A Phase 2 interventional study of Ganaxolone and Placebo in Infantile Spasms, sponsored by Marinus Pharmaceuticals. Completed at 15 sites in United States. Open to participants aged 4 Months to 24 Months. Per ClinicalTrials.gov, last updated 2023-06-02.
Sponsored by Marinus Pharmaceuticals (part of Immedica Pharma) · Phase 2, Interventional, and Treatment
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.
47 studies on the registry are indexed under Spasms, Infantile; 8 are open to participants now.
This study's enrollment of 57 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.
If being treated with concomitant AEDs
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.
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
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.
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
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.
Corticosteroids (steroids)
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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.
ULN (upper limit of normal)
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
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.
ganaxolone
Drug: Ganaxolone
placebo
Other: Placebo
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.
Time frame: Baseline (Day 0) and Day 10
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.
Time frame: Baseline (Day 0) and Day 20
Number of Participants With Absence of Hypsarrhythmia
Absence of hypsarrhythmia was determined by 24-hour vEEG at Day 10 and Day 20.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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
Time frame: 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.
Time frame: At Day 20
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.
| Milestone | Cohort 1: Sequence A: Ganaxolone/Placebo/Ganaxolone | Cohort 1: Sequence B: Placebo/Ganaxolone/Placebo | Cohort 2: Sequence A: Ganaxolone/Placebo/Ganaxolone | Cohort 2: Sequence B: Placebo/Ganaxolone/Placebo | Cohort 3: Sequence A: Ganaxolone/Placebo/Ganaxolone | Cohort 3: Sequence B: Placebo/Ganaxolone/Placebo | Cohort 4: Sequence A: Ganaxolone/Placebo/Ganaxolone | Cohort 4: Sequence B: Placebo/Ganaxolone/Placebo | Cohort 5: Sequence A: Ganaxolone/Placebo/Ganaxolone | Cohort 5: Sequence B: Placebo/Ganaxolone/Placebo |
|---|---|---|---|---|---|---|---|---|---|---|
| Started | 6 | 3 | 7 | 4 | 12 | 3 | 7 | 5 | 6 | 4 |
| Completed | 6 | 3 | 7 | 3 | 11 | 3 | 7 | 5 | 6 | 4 |
| Not completed | 0 | 0 | 0 | 1 | 1 | 0 | 0 | 0 | 0 | 0 |
| Withdrew: Withdrawal by subject | 0 | 0 | 0 | 1 | 0 | 0 | 0 | 0 | 0 | 0 |
| Withdrew: Other | 0 | 0 | 0 | 0 | 1 | 0 | 0 | 0 | 0 | 0 |
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.
| Spasm clusters per day | Cohort 1: Sequence A: Ganaxolone/Placebo/Ganaxolone | Cohort 1: Sequence B: Placebo/Ganaxolone/Placebo | Cohort 2: Sequence A: Ganaxolone/Placebo/Ganaxolone | Cohort 2: Sequence B: Placebo/Ganaxolone/Placebo | Cohort 3: Sequence A: Ganaxolone/Placebo/Ganaxolone | Cohort 3: Sequence B: Placebo/Ganaxolone/Placebo | Cohort 4: Sequence A: Ganaxolone/Placebo/Ganaxolone | Cohort 4: Sequence B: Placebo/Ganaxolone/Placebo | Cohort 5: Sequence A: Ganaxolone/Placebo/Ganaxolone | Cohort 5: Sequence B: Placebo/Ganaxolone/Placebo |
|---|---|---|---|---|---|---|---|---|---|---|
| Change From Baseline in Frequency of Spasm Clusters at Day 10 | 1.0 ± 20.06 | 2.7 ± 6.66 | -5.4 ± 10.97 | -1.0 ± 1.83 | -1.4 ± 13.69 | -5.3 ± 10.12 | -3.7 ± 4.92 | 0.2 ± 13.12 | -3.2 ± 5.02 | -1.7 ± 7.64 |
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.
