A Phase 2 interventional study of Ganaxolone in Infantile Spasms, sponsored by Marinus Pharmaceuticals. Terminated at 14 sites in United States. Open to participants aged 4 Months to 24 Months. Per ClinicalTrials.gov, last updated 2024-05-28.
Sponsored by Marinus Pharmaceuticals (part of Immedica Pharma) · Phase 2, Interventional, and Treatment
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.
47 studies on the registry are indexed under Spasms, Infantile; 8 are open to participants now.
This study's enrollment of 54 is above the median of 40 across 33 interventional studies indexed under Spasms, Infantile.
Browse Spasms, Infantile studies →Marinus Pharmaceuticals is the lead sponsor of 22 studies on the registry; none are open to participants now.
Of its 9 completed or terminated interventional studies of FDA-regulated products, 9 (100%) have results posted.
First submitted before Marinus Pharmaceuticals became part of Immedica Pharma.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ALT and AST (liver enzymes)
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Bilirubin
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Drug: Ganaxolone
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.
Time frame: Weeks 4 through Week 96
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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: Weeks 4 through Week 32
| Milestone | Ganaxolone |
|---|---|
| Started | 54 |
| Completed | 7 |
| Not completed | 47 |
| Withdrew: Adverse event | 5 |
| Withdrew: Death | 2 |
| Withdrew: Lost to follow-up | 3 |
| Withdrew: Withdrawal by subject | 5 |
| Withdrew: Insufficient clinical response | 10 |
| Withdrew: Other | 21 |
| Withdrew: Discretion of the investigator/sponsor | 1 |
Clinical spasms were determined by video-electroencephalography (VEEG). The number of participants with spasm-free duration have been presented.
| percentage of participants | Ganaxolone |
|---|---|
| Percentage of Participants Who Were Free of Spasms | 100 (59.0 to 100.0) |
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.
| Spasm clusters per day | Ganaxolone |
|---|---|
| Spasm Clusters - Week 4 | -2.9 ± 7.94 |
| Spasm Clusters - Week 8 | -4.1 ± 8.35 |
| Spasm Clusters - Week 14 | -4.1 ± 9.12 |
| Spasm Clusters - Week 20 | -5.8 ± 8.58 |
| Spasm Clusters - Week 26 | -5.6 ± 7.98 |
| Spasm Clusters - Week 32 | -5.3 ± 9.96 |
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.
| Individual spasms per day | Ganaxolone |
|---|---|
| Individual Spasms - Week 4 | -6.8 ± 40.70 |
| Individual Spasms - Week 8 | -17.4 ± 31.28 |
| Individual Spasms - Week 14 | -20.8 ± 31.21 |
| Individual Spasms - Week 20 | -15.8 ± 52.46 |
| Individual Spasms - Week 26 | -19.9 ± 39.27 |
| Individual Spasms - Week 32 | -17.7 ± 36.35 |
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.
| participants | Ganaxolone |
|---|---|
| Caregiver's- Week 4 - Marked Improvement | 11 |
| Caregiver's - Week 4 - Moderate Improvement | 11 |
| Caregiver's -Week 4 - Slight Improvement | 16 |
| Caregiver's- Week 4 - No change from Baseline | 6 |
| Caregiver's - Week 4 - Moderate Worsening | 0 |
| Caregiver's - Week 4 - Slight Worsening | 1 |
| Caregiver's - Week 4 - Marked Worsening | 0 |
| Caregiver's - Week 8 - Marked Improvement | 7 |
| Caregiver's- Week 8 - Moderate Improvement | 12 |
| Caregiver's -Week 8 - Slight Improvement | 18 |
| Caregiver's- Week 8 - No change from Baseline | 1 |
| Caregiver's - Week 8 - Slight Worsening | 2 |
| Caregiver's - Week 8 - Moderate Worsening | 0 |
| Caregiver's - Week 8 - Marked Worsening | 0 |
| Caregiver's- Week 14 - Marked Improvement | 8 |
| Caregiver's - Week 14 - Moderate Improvement | 13 |
| Caregiver's -Week 14 - Slight Improvement | 14 |
| Caregiver's - Week 14 - No change from Baseline | 2 |
| Caregiver's - Week 14 - Slight Worsening | 1 |
| Caregiver's - Week 14 - Moderate Worsening | 0 |
| Caregiver's - Week 14 - Marked Worsening | 0 |
| Caregiver's - Week 20 - Marked Improvement | 9 |
| Caregiver's - Week 20 - Moderate Improvement | 13 |
| Caregiver's -Week 20 - Slight Improvement | 10 |
| Caregiver's- Week 20 - No change from Baseline | 3 |
| Caregiver's- Week 20 - Slight Worsening | 1 |
| Caregiver's - Week 20 - Moderate Worsening | 0 |
| Caregiver's - Week 20 - Marked Worsening | 0 |
| Caregiver's- Week 26 - Marked Improvement | 9 |
| Caregiver's- Week 26 - Moderate Improvement | 12 |
| Caregiver's -Week 26 - Slight Improvement | 7 |
| Caregiver's - Week 26 - No change from Baseline | 3 |
| Caregiver's - Week 26 - Slight Worsening | 0 |
| Caregiver's - Week 26 - Moderate Worsening | 0 |
| Caregiver's - Week 26 - Marked Worsening | 0 |
| Caregiver's - Week 32 - Marked Improvement | 8 |
| Caregiver's - Week 32 - Moderate Improvement | 7 |
| Caregiver's-Week 32 - Slight Improvement | 8 |
| Caregiver's - Week 32 - No change from Baseline | 2 |
| Caregiver's - Week 32 - Slight Worsening | 0 |
| Caregiver's - Week 32 - Moderate Worsening | 1 |
| Caregiver's - Week 32 - Marked Worsening | 0 |
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.
