Trial · NCT00442104
Open-label Extension to Protocol 1042-0500
NCT00442104, Phase 2, Terminated, Marinus Pharmaceuticals; results first posted 2023-06-02
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Open-label Extension to Protocol 1042-0500. Trials, record NCT00442104. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00442104
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Open-label Extension to Protocol 1042-0500
- NCT number
- NCT00442104
- Status
- Terminated
- Phases
- Phase 2
- Enrollment
- 54 participants, actual
- Lead sponsor
- Marinus Pharmaceuticals
- Conditions
- Infantile Spasms
- Interventions
- Ganaxolone
- First posted
- 2007-03-01
- Last updated
- 2024-05-28
- Results first posted
- 2023-06-02
- Limitations and caveats
- Seizure-free information is not available
Source ClinicalTrials.gov
Registered outcomes9
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percentage of Participants Who Were Free of Spasms | Weeks 4 through Week 96 | Clinical spasms were determined by video-electroencephalography (VEEG). The number of participants with spasm-free duration have been presented. |
| Secondary | Change From Baseline in Frequency of Spasm Clusters | Baseline and Week 4 through Week 32 | 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. |
| Secondary | Change From Baseline in Frequency of Individual Spasm | Baseline and Week 4 through Week 32 | 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. |
| Secondary | Number of Participants With Change in Clinical Status on Caregiver's Global Assessment | Week 4 through Week 32 | 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. |
| Secondary | Number of Participants With Change in Clinical Status on the Investigator's Global Assessment | Week 4 through Week 32 | 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. |
| Secondary | Number of Participants With Spasm-free Durations | Week 4 through Week 32 | 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. |
| Secondary | Number of Participants With Absence of Spasms | Week 4 through Week 32 | 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. |
| Secondary | Developmental Assessment Using Denver-II Developmental Test | Week 8 through Week 32 | 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. |
| Secondary | Percentage of Total Spasm-free Days and Cumulative Spasm-free Days as Determined From the Seizure Diary. | Weeks 4 through Week 32 | 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. |
Source ClinicalTrials.gov
Outcomes9
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Percentage of Participants Who Were Free of Spasms | Weeks 4 through Week 96 | 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. | percentage of participants | NUMBER |
| SECONDARY | Change From Baseline in Frequency of Spasm Clusters | Baseline and Week 4 through Week 32 | ITT Population. Only those participants with data available at the indicated time point were analyzed. | Spasm clusters per day | MEAN |
| SECONDARY | Change From Baseline in Frequency of Individual Spasm | Baseline and Week 4 through Week 32 | ITT Population. Only those participants with data available at the indicated time point were analyzed. | Individual spasms per day | MEAN |
| SECONDARY | Number of Participants With Change in Clinical Status on Caregiver's Global Assessment | Week 4 through Week 32 | ITT population. Only those participants with data available at the indicated time point were analyzed. | participants | NUMBER |
| SECONDARY | Number of Participants With Change in Clinical Status on the Investigator's Global Assessment | Week 4 through Week 32 | ITT population. Only those participants with data available at the indicated time point were analyzed. | participants | NUMBER |
| SECONDARY | Number of Participants With Spasm-free Durations | Week 4 through Week 32 | ITT Population. Only those participants with data available at the indicated time point were analyzed. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants With Absence of Spasms | Week 4 through Week 32 | ITT Population. Only those participants with data available at the indicated time point were analyzed. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Developmental Assessment Using Denver-II Developmental Test | Week 8 through Week 32 | ITT Population. Only those participants with data available at the indicated time point were analyzed. | Scores on a scale | MEAN |
| SECONDARY | Percentage of Total Spasm-free Days and Cumulative Spasm-free Days as Determined From the Seizure Diary. | Weeks 4 through Week 32 | ITT Population. Only those participants with data available at the indicated time point were analyzed. | Percentage of Days | MEAN |
Source ClinicalTrials.gov
Results by group133
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Percentage of Participants Who Were Free of Spasms | Ganaxolone | 100 | 95% Confidence Interval 59.0 to 100.0 | percentage of participants | |
| Change From Baseline in Frequency of Spasm Clusters | Ganaxolone | Spasm Clusters - Week 4 | -2.9 | Standard Deviation 7.94 | Spasm clusters per day |
| Change From Baseline in Frequency of Spasm Clusters | Ganaxolone | Spasm Clusters - Week 8 | -4.1 | Standard Deviation 8.35 | Spasm clusters per day |
| Change From Baseline in Frequency of Spasm Clusters | Ganaxolone | Spasm Clusters - Week 14 | -4.1 | Standard Deviation 9.12 | Spasm clusters per day |
| Change From Baseline in Frequency of Spasm Clusters | Ganaxolone | Spasm Clusters - Week 20 | -5.8 | Standard Deviation 8.58 | Spasm clusters per day |
| Change From Baseline in Frequency of Spasm Clusters | Ganaxolone | Spasm Clusters - Week 26 | -5.6 | Standard Deviation 7.98 | Spasm clusters per day |
| Change From Baseline in Frequency of Spasm Clusters | Ganaxolone | Spasm Clusters - Week 32 | -5.3 | Standard Deviation 9.96 | Spasm clusters per day |
| Change From Baseline in Frequency of Individual Spasm | Ganaxolone | Individual Spasms - Week 4 | -6.8 | Standard Deviation 40.70 | Individual spasms per day |
| Change From Baseline in Frequency of Individual Spasm | Ganaxolone | Individual Spasms - Week 8 | -17.4 | Standard Deviation 31.28 | Individual spasms per day |
| Change From Baseline in Frequency of Individual Spasm | Ganaxolone | Individual Spasms - Week 14 | -20.8 | Standard Deviation 31.21 | Individual spasms per day |
1 to 10 of 133Source ClinicalTrials.gov
See all 133 results by groupParticipant flow3
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Ganaxolone | 54 | |
| Overall Study | COMPLETED | Ganaxolone | 7 | |
| Overall Study | NOT COMPLETED | Ganaxolone | 47 |
Source ClinicalTrials.gov
Adverse events44
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Pyrexia | General disorders | Ganaxolone | 26 | 54 | |
| other | Cough | Respiratory, thoracic and mediastinal disorders | Ganaxolone | 19 | 54 | |
| other | Upper respiratory tract infection | Infections and infestations | Ganaxolone | 18 | 54 | |
| other | Vomiting | Gastrointestinal disorders | Ganaxolone | 15 | 54 | |
| other | Nasopharyngitis | Infections and infestations | Ganaxolone | 13 | 54 | |
| other | Nasal congestion | Respiratory, thoracic and mediastinal disorders | Ganaxolone | 11 | 54 | |
| other | Convulsion | Nervous system disorders | Ganaxolone | 9 | 54 | |
| other | Urinary tract infection | Infections and infestations | Ganaxolone | 8 | 54 | |
| other | Diarrhea | Gastrointestinal disorders | Ganaxolone | 8 | 54 | |
| other | Constipation | Gastrointestinal disorders | Ganaxolone | 7 | 54 |
1 to 10 of 44Source ClinicalTrials.gov
See all 44 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Ganaxolone | 2 | 54 |
| Total, other adverse events | Ganaxolone | 47 | 54 |
| Total, serious adverse events | Ganaxolone | 24 | 54 |
Source ClinicalTrials.gov
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT00442104
GET https://conductscience.org/api/trials/v1/NCT00442104/results?page=2