| Spasm clusters per day | Cohort 1: Sequence A: Ganaxolone/Placebo/Ganaxolone | Cohort 1: Sequence B: Placebo/Ganaxolone/Placebo | Cohort 2: Sequence A: Ganaxolone/Placebo/Ganaxolone | Cohort 2: Sequence B: Placebo/Ganaxolone/Placebo | Cohort 3: Sequence A: Ganaxolone/Placebo/Ganaxolone | Cohort 3: Sequence B: Placebo/Ganaxolone/Placebo | Cohort 4: Sequence A: Ganaxolone/Placebo/Ganaxolone | Cohort 4: Sequence B: Placebo/Ganaxolone/Placebo | Cohort 5: Sequence A: Ganaxolone/Placebo/Ganaxolone | Cohort 5: Sequence B: Placebo/Ganaxolone/Placebo |
|---|---|---|---|---|---|---|---|---|---|---|
| Change From Baseline in Frequency of Spasm Clusters at Day 20 | -3.2 ± 10.91 | 6.0 ± 11.53 | -5.0 ± 7.55 | -1.5 ± 3.11 | -1.1 ± 11.21 | -12.7 ± 26.27 | -4.4 ± 6.63 | -1.2 ± 13.85 | -3.2 ± 4.92 | -5.3 ± 7.57 |
Absence of hypsarrhythmia was determined by 24-hour vEEG at Day 10 and Day 20.
| Participants | Cohort 1: Sequence A: Ganaxolone/Placebo/Ganaxolone | Cohort 1: Sequence B: Placebo/Ganaxolone/Placebo | Cohort 2: Sequence A: Ganaxolone/Placebo/Ganaxolone | Cohort 2: Sequence B: Placebo/Ganaxolone/Placebo | Cohort 3: Sequence A: Ganaxolone/Placebo/Ganaxolone | Cohort 3: Sequence B: Placebo/Ganaxolone/Placebo | Cohort 4: Sequence A: Ganaxolone/Placebo/Ganaxolone | Cohort 4: Sequence B: Placebo/Ganaxolone/Placebo | Cohort 5: Sequence A: Ganaxolone/Placebo/Ganaxolone | Cohort 5: Sequence B: Placebo/Ganaxolone/Placebo |
|---|---|---|---|---|---|---|---|---|---|---|
| Day 10 | 1 | 0 | 2 | 0 | 2 | 0 | 2 | 2 | 1 | 0 |
| Day 20 | 1 | 0 | 2 | 0 | 2 | 0 | 2 | 2 | 3 | 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 | Cohort 1: Sequence A: Ganaxolone/Placebo/Ganaxolone | Cohort 1: Sequence B: Placebo/Ganaxolone/Placebo | Cohort 2: Sequence A: Ganaxolone/Placebo/Ganaxolone | Cohort 2: Sequence B: Placebo/Ganaxolone/Placebo | Cohort 3: Sequence A: Ganaxolone/Placebo/Ganaxolone | Cohort 3: Sequence B: Placebo/Ganaxolone/Placebo | Cohort 4: Sequence A: Ganaxolone/Placebo/Ganaxolone | Cohort 4: Sequence B: Placebo/Ganaxolone/Placebo | Cohort 5: Sequence A: Ganaxolone/Placebo/Ganaxolone | Cohort 5: Sequence B: Placebo/Ganaxolone/Placebo |
|---|---|---|---|---|---|---|---|---|---|---|
| Marked improvement at Day 10 | 1 | 0 | 0 | 0 | 1 | 0 | 0 | 0 | 0 | 0 |
| Moderate improvement at Day 10 | 0 | 0 | 2 | 0 | 0 | 0 | 1 | 0 | 1 | 0 |
| Slight improvement at Day 10 | 1 | 0 | 1 | 1 | 2 | 1 | 2 | 2 | 2 | 0 |
| No change from Baseline at Day 10 | 4 | 3 | 4 | 3 | 7 | 2 | 4 | 3 | 3 | 3 |
| Slight worsening at Day 10 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 |
| Moderate worsening at Day 10 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 |
| Severe worsening at Day 10 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 |
| Marked improvement at Day 20 | 2 | 1 | 1 | 0 | 1 | 0 | 0 | 0 | 0 | 0 |
| Moderate improvement at Day 20 | 1 | 2 | 1 | 1 | 1 | 0 | 2 | 1 | 1 | 1 |
| Slight improvement at Day 20 | 2 | 0 | 3 | 1 | 6 | 3 | 2 | 3 | 4 | 0 |
