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The study consists of cohorts where participants are randomized, in a 2:1 ratio, to 1 of 2 sequences, A and B. In each cohort, Sequence A, comprised of participants, who will receive ascending doses of ganaxolone and ascending doses of placebo. Sequence B, comprised of participants, who will receive ascending doses of placebo and ascending doses of ganaxolone. The dosing level in each subsequent cohort will be based upon experience gained from previous cohorts.
Male or female, 4 to 24 months of age (inclusive) with a diagnosis of IS with a 24 hour video EEG (vEEG) recording confirming the diagnosis and previously treated with 3 or fewer antiepileptic drugs (AEDs) are eligible for the study. The subject is able to continue treatment with concomitant AEDs (no more than 2; adrenocorticotropic hormone [ACTH], corticosteroids, felbamate, and vigabatrin are not allowed concomitantly). A ketogenic diet is permitted if it can be maintained for the duration of the study.
There will be a total of three weekly 24-hr video EEGs (baseline, end of weeks 1 and 2 of treatment). Dosing titration begins the day after each video EEG during the inpatient stay. All subjects will be receiving ganaxolone the day after the second video EEG.
A Data Monitoring Board (DMB) will determine whether successive cohorts of subjects can be dosed at an increased dose level; up to a maximum of 6 cohorts.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| ganaxolone | Experimental | ganaxolone |
|
| non-active drug | Placebo Comparator | placebo |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Ganaxolone | Drug |
| ||
| Placebo |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in Frequency of Spasm Clusters at Day 10 | 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. | Baseline (Day 0) and Day 10 |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in Frequency of Spasm Clusters at Day 20 | 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. | Baseline (Day 0) and Day 20 |
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Inclusion Criteria:
Be diagnosed with IS (regardless of etiology- except for a progressive neurologic illness). Diagnostic Criteria: Seizures consisting of single or repetitive short muscular contractions leading to flexion or extension. Spasms may be characterized as tonic or myoclonic contractions, may occur singly or in clusters, and typically occur bilaterally and symmetrically. The EEG pattern must be consistent with the diagnosis of IS (hypsarrhythmia, modified hypsarrhythmia, multifocal spike wave discharges, etc).
Have a vEEG recording confirming the diagnosis of IS.
Have had a magnetic resonance imaging (MRI) performed to determine any possible causes of IS.
Have been previously treated with 3 or fewer AEDs.
If being treated with concomitant AEDs
Be a male or female, 4 to 24 months of age (inclusive)
Have a Parent/Guardian who is properly informed of the nature and risks of the clinical study, who is willing and capable of complying with all clinical study procedures, and has given informed consent in writing prior to entering the clinical study
Be able to participate for the full term of the clinical study.
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Children's Hospital of Los Angeles | Los Angeles | California | 90027 | United States | ||
| Mattel Children's Hospital at UCLA |
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| Label | URL |
|---|---|
| information about ganaxolone | View source |
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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.
