Not provided
| ID | Type | Description | Link |
|---|---|---|---|
| Amend 5 (ROW) | |||
| Amend 6 (US) |
Not provided
Not provided
Not provided
Sponsor's decision
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
To allow open-label extension to patients who have completed Protocol 1042-0500
Patient should have completed all scheduled clinical study visits in the double blind, controlled trial (Protocol 1042-0500) and have been deemed eligible (had a response to treatment) by the Investigator. Male or female, with a diagnosis of IS with a video EEG (vEEG) recording confirming the diagnosis.
There will be a total of 14 visits over 99(+or-1)week. A 24-hr vEEG is only required if the subject has been spasm-free for more than 24-hrs.
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| ganaxolone | Experimental |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Ganaxolone | Drug |
|
| Measure | Description | Time Frame |
|---|---|---|
| Percentage of Participants Who Were Free of Spasms | Clinical spasms were determined by video-electroencephalography (VEEG). The number of participants with spasm-free duration have been presented. | Weeks 4 through Week 96 |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in Frequency of Spasm Clusters | Infantile spasms that come one after another in a cluster and lasts several minutes are called Spasm Clusters. Spasm clusters were determined by a 24-hour video-electroencephalography (vEEG). Change from Baseline was calculated as the post-Baseline value minus the Baseline value. Baseline was defined as the Day 0 assessment prior to ganaxolone dosing in Protocol 1042-0500. |
Not provided
Inclusion Criteria:
Exclusion Criteria:
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| 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 |
Participants who completed the previous double-blind controlled trial (Protocol 1042-0500, NCT00441896) were enrolled in this study. No separate analysis was performed to report results by doses.
Not provided
Not provided
| ID | Title | Description |
|---|---|---|
| FG000 | Ganaxolone | Participants were administered a maximum dose of 54 milligrams per kilogram (mg/kg)/day ganaxolone oral suspension. The investigator could adjust (increase or decrease) the dose of ganaxolone by 3 mg/kg three times a day (total daily dose adjustment = 9 mg/kg) until the optimal dose for efficacy and tolerability was achieved |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
Not provided
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | Ganaxolone | Participants were administered a maximum dose of 54 mg/kg/day ganaxolone oral suspension. The investigator could adjust (increase or decrease) the dose of ganaxolone by 3 mg/kg t.i.d. (total daily dose adjustment = 9 mg/kg) until the optimal dose for efficacy and tolerability was achieved. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| 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 | Percentage of Participants Who Were Free of Spasms | Clinical spasms were determined by video-electroencephalography (VEEG). The number of participants with spasm-free duration have been presented. | ITT Population included all participants who received at least 1 dose of open-label study medication. Only those participants with data available at the indicated time point were analyzed. | Posted | Number | 95% Confidence Interval | percentage of participants | Weeks 4 through Week 96 |
|
Up to Week 32
AEs and SAEs were collected in ITT Population. No separate analysis was performed to report results by doses.
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 | Ganaxolone | Participants were administered a maximum dose of 54 mg/kg/day ganaxolone oral suspension. The investigator could adjust (increase or decrease) the dose of ganaxolone by 3 mg/kg t.i.d. (total daily dose adjustment = 9 mg/kg) until the optimal dose for efficacy and tolerability was achieved. |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Infantile spasms | Nervous system disorders | MedDRA (9.0) | Systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Pyrexia | General disorders | MedDRA (9.0) | Systematic Assessment |
