an oral dosing syringe by hospital staff/parents/guardians 3 times a day (tid). Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 1 received starting doses of 3 milligrams per kilogram (mg/kg) tid with maximum allowable doses of 36 mg/kg/day (12 mg/kg tid).
|
Cohort 1: Sequence B: Placebo/Ganaxolone/Placebo
Participants were administered placebo followed by oral suspension of ganaxolone and then placebo using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 1 received starting doses of 3 mg/kg tid with maximum allowable doses of 36 mg/kg/day (12 mg/kg tid).
|
Cohort 2: Sequence A: Ganaxolone/Placebo/Ganaxolone
Participants were administered oral suspension of ganaxolone followed by placebo and then ganaxolone using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 2 received starting doses of 6 mg/kg tid with maximum allowable doses of 45 mg/kg/day (15 mg/kg tid).
|
Cohort 2: Sequence B: Placebo/Ganaxolone/Placebo
Participants were administered placebo followed by oral suspension of ganaxolone and then placebo using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 2 received starting doses of 6 mg/kg tid with maximum allowable doses of 45 mg/kg/day (15 mg/kg tid).
|
Cohort 3: Sequence A: Ganaxolone/Placebo/Ganaxolone
Participants were administered oral suspension of ganaxolone followed by placebo and then ganaxolone using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 3 received starting doses of 9 mg/kg tid with maximum allowable doses of 54 mg/kg/day (18 mg/kg tid).
|
Cohort 3: Sequence B: Placebo/Ganaxolone/Placebo
Participants were administered placebo followed by oral suspension of ganaxolone and then placebo using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 3 received starting doses of 9 mg/kg tid with maximum allowable doses of 54 mg/kg/day (18 mg/kg tid).
|
Cohort 4: Sequence A: Ganaxolone/Placebo/Ganaxolone
Participants were administered oral suspension of ganaxolone followed by placebo and then ganaxolone using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 4 received starting doses of 12 mg/kg tid with maximum allowable doses of 54 mg/kg/day (18 mg/kg tid).
|
Cohort 4: Sequence B: Placebo/Ganaxolone/Placebo
Participants were administered placebo followed by oral suspension of ganaxolone and then placebo using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 4 received starting doses of 12 mg/kg tid with maximum allowable doses of 54 mg/kg/day (18 mg/kg tid).
|
Cohort 5: Sequence A: Ganaxolone/Placebo/Ganaxolone
Participants were administered oral suspension of ganaxolone followed by placebo and then ganaxolone using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 5 received starting doses of 18 mg/kg tid with maximum allowable doses of 54 mg/kg/day (18 mg/kg tid).
|
Cohort 5: Sequence B: Placebo/Ganaxolone/Placebo
Participants were administered placebo followed by oral suspension of ganaxolone and then placebo using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 5 received starting doses of 18 mg/kg tid with maximum allowable doses of 54 mg/kg/day (18 mg/kg tid).
|
|---|---|---|---|---|---|---|---|---|---|---|
|
Overall Study
Withdrawal by Subject
|
0
|
0
|
0
|
1
|
0
|
0
|
0
|
0
|
0
|
0
|
|
Overall Study
Other
|
0
|
0
|
0
|
0
|
1
|
0
|
0
|
0
|
0
|
0
|
Baseline Characteristics
A Randomized, Controlled Trial of Ganaxolone in Patients With Infantile Spasms
Baseline characteristics by cohort
| Measure |
Cohort 1: Sequence A: Ganaxolone/Placebo/Ganaxolone
n=6 Participants
Participants were administered oral suspension of ganaxolone followed by placebo and then ganaxolone using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 1 received starting doses of 3 mg/kg tid with maximum allowable doses of 36 mg/kg/day (12 mg/kg tid).
|
Cohort 1: Sequence B: Placebo/Ganaxolone/Placebo
n=3 Participants
Participants were administered placebo followed by oral suspension of ganaxolone and then placebo using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 1 received starting doses of 3 mg/kg tid with maximum allowable doses of 36 mg/kg/day (12 mg/kg tid).
|
Cohort 2: Sequence A: Ganaxolone/Placebo/Ganaxolone
n=7 Participants
Participants were administered oral suspension of ganaxolone followed by placebo and then ganaxolone using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 2 received starting doses of 6 mg/kg tid with maximum allowable doses of 45 mg/kg/day (15 mg/kg tid).
|
Cohort 2: Sequence B: Placebo/Ganaxolone/Placebo
n=4 Participants
Participants were administered placebo followed by oral suspension of ganaxolone and then placebo using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 2 received starting doses of 6 mg/kg tid with maximum allowable doses of 45 mg/kg/day (15 mg/kg tid).
|
Cohort 3: Sequence A: Ganaxolone/Placebo/Ganaxolone
n=11 Participants
Participants were administered oral suspension of ganaxolone followed by placebo and then ganaxolone using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 3 received starting doses of 9 mg/kg tid with maximum allowable doses of 54 mg/kg/day (18 mg/kg tid).
|
Cohort 3: Sequence B: Placebo/Ganaxolone/Placebo
n=3 Participants
Participants were administered placebo followed by oral suspension of ganaxolone and then placebo using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 3 received starting doses of 9 mg/kg tid with maximum allowable doses of 54 mg/kg/day (18 mg/kg tid).
|
Cohort 4: Sequence A: Ganaxolone/Placebo/Ganaxolone
n=7 Participants
Participants were administered oral suspension of ganaxolone followed by placebo and then ganaxolone using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 4 received starting doses of 12 mg/kg tid with maximum allowable doses of 54 mg/kg/day (18 mg/kg tid).
|
Cohort 4: Sequence B: Placebo/Ganaxolone/Placebo
n=5 Participants
Participants were administered placebo followed by oral suspension of ganaxolone and then placebo using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 4 received starting doses of 12 mg/kg tid with maximum allowable doses of 54 mg/kg/day (18 mg/kg tid).
|
Cohort 5: Sequence A: Ganaxolone/Placebo/Ganaxolone
n=6 Participants
Participants were administered oral suspension of ganaxolone followed by placebo and then ganaxolone using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 5 received starting doses of 18 mg/kg tid with maximum allowable doses of 54 mg/kg/day (18 mg/kg tid).
|
Cohort 5: Sequence B: Placebo/Ganaxolone/Placebo
n=4 Participants
Participants were administered placebo followed by oral suspension of ganaxolone and then placebo using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 5 received starting doses of 18 mg/kg tid with maximum allowable doses of 54 mg/kg/day (18 mg/kg tid).