| No change from Baseline at Day 20 | 1 | 0 | 2 | 2 | 2 | 0 | 3 | 1 | 1 | 2 |
| Slight worsening at Day 20 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 |
| Moderate worsening at Day 20 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 |
| Severe worsening at Day 20 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 |
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 | Cohort 1: Sequence A: Ganaxolone/Placebo/Ganaxolone | Cohort 1: Sequence B: Placebo/Ganaxolone/Placebo | Cohort 2: Sequence A: Ganaxolone/Placebo/Ganaxolone | Cohort 2: Sequence B: Placebo/Ganaxolone/Placebo | Cohort 3: Sequence A: Ganaxolone/Placebo/Ganaxolone | Cohort 3: Sequence B: Placebo/Ganaxolone/Placebo | Cohort 4: Sequence A: Ganaxolone/Placebo/Ganaxolone | Cohort 4: Sequence B: Placebo/Ganaxolone/Placebo | Cohort 5: Sequence A: Ganaxolone/Placebo/Ganaxolone | Cohort 5: Sequence B: Placebo/Ganaxolone/Placebo |
|---|---|---|---|---|---|---|---|---|---|---|
| Marked improvement at Day 10 | 1 | 0 | 0 | 0 | 1 | 0 | 0 | 0 | 0 | 0 |
| Moderate improvement at Day 10 | 0 | 0 | 2 | 0 | 0 | 0 | 1 | 0 | 1 | 0 |
| Slight improvement at Day 10 | 1 | 1 | 1 | 2 | 5 | 1 | 2 | 3 | 2 | 1 |
| No change from Baseline at Day 10 | 4 | 2 | 4 | 2 | 4 | 2 | 3 | 2 | 3 | 2 |
| Slight worsening at Day 10 | 0 | 0 | 0 | 0 | 0 | 0 | 1 | 0 | 0 | 0 |
| Moderate worsening at Day 10 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 |
| Severe worsening at Day 10 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 |
| Marked improvement at Day 20 | 2 | 1 | 1 | 0 | 1 | 0 | 0 | 0 | 0 | 0 |
| Moderate improvement at Day 20 | 1 | 2 | 1 | 1 | 1 | 1 | 2 | 1 | 1 | 0 |
| Slight improvement at Day 20 | 2 | 0 | 2 | 1 | 6 | 2 | 3 | 3 | 4 | 0 |
| No change from Baseline at Day 20 | 1 | 0 | 3 | 2 | 2 | 0 | 2 | 0 | 1 | 3 |
| Slight worsening at Day 20 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 1 | 0 | 0 |
| Moderate worsening at Day 20 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 |
| Severe worsening at Day 20 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 |
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.
| Participants | Cohort 1: Sequence A: Ganaxolone/Placebo/Ganaxolone | Cohort 1: Sequence B: Placebo/Ganaxolone/Placebo | Cohort 2: Sequence A: Ganaxolone/Placebo/Ganaxolone | Cohort 2: Sequence B: Placebo/Ganaxolone/Placebo | Cohort 3: Sequence A: Ganaxolone/Placebo/Ganaxolone | Cohort 3: Sequence B: Placebo/Ganaxolone/Placebo | Cohort 4: Sequence A: Ganaxolone/Placebo/Ganaxolone | Cohort 4: Sequence B: Placebo/Ganaxolone/Placebo | Cohort 5: Sequence A: Ganaxolone/Placebo/Ganaxolone | Cohort 5: Sequence B: Placebo/Ganaxolone/Placebo |
|---|---|---|---|---|---|---|---|---|---|---|
| Spasm-free Durations at Day 10 | 3 | 1 | 3 | 2 | 2 | 0 | 3 | 0 | 1 | 0 |
| Spasm-free Durations at Day 20 | 3 | 1 | 2 | 2 | 2 | 1 | 1 | 1 | 0 | 0 |
Seizure-free days were measured using data obtained from participants' daily dairy. Participants without seizures have been reported.