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| ID | Title | Description |
|---|---|---|
| FG000 | 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). |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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|
| Number of Participants With Absence of Hypsarrhythmia |
Absence of hypsarrhythmia was determined by 24-hour vEEG at Day 10 and Day 20. |
| Day 10 and Day 20 |
| Number of Participants With Change in Clinical Status on the Investigator's Global Assessment | 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. | Baseline (Day 0), Day 10 and Day 20 |
| Number of Participants With Change in Clinical Status on Caregiver's Global Assessment | 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. | Baseline (Day 0), Day10 and Day 20 |
| Number of Participants With Spasm-free Durations | 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. | Day 10 and Day 20 |
| Number of Participants With Seizure-free Days | Seizure-free days were measured using data obtained from participants' daily dairy. Participants without seizures have been reported. | From Day 8 to Day 10 and From Day 18 to Day 20 |
| Number of Responders | 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 | Day 10 and Day 20 |
| Developmental Assessment Using Denver-II Developmental Test at Day 20 | 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. | At Day 20 |
| Los Angeles |
| California |
| 90095 |
| United States |
| Children's National Medical Center | Washington D.C. | District of Columbia | 20010 | United States |
| Miami Children's Hospital, The Brain Institute | Miami | Florida | 33155 | United States |
| Child Neurology Care Center of Northwest Florida | Pensacola | Florida | 32504 | United States |
| Child Neurology Center of Northwest Florida | Pensacola | Florida | United States |
| University of Chicago Comer Children's Hospital | Chicago | Illinois | 60637 | United States |
| Minnesota Epilepsy Group, P.A. | Saint Paul | Minnesota | 55102 | United States |
| Montefiore Medical Center- Albert Einstein College of Medicine | The Bronx | New York | 10467 | United States |
| Le Bonheur Children's Medical Center | Memphis | Tennessee | 38105 | United States |
| Dallas Pediatric Neurology Associates | Dallas | Texas | 75230 | United States |
| Texas Children's Hospital | Houston | Texas | 77030 | United States |
| Virginia Commonwealth University Health System | Richmond | Virginia | 23298 | United States |
| Children's Hospital and Regional Medical Center | Seattle | Washington | 98105 | United States |
| Children's Hospital of Wisconsin | Milwaukee | Wisconsin | 53201 | United States |
| FG001 | 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). |
| FG002 | 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). |
| FG003 | 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). |
| FG004 | 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). |
| FG005 | 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). |
| FG006 | 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). |
| FG007 | 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). |
| FG008 | 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). |
| FG009 | 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). |
| COMPLETED |
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| NOT COMPLETED |
|
|
Intent-to-treat (ITT) population included all randomized participants who received at least 1 dose of study medication
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| ID | Title | Description |
|---|---|---|
| BG000 | 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 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). |
| BG001 | 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). |
| BG002 | 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). |
| BG003 | 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). |
| BG004 | 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). |
| BG005 | 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). |
| BG006 | 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). |
| BG007 | 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). |
| BG008 | 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). |
| BG009 | 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). |
| BG010 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | Months |
| |||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
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| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||
| Race (NIH/OMB) | Count of Participants | Participants |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Change From Baseline in Frequency of Spasm Clusters at Day 10 | 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. | 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. | Posted | Mean | Standard Deviation | Spasm clusters per day | Baseline (Day 0) and Day 10 |
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| Secondary | Change From Baseline in Frequency of Spasm Clusters at Day 20 | 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. | Efficacy Population. | Posted | Mean | Standard Deviation | Spasm clusters per day | Baseline (Day 0) and Day 20 |
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| Secondary | Number of Participants With Absence of Hypsarrhythmia | Absence of hypsarrhythmia was determined by 24-hour vEEG at Day 10 and Day 20. | Efficacy Population. Only those participants with data available at the indicated time point were analyzed. | Posted | Count of Participants | Participants | Day 10 and Day 20 |
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| Secondary | Number of Participants With Change in Clinical Status on the Investigator's Global Assessment | 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. | Efficacy Population. Only those participants with data available at the indicated time point were analyzed. | Posted | Count of Participants | Participants | Baseline (Day 0), Day 10 and Day 20 |
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| Secondary | Number of Participants With Change in Clinical Status on Caregiver's Global Assessment | 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. | Efficacy population. Only those participants with data available at the indicated time point were analyzed. | Posted | Count of Participants | Participants | Baseline (Day 0), Day10 and Day 20 |