|
Total
n=56 Participants
Total of all reporting groups
|
|---|---|---|---|---|---|---|---|---|---|---|---|
|
Age, Continuous
|
8.2 Months
STANDARD_DEVIATION 2.48 • n=5 Participants
|
20.3 Months
STANDARD_DEVIATION 3.06 • n=7 Participants
|
13.4 Months
STANDARD_DEVIATION 4.89 • n=5 Participants
|
23.0 Months
STANDARD_DEVIATION 12.25 • n=4 Participants
|
11.2 Months
STANDARD_DEVIATION 5.47 • n=21 Participants
|
14.0 Months
STANDARD_DEVIATION 6.93 • n=10 Participants
|
10.3 Months
STANDARD_DEVIATION 4.96 • n=115 Participants
|
13.2 Months
STANDARD_DEVIATION 3.03 • n=24 Participants
|
11.3 Months
STANDARD_DEVIATION 3.27 • n=42 Participants
|
11.5 Months
STANDARD_DEVIATION 7.55 • n=42 Participants
|
12.7 Months
STANDARD_DEVIATION 6.41 • n=42 Participants
|
|
Sex: Female, Male
Female
|
3 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
5 Participants
n=5 Participants
|
1 Participants
n=4 Participants
|
6 Participants
n=21 Participants
|
2 Participants
n=10 Participants
|
3 Participants
n=115 Participants
|
4 Participants
n=24 Participants
|
3 Participants
n=42 Participants
|
2 Participants
n=42 Participants
|
32 Participants
n=42 Participants
|
|
Sex: Female, Male
Male
|
3 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
3 Participants
n=4 Participants
|
5 Participants
n=21 Participants
|
1 Participants
n=10 Participants
|
4 Participants
n=115 Participants
|
1 Participants
n=24 Participants
|
3 Participants
n=42 Participants
|
2 Participants
n=42 Participants
|
24 Participants
n=42 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
1 Participants
n=21 Participants
|
2 Participants
n=10 Participants
|
2 Participants
n=115 Participants
|
1 Participants
n=24 Participants
|
0 Participants
n=42 Participants
|
0 Participants
n=42 Participants
|
9 Participants
n=42 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
5 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
5 Participants
n=5 Participants
|
4 Participants
n=4 Participants
|
10 Participants
n=21 Participants
|
1 Participants
n=10 Participants
|
5 Participants
n=115 Participants
|
4 Participants
n=24 Participants
|
6 Participants
n=42 Participants
|
4 Participants
n=42 Participants
|
47 Participants
n=42 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
0 Participants
n=10 Participants
|
0 Participants
n=115 Participants
|
0 Participants
n=24 Participants
|
0 Participants
n=42 Participants
|
0 Participants
n=42 Participants
|
0 Participants
n=42 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
0 Participants
n=10 Participants
|
0 Participants
n=115 Participants
|
0 Participants
n=24 Participants
|
0 Participants
n=42 Participants
|
0 Participants
n=42 Participants
|
0 Participants
n=42 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
2 Participants
n=4 Participants
|
2 Participants
n=21 Participants
|
0 Participants
n=10 Participants
|
1 Participants
n=115 Participants
|
1 Participants
n=24 Participants
|
2 Participants
n=42 Participants
|
0 Participants
n=42 Participants
|
8 Participants
n=42 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
0 Participants
n=10 Participants
|
0 Participants
n=115 Participants
|
0 Participants
n=24 Participants
|
0 Participants
n=42 Participants
|
0 Participants
n=42 Participants
|
0 Participants
n=42 Participants
|
|
Race (NIH/OMB)
Black or African American
|
3 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
1 Participants
n=4 Participants
|
2 Participants
n=21 Participants
|
0 Participants
n=10 Participants
|
1 Participants
n=115 Participants
|
0 Participants
n=24 Participants
|
0 Participants
n=42 Participants
|
1 Participants
n=42 Participants
|
9 Participants
n=42 Participants
|
|
Race (NIH/OMB)
White
|
3 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
7 Participants
n=5 Participants
|
1 Participants
n=4 Participants
|
6 Participants
n=21 Participants
|
3 Participants
n=10 Participants
|
5 Participants
n=115 Participants
|
3 Participants
n=24 Participants
|
1 Participants
n=42 Participants
|
3 Participants
n=42 Participants
|
34 Participants
n=42 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
0 Participants
n=10 Participants
|
0 Participants
n=115 Participants
|
0 Participants
n=24 Participants
|
0 Participants
n=42 Participants
|
0 Participants
n=42 Participants
|
0 Participants
n=42 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
1 Participants
n=21 Participants
|
0 Participants
n=10 Participants
|
0 Participants
n=115 Participants
|
1 Participants
n=24 Participants
|
3 Participants
n=42 Participants
|
0 Participants
n=42 Participants
|
5 Participants
n=42 Participants
|
PRIMARY outcome
Timeframe: Baseline (Day 0) and Day 10Population: Efficacy Population included all randomized participants who received at least 1 evaluation of efficacy at both Baseline (Day 0) and at either Visit 5 (Day 10) or Visit 8 (Day 20). Only those participants with data available at the indicated time point were analyzed.
Spasm clusters were determined by a 24-hour video-electroencephalography (vEEG) at Day 10. Change from Baseline was calculated as the post-Baseline value minus the Baseline value. Baseline was defined as the Day 0 assessment before study drug infusion.
Outcome measures
| Measure |
Cohort 1: Sequence A: Ganaxolone/Placebo/Ganaxolone
n=6 Participants
Participants were administered oral suspension of ganaxolone followed by placebo and then ganaxolone using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 1 received starting doses of 3 mg/kg tid with maximum allowable doses of 36 mg/kg/day (12 mg/kg tid).
|
Cohort 1: Sequence B: Placebo/Ganaxolone/Placebo
n=3 Participants
Participants were administered placebo followed by oral suspension of ganaxolone and then placebo using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 1 received starting doses of 3 mg/kg tid with maximum allowable doses of 36 mg/kg/day (12 mg/kg tid).
|
Cohort 2: Sequence A: Ganaxolone/Placebo/Ganaxolone
n=7 Participants
Participants were administered oral suspension of ganaxolone followed by placebo and then ganaxolone using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 2 received starting doses of 6 mg/kg tid with maximum allowable doses of 45 mg/kg/day (15 mg/kg tid).
|
Cohort 2: Sequence B: Placebo/Ganaxolone/Placebo
n=4 Participants
Participants were administered placebo followed by oral suspension of ganaxolone and then placebo using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 2 received starting doses of 6 mg/kg tid with maximum allowable doses of 45 mg/kg/day (15 mg/kg tid).
|
Cohort 3: Sequence A: Ganaxolone/Placebo/Ganaxolone
n=11 Participants
Participants were administered oral suspension of ganaxolone followed by placebo and then ganaxolone using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 3 received starting doses of 9 mg/kg tid with maximum allowable doses of 54 mg/kg/day (18 mg/kg tid).
|
Cohort 3: Sequence B: Placebo/Ganaxolone/Placebo
n=3 Participants
Participants were administered placebo followed by oral suspension of ganaxolone and then placebo using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 3 received starting doses of 9 mg/kg tid with maximum allowable doses of 54 mg/kg/day (18 mg/kg tid).
|
Cohort 4: Sequence A: Ganaxolone/Placebo/Ganaxolone
n=7 Participants
Participants were administered oral suspension of ganaxolone followed by placebo and then ganaxolone using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 4 received starting doses of 12 mg/kg tid with maximum allowable doses of 54 mg/kg/day (18 mg/kg tid).
|
Cohort 4: Sequence B: Placebo/Ganaxolone/Placebo
n=5 Participants
Participants were administered placebo followed by oral suspension of ganaxolone and then placebo using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 4 received starting doses of 12 mg/kg tid with maximum allowable doses of 54 mg/kg/day (18 mg/kg tid).
|
Cohort 5: Sequence A: Ganaxolone/Placebo/Ganaxolone
n=5 Participants
Participants were administered oral suspension of ganaxolone followed by placebo and then ganaxolone using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 5 received starting doses of 18 mg/kg tid with maximum allowable doses of 54 mg/kg/day (18 mg/kg tid).
|
Cohort 5: Sequence B: Placebo/Ganaxolone/Placebo
n=3 Participants
Participants were administered placebo followed by oral suspension of ganaxolone and then placebo using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 5 received starting doses of 18 mg/kg tid with maximum allowable doses of 54 mg/kg/day (18 mg/kg tid).
|
|---|---|---|---|---|---|---|---|---|---|---|
|
Change From Baseline in Frequency of Spasm Clusters at Day 10
|
1.0 Spasm clusters per day
Standard Deviation 20.06
|
2.7 Spasm clusters per day
Standard Deviation 6.66
|
-5.4 Spasm clusters per day
Standard Deviation 10.97
|
-1.0 Spasm clusters per day
Standard Deviation 1.83
|
-1.4 Spasm clusters per day
Standard Deviation 13.69
|
-5.3 Spasm clusters per day
Standard Deviation 10.12
|
-3.7 Spasm clusters per day
Standard Deviation 4.92
|
0.2 Spasm clusters per day
Standard Deviation 13.12
|
-3.2 Spasm clusters per day
Standard Deviation 5.02
|
-1.7 Spasm clusters per day
Standard Deviation 7.64
|
SECONDARY outcome
Timeframe: Baseline (Day 0) and Day 20Population: Efficacy Population.
Spasm clusters were determined by a 24-hour vEEG at Day 20. Change from Baseline was calculated as the post-Baseline value minus the Baseline value. Baseline was defined as the Day 0 assessment before study drug infusion.
Outcome measures
| Measure |
Cohort 1: Sequence A: Ganaxolone/Placebo/Ganaxolone
n=6 Participants
Participants were administered oral suspension of ganaxolone followed by placebo and then ganaxolone using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 1 received starting doses of 3 mg/kg tid with maximum allowable doses of 36 mg/kg/day (12 mg/kg tid).
|
Cohort 1: Sequence B: Placebo/Ganaxolone/Placebo
n=3 Participants
Participants were administered placebo followed by oral suspension of ganaxolone and then placebo using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 1 received starting doses of 3 mg/kg tid with maximum allowable doses of 36 mg/kg/day (12 mg/kg tid).
|
Cohort 2: Sequence A: Ganaxolone/Placebo/Ganaxolone
n=7 Participants
Participants were administered oral suspension of ganaxolone followed by placebo and then ganaxolone using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 2 received starting doses of 6 mg/kg tid with maximum allowable doses of 45 mg/kg/day (15 mg/kg tid).
|
Cohort 2: Sequence B: Placebo/Ganaxolone/Placebo
n=4 Participants
Participants were administered placebo followed by oral suspension of ganaxolone and then placebo using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 2 received starting doses of 6 mg/kg tid with maximum allowable doses of 45 mg/kg/day (15 mg/kg tid).
|
Cohort 3: Sequence A: Ganaxolone/Placebo/Ganaxolone
n=11 Participants
Participants were administered oral suspension of ganaxolone followed by placebo and then ganaxolone using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 3 received starting doses of 9 mg/kg tid with maximum allowable doses of 54 mg/kg/day (18 mg/kg tid).