| Participants | Cohort 1: Sequence A: Ganaxolone/Placebo/Ganaxolone | Cohort 1: Sequence B: Placebo/Ganaxolone/Placebo | Cohort 2: Sequence A: Ganaxolone/Placebo/Ganaxolone | Cohort 2: Sequence B: Placebo/Ganaxolone/Placebo | Cohort 3: Sequence A: Ganaxolone/Placebo/Ganaxolone | Cohort 3: Sequence B: Placebo/Ganaxolone/Placebo | Cohort 4: Sequence A: Ganaxolone/Placebo/Ganaxolone | Cohort 4: Sequence B: Placebo/Ganaxolone/Placebo | Cohort 5: Sequence A: Ganaxolone/Placebo/Ganaxolone | Cohort 5: Sequence B: Placebo/Ganaxolone/Placebo |
|---|---|---|---|---|---|---|---|---|---|---|
| From Day 8 to Day 10 | 6 | 2 | 6 | 3 | 10 | 3 | 6 | 5 | 6 | 3 |
| From Day 18 to Day 20 | 6 | 1 | 6 | 4 | 10 | 3 | 6 | 5 | 6 | 3 |
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
| Participants | Cohort 1: Sequence A: Ganaxolone/Placebo/Ganaxolone | Cohort 1: Sequence B: Placebo/Ganaxolone/Placebo | Cohort 2: Sequence A: Ganaxolone/Placebo/Ganaxolone | Cohort 2: Sequence B: Placebo/Ganaxolone/Placebo | Cohort 3: Sequence A: Ganaxolone/Placebo/Ganaxolone | Cohort 3: Sequence B: Placebo/Ganaxolone/Placebo | Cohort 4: Sequence A: Ganaxolone/Placebo/Ganaxolone | Cohort 4: Sequence B: Placebo/Ganaxolone/Placebo | Cohort 5: Sequence A: Ganaxolone/Placebo/Ganaxolone | Cohort 5: Sequence B: Placebo/Ganaxolone/Placebo |
|---|---|---|---|---|---|---|---|---|---|---|
| Day 10 | 2 | 1 | 3 | 0 | 3 | 0 | 2 | 2 | 2 | 1 |
| Day 20 | 2 | 0 | 4 | 2 | 3 | 1 | 4 | 1 | 1 | 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 | Cohort 1: Sequence A: Ganaxolone/Placebo/Ganaxolone | Cohort 1: Sequence B: Placebo/Ganaxolone/Placebo | Cohort 2: Sequence A: Ganaxolone/Placebo/Ganaxolone | Cohort 2: Sequence B: Placebo/Ganaxolone/Placebo | Cohort 3: Sequence A: Ganaxolone/Placebo/Ganaxolone | Cohort 3: Sequence B: Placebo/Ganaxolone/Placebo | Cohort 4: Sequence A: Ganaxolone/Placebo/Ganaxolone | Cohort 4: Sequence B: Placebo/Ganaxolone/Placebo | Cohort 5: Sequence A: Ganaxolone/Placebo/Ganaxolone | Cohort 5: Sequence B: Placebo/Ganaxolone/Placebo |
|---|---|---|---|---|---|---|---|---|---|---|
| Personal Social at Day 20 | -5.48 ± 1.618 | -16.90 ± 2.563 | -8.79 ± 5.874 | -8.73 ± 6.071 | -7.76 ± 3.727 | -5.35 ± 0.354 | -5.99 ± 4.354 | -10.22 ± 4.925 | -7.72 ± 4.581 | -12.93 ± 7.336 |
| Fine Motor Adaptive at Day 20 | -4.38 ± 1.834 | -14.63 ± 2.409 | -7.49 ± 5.414 | -7.70 ± 5.624 | -6.40 ± 4.224 | -5.05 ± 2.051 | -6.16 ± 4.484 | -10.24 ± 6.080 | -8.32 ± 3.637 | -12.87 ± 7.731 |
| Language at Day 20 | -1.36 ± 2.681 | -12.33 ± 4.196 | -7.23 ± 7.020 | -5.17 ± 3.017 | -5.82 ± 3.880 | -4.95 ± 0.778 | -6.09 ± 4.674 | -8.22 ± 5.644 | -7.44 ± 3.089 | -10.77 ± 7.223 |