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| Secondary | Number of Participants With Spasm-free Durations | 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. | Efficacy Population. Only those participants with data available at the indicated time point were analyzed. | Posted | Count of Participants | Participants | Day 10 and Day 20 |
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| Secondary | Number of Participants With Seizure-free Days | Seizure-free days were measured using data obtained from participants' daily dairy. Participants without seizures have been reported. | Efficacy Population. Only those participants with data available at the indicated time point were analyzed. | Posted | Count of Participants | Participants | From Day 8 to Day 10 and From Day 18 to Day 20 |
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| Secondary | Number of Responders | 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 | Efficacy Population. Only those participants with data available at the indicated time point were analyzed. | Posted | Count of Participants | Participants | Day 10 and Day 20 |
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| Secondary | Developmental Assessment Using Denver-II Developmental Test at Day 20 | 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. | Efficacy Population. Only those participants with data available at the indicated time point were analyzed. | Posted | Mean | Standard Deviation | Scores on a scale | At Day 20 |
|
Up to 25 days
AEs and SAEs were collected in ITT Population, which included all randomized participants who received at least 1 dose of study medication.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | 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 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). | 0 | 6 | 2 | 6 | 6 | 6 |
| EG001 | 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). | 0 | 3 | 0 | 3 | 3 | 3 |
| EG002 | 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). | 0 | 7 | 0 | 7 | 5 | 7 |
| EG003 | 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). | 0 | 4 | 0 | 4 | 1 | 4 |
| EG004 | 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). | 0 | 11 | 0 | 11 | 9 | 11 |
| EG005 | 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). | 0 | 3 | 0 | 3 | 1 | 3 |
| EG006 | 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). | 0 | 7 | 2 | 7 | 4 | 7 |
| EG007 | 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). | 0 | 5 | 0 | 5 | 2 | 5 |
| EG008 | 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). | 0 | 6 | 0 | 6 | 4 | 6 |
| EG009 | 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). | 0 | 4 | 0 | 4 | 4 | 4 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Convulsion | Nervous system disorders | MedDRA (9.0) | Systematic Assessment |
| |
| Hydrocephalus | Nervous system disorders | MedDRA (9.0) | Systematic Assessment |
| |
| Bradycardia | Cardiac disorders | MedDRA (9.0) | Systematic Assessment |
| |
| Lethargy | Nervous system disorders | MedDRA (9.0) | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Somnolence | Nervous system disorders | MedDRA (9.0) | Systematic Assessment |
| |
| Convulsion | Nervous system disorders | MedDRA (9.0) | Systematic Assessment |
| |
| Hydrocephalus | Nervous system disorders | MedDRA (9.0) | Systematic Assessment |
| |
| Irritability | General disorders | MedDRA (9.0) | Systematic Assessment |
| |
| Puncture site haemorrhage | General disorders | MedDRA (9.0) | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA (9.0) | Systematic Assessment |
| |
| Teething | Gastrointestinal disorders | MedDRA (9.0) | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA (9.0) | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA (9.0) | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA (9.0) | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA (9.0) | Systematic Assessment |
| |
| Upper respiratory tract congestion | Respiratory, thoracic and mediastinal disorders | MedDRA (9.0) | Systematic Assessment |
| |
| Pityriasis | Skin and subcutaneous tissue disorders | MedDRA (9.0) | Systematic Assessment |
| |
| Infantile spitting up | Gastrointestinal disorders | MedDRA (9.0) | Systematic Assessment |
| |
| Lethargy | Nervous system disorders | MedDRA (9.0) | Systematic Assessment |
| |
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA (9.0) | Systematic Assessment |
| |
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA (9.0) | Systematic Assessment |
| |
| Sinus congestion | Respiratory, thoracic and mediastinal disorders | MedDRA (9.0) | Systematic Assessment |
| |
| Thrombocytopenia | Blood and lymphatic system disorders | MedDRA (9.0) | Systematic Assessment |
| |
| Rhinitis | Infections and infestations | MedDRA (9.0) | Systematic Assessment |
| |
| Skin irritation | Skin and subcutaneous tissue disorders | MedDRA (9.0) | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA (9.0) | Systematic Assessment |
| |
| Gastrooesophageal reflux disease | Gastrointestinal disorders | MedDRA (9.0) | Systematic Assessment |
| |
| Ear infection | Infections and infestations | MedDRA (9.0) | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA (9.0) | Systematic Assessment |
| |
| Muscle spasms | Musculoskeletal and connective tissue disorders | MedDRA (9.0) | Systematic Assessment |
| |
| Muscle twitching | Musculoskeletal and connective tissue disorders | MedDRA (9.0) | Systematic Assessment |
| |
| Dry skin | Skin and subcutaneous tissue disorders | MedDRA (9.0) | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA (9.0) | Systematic Assessment |
| |
| Erythema of eyelid | Eye disorders | MedDRA (9.0) | Systematic Assessment |
| |
| Procedural site reaction | Injury, poisoning and procedural complications | MedDRA (9.0) | Systematic Assessment |
| |
| Hypernatraemia | Metabolism and nutrition disorders | MedDRA (9.0) | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA (9.0) | Systematic Assessment |
| |
| Flatulence | Gastrointestinal disorders | MedDRA (9.0) | Systematic Assessment |
| |
| Body temperature increased | Investigations | MedDRA (9.0) | Systematic Assessment |