|
Cohort 3: Sequence B: Placebo/Ganaxolone/Placebo
n=3 Participants
Participants were administered placebo followed by oral suspension of ganaxolone and then placebo using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 3 received starting doses of 9 mg/kg tid with maximum allowable doses of 54 mg/kg/day (18 mg/kg tid).
|
Cohort 4: Sequence A: Ganaxolone/Placebo/Ganaxolone
n=7 Participants
Participants were administered oral suspension of ganaxolone followed by placebo and then ganaxolone using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 4 received starting doses of 12 mg/kg tid with maximum allowable doses of 54 mg/kg/day (18 mg/kg tid).
|
Cohort 4: Sequence B: Placebo/Ganaxolone/Placebo
n=5 Participants
Participants were administered placebo followed by oral suspension of ganaxolone and then placebo using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 4 received starting doses of 12 mg/kg tid with maximum allowable doses of 54 mg/kg/day (18 mg/kg tid).
|
Cohort 5: Sequence A: Ganaxolone/Placebo/Ganaxolone
n=6 Participants
Participants were administered oral suspension of ganaxolone followed by placebo and then ganaxolone using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 5 received starting doses of 18 mg/kg tid with maximum allowable doses of 54 mg/kg/day (18 mg/kg tid).
|
Cohort 5: Sequence B: Placebo/Ganaxolone/Placebo
n=3 Participants
Participants were administered placebo followed by oral suspension of ganaxolone and then placebo using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 5 received starting doses of 18 mg/kg tid with maximum allowable doses of 54 mg/kg/day (18 mg/kg tid).
|
|---|---|---|---|---|---|---|---|---|---|---|
|
Change From Baseline in Frequency of Spasm Clusters at Day 20
|
-3.2 Spasm clusters per day
Standard Deviation 10.91
|
6.0 Spasm clusters per day
Standard Deviation 11.53
|
-5.0 Spasm clusters per day
Standard Deviation 7.55
|
-1.5 Spasm clusters per day
Standard Deviation 3.11
|
-1.1 Spasm clusters per day
Standard Deviation 11.21
|
-12.7 Spasm clusters per day
Standard Deviation 26.27
|
-4.4 Spasm clusters per day
Standard Deviation 6.63
|
-1.2 Spasm clusters per day
Standard Deviation 13.85
|
-3.2 Spasm clusters per day
Standard Deviation 4.92
|
-5.3 Spasm clusters per day
Standard Deviation 7.57
|
SECONDARY outcome
Timeframe: Day 10 and Day 20Population: Efficacy Population. Only those participants with data available at the indicated time point were analyzed.
Absence of hypsarrhythmia was determined by 24-hour vEEG at Day 10 and Day 20.
Outcome measures
| Measure |
Cohort 1: Sequence A: Ganaxolone/Placebo/Ganaxolone
n=6 Participants
Participants were administered oral suspension of ganaxolone followed by placebo and then ganaxolone using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 1 received starting doses of 3 mg/kg tid with maximum allowable doses of 36 mg/kg/day (12 mg/kg tid).
|
Cohort 1: Sequence B: Placebo/Ganaxolone/Placebo
n=3 Participants
Participants were administered placebo followed by oral suspension of ganaxolone and then placebo using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 1 received starting doses of 3 mg/kg tid with maximum allowable doses of 36 mg/kg/day (12 mg/kg tid).
|
Cohort 2: Sequence A: Ganaxolone/Placebo/Ganaxolone
n=7 Participants
Participants were administered oral suspension of ganaxolone followed by placebo and then ganaxolone using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 2 received starting doses of 6 mg/kg tid with maximum allowable doses of 45 mg/kg/day (15 mg/kg tid).
|
Cohort 2: Sequence B: Placebo/Ganaxolone/Placebo
n=4 Participants
Participants were administered placebo followed by oral suspension of ganaxolone and then placebo using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 2 received starting doses of 6 mg/kg tid with maximum allowable doses of 45 mg/kg/day (15 mg/kg tid).
|
Cohort 3: Sequence A: Ganaxolone/Placebo/Ganaxolone
n=11 Participants
Participants were administered oral suspension of ganaxolone followed by placebo and then ganaxolone using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 3 received starting doses of 9 mg/kg tid with maximum allowable doses of 54 mg/kg/day (18 mg/kg tid).
|
Cohort 3: Sequence B: Placebo/Ganaxolone/Placebo
n=3 Participants
Participants were administered placebo followed by oral suspension of ganaxolone and then placebo using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 3 received starting doses of 9 mg/kg tid with maximum allowable doses of 54 mg/kg/day (18 mg/kg tid).
|
Cohort 4: Sequence A: Ganaxolone/Placebo/Ganaxolone
n=7 Participants
Participants were administered oral suspension of ganaxolone followed by placebo and then ganaxolone using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 4 received starting doses of 12 mg/kg tid with maximum allowable doses of 54 mg/kg/day (18 mg/kg tid).
|
Cohort 4: Sequence B: Placebo/Ganaxolone/Placebo
n=5 Participants
Participants were administered placebo followed by oral suspension of ganaxolone and then placebo using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 4 received starting doses of 12 mg/kg tid with maximum allowable doses of 54 mg/kg/day (18 mg/kg tid).
|
Cohort 5: Sequence A: Ganaxolone/Placebo/Ganaxolone
n=6 Participants
Participants were administered oral suspension of ganaxolone followed by placebo and then ganaxolone using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 5 received starting doses of 18 mg/kg tid with maximum allowable doses of 54 mg/kg/day (18 mg/kg tid).
|
Cohort 5: Sequence B: Placebo/Ganaxolone/Placebo
n=3 Participants
Participants were administered placebo followed by oral suspension of ganaxolone and then placebo using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 5 received starting doses of 18 mg/kg tid with maximum allowable doses of 54 mg/kg/day (18 mg/kg tid).
|
|---|---|---|---|---|---|---|---|---|---|---|
|
Number of Participants With Absence of Hypsarrhythmia
Day 10
|
1 Participants
|
0 Participants
|
2 Participants
|
0 Participants
|
2 Participants
|
0 Participants
|
2 Participants
|
2 Participants
|
1 Participants
|
0 Participants
|
|
Number of Participants With Absence of Hypsarrhythmia
Day 20
|
1 Participants
|
0 Participants
|
2 Participants
|
0 Participants
|
2 Participants
|
0 Participants
|
2 Participants
|
2 Participants
|
3 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: Baseline (Day 0), Day 10 and Day 20Population: Efficacy Population. Only those participants with data available at the indicated time point were analyzed.
The investigators rated the participants' overall clinical status based on 7 clinical factors: seizure frequency, duration, and intensity; adverse experiences; social, intellectual, and motor functioning. Using a 7-point scale (\[1\] marked improvement, \[2\] moderate improvement, \[3\] slight improvement, \[4\] no change from Baseline, \[5\] slight worsening, \[6\] moderate worsening, or \[7\] marked worsening). Higher score indicated worse symptoms. The investigators assessed the participants' status compared to their condition prior to initiating study medication.
Outcome measures
| Measure |
Cohort 1: Sequence A: Ganaxolone/Placebo/Ganaxolone
n=6 Participants
Participants were administered oral suspension of ganaxolone followed by placebo and then ganaxolone using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 1 received starting doses of 3 mg/kg tid with maximum allowable doses of 36 mg/kg/day (12 mg/kg tid).
|
Cohort 1: Sequence B: Placebo/Ganaxolone/Placebo
n=3 Participants
Participants were administered placebo followed by oral suspension of ganaxolone and then placebo using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 1 received starting doses of 3 mg/kg tid with maximum allowable doses of 36 mg/kg/day (12 mg/kg tid).
|
Cohort 2: Sequence A: Ganaxolone/Placebo/Ganaxolone
n=7 Participants
Participants were administered oral suspension of ganaxolone followed by placebo and then ganaxolone using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 2 received starting doses of 6 mg/kg tid with maximum allowable doses of 45 mg/kg/day (15 mg/kg tid).
|
Cohort 2: Sequence B: Placebo/Ganaxolone/Placebo
n=4 Participants
Participants were administered placebo followed by oral suspension of ganaxolone and then placebo using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 2 received starting doses of 6 mg/kg tid with maximum allowable doses of 45 mg/kg/day (15 mg/kg tid).
|
Cohort 3: Sequence A: Ganaxolone/Placebo/Ganaxolone
n=10 Participants
Participants were administered oral suspension of ganaxolone followed by placebo and then ganaxolone using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 3 received starting doses of 9 mg/kg tid with maximum allowable doses of 54 mg/kg/day (18 mg/kg tid).
|
Cohort 3: Sequence B: Placebo/Ganaxolone/Placebo
n=3 Participants
Participants were administered placebo followed by oral suspension of ganaxolone and then placebo using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 3 received starting doses of 9 mg/kg tid with maximum allowable doses of 54 mg/kg/day (18 mg/kg tid).
