Trial · NCT01405053
Study of Rufinamide in Pediatric Subjects 1 to Less Than 4 Years of Age With Lennox-Gastaut Syndrome Inadequately Controlled With Other Anti-epileptic Drugs
NCT01405053, Phase 3, Completed, Eisai Inc.; results first posted 2019-08-06
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ConductScience Library. Study of Rufinamide in Pediatric Subjects 1 to Less Than 4 Years of Age With Lennox-Gastaut Syndrome Inadequately Controlled With Other Anti-epileptic Drugs. Trials, record NCT01405053. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01405053
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Study of Rufinamide in Pediatric Subjects 1 to Less Than 4 Years of Age With Lennox-Gastaut Syndrome Inadequately Controlled With Other Anti-epileptic Drugs
- NCT number
- NCT01405053
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 37 participants, actual
- Lead sponsor
- Eisai Inc.
- Conditions
- Lennox-Gastaut Syndrome
- Interventions
- Rufinamide, Any other approved Antiepileptic Drug
- First posted
- 2011-07-29
- Last updated
- 2019-08-06
- Results first posted
- 2019-08-06
Source ClinicalTrials.gov
Registered outcomes10
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Child Behavior Checklist (CBCL) Total Problem T-scores at the End of 2-year Treatment Period | End of Treatment Period (up to approximately Week 106) | CBCL: 99-item questionnaire measures specific behavioral problems or developmental delays, answered by a parent/legal guardian or suitable caregiver. Each item were rated using 3-point scale (0=Not True, 1=Somewhat/Sometimes True, 2=Very True/ Often True) to indicate how often or typical the behavior was. The 99 items were combined to yield scores for 8 problem area scales (emotionally reactive, anxious/depressed, somatic complaints, withdrawn, sleep problems, attention problems, aggressive behavior, and other problems) and 3 summary scores (internalizing, externalizing, and total problems). Total Problem score was sum of all the problem areas plus 1 additional item, ranging from 0 to 198. Total raw scores are converted to t-scores with mean of 50 and standard deviation (SD) of 10. T-scores were standardized test scores that indicate same degree of elevation in problems relative to the normative sample of peers. Higher scores were indicative of more problems. |
| Primary | Change From Baseline in CBCL Total Problem T-Scores at End of 2-year Treatment Period | Baseline and End of Treatment Period (up to approximately Week 106) | CBCL: 99-item questionnaire measures specific behavioral problems or developmental delays, answered by a parent/legal guardian or suitable caregiver. Each item were rated using 3-point scale (0=Not True, 1=Somewhat/Sometimes True, 2=Very True/ Often True) to indicate how often or typical the behavior was. The 99 items were combined to yield scores for 8 problem area scales (emotionally reactive, anxious/depressed, somatic complaints, withdrawn, sleep problems, attention problems, aggressive behavior, and other problems) and 3 summary scores (internalizing, externalizing, and total problems). Total Problem score was sum of all the problem areas plus 1 additional item, ranging from 0 to 198. Total raw scores are converted to t-scores with mean of 50 and standard deviation (SD) of 10. T-scores were standardized test scores that indicate same degree of elevation in problems relative to the normative sample of peers. Higher scores were indicative of more problems. |
| Other | Time to Withdrawal From Treatment Due to an Adverse Event or Lack of Efficacy | Baseline up to the End of the Treatment Period (up to approximately Week 106) | Withdrawal from either rufinamide or other AED was due to the occurrence of an adverse event or for lack of efficacy. Data was obtained till Week 106 and was extrapolated using Kaplan-Meier method to determine the overall survival time (in weeks) to withdrawal from treatment (excluding taper) due to an adverse event or lack efficacy. |
| Other | Percent Change in Total Seizure Frequency Per 28 Days | Baseline up to End of the Treatment Period (up to approximately Week 106) | The frequency per 28 days was defined as (S/D)\*28 where, S was equal to the sum of the seizures reported in the participant seizure diary during the specified time interval and D was equal to the number of days with non-missing data in the participant seizure diary for the specified study phase. The number of seizures was assessed and recorded by the participant's parent(s)/caregiver(s) in the participant seizure diary. |