| Gross Motor at Day 20 | -3.96 ± 2.532 | -13.63 ± 2.079 | -7.16 ± 5.298 | -7.00 ± 5.828 | -5.58 ± 3.329 | -3.00 ± 2.546 | -6.89 ± 4.282 | -8.74 ± 5.546 | -8.76 ± 3.260 | -13.43 ± 8.618 |
Collected over Up to 25 days. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Cohort 1: Sequence A: Ganaxolone/Placebo/Ganaxolone | 0/6 (0%) | 2/6 (33.3%) | 6/6 (100%) |
| Cohort 1: Sequence B: Placebo/Ganaxolone/Placebo | 0/3 (0%) | 0/3 (0%) | 3/3 (100%) |
| Cohort 2: Sequence A: Ganaxolone/Placebo/Ganaxolone | 0/7 (0%) | 0/7 (0%) | 5/7 (71.4%) |
| Cohort 2: Sequence B: Placebo/Ganaxolone/Placebo | 0/4 (0%) | 0/4 (0%) | 1/4 (25%) |
| Cohort 3: Sequence A: Ganaxolone/Placebo/Ganaxolone | 0/11 (0%) | 0/11 (0%) | 9/11 (81.8%) |
| Cohort 3: Sequence B: Placebo/Ganaxolone/Placebo | 0/3 (0%) | 0/3 (0%) | 1/3 (33.3%) |
| Cohort 4: Sequence A: Ganaxolone/Placebo/Ganaxolone | 0/7 (0%) | 2/7 (28.6%) | 4/7 (57.1%) |
| Cohort 4: Sequence B: Placebo/Ganaxolone/Placebo | 0/5 (0%) | 0/5 (0%) | 2/5 (40%) |
| Cohort 5: Sequence A: Ganaxolone/Placebo/Ganaxolone | 0/6 (0%) | 0/6 (0%) | 4/6 (66.7%) |
| Cohort 5: Sequence B: Placebo/Ganaxolone/Placebo | 0/4 (0%) | 0/4 (0%) | 4/4 (100%) |
| Event | Cohort 1: Sequence A: Ganaxolone/Placebo/Ganaxolone | Cohort 1: Sequence B: Placebo/Ganaxolone/Placebo | Cohort 2: Sequence A: Ganaxolone/Placebo/Ganaxolone | Cohort 2: Sequence B: Placebo/Ganaxolone/Placebo | Cohort 3: Sequence A: Ganaxolone/Placebo/Ganaxolone | Cohort 3: Sequence B: Placebo/Ganaxolone/Placebo | Cohort 4: Sequence A: Ganaxolone/Placebo/Ganaxolone | Cohort 4: Sequence B: Placebo/Ganaxolone/Placebo | Cohort 5: Sequence A: Ganaxolone/Placebo/Ganaxolone | Cohort 5: Sequence B: Placebo/Ganaxolone/Placebo |
|---|---|---|---|---|---|---|---|---|---|---|
| ConvulsionNervous system disorders | 1/6 | 0/3 | 0/7 | 0/4 | 0/11 | 0/3 | 0/7 | 0/5 | 0/6 | 0/4 |
| HydrocephalusNervous system disorders | 1/6 | 0/3 | 0/7 | 0/4 | 0/11 | 0/3 | 0/7 | 0/5 | 0/6 | 0/4 |
| BradycardiaCardiac disorders | 0/6 | 0/3 | 0/7 | 0/4 | 0/11 | 0/3 | 1/7 | 0/5 | 0/6 | 0/4 |
| LethargyNervous system disorders | 0/6 | 0/3 | 0/7 | 0/4 | 0/11 | 0/3 | 1/7 | 0/5 | 0/6 | 0/4 |
| Event | Cohort 1: Sequence A: Ganaxolone/Placebo/Ganaxolone | Cohort 1: Sequence B: Placebo/Ganaxolone/Placebo | Cohort 2: Sequence A: Ganaxolone/Placebo/Ganaxolone | Cohort 2: Sequence B: Placebo/Ganaxolone/Placebo | Cohort 3: Sequence A: Ganaxolone/Placebo/Ganaxolone | Cohort 3: Sequence B: Placebo/Ganaxolone/Placebo | Cohort 4: Sequence A: Ganaxolone/Placebo/Ganaxolone | Cohort 4: Sequence B: Placebo/Ganaxolone/Placebo | Cohort 5: Sequence A: Ganaxolone/Placebo/Ganaxolone | Cohort 5: Sequence B: Placebo/Ganaxolone/Placebo |