|
Cohort 4: Sequence A: Ganaxolone/Placebo/Ganaxolone
n=7 Participants
Participants were administered oral suspension of ganaxolone followed by placebo and then ganaxolone using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 4 received starting doses of 12 mg/kg tid with maximum allowable doses of 54 mg/kg/day (18 mg/kg tid).
|
Cohort 4: Sequence B: Placebo/Ganaxolone/Placebo
n=5 Participants
Participants were administered placebo followed by oral suspension of ganaxolone and then placebo using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 4 received starting doses of 12 mg/kg tid with maximum allowable doses of 54 mg/kg/day (18 mg/kg tid).
|
Cohort 5: Sequence A: Ganaxolone/Placebo/Ganaxolone
n=6 Participants
Participants were administered oral suspension of ganaxolone followed by placebo and then ganaxolone using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 5 received starting doses of 18 mg/kg tid with maximum allowable doses of 54 mg/kg/day (18 mg/kg tid).
|
Cohort 5: Sequence B: Placebo/Ganaxolone/Placebo
n=3 Participants
Participants were administered placebo followed by oral suspension of ganaxolone and then placebo using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 5 received starting doses of 18 mg/kg tid with maximum allowable doses of 54 mg/kg/day (18 mg/kg tid).
|
|---|---|---|---|---|---|---|---|---|---|---|
|
Number of Participants With Change in Clinical Status on the Investigator's Global Assessment
Severe worsening at Day 20
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants With Change in Clinical Status on the Investigator's Global Assessment
Marked improvement at Day 10
|
1 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
1 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants With Change in Clinical Status on the Investigator's Global Assessment
Moderate improvement at Day 10
|
0 Participants
|
0 Participants
|
2 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
1 Participants
|
0 Participants
|
1 Participants
|
0 Participants
|
|
Number of Participants With Change in Clinical Status on the Investigator's Global Assessment
Slight improvement at Day 10
|
1 Participants
|
0 Participants
|
1 Participants
|
1 Participants
|
2 Participants
|
1 Participants
|
2 Participants
|
2 Participants
|
2 Participants
|
0 Participants
|
|
Number of Participants With Change in Clinical Status on the Investigator's Global Assessment
No change from Baseline at Day 10
|
4 Participants
|
3 Participants
|
4 Participants
|
3 Participants
|
7 Participants
|
2 Participants
|
4 Participants
|
3 Participants
|
3 Participants
|
3 Participants
|
|
Number of Participants With Change in Clinical Status on the Investigator's Global Assessment
Slight worsening at Day 10
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants With Change in Clinical Status on the Investigator's Global Assessment
Moderate worsening at Day 10
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants With Change in Clinical Status on the Investigator's Global Assessment
Severe worsening at Day 10
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants With Change in Clinical Status on the Investigator's Global Assessment
Marked improvement at Day 20
|
2 Participants
|
1 Participants
|
1 Participants
|
0 Participants
|
1 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants With Change in Clinical Status on the Investigator's Global Assessment
Moderate improvement at Day 20
|
1 Participants
|
2 Participants
|
1 Participants
|
1 Participants
|
1 Participants
|
0 Participants
|
2 Participants
|
1 Participants
|
1 Participants
|
1 Participants
|
|
Number of Participants With Change in Clinical Status on the Investigator's Global Assessment
Slight improvement at Day 20
|
2 Participants
|
0 Participants
|
3 Participants
|
1 Participants
|
6 Participants
|
3 Participants
|
2 Participants
|
3 Participants
|
4 Participants
|
0 Participants
|
|
Number of Participants With Change in Clinical Status on the Investigator's Global Assessment
No change from Baseline at Day 20
|
1 Participants
|
0 Participants
|
2 Participants
|
2 Participants
|
2 Participants
|
0 Participants
|
3 Participants
|
1 Participants
|
1 Participants
|
2 Participants
|
|
Number of Participants With Change in Clinical Status on the Investigator's Global Assessment
Slight worsening at Day 20
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants With Change in Clinical Status on the Investigator's Global Assessment
Moderate worsening at Day 20
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: Baseline (Day 0), Day10 and Day 20Population: Efficacy population. Only those participants with data available at the indicated time point were analyzed.
Caregiver global assessment of seizure severity and response to treatment rated the participants' based on 7 clinical factors: seizure frequency, duration, and intensity; adverse experiences; social, intellectual, and motor functioning. Using a 7-point scale (\[1\] marked improvement, \[2\] moderate improvement, \[3\] slight improvement, \[4\] no change from Baseline, \[5\] slight worsening, \[6\] moderate worsening, or \[7\] marked worsening). Higher score indicated worse symptoms. The assessment compared the participants' current status to their condition prior to initiating study medication.
Outcome measures
| Measure |
Cohort 1: Sequence A: Ganaxolone/Placebo/Ganaxolone
n=6 Participants
Participants were administered oral suspension of ganaxolone followed by placebo and then ganaxolone using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 1 received starting doses of 3 mg/kg tid with maximum allowable doses of 36 mg/kg/day (12 mg/kg tid).
|
Cohort 1: Sequence B: Placebo/Ganaxolone/Placebo
n=3 Participants
Participants were administered placebo followed by oral suspension of ganaxolone and then placebo using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 1 received starting doses of 3 mg/kg tid with maximum allowable doses of 36 mg/kg/day (12 mg/kg tid).
|
Cohort 2: Sequence A: Ganaxolone/Placebo/Ganaxolone
n=7 Participants
Participants were administered oral suspension of ganaxolone followed by placebo and then ganaxolone using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 2 received starting doses of 6 mg/kg tid with maximum allowable doses of 45 mg/kg/day (15 mg/kg tid).
|
Cohort 2: Sequence B: Placebo/Ganaxolone/Placebo
n=4 Participants
Participants were administered placebo followed by oral suspension of ganaxolone and then placebo using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 2 received starting doses of 6 mg/kg tid with maximum allowable doses of 45 mg/kg/day (15 mg/kg tid).
|
Cohort 3: Sequence A: Ganaxolone/Placebo/Ganaxolone
n=10 Participants
Participants were administered oral suspension of ganaxolone followed by placebo and then ganaxolone using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 3 received starting doses of 9 mg/kg tid with maximum allowable doses of 54 mg/kg/day (18 mg/kg tid).
|
Cohort 3: Sequence B: Placebo/Ganaxolone/Placebo
n=3 Participants
Participants were administered placebo followed by oral suspension of ganaxolone and then placebo using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 3 received starting doses of 9 mg/kg tid with maximum allowable doses of 54 mg/kg/day (18 mg/kg tid).
|
Cohort 4: Sequence A: Ganaxolone/Placebo/Ganaxolone
n=7 Participants
Participants were administered oral suspension of ganaxolone followed by placebo and then ganaxolone using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 4 received starting doses of 12 mg/kg tid with maximum allowable doses of 54 mg/kg/day (18 mg/kg tid).
|
Cohort 4: Sequence B: Placebo/Ganaxolone/Placebo
n=5 Participants
Participants were administered placebo followed by oral suspension of ganaxolone and then placebo using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 4 received starting doses of 12 mg/kg tid with maximum allowable doses of 54 mg/kg/day (18 mg/kg tid).
|
Cohort 5: Sequence A: Ganaxolone/Placebo/Ganaxolone
n=6 Participants
Participants were administered oral suspension of ganaxolone followed by placebo and then ganaxolone using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 5 received starting doses of 18 mg/kg tid with maximum allowable doses of 54 mg/kg/day (18 mg/kg tid).
|
Cohort 5: Sequence B: Placebo/Ganaxolone/Placebo
n=3 Participants
Participants were administered placebo followed by oral suspension of ganaxolone and then placebo using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 5 received starting doses of 18 mg/kg tid with maximum allowable doses of 54 mg/kg/day (18 mg/kg tid).
|
|---|---|---|---|---|---|---|---|---|---|---|
|
Number of Participants With Change in Clinical Status on Caregiver's Global Assessment
Marked improvement at Day 10
|
1 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
1 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants With Change in Clinical Status on Caregiver's Global Assessment
Moderate improvement at Day 10
|
0 Participants
|
0 Participants
|
2 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
1 Participants
|
0 Participants
|
1 Participants
|
0 Participants
|
|
Number of Participants With Change in Clinical Status on Caregiver's Global Assessment
Slight improvement at Day 10
|
1 Participants
|
1 Participants
|
1 Participants
|
2 Participants
|
5 Participants
|
1 Participants
|
2 Participants
|
3 Participants
|
2 Participants
|
1 Participants
|
|
Number of Participants With Change in Clinical Status on Caregiver's Global Assessment
No change from Baseline at Day 10
|
4 Participants
|
2 Participants
|
4 Participants
|
2 Participants
|
4 Participants
|
2 Participants
|
3 Participants
|
2 Participants
|
3 Participants
|
2 Participants
|
|
Number of Participants With Change in Clinical Status on Caregiver's Global Assessment
Slight worsening at Day 10
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
1 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants With Change in Clinical Status on Caregiver's Global Assessment
Moderate worsening at Day 10
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants With Change in Clinical Status on Caregiver's Global Assessment
Severe worsening at Day 10
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants With Change in Clinical Status on Caregiver's Global Assessment
Marked improvement at Day 20
|
2 Participants
|
1 Participants
|
1 Participants
|
0 Participants
|
1 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants With Change in Clinical Status on Caregiver's Global Assessment
Moderate improvement at Day 20
|
1 Participants
|
2 Participants
|
1 Participants
|
1 Participants
|
1 Participants
|
1 Participants
|
2 Participants
|
1 Participants
|
1 Participants
|
0 Participants
|
|
Number of Participants With Change in Clinical Status on Caregiver's Global Assessment
Slight improvement at Day 20
|
2 Participants
|
0 Participants
|
2 Participants
|
1 Participants
|
6 Participants
|
2 Participants
|
3 Participants
|
3 Participants
|
4 Participants
|
0 Participants
|
|
Number of Participants With Change in Clinical Status on Caregiver's Global Assessment
No change from Baseline at Day 20
|
1 Participants
|
0 Participants
|
3 Participants
|
2 Participants
|
2 Participants
|
0 Participants
|
2 Participants
|
0 Participants
|
1 Participants
|
3 Participants
|
|
Number of Participants With Change in Clinical Status on Caregiver's Global Assessment
Slight worsening at Day 20
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
1 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants With Change in Clinical Status on Caregiver's Global Assessment
Moderate worsening at Day 20
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants With Change in Clinical Status on Caregiver's Global Assessment
Severe worsening at Day 20
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: Day 10 and Day 20Population: Efficacy Population. Only those participants with data available at the indicated time point were analyzed.
Clinical spasms were determined by vEEG for at least 24 hours at Day 10 and Day 20. The number of participants with spasm-free duration have been presented.
Outcome measures
| Measure |
Cohort 1: Sequence A: Ganaxolone/Placebo/Ganaxolone
n=6 Participants
Participants were administered oral suspension of ganaxolone followed by placebo and then ganaxolone using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 1 received starting doses of 3 mg/kg tid with maximum allowable doses of 36 mg/kg/day (12 mg/kg tid).
|
Cohort 1: Sequence B: Placebo/Ganaxolone/Placebo
n=3 Participants
Participants were administered placebo followed by oral suspension of ganaxolone and then placebo using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 1 received starting doses of 3 mg/kg tid with maximum allowable doses of 36 mg/kg/day (12 mg/kg tid).
|
Cohort 2: Sequence A: Ganaxolone/Placebo/Ganaxolone
n=7 Participants
Participants were administered oral suspension of ganaxolone followed by placebo and then ganaxolone using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 2 received starting doses of 6 mg/kg tid with maximum allowable doses of 45 mg/kg/day (15 mg/kg tid).
|
Cohort 2: Sequence B: Placebo/Ganaxolone/Placebo
n=4 Participants
Participants were administered placebo followed by oral suspension of ganaxolone and then placebo using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 2 received starting doses of 6 mg/kg tid with maximum allowable doses of 45 mg/kg/day (15 mg/kg tid).
|
Cohort 3: Sequence A: Ganaxolone/Placebo/Ganaxolone
n=10 Participants
Participants were administered oral suspension of ganaxolone followed by placebo and then ganaxolone using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 3 received starting doses of 9 mg/kg tid with maximum allowable doses of 54 mg/kg/day (18 mg/kg tid).
|
Cohort 3: Sequence B: Placebo/Ganaxolone/Placebo
n=3 Participants
Participants were administered placebo followed by oral suspension of ganaxolone and then placebo using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 3 received starting doses of 9 mg/kg tid with maximum allowable doses of 54 mg/kg/day (18 mg/kg tid).
|
Cohort 4: Sequence A: Ganaxolone/Placebo/Ganaxolone
n=7 Participants
Participants were administered oral suspension of ganaxolone followed by placebo and then ganaxolone using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 4 received starting doses of 12 mg/kg tid with maximum allowable doses of 54 mg/kg/day (18 mg/kg tid).
|
Cohort 4: Sequence B: Placebo/Ganaxolone/Placebo
n=5 Participants
Participants were administered placebo followed by oral suspension of ganaxolone and then placebo using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 4 received starting doses of 12 mg/kg tid with maximum allowable doses of 54 mg/kg/day (18 mg/kg tid).
|
Cohort 5: Sequence A: Ganaxolone/Placebo/Ganaxolone
n=6 Participants
Participants were administered oral suspension of ganaxolone followed by placebo and then ganaxolone using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 5 received starting doses of 18 mg/kg tid with maximum allowable doses of 54 mg/kg/day (18 mg/kg tid).
|
Cohort 5: Sequence B: Placebo/Ganaxolone/Placebo
n=3 Participants
Participants were administered placebo followed by oral suspension of ganaxolone and then placebo using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 5 received starting doses of 18 mg/kg tid with maximum allowable doses of 54 mg/kg/day (18 mg/kg tid).
|
|---|---|---|---|---|---|---|---|---|---|---|
|
Number of Participants With Spasm-free Durations
Spasm-free Durations at Day 10
|
3 Participants
|
1 Participants
|
3 Participants
|
2 Participants
|
2 Participants
|
0 Participants
|
3 Participants
|
0 Participants
|
1 Participants
|
0 Participants
|
|
Number of Participants With Spasm-free Durations
Spasm-free Durations at Day 20
|
3 Participants
|
1 Participants
|
2 Participants
|
2 Participants
|
2 Participants
|
1 Participants
|
1 Participants
|
1 Participants
|
0 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: From Day 8 to Day 10 and From Day 18 to Day 20Population: Efficacy Population. Only those participants with data available at the indicated time point were analyzed.
Seizure-free days were measured using data obtained from participants' daily dairy. Participants without seizures have been reported.
Outcome measures
| Measure |
Cohort 1: Sequence A: Ganaxolone/Placebo/Ganaxolone
n=6 Participants
Participants were administered oral suspension of ganaxolone followed by placebo and then ganaxolone using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 1 received starting doses of 3 mg/kg tid with maximum allowable doses of 36 mg/kg/day (12 mg/kg tid).
|
Cohort 1: Sequence B: Placebo/Ganaxolone/Placebo
n=3 Participants
Participants were administered placebo followed by oral suspension of ganaxolone and then placebo using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 1 received starting doses of 3 mg/kg tid with maximum allowable doses of 36 mg/kg/day (12 mg/kg tid).
|
Cohort 2: Sequence A: Ganaxolone/Placebo/Ganaxolone
n=7 Participants
Participants were administered oral suspension of ganaxolone followed by placebo and then ganaxolone using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 2 received starting doses of 6 mg/kg tid with maximum allowable doses of 45 mg/kg/day (15 mg/kg tid).
|
Cohort 2: Sequence B: Placebo/Ganaxolone/Placebo
n=4 Participants
Participants were administered placebo followed by oral suspension of ganaxolone and then placebo using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 2 received starting doses of 6 mg/kg tid with maximum allowable doses of 45 mg/kg/day (15 mg/kg tid).
|
Cohort 3: Sequence A: Ganaxolone/Placebo/Ganaxolone
n=10 Participants
Participants were administered oral suspension of ganaxolone followed by placebo and then ganaxolone using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 3 received starting doses of 9 mg/kg tid with maximum allowable doses of 54 mg/kg/day (18 mg/kg tid).
|
Cohort 3: Sequence B: Placebo/Ganaxolone/Placebo
n=3 Participants
Participants were administered placebo followed by oral suspension of ganaxolone and then placebo using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 3 received starting doses of 9 mg/kg tid with maximum allowable doses of 54 mg/kg/day (18 mg/kg tid).
|
Cohort 4: Sequence A: Ganaxolone/Placebo/Ganaxolone
n=7 Participants
Participants were administered oral suspension of ganaxolone followed by placebo and then ganaxolone using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 4 received starting doses of 12 mg/kg tid with maximum allowable doses of 54 mg/kg/day (18 mg/kg tid).
|
Cohort 4: Sequence B: Placebo/Ganaxolone/Placebo
n=5 Participants
Participants were administered placebo followed by oral suspension of ganaxolone and then placebo using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 4 received starting doses of 12 mg/kg tid with maximum allowable doses of 54 mg/kg/day (18 mg/kg tid).
|
Cohort 5: Sequence A: Ganaxolone/Placebo/Ganaxolone
n=6 Participants
Participants were administered oral suspension of ganaxolone followed by placebo and then ganaxolone using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 5 received starting doses of 18 mg/kg tid with maximum allowable doses of 54 mg/kg/day (18 mg/kg tid).
|
Cohort 5: Sequence B: Placebo/Ganaxolone/Placebo
n=3 Participants
Participants were administered placebo followed by oral suspension of ganaxolone and then placebo using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 5 received starting doses of 18 mg/kg tid with maximum allowable doses of 54 mg/kg/day (18 mg/kg tid).
|
|---|---|---|---|---|---|---|---|---|---|---|
|
Number of Participants With Seizure-free Days
From Day 18 to Day 20
|
6 Participants
|
1 Participants
|
6 Participants
|
4 Participants
|
10 Participants
|
3 Participants
|
6 Participants
|
5 Participants
|
6 Participants
|
3 Participants
|
|
Number of Participants With Seizure-free Days
From Day 8 to Day 10
|
6 Participants
|
2 Participants
|
6 Participants
|
3 Participants
|
10 Participants
|
3 Participants
|
6 Participants
|
5 Participants
|
6 Participants
|
3 Participants
|
SECONDARY outcome
Timeframe: Day 10 and Day 20Population: Efficacy Population. Only those participants with data available at the indicated time point were analyzed.
