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| ID | Type | Description | Link |
|---|---|---|---|
| 2023-504104-29-00 | EU Trial (CTIS) Number |
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Business decision unrelated to patient safety.
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The purpose of this study is to check how soticlestat impacts symptoms of Dravet syndrome [DS] and Lennox-Gastaut syndrome [LGS] in participants who have been exposed to fenfluramine.
The drug being tested in this study is called soticlestat. Soticlestat is being tested to treat people who have DS or LGS and have been exposed to fenfluramine. This study will assess the efficacy and safety of soticlestat in addition to standard care in the treatment of DS or LGS.
The study will enroll approximately 45 patients. This study comprises a screening period of up to 6 weeks, a 4-week titration period, a 48-week maintenance period, a taper period of up to 1 week and a follow-up safety visit. Participants will be enrolled to receive soticlestat along with the standard of care:
• Soticlestat 100-300 milligrams (mg)
Participants will receive oral administration of soticlestat Dose 1 (days 1 to 7), Dose 2 (days 8 to 14), and Dose 3 (Days 15 to 28) with a minimum dose of 100 mg to a maximum dose of 300 mg depending on participant's body weight in the titration period followed by maintenance period up to end of treatment (up to approximately 52 weeks). Percent change from baseline in convulsive in participants with DS and major motor drop (MMD) in participants with LGS seizure frequency per 28 days during the initial 12 weeks of the maintenance period will be assessed.
This multi-center trial will be conducted in the United Kingdom and Europe. The overall time to participate in this study is approximately 60 weeks. Participants will make multiple visits to the clinic and will be followed up for safety by visiting the clinic or by telephone approximately 2 weeks after the last dose of the study drug.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Soticlestat | Experimental | Participant received soticlestat at a starting dose of 100 mg to 200 mg in the 4-week titration. As a part of maintenance (initially planned for 48 weeks per protocol), participant remained on the 200 mg BID dose for 9 days followed by a 1-week taper to receive soticlestat 100 mg BID. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Soticlestat | Drug | Soticlestat tablets or mini-tablets |
|
|
| Measure | Description | Time Frame |
|---|---|---|
| Percent Change From Baseline in Convulsive Seizure Frequency Per 28 Days During First 12 Weeks of Maintenance Period for DS Participants | Baseline to Week 12 of Maintenance Period | |
| Percent Change From Baseline in Major Motor Drop (MMD) Seizure Frequency Per 28 Days During First 12 Weeks of Maintenance Period for LGS Participants | Baseline to Week 12 of Maintenance Period |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Medical Director | Takeda | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Epilepsihospitalet Filadelfia | Dianalund | Region Sjælland | 4293 | Denmark |
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| Label | URL |
|---|---|
| To obtain more information on the study, click this link. | View source |
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De-identified individual participant data from this particular study will not be shared as there is a reasonable likelihood that individual patients could be re-identified (due to the limited number of study participants/study sites, …).
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The study was terminated by the Sponsor due to a business decision (no safety concerns) as sufficient results were obtained from the previous studies and supplementary data from this study was not required. As there was only 1 participant in this study, no data is reported in order to protect and maintain participant privacy/confidentiality.
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| ID | Title | Description |
|---|---|---|
| FG000 | Soticlestat | Participant received soticlestat at a starting dose of 100 milligrams (mg) to 200 mg in the 4-week titration. As a part of maintenance (initially planned for 48 weeks per protocol), participant remained on the 200 mg twice daily (BID) dose for 9 days followed by a 1-week taper to receive soticlestat 100 mg BID. |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
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|
The study was terminated by the Sponsor due to a business decision (no safety concerns) as sufficient results were obtained from the previous studies and supplementary data from this study was not required. As there was only 1 participant in this study, no data is reported in order to protect and maintain participant privacy/confidentiality.
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| ID | Title | Description |
|---|---|---|
| BG000 | Soticlestat | Participant received soticlestat at a starting dose of 100 mg to 200 mg in the 4-week titration. As a part of maintenance (initially planned for 48 weeks per protocol), participant remained on the 200 mg BID dose for 9 days followed by a 1-week taper to receive soticlestat 100 mg BID. |
| Units | Counts |
|---|---|
| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Percent Change From Baseline in Convulsive Seizure Frequency Per 28 Days During First 12 Weeks of Maintenance Period for DS Participants | The study was terminated by the Sponsor due to a business decision (no safety concerns) as sufficient results were obtained from the previous studies and supplementary data from this study was not required. As there was only 1 participant in this study, no data is reported in order to protect and maintain participant privacy/confidentiality. | Posted | Baseline to Week 12 of Maintenance Period |
|
Up to Day 82
The study was terminated by the Sponsor due to a business decision (no safety concerns) as sufficient results were obtained from the previous studies and supplementary data from this study was not required. As there was only 1 participant in this study, no data is reported in order to protect and maintain participant privacy/confidentiality.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Soticlestat | Participant received soticlestat at a starting dose of 100 mg to 200 mg in the 4-week titration. As a part of maintenance (initially planned for 48 weeks per protocol), participant remained on the 200 mg BID dose for 9 days followed by a 1-week taper to receive soticlestat 100 mg BID. |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Study Director | Takeda | +1-877-825-3327 | TrialDisclosures@takeda.com |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Jun 27, 2023 | Feb 14, 2025 | Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Jun 27, 2023 | Feb 14, 2025 | SAP_001.pdf |
| ID | Term |
|---|---|
| D004831 | Epilepsies, Myoclonic |
| D065768 | Lennox Gastaut Syndrome |
| ID | Term |
|---|---|
| D004829 | Epilepsy, Generalized |
| D004827 | Epilepsy |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
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| ID | Term |
|---|---|
| C000712808 | soticlestat |
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| years |
| Sex: Female, Male | participants |
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| Ethnicity (NIH/OMB) | participants |
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| Race (NIH/OMB) | participants |
|
| Units | Counts |
|---|---|
| Participants |
|
| Primary | Percent Change From Baseline in Major Motor Drop (MMD) Seizure Frequency Per 28 Days During First 12 Weeks of Maintenance Period for LGS Participants | The study was terminated by the Sponsor due to a business decision (no safety concerns) as sufficient results were obtained from the previous studies and supplementary data from this study was not required. As there was only 1 participant in this study, no data is reported in order to protect and maintain participant privacy/confidentiality. | Posted | Baseline to Week 12 of Maintenance Period |
|
|
| 0 |
| 0 |
| 0 |
| 0 |
| 0 |
| 0 |
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| D009422 |
| Nervous System Diseases |
| D000073376 | Epileptic Syndromes |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |