Trial · NCT04940624
A Study of Soticlestat as an Add-on Therapy in Children and Young Adults With Dravet Syndrome
NCT04940624, Phase 3, Completed, Takeda; results first posted 2025-01-01
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. A Study of Soticlestat as an Add-on Therapy in Children and Young Adults With Dravet Syndrome. Trials, record NCT04940624. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04940624
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Study of Soticlestat as an Add-on Therapy in Children and Young Adults With Dravet Syndrome
- NCT number
- NCT04940624
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 144 participants, actual
- Lead sponsor
- Takeda
- Conditions
- Dravet Syndrome (DS)
- Interventions
- Soticlestat, Placebo
- First posted
- 2021-06-25
- Last updated
- 2026-09-04
- Other registries' IDs
- eudract:2021-002480-22
- Results first posted
- 2025-01-01
Source ClinicalTrials.gov
Registered outcomes15
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percent Change From Baseline in Convulsive Seizure Frequency Per 28 Days During the Full Treatment Period | Baseline; Full Treatment Period: Weeks 1 to 16 | Convulsive seizure frequency per 28 days was defined as the total number of convulsive seizures reported during the period divided by the number of days during the period seizures were assessed multiplied by 28. Percent change from Baseline was defined as (frequency of seizures per 28 days during the Full Treatment Period - frequency of seizures per 28 days at Baseline) divided by the frequency of seizures per 28 days at Baseline multiplied by 100. |
| Primary | Percent Change From Baseline in Convulsive Seizure Frequency Per 28 Days During the Maintenance Period | Baseline; Maintenance Period: Weeks 5 to 16 | Convulsive seizure frequency per 28 days was defined as the total number of convulsive seizures reported during the period divided by number of days during the period seizures were assessed multiplied by 28. Percent change from Baseline was defined as (frequency of seizures per 28 days during Maintenance Period - frequency of seizures per 28 days at Baseline) divided by the frequency of seizures per 28 days at Baseline multiplied by 100. |
| Secondary | Percentage of Responders During Maintenance Period | Maintenance Period: Weeks 5 to 16 | Responders were defined as those with ≥50% reduction from Baseline in convulsive seizures during the Maintenance Period. Percentages were rounded off to the nearest single decimal place. |
| Secondary | Percentage of Responders During the Full Treatment Period | Full Treatment Period: Weeks 1 to 16 | Responders were defined as those with ≥50% reduction from Baseline in convulsive seizures during the Full Treatment Period. Percentages were rounded off to the nearest single decimal place. |
| Secondary | Percentage of Participants With ≤0%, >0% to ≤25%, >25% to ≤50%, >50% to ≤75%, and >75% to ≤100% Reduction in Convulsive Seizures During the Full Treatment Period | Full Treatment Period: Weeks 1 to 16 | Percent reduction from Baseline (%) was defined as \[(Full Treatment Period Convulsive Seizure Frequency - Baseline Convulsive Seizure Frequency) divided by Baseline Convulsive Seizure Frequency\] multiplied by 100. Data was reported as reduction of ≤0%, \>0% to ≤25%, \>25% to ≤50%, \>50% to ≤75%, \>75% to ≤100% or more in seizures from Baseline. Percentages were rounded off to the nearest single decimal place. |
| Secondary | Percentage of Participants With Caregiver Global Impression of Improvement (Care GI-I) Scale Responses as Per the Parent/Caregiver Reported Impression at Week 16 | Week 16 | The Care GI-I is a 7-point Likert scale that the caregiver used to rate improvement in overall seizure control, behavior, safety and tolerability after the initiation of study drug relative to Baseline (before treatment with the study drug). The participant was rated as follows: 1 (very much improved), 2 (much improved), 3 (minimally improved), 4 (no change), 5 (minimally worse), 6 (much worse), and 7 (very much worse). The parent/caregiver completed the Care GI-I via interview. Lower scores indicate improvement. Percentages were rounded off to the nearest single decimal place. |
| Secondary | Percentage of Participants With Clinical Global Impression of Improvement (CGI-I) Scale Responses as Per the Investigator Reported Impression at Week 16 | Week 16 | The CGI-I (Clinician) is a 7-point Likert scale that the investigator used to rate a participant's change (improvement) in overall seizure control, behavior, safety and tolerability, after the initiation of study drug relative to Baseline (before treatment with the study drug). The participant was rated as follows: 1 (very much improved), 2 (much improved), 3 (minimally improved), 4 (no change), 5 (minimally worse), 6 (much worse), and 7 (very much worse). The investigator or designee completed the CGI-I. Lower scores indicate improvement. Percentages were rounded off to the nearest single decimal place. |
| Secondary | Percentage of Participants With CGI-I Nonseizure Symptoms Instrument Responses for Each Domain as Per the Investigator Reported Impression at Week 16 | Week 16 | The CGI-I non-seizure symptoms instrument is a series of single-item assessments that the investigator used to rate improvement in the symptoms and impacts in select non-seizure domains (alertness, communication, and disruptive behaviors) since initiating the study drug. The participant was rated by the investigator for each domain as follows: 1 (very much improved), 2 (much improved), 3 (minimally improved), 4 (no change), 5 (minimally worse), 6 (much worse), and 7 (very much worse). Lower scores indicated improvement. Data for percentage of participants categorized based on the responses for each domain are presented. Percentages were rounded off to the nearest single decimal place. |
| Secondary | Change From Baseline in Quality of Life Inventory-Disability (QI-Disability) Total Score at Week 16 | Baseline, Week 16 | The QI-Disability tool is a parent/caregiver-reported questionnaire that evaluated the quality of life in children with intellectual disabilities. It contains 32 items covering 6 domains of quality of life: physical health, positive emotions, negative emotions, social interaction, leisure and the outdoors, and independence. Each QI-Disability item is rated on a Likert scale of: Never, Rarely, Sometimes, Often, and Very Often. Items were linearly transformed to a scale of 0 to 100, with higher scores representing better quality of life. Domain scores are calculated by averaging item scores. The domain scores are summed and divided by 6 to yield a total score. The total score ranges from 0 to 100, with higher scores indicating a better quality of life. A negative change from Baseline implies deteriorating quality of life. Mixed-effects model for repeated measures (MMRM) was used for analysis. |
| Secondary | Percentage of Participants With CGI-I Seizure Intensity and Duration Instrument Responses as Per the Parent/Caregiver Reported Impression at Week 16 | Week 16 | The CGI-I seizure intensity and duration instrument was used by the parent/caregiver to rate changes in intensity and/or duration of the most impactful seizures from the first assessment. The participant's symptoms were rated on 7-point scale as follows: 1 (very much improved), 2 (much improved), 3 (minimally improved), 4 (no change), 5 (minimally worse), 6 (much worse), and 7 (very much worse). Lower scores indicate improvement. Percentages were rounded off to the nearest single decimal place. |
1 to 10 of 15Source ClinicalTrials.gov
See all 15 registered outcomesOutcomes15
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Percent Change From Baseline in Convulsive Seizure Frequency Per 28 Days During the Full Treatment Period | Baseline; Full Treatment Period: Weeks 1 to 16 | Modified Intent-to-Treat (mITT) Analysis Set included all randomized participants who had received at least one dose of study drug and had been assessed for seizures for at least one day in the Full Treatment Period. | percent change | MEDIAN |
| PRIMARY | Percent Change From Baseline in Convulsive Seizure Frequency Per 28 Days During the Maintenance Period | Baseline; Maintenance Period: Weeks 5 to 16 | mITT Analysis Set included all randomized participants who had received at least one dose of study drug and had been assessed for seizures for at least one day in the Full Treatment Period. Overall number of participants analyzed indicates the number of participants with data available for analyses. | percent change | MEDIAN |
| SECONDARY | Percentage of Responders During Maintenance Period | Maintenance Period: Weeks 5 to 16 | mITT Analysis Set included all randomized participants who had received at least one dose of study drug and had been assessed for seizures for at least one day in the Full Treatment Period. Overall number of participants analyzed indicates the number of participants with data available for analyses. | percentage of participants | NUMBER |
| SECONDARY | Percentage of Responders During the Full Treatment Period | Full Treatment Period: Weeks 1 to 16 | mITT Analysis Set included all randomized participants who had received at least one dose of study drug and had been assessed for seizures for at least one day in the Full Treatment Period. | percentage of participants | NUMBER |
| SECONDARY | Percentage of Participants With ≤0%, >0% to ≤25%, >25% to ≤50%, >50% to ≤75%, and >75% to ≤100% Reduction in Convulsive Seizures During the Full Treatment Period | Full Treatment Period: Weeks 1 to 16 | mITT Analysis Set included all randomized participants who had received at least one dose of study drug and had been assessed for seizures for at least one day in the Full Treatment Period. | percentage of participants | NUMBER |
| SECONDARY | Percentage of Participants With Caregiver Global Impression of Improvement (Care GI-I) Scale Responses as Per the Parent/Caregiver Reported Impression at Week 16 | Week 16 | mITT Analysis Set included all randomized participants who had received at least one dose of study drug and had been assessed for seizures for at least one day in the Full Treatment Period. Overall number of participants analyzed indicates the number of participants with data available for analyses. | percentage of participants | NUMBER |
| SECONDARY | Percentage of Participants With Clinical Global Impression of Improvement (CGI-I) Scale Responses as Per the Investigator Reported Impression at Week 16 | Week 16 | mITT Analysis Set included all randomized participants who had received at least one dose of study drug and had been assessed for seizures for at least one day in the Full Treatment Period. Overall number of participants analyzed indicates the number of participants with data available for analyses. | percentage of participants | NUMBER |