A responder is defined as a participant experiencing a greater than equal to (\>=) 50 percent (%) decrease in spasm frequency. Test for responders was conducted by vEEG for up to 24 hours at Day 10 and Day 20
Outcome measures
| Measure |
Cohort 1: Sequence A: Ganaxolone/Placebo/Ganaxolone
n=6 Participants
Participants were administered oral suspension of ganaxolone followed by placebo and then ganaxolone using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 1 received starting doses of 3 mg/kg tid with maximum allowable doses of 36 mg/kg/day (12 mg/kg tid).
|
Cohort 1: Sequence B: Placebo/Ganaxolone/Placebo
n=3 Participants
Participants were administered placebo followed by oral suspension of ganaxolone and then placebo using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 1 received starting doses of 3 mg/kg tid with maximum allowable doses of 36 mg/kg/day (12 mg/kg tid).
|
Cohort 2: Sequence A: Ganaxolone/Placebo/Ganaxolone
n=7 Participants
Participants were administered oral suspension of ganaxolone followed by placebo and then ganaxolone using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 2 received starting doses of 6 mg/kg tid with maximum allowable doses of 45 mg/kg/day (15 mg/kg tid).
|
Cohort 2: Sequence B: Placebo/Ganaxolone/Placebo
n=4 Participants
Participants were administered placebo followed by oral suspension of ganaxolone and then placebo using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 2 received starting doses of 6 mg/kg tid with maximum allowable doses of 45 mg/kg/day (15 mg/kg tid).
|
Cohort 3: Sequence A: Ganaxolone/Placebo/Ganaxolone
n=11 Participants
Participants were administered oral suspension of ganaxolone followed by placebo and then ganaxolone using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 3 received starting doses of 9 mg/kg tid with maximum allowable doses of 54 mg/kg/day (18 mg/kg tid).
|
Cohort 3: Sequence B: Placebo/Ganaxolone/Placebo
n=3 Participants
Participants were administered placebo followed by oral suspension of ganaxolone and then placebo using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 3 received starting doses of 9 mg/kg tid with maximum allowable doses of 54 mg/kg/day (18 mg/kg tid).
|
Cohort 4: Sequence A: Ganaxolone/Placebo/Ganaxolone
n=7 Participants
Participants were administered oral suspension of ganaxolone followed by placebo and then ganaxolone using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 4 received starting doses of 12 mg/kg tid with maximum allowable doses of 54 mg/kg/day (18 mg/kg tid).
|
Cohort 4: Sequence B: Placebo/Ganaxolone/Placebo
n=5 Participants
Participants were administered placebo followed by oral suspension of ganaxolone and then placebo using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 4 received starting doses of 12 mg/kg tid with maximum allowable doses of 54 mg/kg/day (18 mg/kg tid).
|
Cohort 5: Sequence A: Ganaxolone/Placebo/Ganaxolone
n=6 Participants
Participants were administered oral suspension of ganaxolone followed by placebo and then ganaxolone using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 5 received starting doses of 18 mg/kg tid with maximum allowable doses of 54 mg/kg/day (18 mg/kg tid).
|
Cohort 5: Sequence B: Placebo/Ganaxolone/Placebo
n=3 Participants
Participants were administered placebo followed by oral suspension of ganaxolone and then placebo using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 5 received starting doses of 18 mg/kg tid with maximum allowable doses of 54 mg/kg/day (18 mg/kg tid).
|
|---|---|---|---|---|---|---|---|---|---|---|
|
Number of Responders
Day 10
|
2 Participants
|
1 Participants
|
3 Participants
|
0 Participants
|
3 Participants
|
0 Participants
|
2 Participants
|
2 Participants
|
2 Participants
|
1 Participants
|
|
Number of Responders
Day 20
|
2 Participants
|
0 Participants
|
4 Participants
|
2 Participants
|
3 Participants
|
1 Participants
|
4 Participants
|
1 Participants
|
1 Participants
|
1 Participants
|
SECONDARY outcome
Timeframe: At Day 20Population: Efficacy Population. Only those participants with data available at the indicated time point were analyzed.
Denver-II Developmental Test measures a child's development in several areas:Personal-Social,Fine Motor-Adaptive,Language,and Gross Motor,from birth to 6 years old.It consists of 125 items that are organized into subscales and scored as pass,fail,or refused.To evaluate a child's progress,test compares their performance to a normative sample of children of same age.For each item,age at which 90% of children in normative sample pass it is determined.Derived score for each subscale is sum of item scores and represents difference between child's chronological age and age at which 90% of children in normative sample pass the items in that subscale.A higher derived score on a subscale indicates better performance on items in that subscale relative to other children of same age who have taken the test.Among subscales,Personal-Social subscale ranges from -16 months to 24 months;others range from - 12 months to 24 months.All subscales have a population mean of 0 and a standard deviation of 3.
Outcome measures
| Measure |
Cohort 1: Sequence A: Ganaxolone/Placebo/Ganaxolone
n=5 Participants
Participants were administered oral suspension of ganaxolone followed by placebo and then ganaxolone using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 1 received starting doses of 3 mg/kg tid with maximum allowable doses of 36 mg/kg/day (12 mg/kg tid).
|
Cohort 1: Sequence B: Placebo/Ganaxolone/Placebo
n=3 Participants
Participants were administered placebo followed by oral suspension of ganaxolone and then placebo using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 1 received starting doses of 3 mg/kg tid with maximum allowable doses of 36 mg/kg/day (12 mg/kg tid).
|
Cohort 2: Sequence A: Ganaxolone/Placebo/Ganaxolone
n=7 Participants
Participants were administered oral suspension of ganaxolone followed by placebo and then ganaxolone using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 2 received starting doses of 6 mg/kg tid with maximum allowable doses of 45 mg/kg/day (15 mg/kg tid).
|
Cohort 2: Sequence B: Placebo/Ganaxolone/Placebo
n=3 Participants
Participants were administered placebo followed by oral suspension of ganaxolone and then placebo using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 2 received starting doses of 6 mg/kg tid with maximum allowable doses of 45 mg/kg/day (15 mg/kg tid).
|
Cohort 3: Sequence A: Ganaxolone/Placebo/Ganaxolone
n=11 Participants
Participants were administered oral suspension of ganaxolone followed by placebo and then ganaxolone using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 3 received starting doses of 9 mg/kg tid with maximum allowable doses of 54 mg/kg/day (18 mg/kg tid).
|
Cohort 3: Sequence B: Placebo/Ganaxolone/Placebo
n=2 Participants
Participants were administered placebo followed by oral suspension of ganaxolone and then placebo using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 3 received starting doses of 9 mg/kg tid with maximum allowable doses of 54 mg/kg/day (18 mg/kg tid).
|
Cohort 4: Sequence A: Ganaxolone/Placebo/Ganaxolone
n=7 Participants
Participants were administered oral suspension of ganaxolone followed by placebo and then ganaxolone using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 4 received starting doses of 12 mg/kg tid with maximum allowable doses of 54 mg/kg/day (18 mg/kg tid).
|
Cohort 4: Sequence B: Placebo/Ganaxolone/Placebo
n=5 Participants
Participants were administered placebo followed by oral suspension of ganaxolone and then placebo using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 4 received starting doses of 12 mg/kg tid with maximum allowable doses of 54 mg/kg/day (18 mg/kg tid).
|
Cohort 5: Sequence A: Ganaxolone/Placebo/Ganaxolone
n=5 Participants
Participants were administered oral suspension of ganaxolone followed by placebo and then ganaxolone using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 5 received starting doses of 18 mg/kg tid with maximum allowable doses of 54 mg/kg/day (18 mg/kg tid).
|
Cohort 5: Sequence B: Placebo/Ganaxolone/Placebo
n=3 Participants
Participants were administered placebo followed by oral suspension of ganaxolone and then placebo using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 5 received starting doses of 18 mg/kg tid with maximum allowable doses of 54 mg/kg/day (18 mg/kg tid).