| Other | Percent Change in Seizure Frequency by Individual Seizure Type Per 28 Days | Baseline up to End of Treatment Period (up to approximately Week 106) | The frequency per 28 days was defined as (S/D)\*28 where, S was equal to the sum of the seizures reported in the participant seizure diary during the specified time interval and D was equal to the number of days with non-missing data in the participant seizure diary for the specified study phase. The number of seizures was assessed and recorded by the participant's parent(s)/caregiver(s) in the participant seizure diary. |
| Other | Incidence of Worsening of Seizures | Baseline up to End of Treatment Period (up to approximately Week 106) | Worsening of seizures was summarized by the incidence of participants with doubling in total seizure frequency, doubling in frequency of major seizures (generalized tonic-clonic, drop attacks), or occurrence of new seizure type during each successive 3 to 4 month visit interval of the Maintenance Period relative to baseline. |
| Other | Change From Baseline in CBCL Sub Scores at Week 106 | Baseline and Week 106 | CBCL: 99-item questionnaire, measures behavioral problems/developmental delays, answered by parent/guardian/caregiver. Each item rated on 3-point scale (0=Not True,1=Somewhat/Sometimes True, 2=Very/Often True). 99 items were combined to give scores for 8 problem area scales, where 1 for each 8 syndrome (emotionally reactive, anxious/depressed, somatic, withdrawn, sleep, attention, aggressive behavior, and other problems) were calculated, range: 0 (normal) to 16 (clinical behavior) and 3 summary scores (internalizing, externalizing, and total problems). All 3 summary scores reported scaled to T-scores. Total Problem score was sum of all the problem areas plus 1 additional item, ranging from 0 to 198. Total raw score were converted to t-scores with mean of 50 and SD of 10. T-scores were standardized test scores that indicate same degree of elevation in problems relative to normative sample of peers. Higher scores were indicative of more problems. |
| Other | Change From Baseline in Language Development Survey (LDS) Scores During Maintenance Period | Baseline, Weeks 24, 56, 88, and 106 | LDS, a caregiver-administered survey consisted of 8-item questionnaire and vocabulary list of 310 words organized within 14 semantic categories. List contained high frequency words (e.g. more), less common words (e.g. hamburger), and lexical chunks (e.g. Sesame Street). Average LDS score, calculated by dividing total number of words across all valid phrases by number of phrases with greater than (\>) 0words; for participants with no words, average was 0. This value was compared to standardized chart to obtain percentile rating. LDS provided 2 scores: average phrase length (number of words/phrase) and number of endorsed vocabulary words. LDS phrase length was categorized into delay (less than or equal to \[\<=\] 20th percentile) and no delay (\>20th percentile). LDS vocabulary was categorized into delay(\<=15th percentile)and no delay(\>15th percentile). Both raw scores were used to provide 2 normative scores based on child's age in months. Higher scores indicated better language development. |
| Other | Change From Baseline in Total Score of Quality of Life in Childhood Epilepsy (QoLCE) Scale | Baseline and Week 106 | The QoLCE was a 76-item questionnaire designed specifically to measure quality of life in children with epilepsy. QOLCE consists of 16 quality of life subscales (14 multi-item and 2 single item). Each subscales had number of items or questions with responses as excellent, very good, good, fair, and poor. They were changed to 1, 2, 3, 4, and 5 as per instructions. Then changed on a scale of 100, where 1 is equal to (=) 0, 2=25, 3=50, 4=75, and 5=100. Items corresponding to each subscale were marked and there mean score was score of that subscale. The form was completed by a parent or caregiver who interacted with the child on a consistent, daily basis and took about 20 to 30 minutes to complete. The higher the score, the better the child's quality of life. |
| Other | Change From Baseline in Sub-scores in QoLCE | Baseline and Week 106 | The QoLCE was a 76-item questionnaire designed specifically to measure quality of life in children with epilepsy. QOLCE consists of 16 quality of life subscales (14 multi-item and 2 single item). Each subscales had number of items or questions with responses as excellent, very good, good, fair, and poor. They were changed to 1, 2, 3, 4, and 5 as per instructions. Then changed on a scale of 100, where 1=0, 2=25, 3=50, 4=75, and 5=100. Items corresponding to each subscale were marked and there mean score was score of that subscale. The form was completed by a parent or caregiver who interacted with the child on a consistent, daily basis and took about 20 to 30 minutes to complete. The higher the score, the better the child's quality of life. |
Source ClinicalTrials.gov
Outcomes10
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Child Behavior Checklist (CBCL) Total Problem T-scores at the End of 2-year Treatment Period | End of Treatment Period (up to approximately Week 106) | The full analysis set for primary efficacy variable included randomized participants who received rufinamide or any other approved add-on AED of the investigator's choice and had baseline and at least 1 postdose cognition measurement. Participants who were evaluable for this outcome measure at given time period were included for assessment. | score on a scale | MEAN |
| PRIMARY | Change From Baseline in CBCL Total Problem T-Scores at End of 2-year Treatment Period | Baseline and End of Treatment Period (up to approximately Week 106) | The full analysis set for primary efficacy variable included randomized participants who received rufinamide or any other approved add-on AED of the investigator's choice and had baseline and at least 1 postdose cognition measurement. Participants who were evaluable for this outcome measure at given time period were included for assessment. | score on a scale | GEOMETRIC_MEAN |
| OTHER_PRE_SPECIFIED | Time to Withdrawal From Treatment Due to an Adverse Event or Lack of Efficacy | Baseline up to the End of the Treatment Period (up to approximately Week 106) | The full analysis set for other efficacy variable included randomized participants who received rufinamide or any other add-on AED of the investigator's choice and had a baseline efficacy assessment and at least 1 post baseline efficacy assessment. Participants who were evaluable at a given time point were included for this assessment. | weeks | MEDIAN |
| OTHER_PRE_SPECIFIED | Percent Change in Total Seizure Frequency Per 28 Days | Baseline up to End of the Treatment Period (up to approximately Week 106) | The full analysis set for other efficacy variable included randomized participants who received rufinamide or any other add-on AED of the investigator's choice and had a baseline efficacy assessment and at least 1 postbaseline efficacy assessment. Participants evaluable for this outcome measure at given time period were included for assessment. | percent change in seizure frequency | MEDIAN |
| OTHER_PRE_SPECIFIED | Percent Change in Seizure Frequency by Individual Seizure Type Per 28 Days | Baseline up to End of Treatment Period (up to approximately Week 106) | The full analysis set for other efficacy variable included randomized participants who received rufinamide or any other add-on AED of the investigator's choice and had a baseline efficacy assessment and at least 1 postbaseline efficacy assessment. Participants evaluable for this outcome measure at given time period were included for assessment. | percent change in seizure frequency | MEDIAN |
| OTHER_PRE_SPECIFIED | Incidence of Worsening of Seizures | Baseline up to End of Treatment Period (up to approximately Week 106) | The full analysis set for other efficacy variable included randomized participants who received rufinamide or any other add-on AED of the investigator's choice and had a baseline efficacy assessment and at least 1 postbaseline efficacy assessment. | Participants | COUNT_OF_PARTICIPANTS |
| OTHER_PRE_SPECIFIED | Change From Baseline in CBCL Sub Scores at Week 106 | Baseline and Week 106 | The full analysis set for other efficacy variable included randomized participants who received rufinamide or any other approved add-on AED of the investigator's choice and had a baseline efficacy and at least 1 postbaseline efficacy assessment. Participants evaluable for this outcome measure at given time period were included for assessment. | score on a scale | MEAN |
| OTHER_PRE_SPECIFIED | Change From Baseline in Language Development Survey (LDS) Scores During Maintenance Period | Baseline, Weeks 24, 56, 88, and 106 | The full analysis set for other efficacy variable included randomized participants who received rufinamide or any other approved add-on AED of the investigator's choice and had a baseline efficacy and at least 1 postbaseline efficacy assessment. Participants evaluable for this outcome measure at given time period were included for assessment. | words | MEAN |
| OTHER_PRE_SPECIFIED | Change From Baseline in Total Score of Quality of Life in Childhood Epilepsy (QoLCE) Scale | Baseline and Week 106 | The full analysis set for other efficacy variable included randomized participants who received rufinamide or any other add-on AED of the investigator's choice and had a baseline efficacy assessment and at least 1 postbaseline efficacy assessment. Participants evaluable for this outcome measure at given time period were included for assessment. | score on a scale | MEAN |