Seizure-free information is not available
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Marinus | Marinus Pharmaceuticals, Inc. | 484-801-4670 | clinicaltrials@marinuspharma.com |
Not provided
| ID | Term |
|---|---|
| D013036 | Spasms, Infantile |
| ID | Term |
|---|---|
| D004829 | Epilepsy, Generalized |
| D004827 | Epilepsy |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
Not provided
Not provided
| ID | Term |
|---|---|
| C105051 | ganaxolone |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Baseline and Week 4 through Week 32 |
| Change From Baseline in Frequency of Individual Spasm | Individual Spasm are seizures that may last only a second or two and were determined by a 24-hour video-electroencephalography (vEEG). Change from Baseline was calculated as the post-Baseline value minus the Baseline value. Baseline was defined as the Day 0 assessment prior to ganaxolone dosing in Protocol 1042-0500. | Baseline and Week 4 through Week 32 |
| 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. | Week 4 through Week 32 |
| 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. | Week 4 through Week 32 |
| Number of Participants With Spasm-free Durations | Spasm-free duration is defined as total number of spasm-free days recorded in the spasm/seizure diary. Parents/Guardians or nursing staff maintained a spasm/seizure diary to record the number and type of seizures each day, including spasms, throughout the entire study. The number of participants with spasm-free duration of at least 24 hours have been presented. | Week 4 through Week 32 |
| Number of Participants With Absence of Spasms | Absence of spasms is defined as percentage of total spasm-free days during a visit period=100%. Parents/Guardians or nursing staff maintained a spasm/seizure diary to record the number and type of seizures each day, including spasms, throughout the entire study. The participants achieving absence of spasms have been presented. | Week 4 through Week 32 |
| Developmental Assessment Using Denver-II Developmental Test | 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. | Week 8 through Week 32 |
| Percentage of Total Spasm-free Days and Cumulative Spasm-free Days as Determined From the Seizure Diary. | Percentage of total spasm-free days during a visit period is defined as total number of spasm free days as recorded in the Seizure Diary/ total number of days during that period, multiplied by 100. Percentage of cumulative total spasm-free days during a visit period is defined as sum of total number of spasm-free days during this period as recorded in the Seizure Diary/ total number of days in the treatment period, multiplied by 100. | Weeks 4 through Week 32 |
| 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 Center of Nrothwest Florida, P.A. | Pensacola | Florida | 32504 | 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 Systems | 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 |
| Withdrawal by Subject |
|
| Insufficient Clinical Response |
|
| Other |
|
| Discretion of the Investigator/Sponsor |
|
| months |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
| Units | Counts |
|---|---|
| Participants |
|
|
| Secondary | Change From Baseline in Frequency of Spasm Clusters | Infantile spasms that come one after another in a cluster and lasts several minutes are called Spasm Clusters. Spasm clusters were determined by a 24-hour video-electroencephalography (vEEG). Change from Baseline was calculated as the post-Baseline value minus the Baseline value. Baseline was defined as the Day 0 assessment prior to ganaxolone dosing in Protocol 1042-0500. | ITT Population. Only those participants with data available at the indicated time point were analyzed. | Posted | Mean | Standard Deviation | Spasm clusters per day | Baseline and Week 4 through Week 32 |
|
|
|
| Secondary | Change From Baseline in Frequency of Individual Spasm | Individual Spasm are seizures that may last only a second or two and were determined by a 24-hour video-electroencephalography (vEEG). Change from Baseline was calculated as the post-Baseline value minus the Baseline value. Baseline was defined as the Day 0 assessment prior to ganaxolone dosing in Protocol 1042-0500. | ITT Population. Only those participants with data available at the indicated time point were analyzed. | Posted | Mean | Standard Deviation | Individual spasms per day | Baseline and Week 4 through Week 32 |