|
|---|---|---|---|---|---|---|---|---|---|---|
|
Developmental Assessment Using Denver-II Developmental Test at Day 20
Personal Social at Day 20
|
-5.48 Scores on a scale
Standard Deviation 1.618
|
-16.90 Scores on a scale
Standard Deviation 2.563
|
-8.79 Scores on a scale
Standard Deviation 5.874
|
-8.73 Scores on a scale
Standard Deviation 6.071
|
-7.76 Scores on a scale
Standard Deviation 3.727
|
-5.35 Scores on a scale
Standard Deviation 0.354
|
-5.99 Scores on a scale
Standard Deviation 4.354
|
-10.22 Scores on a scale
Standard Deviation 4.925
|
-7.72 Scores on a scale
Standard Deviation 4.581
|
-12.93 Scores on a scale
Standard Deviation 7.336
|
|
Developmental Assessment Using Denver-II Developmental Test at Day 20
Fine Motor Adaptive at Day 20
|
-4.38 Scores on a scale
Standard Deviation 1.834
|
-14.63 Scores on a scale
Standard Deviation 2.409
|
-7.49 Scores on a scale
Standard Deviation 5.414
|
-7.70 Scores on a scale
Standard Deviation 5.624
|
-6.40 Scores on a scale
Standard Deviation 4.224
|
-5.05 Scores on a scale
Standard Deviation 2.051
|
-6.16 Scores on a scale
Standard Deviation 4.484
|
-10.24 Scores on a scale
Standard Deviation 6.080
|
-8.32 Scores on a scale
Standard Deviation 3.637
|
-12.87 Scores on a scale
Standard Deviation 7.731
|
|
Developmental Assessment Using Denver-II Developmental Test at Day 20
Language at Day 20
|
-1.36 Scores on a scale
Standard Deviation 2.681
|
-12.33 Scores on a scale
Standard Deviation 4.196
|
-7.23 Scores on a scale
Standard Deviation 7.020
|
-5.17 Scores on a scale
Standard Deviation 3.017
|
-5.82 Scores on a scale
Standard Deviation 3.880
|
-4.95 Scores on a scale
Standard Deviation 0.778
|
-6.09 Scores on a scale
Standard Deviation 4.674
|
-8.22 Scores on a scale
Standard Deviation 5.644
|
-7.44 Scores on a scale
Standard Deviation 3.089
|
-10.77 Scores on a scale
Standard Deviation 7.223
|
|
Developmental Assessment Using Denver-II Developmental Test at Day 20
Gross Motor at Day 20
|
-3.96 Scores on a scale
Standard Deviation 2.532
|
-13.63 Scores on a scale
Standard Deviation 2.079
|
-7.16 Scores on a scale
Standard Deviation 5.298
|
-7.00 Scores on a scale
Standard Deviation 5.828
|
-5.58 Scores on a scale
Standard Deviation 3.329
|
-3.00 Scores on a scale
Standard Deviation 2.546
|
-6.89 Scores on a scale
Standard Deviation 4.282
|
-8.74 Scores on a scale
Standard Deviation 5.546
|
-8.76 Scores on a scale
Standard Deviation 3.260
|
-13.43 Scores on a scale
Standard Deviation 8.618
|
Adverse Events
Cohort 1: Sequence A: Ganaxolone/Placebo/Ganaxolone
Serious events: 2 serious events
Other events: 6 other events
Deaths: 0 deaths
Cohort 1: Sequence B: Placebo/Ganaxolone/Placebo
Serious events: 0 serious events
Other events: 3 other events
Deaths: 0 deaths
Cohort 2: Sequence A: Ganaxolone/Placebo/Ganaxolone
Serious events: 0 serious events
Other events: 5 other events
Deaths: 0 deaths
Cohort 2: Sequence B: Placebo/Ganaxolone/Placebo
Serious events: 0 serious events
Other events: 1 other events
Deaths: 0 deaths
Cohort 3: Sequence A: Ganaxolone/Placebo/Ganaxolone
Serious events: 0 serious events
Other events: 9 other events
Deaths: 0 deaths
Cohort 3: Sequence B: Placebo/Ganaxolone/Placebo
Serious events: 0 serious events
Other events: 1 other events
Deaths: 0 deaths
Cohort 4: Sequence A: Ganaxolone/Placebo/Ganaxolone
Serious events: 2 serious events
Other events: 4 other events
Deaths: 0 deaths
Cohort 4: Sequence B: Placebo/Ganaxolone/Placebo
Serious events: 0 serious events
Other events: 2 other events
Deaths: 0 deaths
Cohort 5: Sequence A: Ganaxolone/Placebo/Ganaxolone
Serious events: 0 serious events
Other events: 4 other events
Deaths: 0 deaths
Cohort 5: Sequence B: Placebo/Ganaxolone/Placebo
Serious events: 0 serious events
Other events: 4 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Cohort 1: Sequence A: Ganaxolone/Placebo/Ganaxolone
n=6 participants at risk
Participants were administered oral suspension of ganaxolone followed by placebo and then ganaxolone using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 1 received starting doses of 3 mg/kg tid with maximum allowable doses of 36 mg/kg/day (12 mg/kg tid).
|
Cohort 1: Sequence B: Placebo/Ganaxolone/Placebo
n=3 participants at risk
Participants were administered placebo followed by oral suspension of ganaxolone and then placebo using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 1 received starting doses of 3 mg/kg tid with maximum allowable doses of 36 mg/kg/day (12 mg/kg tid).
|
Cohort 2: Sequence A: Ganaxolone/Placebo/Ganaxolone
n=7 participants at risk
Participants were administered oral suspension of ganaxolone followed by placebo and then ganaxolone using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 2 received starting doses of 6 mg/kg tid with maximum allowable doses of 45 mg/kg/day (15 mg/kg tid).
|
Cohort 2: Sequence B: Placebo/Ganaxolone/Placebo
n=4 participants at risk
Participants were administered placebo followed by oral suspension of ganaxolone and then placebo using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 2 received starting doses of 6 mg/kg tid with maximum allowable doses of 45 mg/kg/day (15 mg/kg tid).
|
Cohort 3: Sequence A: Ganaxolone/Placebo/Ganaxolone
n=11 participants at risk
Participants were administered oral suspension of ganaxolone followed by placebo and then ganaxolone using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 3 received starting doses of 9 mg/kg tid with maximum allowable doses of 54 mg/kg/day (18 mg/kg tid).
|
Cohort 3: Sequence B: Placebo/Ganaxolone/Placebo
n=3 participants at risk
Participants were administered placebo followed by oral suspension of ganaxolone and then placebo using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 3 received starting doses of 9 mg/kg tid with maximum allowable doses of 54 mg/kg/day (18 mg/kg tid).
|
Cohort 4: Sequence A: Ganaxolone/Placebo/Ganaxolone
n=7 participants at risk
Participants were administered oral suspension of ganaxolone followed by placebo and then ganaxolone using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 4 received starting doses of 12 mg/kg tid with maximum allowable doses of 54 mg/kg/day (18 mg/kg tid).
|
Cohort 4: Sequence B: Placebo/Ganaxolone/Placebo
n=5 participants at risk
Participants were administered placebo followed by oral suspension of ganaxolone and then placebo using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 4 received starting doses of 12 mg/kg tid with maximum allowable doses of 54 mg/kg/day (18 mg/kg tid).
|
Cohort 5: Sequence A: Ganaxolone/Placebo/Ganaxolone
n=6 participants at risk
Participants were administered oral suspension of ganaxolone followed by placebo and then ganaxolone using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 5 received starting doses of 18 mg/kg tid with maximum allowable doses of 54 mg/kg/day (18 mg/kg tid).
|
Cohort 5: Sequence B: Placebo/Ganaxolone/Placebo
n=4 participants at risk
Participants were administered placebo followed by oral suspension of ganaxolone and then placebo using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 5 received starting doses of 18 mg/kg tid with maximum allowable doses of 54 mg/kg/day (18 mg/kg tid).
|
|---|---|---|---|---|---|---|---|---|---|---|
|
Nervous system disorders
Convulsion
|
16.7%
1/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/11 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/5 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
|
Nervous system disorders
Hydrocephalus
|
16.7%
1/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/11 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/5 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
|
Cardiac disorders
Bradycardia
|
0.00%
0/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/11 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
14.3%
1/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/5 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
|
Nervous system disorders
Lethargy
|
0.00%
0/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/11 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
14.3%
1/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/5 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
Other adverse events
| Measure |
Cohort 1: Sequence A: Ganaxolone/Placebo/Ganaxolone
n=6 participants at risk
Participants were administered oral suspension of ganaxolone followed by placebo and then ganaxolone using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 1 received starting doses of 3 mg/kg tid with maximum allowable doses of 36 mg/kg/day (12 mg/kg tid).
|
Cohort 1: Sequence B: Placebo/Ganaxolone/Placebo
n=3 participants at risk
Participants were administered placebo followed by oral suspension of ganaxolone and then placebo using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 1 received starting doses of 3 mg/kg tid with maximum allowable doses of 36 mg/kg/day (12 mg/kg tid).