| SECONDARY | Percentage of Participants With CGI-I Nonseizure Symptoms Instrument Responses for Each Domain as Per the Investigator Reported Impression at Week 16 | Week 16 | mITT Analysis Set included all randomized participants who had received at least one dose of study drug and had been assessed for seizures for at least one day in the Full Treatment Period. Overall number of participants analyzed indicates the number of participants with data available for analyses. | percentage of participants | NUMBER |
| SECONDARY | Change From Baseline in Quality of Life Inventory-Disability (QI-Disability) Total Score at Week 16 | Baseline, Week 16 | mITT Analysis Set included all randomized participants who had received at least one dose of study drug and had been assessed for seizures for at least one day in the Full Treatment Period. Overall number of participants analyzed indicates the number of participants with data available for analyses. | score on a scale | MEAN |
| SECONDARY | Percentage of Participants With CGI-I Seizure Intensity and Duration Instrument Responses as Per the Parent/Caregiver Reported Impression at Week 16 | Week 16 | mITT Analysis Set included all randomized participants who had received at least one dose of study drug and had been assessed for seizures for at least one day in the Full Treatment Period. Overall number of participants analyzed indicates the number of participants with data available for analyses. | percentage of participants | NUMBER |
1 to 10 of 15Source ClinicalTrials.gov
See all 15 outcomesResults by group114
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Percent Change From Baseline in Convulsive Seizure Frequency Per 28 Days During the Full Treatment Period | Placebo | -8.64 | Inter-Quartile Range -30.13 to 14.83 | percent change | |
| Percent Change From Baseline in Convulsive Seizure Frequency Per 28 Days During the Full Treatment Period | Soticlestat | -22.16 | Inter-Quartile Range -53.64 to 22.73 | percent change | |
| Percent Change From Baseline in Convulsive Seizure Frequency Per 28 Days During the Maintenance Period | Placebo | -11.99 | Inter-Quartile Range -31.93 to 9.75 | percent change | |
| Percent Change From Baseline in Convulsive Seizure Frequency Per 28 Days During the Maintenance Period | Soticlestat | -23.29 | Inter-Quartile Range -56.92 to 14.15 | percent change | |
| Percentage of Responders During Maintenance Period | Placebo | 11.8 | percentage of participants | ||
| Percentage of Responders During Maintenance Period | Soticlestat | 30.4 | percentage of participants | ||
| Percentage of Responders During the Full Treatment Period | Placebo | 9.9 | percentage of participants | ||
| Percentage of Responders During the Full Treatment Period | Soticlestat | 27.4 | percentage of participants | ||
| Percentage of Participants With ≤0%, >0% to ≤25%, >25% to ≤50%, >50% to ≤75%, and >75% to ≤100% Reduction in Convulsive Seizures During the Full Treatment Period | Placebo | ≤0% Reduction | 39.4 | percentage of participants | |
| Percentage of Participants With ≤0%, >0% to ≤25%, >25% to ≤50%, >50% to ≤75%, and >75% to ≤100% Reduction in Convulsive Seizures During the Full Treatment Period | Soticlestat | ≤0% Reduction | 31.5 | percentage of participants |
1 to 10 of 114Source ClinicalTrials.gov
See all 114 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Placebo | 71 | |
| Overall Study | STARTED | Soticlestat | 73 | |
| Overall Study | COMPLETED | Placebo | 63 | |
| Overall Study | COMPLETED | Soticlestat | 60 | |
| Overall Study | NOT COMPLETED | Placebo | 8 | |
| Overall Study | NOT COMPLETED | Soticlestat | 13 |
Source ClinicalTrials.gov
Adverse events40
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 | Change in seizure presentation | Nervous system disorders | Placebo | 9 | 71 | |
| other | Change in seizure presentation | Nervous system disorders | Soticlestat | 10 | 73 | |
| other | Constipation | Gastrointestinal disorders | Placebo | 0 | 71 | |
| other | Constipation | Gastrointestinal disorders | Soticlestat | 6 | 73 | |
| other | Decreased appetite | Metabolism and nutrition disorders | Placebo | 4 | 71 | |
| other | Decreased appetite | Metabolism and nutrition disorders | Soticlestat | 5 | 73 | |
| other | Fatigue | General disorders | Placebo | 4 | 71 | |
| other | Fatigue | General disorders | Soticlestat | 2 | 73 | |
| other | Insomnia | Psychiatric disorders | Placebo | 1 | 71 | |
| other | Insomnia | Psychiatric disorders | Soticlestat | 6 | 73 |
1 to 10 of 40Source ClinicalTrials.gov
See all 40 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Placebo | 0 | 71 |
| Total, all-cause mortality | Soticlestat | 1 | 73 |
| Total, other adverse events | Placebo | 37 | 71 |
| Total, other adverse events | Soticlestat | 40 | 73 |
| Total, serious adverse events | Placebo | 10 | 71 |
| Total, serious adverse events | Soticlestat | 7 | 73 |
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 comparisons2
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Soticlestat vs Placebo: deaths from any cause | Trial comparisons |
| Soticlestat vs Placebo: 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.
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