|
|
|
| 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. | ITT population. Only those participants with data available at the indicated time point were analyzed. | Posted | Number | participants | Week 4 through Week 32 |
|
|
|
| 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. | ITT population. Only those participants with data available at the indicated time point were analyzed. | Posted | Number | participants | Week 4 through Week 32 |
|
|
|
| Secondary | Number of Participants With Spasm-free Durations | Spasm-free duration is defined as total number of spasm-free days recorded in the spasm/seizure diary. Parents/Guardians or nursing staff maintained a spasm/seizure diary to record the number and type of seizures each day, including spasms, throughout the entire study. The number of participants with spasm-free duration of at least 24 hours have been presented. | ITT Population. Only those participants with data available at the indicated time point were analyzed. | Posted | Count of Participants | Participants | Week 4 through Week 32 |
|
|
|
| Secondary | Number of Participants With Absence of Spasms | Absence of spasms is defined as percentage of total spasm-free days during a visit period=100%. Parents/Guardians or nursing staff maintained a spasm/seizure diary to record the number and type of seizures each day, including spasms, throughout the entire study. The participants achieving absence of spasms have been presented. | ITT Population. Only those participants with data available at the indicated time point were analyzed. | Posted | Count of Participants | Participants | Week 4 through Week 32 |
|
|
|
| Secondary | Developmental Assessment Using Denver-II Developmental Test | 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. | ITT Population. Only those participants with data available at the indicated time point were analyzed. | Posted | Mean | Standard Deviation | Scores on a scale | Week 8 through Week 32 |
|
|
|
| Secondary | Percentage of Total Spasm-free Days and Cumulative Spasm-free Days as Determined From the Seizure Diary. | Percentage of total spasm-free days during a visit period is defined as total number of spasm free days as recorded in the Seizure Diary/ total number of days during that period, multiplied by 100. Percentage of cumulative total spasm-free days during a visit period is defined as sum of total number of spasm-free days during this period as recorded in the Seizure Diary/ total number of days in the treatment period, multiplied by 100. | ITT Population. Only those participants with data available at the indicated time point were analyzed. | Posted | Mean | Standard Deviation | Percentage of Days | Weeks 4 through Week 32 |
|
|
|
| 2 |
| 54 |
| 24 |
| 54 |
| 47 |
| 54 |
| Convulsion | Nervous system disorders | MedDRA (9.0) | Systematic Assessment |
|
| Pyrexia | General disorders | MedDRA (9.0) | Systematic Assessment |
|
| Pneumonia aspiration | Respiratory, thoracic and mediastinal disorders | MedDRA (9.0) | Systematic Assessment |
|
| Gastroesophageal reflux disease | Gastrointestinal disorders | MedDRA (9.0) | Systematic Assessment |
|
| Epilepsy | Nervous system disorders | MedDRA (9.0) | Systematic Assessment |
|
| Gastroenteritis | Infections and infestations | MedDRA (9.0) | Systematic Assessment |
|
| Nasopharyngitis | Infections and infestations | MedDRA (9.0) | Systematic Assessment |
|
| Upper respiratory tract infection | Infections and infestations | MedDRA (9.0) | Systematic Assessment |
|
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA (9.0) | Systematic Assessment |
|
| Respiratory distress | Respiratory, thoracic and mediastinal disorders | MedDRA (9.0) | Systematic Assessment |
|
| Failure to thrive | Metabolism and nutrition disorders | MedDRA (9.0) | Systematic Assessment |
|
| Oral intake reduced | Metabolism and nutrition disorders | MedDRA (9.0) | Systematic Assessment |
|