|---|---|---|---|---|---|---|---|---|---|---|
| SomnolenceNervous system disorders | 2/6 | 0/3 | 1/7 | 1/4 | 0/11 | 0/3 | 1/7 | 0/5 | 0/6 | 0/4 |
| IrritabilityGeneral disorders | 2/6 | 0/3 | 0/7 | 0/4 | 1/11 | 0/3 | 0/7 | 0/5 | 0/6 | 0/4 |
| PyrexiaGeneral disorders | 0/6 | 1/3 | 0/7 | 0/4 | 1/11 | 0/3 | 0/7 | 0/5 | 1/6 | 1/4 |
| VomitingGastrointestinal disorders | 0/6 | 1/3 | 1/7 | 0/4 | 3/11 | 1/3 | 1/7 | 0/5 | 0/6 | 0/4 |
| Upper respiratory tract infectionInfections and infestations | 0/6 | 1/3 | 0/7 | 0/4 | 0/11 | 0/3 | 1/7 | 1/5 | 0/6 | 0/4 |
| CoughRespiratory, thoracic and mediastinal disorders | 1/6 | 1/3 | 1/7 | 0/4 | 1/11 | 0/3 | 0/7 | 0/5 | 1/6 | 0/4 |
| Upper respiratory tract congestionRespiratory, thoracic and mediastinal disorders | 0/6 | 1/3 | 0/7 | 0/4 | 0/11 | 0/3 | 0/7 | 0/5 | 0/6 | 0/4 |
| PityriasisSkin and subcutaneous tissue disorders | 0/6 | 1/3 | 0/7 | 0/4 | 0/11 | 0/3 | 0/7 | 0/5 | 0/6 | 0/4 |
| TeethingGastrointestinal disorders | 1/6 | 0/3 | 1/7 | 0/4 | 1/11 | 0/3 | 0/7 | 0/5 | 0/6 | 1/4 |
| RhinorrhoeaRespiratory, thoracic and mediastinal disorders | 0/6 | 0/3 | 0/7 | 1/4 | 0/11 | 0/3 | 0/7 | 0/5 | 0/6 | 0/4 |
Intent-to-treat (ITT) population included all randomized participants who received at least 1 dose of study medication
| Age, Continuous(Months) | Cohort 1: Sequence A: Ganaxolone/Placebo/Ganaxolone | Cohort 1: Sequence B: Placebo/Ganaxolone/Placebo | Cohort 2: Sequence A: Ganaxolone/Placebo/Ganaxolone | Cohort 2: Sequence B: Placebo/Ganaxolone/Placebo | Cohort 3: Sequence A: Ganaxolone/Placebo/Ganaxolone | Cohort 3: Sequence B: Placebo/Ganaxolone/Placebo | Cohort 4: Sequence A: Ganaxolone/Placebo/Ganaxolone | Cohort 4: Sequence B: Placebo/Ganaxolone/Placebo | Cohort 5: Sequence A: Ganaxolone/Placebo/Ganaxolone | Cohort 5: Sequence B: Placebo/Ganaxolone/Placebo | Total |
|---|---|---|---|---|---|---|---|---|---|---|---|
| Mean | 8.2 ± 2.48 | 20.3 ± 3.06 | 13.4 ± 4.89 | 23.0 ± 12.25 | 11.2 ± 5.47 | 14.0 ± 6.93 | 10.3 ± 4.96 | 13.2 ± 3.03 | 11.3 ± 3.27 | 11.5 ± 7.55 | 12.7 ± 6.41 |
| Sex: Female, Male(Participants) | Cohort 1: Sequence A: Ganaxolone/Placebo/Ganaxolone | Cohort 1: Sequence B: Placebo/Ganaxolone/Placebo | Cohort 2: Sequence A: Ganaxolone/Placebo/Ganaxolone | Cohort 2: Sequence B: Placebo/Ganaxolone/Placebo | Cohort 3: Sequence A: Ganaxolone/Placebo/Ganaxolone | Cohort 3: Sequence B: Placebo/Ganaxolone/Placebo | Cohort 4: Sequence A: Ganaxolone/Placebo/Ganaxolone | Cohort 4: Sequence B: Placebo/Ganaxolone/Placebo | Cohort 5: Sequence A: Ganaxolone/Placebo/Ganaxolone | Cohort 5: Sequence B: Placebo/Ganaxolone/Placebo | Total |