| |
| Red blood cells urine positive | Investigations | MedDRA (9.0) | Systematic Assessment |
| |
| White blood cells urine positive | Investigations | MedDRA (9.0) | Systematic Assessment |
| |
| Bradycardia | Cardiac disorders | MedDRA (9.0) | Systematic Assessment |
| |
| Eye discharge | Eye disorders | MedDRA (9.0) | Systematic Assessment |
| |
| Asthma | Respiratory, thoracic and mediastinal disorders | MedDRA (9.0) | Systematic Assessment |
| |
| Dermatitis atopic | Skin and subcutaneous tissue disorders | MedDRA (9.0) | Systematic Assessment |
| |
| Blood bicarbonate decreased | Investigations | MedDRA (9.0) | Systematic Assessment |
| |
| Urinary sediment present | Investigations | MedDRA (9.0) | Systematic Assessment |
| |
| Epilepsy | Nervous system disorders | MedDRA (9.0) | Systematic Assessment |
| |
| Insomnia | Psychiatric disorders | MedDRA (9.0) | Systematic Assessment |
| |
| Pharyngolaryngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA (9.0) | Systematic Assessment |
| |
| Eye irritation | Eye disorders | MedDRA (9.0) | Systematic Assessment |
|
Not provided
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Marinus | Marinus Pharmaceuticals, Inc. | 484-801-4670 | clinicaltrials@marinuspharma.com |
| ID | Term |
|---|---|
| D013036 | Spasms, Infantile |
| ID | Term |
|---|---|
| D004829 | Epilepsy, Generalized |
| D004827 | Epilepsy |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D000073376 | Epileptic Syndromes |
Not provided
Not provided
| ID | Term |
|---|---|
| C105051 | ganaxolone |
Not provided
Not provided
Not provided
| Male |
|
| Not Hispanic or Latino |
|
| Unknown or Not Reported |
|
| Asian |
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|
| Superiority |
| ANCOVA | 0.9080 | Superiority |
| ANCOVA | 0.2539 | Superiority |
| ANCOVA | 0.6657 | Superiority |
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). |
| OG003 | 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). |
| OG004 | 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). |
| OG005 | 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). |
| OG006 | 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). |
| OG007 | 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). |
| OG008 | 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). |
| OG009 | 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). |
|
|
|
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).
| OG003 | 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). |
| OG004 | 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). |
| OG005 | 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). |
| OG006 | 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). |
| OG007 | 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). |
| OG008 | 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). |
| OG009 | 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). |
|
|
| OG002 | 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). |
| OG003 | 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). |
| OG004 | 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). |
| OG005 | 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). |
| OG006 | 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). |
| OG007 | 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). |
| OG008 | 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). |
| OG009 | 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). |
|
|
| OG002 | 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). |
| OG003 | 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). |
| OG004 | 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). |
| OG005 | 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). |
| OG006 | 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). |
| OG007 | 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). |
| OG008 | 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). |
| OG009 | 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). |
|
|
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). |
| OG003 | 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). |
| OG004 | 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). |
| OG005 | 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). |
| OG006 | 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). |
| OG007 | 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). |
| OG008 | 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). |
| OG009 | 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). |
|
|
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). |
| OG003 | 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). |
| OG004 | 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). |
| OG005 | 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). |
| OG006 | 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). |
| OG007 | 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). |
| OG008 | 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). |
| OG009 | 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). |
|
|
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). |
| OG003 | 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). |
| OG004 | 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). |
| OG005 | 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). |
| OG006 | 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). |
| OG007 | 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). |
| OG008 | 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). |
| OG009 | 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). |
|
|
| OG001 | 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). |
| OG002 | 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). |
| OG003 | 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). |
| OG004 | 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). |
| OG005 | 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). |
| OG006 | 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). |
| OG007 | 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). |
| OG008 | 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). |
| OG009 | 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). |
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