Dravet Syndrome (DS), Lennox-Gastaut Syndrome (LGS)
Conditions
Keywords
Drug Therapy
Brief summary
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.
Detailed description
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.
Interventions
DRUGSoticlestat
Soticlestat tablets or mini-tablets
Sponsors
Takeda
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
2 Years to 65 Years
Healthy volunteers
No
Inclusion criteria
1. The participant has been exposed to fenfluramine (currently on or used previously). 2. The participant has a clinical diagnosis of LGS and a history of, on average, ≥12 MMD seizures in the last 90 days immediately before screening based on historical information, and the participant has ≥4 MMD seizures during a minimum of 4 weeks of seizure data collection during the prospective baseline period. 3. The participant is currently taking 0 to 5 antiseizure treatments (eg. antiseizure medications \[ASMs\], vagus nerve stimulation \[VNS\], ketogenic diet) at stable doses.
Exclusion criteria
1. The participant is currently enrolled in a clinical study involving an investigational product or treatment device (ie, not approved in that country, other than soticlestat), or concurrently enrolled in any other type of medical research judged not to be scientifically or medically compatible with this study. Note: Compatibility will be determined on the basis of consultation with the sponsor/designee. 2. The participant has a known hypersensitivity to any component of the soticlestat formulation. 3. Participants aged ≥6 years who have positive answers on item numbers 4 or 5 on the Columbia-Suicide Severity Rating Scale (C-SSRS) before dosing are excluded. This scale will only be administered to participants aged ≥6 years at the time of enrollment or participants who turn 6 after enrollment.
Design outcomes
Primary
| Measure | 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 |
Countries
Denmark
Participant flow
Pre-assignment details
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.
Participants by arm
| Arm | Count |
|---|---|
| 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. | 0 |
| Total | 0 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Study Terminated by Sponsor | 1 |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 0 |
| other Total, other adverse events | 0 / 0 |
| serious Total, serious adverse events | 0 / 0 |
Outcome results
Primary
Percent Change From Baseline in Convulsive Seizure Frequency Per 28 Days During First 12 Weeks of Maintenance Period for DS Participants
Time frame: Baseline to Week 12 of Maintenance Period
Population: 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.
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
Time frame: Baseline to Week 12 of Maintenance Period
Population: 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.