| participants | Ganaxolone |
|---|---|
| Investigator's- Week 4 - Marked Improvement | 7 |
| Investigator's - Week 4 - Moderate Improvement | 15 |
| Investigator's -Week 4 - Slight Improvement | 18 |
| Investigator's- Week 4 - No change from Baseline | 4 |
| Investigator's - Week 4 - Moderate Worsening | 1 |
| Investigator's - Week 4 - Slight Worsening | 0 |
| Investigator's - Week 4 - Marked Worsening | 0 |
| Investigator's - Week 8 - Marked Improvement | 6 |
| Investigator's- Week 8 - Moderate Improvement | 12 |
| Investigator's -Week 8 - Slight Improvement | 19 |
| Investigator's- Week 8 - No change from Baseline | 3 |
| Investigator's - Week 8 - Slight Worsening | 0 |
| Investigator's - Week 8 - Moderate Worsening | 0 |
| Investigator's - Week 8 - Marked Worsening | 0 |
| Investigator's- Week 14 - Marked Improvement | 6 |
| Investigator's - Week 14 - Moderate Improvement | 16 |
| Investigator's -Week 14 - Slight Improvement | 15 |
| Investigator's - Week 14 - No change from Baseline | 1 |
| Investigator's - Week 14 - Slight Worsening | 0 |
| Investigator's - Week 14 - Moderate Worsening | 0 |
| Investigator's - Week 14 - Marked Worsening | 0 |
| Investigator's - Week 20 - Marked Improvement | 7 |
| Investigator's - Week 20 - Moderate Improvement | 14 |
| Investigator's -Week 20 - Slight Improvement | 11 |
| Investigator's- Week 20 - No change from Baseline | 1 |
| Investigator's- Week 20 - Slight Worsening | 1 |
| Investigator's - Week 20 - Moderate Worsening | 0 |
| Investigator's - Week 20 - Marked Worsening | 0 |
| Investigator's- Week 26 - Marked Improvement | 5 |
| Investigator's- Week 26 - Moderate Improvement | 14 |
| Investigator's -Week 26 - Slight Improvement | 9 |
| Investigator's - Week 26 - No change from Baseline | 4 |
| Investigator's - Week 26 - Slight Worsening | 0 |
| Investigator's - Week 26 - Moderate Worsening | 0 |
| Investigator's - Week 26 - Marked Worsening | 0 |
| Investigator's - Week 32 - Marked Improvement | 6 |
| Investigator's - Week 32 - Moderate Improvement | 9 |
| Investigator's -Week 32 - Slight Improvement | 8 |
| Investigator's - Week 32 - No change from Baseline | 3 |
| Investigator's - Week 32 - Slight Worsening | 1 |
| Investigator's - Week 32 - Moderate Worsening | 0 |
| Investigator's - Week 32 - Marked Worsening | 0 |
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.
| Participants | Ganaxolone |
|---|---|
| Spasm Free at Week 4 | 19 |
| Spasm Free at Week 8 | 18 |
| Spasm Free at Week 14 | 18 |
| Spasm Free at Week 20 | 18 |
| Spasm Free at Week 26 | 17 |
| Spasm Free at Week 32 | 13 |
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.
| Participants | Ganaxolone |
|---|---|
| Achieved absence of spasms at Week 4 | 2 |
| Achieved absence of spasms at Week 8 | 3 |
| Achieved absence of spasms at Week 14 | 3 |
| Achieved absence of spasms at Week 20 | 4 |
| Achieved absence of spasms at Week 26 | 5 |
| Achieved absence of spasms at Week 32 | 1 |
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.