|---|---|---|---|---|---|---|---|---|---|---|---|
| Female | 3 | 3 | 5 | 1 | 6 | 2 | 3 | 4 | 3 | 2 | 32 |
| Male | 3 | 0 | 2 | 3 | 5 | 1 | 4 | 1 | 3 | 2 | 24 |
| Ethnicity (NIH/OMB)(Participants) | Cohort 1: Sequence A: Ganaxolone/Placebo/Ganaxolone | Cohort 1: Sequence B: Placebo/Ganaxolone/Placebo | Cohort 2: Sequence A: Ganaxolone/Placebo/Ganaxolone | Cohort 2: Sequence B: Placebo/Ganaxolone/Placebo | Cohort 3: Sequence A: Ganaxolone/Placebo/Ganaxolone | Cohort 3: Sequence B: Placebo/Ganaxolone/Placebo | Cohort 4: Sequence A: Ganaxolone/Placebo/Ganaxolone | Cohort 4: Sequence B: Placebo/Ganaxolone/Placebo | Cohort 5: Sequence A: Ganaxolone/Placebo/Ganaxolone | Cohort 5: Sequence B: Placebo/Ganaxolone/Placebo | Total |
|---|---|---|---|---|---|---|---|---|---|---|---|
| Hispanic or Latino | 1 | 0 | 2 | 0 | 1 | 2 | 2 | 1 | 0 | 0 | 9 |
| Not Hispanic or Latino | 5 | 3 | 5 | 4 | 10 | 1 | 5 | 4 | 6 | 4 | 47 |
| Unknown or Not Reported | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 |
| Race (NIH/OMB)(Participants) | Cohort 1: Sequence A: Ganaxolone/Placebo/Ganaxolone | Cohort 1: Sequence B: Placebo/Ganaxolone/Placebo | Cohort 2: Sequence A: Ganaxolone/Placebo/Ganaxolone | Cohort 2: Sequence B: Placebo/Ganaxolone/Placebo | Cohort 3: Sequence A: Ganaxolone/Placebo/Ganaxolone | Cohort 3: Sequence B: Placebo/Ganaxolone/Placebo | Cohort 4: Sequence A: Ganaxolone/Placebo/Ganaxolone | Cohort 4: Sequence B: Placebo/Ganaxolone/Placebo | Cohort 5: Sequence A: Ganaxolone/Placebo/Ganaxolone | Cohort 5: Sequence B: Placebo/Ganaxolone/Placebo | Total |
|---|---|---|---|---|---|---|---|---|---|---|---|
| American Indian or Alaska Native | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 |
| Asian | 0 | 0 | 0 | 2 | 2 | 0 | 1 | 1 | 2 | 0 | 8 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 |
| Black or African American | 3 | 1 | 0 | 1 | 2 | 0 | 1 | 0 | 0 | 1 | 9 |
| White | 3 | 2 | 7 | 1 | 6 | 3 | 5 | 3 | 1 | 3 | 34 |
| More than one race | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 |
| Unknown or Not Reported | 0 | 0 | 0 | 0 | 1 | 0 | 0 | 1 | 3 | 0 | 5 |
The record gives a start of Jan 2007, a primary completion of May 2008 and a completion of May 2008. It does not say whether these dates are actual or estimated.
Results were first posted in Jun 2023, 15 years 1 month after primary completion. A results delay was requested in Oct 2009.
How these figures are worked out, and which studies are left out of them.
This study is completed, as verified in May 2023. You cannot join it, but the record below documents what was studied.
Get an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.
Studies of the drug tested here: Ganaxolone