| Dehydration | Metabolism and nutrition disorders | MedDRA (9.0) | Systematic Assessment |
|
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA (9.0) | Systematic Assessment |
|
| Upper respiratory tract infection | Infections and infestations | MedDRA (9.0) | Systematic Assessment |
|
| Vomiting | Gastrointestinal disorders | MedDRA (9.0) | Systematic Assessment |
|
| Nasopharyngitis | Infections and infestations | MedDRA (9.0) | Systematic Assessment |
|
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA (9.0) | Systematic Assessment |
|
| Convulsion | Nervous system disorders | MedDRA (9.0) | Systematic Assessment |
|
| Urinary tract infection | Infections and infestations | MedDRA (9.0) | Systematic Assessment |
|
| Diarrhea | Gastrointestinal disorders | MedDRA (9.0) | Systematic Assessment |
|
| Constipation | Gastrointestinal disorders | MedDRA (9.0) | Systematic Assessment |
|
| Teething | Gastrointestinal disorders | MedDRA (9.0) | Systematic Assessment |
|
| Somnolence | Nervous system disorders | MedDRA (9.0) | Systematic Assessment |
|
| Gastroesophageal reflux disease | Gastrointestinal disorders | MedDRA (9.0) | Systematic Assessment |
|
| Irritability | General disorders | MedDRA (9.0) | Systematic Assessment |
|
| Infantile spasms | Nervous system disorders | MedDRA (9.0) | Systematic Assessment |
|
| Ear infection | Infections and infestations | MedDRA (9.0) | Systematic Assessment |
|
| Influenza | Infections and infestations | MedDRA (9.0) | Systematic Assessment |
|
| Rhinitis | Infections and infestations | MedDRA (9.0) | Systematic Assessment |
|
| Dehydration | Metabolism and nutrition disorders | MedDRA (9.0) | Systematic Assessment |
|
| Tonic convulsion | Nervous system disorders | MedDRA (9.0) | Systematic Assessment |
|
| Oral intake reduced | Metabolism and nutrition disorders | MedDRA (9.0) | Systematic Assessment |
|
| Insomnia | Psychiatric disorders | MedDRA (9.0) | Systematic Assessment |
|
| Bronchitis | Infections and infestations | MedDRA (9.0) | Systematic Assessment |
|
| Gastroenteritis | Infections and infestations | MedDRA (9.0) | Systematic Assessment |
|
| Otitis media | Infections and infestations | MedDRA (9.0) | Systematic Assessment |
|
| Pneumonia aspiration | Respiratory, thoracic and mediastinal disorders | MedDRA (9.0) | Systematic Assessment |
|
| Lethargy | Nervous system disorders | MedDRA (9.0) | Systematic Assessment |
|
| Metabolic acidosis | Metabolism and nutrition disorders | MedDRA (9.0) | Systematic Assessment |
|
| Hemoglobin decreased | Investigations | MedDRA (9.0) | Systematic Assessment |
|
| Anemia | Blood and lymphatic system disorders | MedDRA (9.0) | Systematic Assessment |
|
Not provided
Not provided
| D009422 |
| Nervous System Diseases |
| D000073376 | Epileptic Syndromes |
|
| Spasm Clusters - Week 14 |
|
|
| Spasm Clusters - Week 20 |
|
|
| Spasm Clusters - Week 26 |
|
|
| Spasm Clusters - Week 32 |
|
|
|
| Individual Spasms - Week 14 |
|
|
| Individual Spasms - Week 20 |
|
|
| Individual Spasms - Week 26 |
|
|
| Individual Spasms - Week 32 |
|
|
|
| Caregiver's -Week 4 - Slight Improvement |
|
|
| Caregiver's- Week 4 - No change from Baseline |
|
|
| Caregiver's - Week 4 - Moderate Worsening |
|
|
| Caregiver's - Week 4 - Slight Worsening |
|
|
| Caregiver's - Week 4 - Marked Worsening |
|
|
| Caregiver's - Week 8 - Marked Improvement |
|
|
| Caregiver's- Week 8 - Moderate Improvement |
|
|
| Caregiver's -Week 8 - Slight Improvement |
|
|
| Caregiver's- Week 8 - No change from Baseline |
|
|
| Caregiver's - Week 8 - Slight Worsening |
|
|
| Caregiver's - Week 8 - Moderate Worsening |
|
|
| Caregiver's - Week 8 - Marked Worsening |
|
|
| Caregiver's- Week 14 - Marked Improvement |
|
|
| Caregiver's - Week 14 - Moderate Improvement |
|
|
| Caregiver's -Week 14 - Slight Improvement |
|
|
| Caregiver's - Week 14 - No change from Baseline |
|
|
| Caregiver's - Week 14 - Slight Worsening |
|
|
| Caregiver's - Week 14 - Moderate Worsening |
|
|
| Caregiver's - Week 14 - Marked Worsening |
|
|
| Caregiver's - Week 20 - Marked Improvement |
|
|
| Caregiver's - Week 20 - Moderate Improvement |
|
|
| Caregiver's -Week 20 - Slight Improvement |
|
|