| Scores on a scale | Ganaxolone |
|---|---|
| Week 8 - Personal Social | -0.72 ± 3.899 |
| Week 8 - Fine Motor-Adaptive | -1.56 ± 3.131 |
| Week 8 - Language | -1.45 ± 2.798 |
| Week 8 - Gross Motor | -1.05 ± 2.285 |
| Week 20- Personal Social | -2.82 ± 6.081 |
| Week 20- Fine Motor-Adaptive | -3.73 ± 5.123 |
| Week 20- Language | -2.39 ± 5.615 |
| Week 20 - Gross Motor | -2.69 ± 3.584 |
| Week 32-Personal Social | -4.86 ± 5.659 |
| Week 32-Fine Motor-Adaptive | -6.80 ± 4.952 |
| Week 32-Language | -6.58 ± 4.336 |
| Week 32-Gross Motor | -6.13 ± 4.509 |
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.
| Percentage of Days | Ganaxolone |
|---|---|
| Total Spasm-free days during Week 4 visit | 11.10 ± 23.657 |
| Total Spasm-free days during Week 8 visit | 18.74 ± 32.654 |
| Total Spasm-free days during Week 14 visit | 20.74 ± 34.817 |
| Total Spasm-free days during Week 20 visit | 28.79 ± 40.163 |
| Total Spasm-free days during Week 26 visit | 28.73 ± 39.711 |
| Total Spasm-free days during week 32 visit | 27.99 ± 39.259 |
| Cumulative Spasm-free days during Week 4 visit | 10.86 ± 20.374 |
| Cumulative Spasm-free days during Week 8 visit | 13.82 ± 23.433 |
| Cumulative Spasm-free days during Week 14 visit | 15.70 ± 25.769 |
| Cumulative Spasm-free days during Week 20 visit | 19.97 ± 28.501 |
| Cumulative Spasm-free days during Week 26 visit | 22.07 ± 31.105 |
| Cumulative Spasm-free days during Week 32 visit | 22.99 ± 30.140 |
Collected over Up to Week 32. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Ganaxolone | 2/54 (3.7%) | 24/54 (44.4%) | 47/54 (87%) |
| Event | Ganaxolone |
|---|---|
| Infantile spasmsNervous system disorders | 5/54 |
| ConvulsionNervous system disorders | 4/54 |
| PyrexiaGeneral disorders | 4/54 |
| Pneumonia aspirationRespiratory, thoracic and mediastinal disorders | 3/54 |
| Gastroesophageal reflux diseaseGastrointestinal disorders | 3/54 |
| DehydrationMetabolism and nutrition disorders | 3/54 |
| EpilepsyNervous system disorders | 2/54 |
| GastroenteritisInfections and infestations | 2/54 |
| NasopharyngitisInfections and infestations | 2/54 |
| Upper respiratory tract infectionInfections and infestations | 2/54 |
| Event | Ganaxolone |
|---|---|
| PyrexiaGeneral disorders | 26/54 |
| CoughRespiratory, thoracic and mediastinal disorders | 19/54 |
| Upper respiratory tract infectionInfections and infestations | 18/54 |
| VomitingGastrointestinal disorders | 15/54 |
| NasopharyngitisInfections and infestations | 13/54 |
| Nasal congestionRespiratory, thoracic and mediastinal disorders | 11/54 |
| ConvulsionNervous system disorders | 9/54 |
| Urinary tract infectionInfections and infestations | 8/54 |
| DiarrheaGastrointestinal disorders | 8/54 |
| ConstipationGastrointestinal disorders | 7/54 |
| Age, Continuous(months) | Ganaxolone |
|---|---|
| Mean | 13.4 ± 6.38 |
| Sex: Female, Male(Participants) | Ganaxolone |
|---|---|
| Female | 32 |
| Male | 22 |
| Ethnicity (NIH/OMB)(Participants) | Ganaxolone |
|---|---|
| Hispanic or Latino | 7 |
| Not Hispanic or Latino | 47 |
| Unknown or Not Reported | 0 |
| Race (NIH/OMB)(Participants) | Ganaxolone |
|---|---|
| American Indian or Alaska Native | 0 |
| Asian | 6 |
| Native Hawaiian or Other Pacific Islander | 0 |
| Black or African American | 8 |
| White | 33 |
| More than one race | 0 |
| Unknown or Not Reported | 7 |
This study is terminated, as verified in May 2024. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Ganaxolone