|
Cohort 2: Sequence A: Ganaxolone/Placebo/Ganaxolone
n=7 participants at risk
Participants were administered oral suspension of ganaxolone followed by placebo and then ganaxolone using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 2 received starting doses of 6 mg/kg tid with maximum allowable doses of 45 mg/kg/day (15 mg/kg tid).
|
Cohort 2: Sequence B: Placebo/Ganaxolone/Placebo
n=4 participants at risk
Participants were administered placebo followed by oral suspension of ganaxolone and then placebo using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 2 received starting doses of 6 mg/kg tid with maximum allowable doses of 45 mg/kg/day (15 mg/kg tid).
|
Cohort 3: Sequence A: Ganaxolone/Placebo/Ganaxolone
n=11 participants at risk
Participants were administered oral suspension of ganaxolone followed by placebo and then ganaxolone using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 3 received starting doses of 9 mg/kg tid with maximum allowable doses of 54 mg/kg/day (18 mg/kg tid).
|
Cohort 3: Sequence B: Placebo/Ganaxolone/Placebo
n=3 participants at risk
Participants were administered placebo followed by oral suspension of ganaxolone and then placebo using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 3 received starting doses of 9 mg/kg tid with maximum allowable doses of 54 mg/kg/day (18 mg/kg tid).
|
Cohort 4: Sequence A: Ganaxolone/Placebo/Ganaxolone
n=7 participants at risk
Participants were administered oral suspension of ganaxolone followed by placebo and then ganaxolone using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 4 received starting doses of 12 mg/kg tid with maximum allowable doses of 54 mg/kg/day (18 mg/kg tid).
|
Cohort 4: Sequence B: Placebo/Ganaxolone/Placebo
n=5 participants at risk
Participants were administered placebo followed by oral suspension of ganaxolone and then placebo using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 4 received starting doses of 12 mg/kg tid with maximum allowable doses of 54 mg/kg/day (18 mg/kg tid).
|
Cohort 5: Sequence A: Ganaxolone/Placebo/Ganaxolone
n=6 participants at risk
Participants were administered oral suspension of ganaxolone followed by placebo and then ganaxolone using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 5 received starting doses of 18 mg/kg tid with maximum allowable doses of 54 mg/kg/day (18 mg/kg tid).
|
Cohort 5: Sequence B: Placebo/Ganaxolone/Placebo
n=4 participants at risk
Participants were administered placebo followed by oral suspension of ganaxolone and then placebo using an oral dosing syringe by hospital staff/parents/guardians tid. Each dose was separated by a minimum of 4 hours and a maximum of 8 hours. Participants in Cohort 5 received starting doses of 18 mg/kg tid with maximum allowable doses of 54 mg/kg/day (18 mg/kg tid).
|
|---|---|---|---|---|---|---|---|---|---|---|
|
Gastrointestinal disorders
Gastrooesophageal reflux disease
|
0.00%
0/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
9.1%
1/11 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/5 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
|
Infections and infestations
Ear infection
|
0.00%
0/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
9.1%
1/11 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/5 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
|
Infections and infestations
Nasopharyngitis
|
0.00%
0/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
9.1%
1/11 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
20.0%
1/5 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
|
Musculoskeletal and connective tissue disorders
Muscle spasms
|
0.00%
0/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
9.1%
1/11 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/5 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
|
Musculoskeletal and connective tissue disorders
Muscle twitching
|
0.00%
0/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
9.1%
1/11 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/5 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
|
Skin and subcutaneous tissue disorders
Dry skin
|
0.00%
0/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
9.1%
1/11 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/5 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
|
Skin and subcutaneous tissue disorders
Rash
|
0.00%
0/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
9.1%
1/11 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/5 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
|
Eye disorders
Erythema of eyelid
|
0.00%
0/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
9.1%
1/11 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/5 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
|
Injury, poisoning and procedural complications
Procedural site reaction
|
0.00%
0/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
9.1%
1/11 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/5 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
|
Metabolism and nutrition disorders
Hypernatraemia
|
0.00%
0/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
9.1%
1/11 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/5 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
|
Gastrointestinal disorders
Diarrhoea
|
0.00%
0/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/11 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
14.3%
1/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/5 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
|
Gastrointestinal disorders
Flatulence
|
0.00%
0/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/11 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
14.3%
1/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/5 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
|
Investigations
Body temperature increased
|
0.00%
0/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/11 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
20.0%
1/5 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
|
Investigations
Red blood cells urine positive
|
0.00%
0/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/11 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
20.0%
1/5 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
|
Investigations
White blood cells urine positive
|
0.00%
0/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/11 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
20.0%
1/5 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
16.7%
1/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
|
Cardiac disorders
Bradycardia
|
0.00%
0/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/11 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
14.3%
1/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/5 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
|
Eye disorders
Eye discharge
|
0.00%
0/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/11 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
14.3%
1/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/5 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
25.0%
1/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
|
Respiratory, thoracic and mediastinal disorders
Asthma
|
0.00%
0/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/11 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
14.3%
1/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/5 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
|
Skin and subcutaneous tissue disorders
Dermatitis atopic
|
0.00%
0/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/11 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
20.0%
1/5 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
|
Nervous system disorders
Epilepsy
|
0.00%
0/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/11 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/5 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
25.0%
1/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
|
Psychiatric disorders
Insomnia
|
0.00%
0/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/11 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/5 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
16.7%
1/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
25.0%
1/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
|
Investigations
Blood bicarbonate decreased
|
0.00%
0/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/11 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/5 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
16.7%
1/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
|
Investigations
Urinary sediment present
|
0.00%
0/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/11 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/5 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
16.7%
1/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
|
Respiratory, thoracic and mediastinal disorders
Pharyngolaryngeal pain
|
0.00%
0/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/11 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/5 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
16.7%
1/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
|
Eye disorders
Eye irritation
|
0.00%
0/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/11 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/5 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
25.0%
1/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
|
Gastrointestinal disorders
Constipation
|
0.00%
0/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
9.1%
1/11 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
14.3%
1/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/5 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
|
Nervous system disorders
Somnolence
|
33.3%
2/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
14.3%
1/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
25.0%
1/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/11 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
14.3%
1/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/5 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
|
Nervous system disorders
Convulsion
|
16.7%
1/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/11 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/5 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
|
Nervous system disorders
Hydrocephalus
|
16.7%
1/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/11 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/5 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
|
General disorders
Irritability
|
33.3%
2/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
9.1%
1/11 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/5 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
|
General disorders
Puncture site haemorrhage
|
16.7%
1/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/11 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/5 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
|
General disorders
Pyrexia
|
0.00%
0/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
33.3%
1/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
9.1%
1/11 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/5 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
16.7%
1/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
25.0%
1/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
|
Gastrointestinal disorders
Teething
|
16.7%
1/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
14.3%
1/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
9.1%
1/11 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/5 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
25.0%
1/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
|
Gastrointestinal disorders
Vomiting
|
0.00%
0/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
33.3%
1/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
14.3%
1/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
27.3%
3/11 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
33.3%
1/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
14.3%
1/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/5 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
|
Infections and infestations
Upper respiratory tract infection
|
0.00%
0/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
33.3%
1/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/11 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
14.3%
1/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
20.0%
1/5 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
|
Infections and infestations
Urinary tract infection
|
16.7%
1/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
9.1%
1/11 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/5 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
16.7%
1/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
33.3%
1/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
14.3%
1/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
9.1%
1/11 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/5 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
16.7%
1/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
|
Respiratory, thoracic and mediastinal disorders
Upper respiratory tract congestion
|
0.00%
0/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
33.3%
1/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/11 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/5 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
|
Skin and subcutaneous tissue disorders
Pityriasis
|
0.00%
0/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
33.3%
1/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/11 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/5 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
|
Gastrointestinal disorders
Infantile spitting up
|
0.00%
0/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
14.3%
1/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/11 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/5 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
|
Nervous system disorders
Lethargy
|
0.00%
0/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
14.3%
1/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/11 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
14.3%
1/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/5 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
16.7%
1/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
0.00%
0/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
14.3%
1/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/11 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/5 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrhoea
|
0.00%
0/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
25.0%
1/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/11 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/5 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
|
Respiratory, thoracic and mediastinal disorders
Sinus congestion
|
0.00%
0/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
14.3%
1/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/11 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/5 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
|
Blood and lymphatic system disorders
Thrombocytopenia
|
0.00%
0/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
14.3%
1/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/11 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/5 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
|
Infections and infestations
Rhinitis
|
0.00%
0/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
14.3%
1/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/11 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/5 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
|
Skin and subcutaneous tissue disorders
Skin irritation
|
0.00%
0/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
14.3%
1/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/11 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/3 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/7 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/5 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/6 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
0.00%
0/4 • Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place