| Caregiver's- Week 20 - No change from Baseline |
|
|
| Caregiver's- Week 20 - Slight Worsening |
|
|
| Caregiver's - Week 20 - Moderate Worsening |
|
|
| Caregiver's - Week 20 - Marked Worsening |
|
|
| Caregiver's- Week 26 - Marked Improvement |
|
|
| Caregiver's- Week 26 - Moderate Improvement |
|
|
| Caregiver's -Week 26 - Slight Improvement |
|
|
| Caregiver's - Week 26 - No change from Baseline |
|
|
| Caregiver's - Week 26 - Slight Worsening |
|
|
| Caregiver's - Week 26 - Moderate Worsening |
|
|
| Caregiver's - Week 26 - Marked Worsening |
|
|
| Caregiver's - Week 32 - Marked Improvement |
|
|
| Caregiver's - Week 32 - Moderate Improvement |
|
|
| Caregiver's-Week 32 - Slight Improvement |
|
|
| Caregiver's - Week 32 - No change from Baseline |
|
|
| Caregiver's - Week 32 - Slight Worsening |
|
|
| Caregiver's - Week 32 - Moderate Worsening |
|
|
| Caregiver's - Week 32 - Marked Worsening |
|
|
|
| Investigator's -Week 4 - Slight Improvement |
|
|
| Investigator's- Week 4 - No change from Baseline |
|
|
| Investigator's - Week 4 - Moderate Worsening |
|
|
| Investigator's - Week 4 - Slight Worsening |
|
|
| Investigator's - Week 4 - Marked Worsening |
|
|
| Investigator's - Week 8 - Marked Improvement |
|
|
| Investigator's- Week 8 - Moderate Improvement |
|
|
| Investigator's -Week 8 - Slight Improvement |
|
|
| Investigator's- Week 8 - No change from Baseline |
|
|
| Investigator's - Week 8 - Slight Worsening |
|
|
| Investigator's - Week 8 - Moderate Worsening |
|
|
| Investigator's - Week 8 - Marked Worsening |
|
|
| Investigator's- Week 14 - Marked Improvement |
|
|
| Investigator's - Week 14 - Moderate Improvement |
|
|
| Investigator's -Week 14 - Slight Improvement |
|
|
| Investigator's - Week 14 - No change from Baseline |
|
|
| Investigator's - Week 14 - Slight Worsening |
|
|
| Investigator's - Week 14 - Moderate Worsening |
|
|
| Investigator's - Week 14 - Marked Worsening |
|
|
| Investigator's - Week 20 - Marked Improvement |
|
|
| Investigator's - Week 20 - Moderate Improvement |
|
|
| Investigator's -Week 20 - Slight Improvement |
|
|
| Investigator's- Week 20 - No change from Baseline |
|
|
| Investigator's- Week 20 - Slight Worsening |
|
|
| Investigator's - Week 20 - Moderate Worsening |
|
|
| Investigator's - Week 20 - Marked Worsening |
|
|
| Investigator's- Week 26 - Marked Improvement |
|
|
| Investigator's- Week 26 - Moderate Improvement |
|
|
| Investigator's -Week 26 - Slight Improvement |
|
|
| Investigator's - Week 26 - No change from Baseline |
|
|
| Investigator's - Week 26 - Slight Worsening |
|
|
| Investigator's - Week 26 - Moderate Worsening |
|
|
| Investigator's - Week 26 - Marked Worsening |
|
|
| Investigator's - Week 32 - Marked Improvement |
|
|
| Investigator's - Week 32 - Moderate Improvement |
|
|
| Investigator's -Week 32 - Slight Improvement |
|
|
| Investigator's - Week 32 - No change from Baseline |
|
|
| Investigator's - Week 32 - Slight Worsening |
|
|
| Investigator's - Week 32 - Moderate Worsening |
|
|
| Investigator's - Week 32 - Marked Worsening |
|
|
|
| Spasm Free at Week 14 |
|
|
| Spasm Free at Week 20 |
|
|
| Spasm Free at Week 26 |
|
|
| Spasm Free at Week 32 |
|
|
|
| Achieved absence of spasms at Week 14 |
|
|
| Achieved absence of spasms at Week 20 |
|
|
| Achieved absence of spasms at Week 26 |
|
|
| Achieved absence of spasms at Week 32 |
|
|
|
| Week 8 - Language |
|
|
| Week 8 - Gross Motor |
|
|
| Week 20- Personal Social |
|
|
| Week 20- Fine Motor-Adaptive |
|
|
| Week 20- Language |
|
|
| Week 20 - Gross Motor |
|
|
| Week 32-Personal Social |
|
|
| Week 32-Fine Motor-Adaptive |
|
|
| Week 32-Language |
|
|
| Week 32-Gross Motor |
|
|
|
| Total Spasm-free days during Week 14 visit |
|
|
| Total Spasm-free days during Week 20 visit |
|
|
| Total Spasm-free days during Week 26 visit |
|
|
| Total Spasm-free days during week 32 visit |
|
|
| Cumulative Spasm-free days during Week 4 visit |
|
|
| Cumulative Spasm-free days during Week 8 visit |
|
|
| Cumulative Spasm-free days during Week 14 visit |
|
|
| Cumulative Spasm-free days during Week 20 visit |
|
|
| Cumulative Spasm-free days during Week 26 visit |
|
|
| Cumulative Spasm-free days during Week 32 visit |
|
|