| OTHER_PRE_SPECIFIED | Change From Baseline in Sub-scores in QoLCE | Baseline and Week 106 | The full analysis set for other efficacy variable included randomized participants who received rufinamide or any other approved add-on AED of the investigator's choice and had a baseline efficacy and at least 1 postbaseline efficacy assessment. Participants evaluable for this outcome measure at given time period were included for assessment. | score on a scale | MEAN |
Source ClinicalTrials.gov
Results by group156
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Child Behavior Checklist (CBCL) Total Problem T-scores at the End of 2-year Treatment Period | Rufinamide | 55.7 | Standard Deviation 15.81 | score on a scale | |
| Child Behavior Checklist (CBCL) Total Problem T-scores at the End of 2-year Treatment Period | Any Other Approved Antiepileptic Drug | 54.8 | Standard Deviation 4.50 | score on a scale | |
| Change From Baseline in CBCL Total Problem T-Scores at End of 2-year Treatment Period | Rufinamide | Baseline | 56.6 | Standard Deviation 11.27 | score on a scale |
| Change From Baseline in CBCL Total Problem T-Scores at End of 2-year Treatment Period | Any Other Approved Antiepileptic Drug | Baseline | 62.8 | Standard Deviation 13.07 | score on a scale |
| Change From Baseline in CBCL Total Problem T-Scores at End of 2-year Treatment Period | Rufinamide | Change at Week 106 | -0.3 | Standard Deviation 15.72 | score on a scale |
| Change From Baseline in CBCL Total Problem T-Scores at End of 2-year Treatment Period | Any Other Approved Antiepileptic Drug | Change at Week 106 | -6.7 | Standard Deviation 0.58 | score on a scale |
| Time to Withdrawal From Treatment Due to an Adverse Event or Lack of Efficacy | Rufinamide | 142.0 | 95% Confidence Interval NA to NA | weeks | |
| Time to Withdrawal From Treatment Due to an Adverse Event or Lack of Efficacy | Any Other Approved Antiepileptic Drug | 28.0 | 95% Confidence Interval 17.7 to NA | weeks | |
| Percent Change in Total Seizure Frequency Per 28 Days | Rufinamide | -7.05 | Full Range -79.2 to 3644.1 | percent change in seizure frequency | |
| Percent Change in Total Seizure Frequency Per 28 Days | Any Other Approved Antiepileptic Drug | -20.15 | Full Range -83.3 to 143.1 | percent change in seizure frequency |
1 to 10 of 156Source ClinicalTrials.gov
See all 156 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Rufinamide | 25 | |
| Overall Study | STARTED | Any Other Approved Antiepileptic Drug | 12 | |
| Overall Study | COMPLETED | Rufinamide | 15 | |
| Overall Study | COMPLETED | Any Other Approved Antiepileptic Drug | 4 | |
| Overall Study | NOT COMPLETED | Rufinamide | 10 | |
| Overall Study | NOT COMPLETED | Any Other Approved Antiepileptic Drug | 8 |
Source ClinicalTrials.gov
Adverse events122
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 | Constipation | Gastrointestinal disorders | Rufinamide | 3 | 25 | |
| other | Constipation | Gastrointestinal disorders | Any Other Approved Antiepileptic Drug | 1 | 12 | |
| other | Diarrhoea | Gastrointestinal disorders | Rufinamide | 4 | 25 | |
| other | Diarrhoea | Gastrointestinal disorders | Any Other Approved Antiepileptic Drug | 3 | 12 | |
| other | Nausea | Gastrointestinal disorders | Rufinamide | 1 | 25 | |
| other | Nausea | Gastrointestinal disorders | Any Other Approved Antiepileptic Drug | 1 | 12 | |
| other | Vomiting | Gastrointestinal disorders | Rufinamide | 7 | 25 | |
| other | Vomiting | Gastrointestinal disorders | Any Other Approved Antiepileptic Drug | 1 | 12 | |
| other | Gait disturbance | General disorders | Rufinamide | 2 | 25 | |
| other | Gait disturbance | General disorders | Any Other Approved Antiepileptic Drug | 1 | 12 |
1 to 10 of 122Source ClinicalTrials.gov
See all 122 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Rufinamide | 1 | 25 |
| Total, all-cause mortality | Any Other Approved Antiepileptic Drug | 0 | 12 |
| Total, other adverse events | Rufinamide | 21 | 25 |
| Total, other adverse events | Any Other Approved Antiepileptic Drug | 10 | 12 |
| Total, serious adverse events | Rufinamide | 10 | 25 |
| Total, serious adverse events | Any Other Approved Antiepileptic Drug | 5 | 12 |
Source ClinicalTrials.gov
Papers4
The papers the trial lists on ClinicalTrials.gov.
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 comparisons2
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Rufinamide vs Any Other Approved Antiepileptic Drug: deaths from any cause | Trial comparisons |
| Rufinamide vs Any Other Approved Antiepileptic Drug: serious adverse events | Trial comparisons |
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/NCT01405053
GET https://conductscience.org/api/trials/v1/NCT01405053/results?page=2