The purpose of this study is to investigate the effect of soticlestat on the frequency of motor seizures for participants with Dup15q or CDD during the Maintenance Period.
The drug being tested in this study is called soticlestat. Soticlestat is being tested to treat people with Dup 15q or CDD. This study will assess the effects of TAK-935 on seizure frequency, safety. The study will enroll approximately 30 participants. Participants will be enrolled into 2 groups based on their diagnosis as: Dup 15q or CDD. All participants will be asked to take soticlestat tablets twice daily with or without food. The study comprises of 2 periods: Screening/Baseline Period and Treatment Period (Dose Optimization and Maintenance). The overall time to participate in this study is approximately 30 weeks, including 4 to 6 weeks Screening/Baseline Period, 20 weeks Treatment Period, 2 weeks Taper, and 2 weeks safety follow up period. Participants completing this study will have an option to enroll in the open-label extension (OLE) study, under a separate protocol.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
20
TAK-935 tablets
UCLA
Los Angeles, California, United States
Research Institute Children's Hospital Colorado
Aurora, Colorado, United States
Center for Rare Neurological Diseases
Norcross, Georgia, United States
Center for Rare Neurological Diseases (CRND)--Massachusetts General Hospital
Percent Change From Baseline in Motor Seizure Frequency Per 28 Days During the Maintenance Period
Seizure frequency per 28 days is defined as total number of Seizures reported during the period divided by number of days with no missing seizure count during the period multiplied by 28. Percent Change from Baseline is defined as (frequency of seizures per 28 days during maintenance period - frequency of seizures per 28 days at Baseline) divided by frequency of seizures per 28 days at Baseline multiplied by 100. Positive percent change from Baseline indicates seizure increase and negative percent change from Baseline indicates seizure decrease.
Time frame: Maintenance Period: Weeks 9 to 20
Percent Change From Baseline in Motor Seizure Frequency Per 28 Days During the Treatment Period
Seizure frequency per 28 days is defined as total number of Seizures reported during the period divided by number of days with no missing seizure count during the period multiplied by 28. Percent Change from Baseline is defined as (frequency of seizures per 28 days during the treatment period - frequency of seizures per 28 days at Baseline) divided by frequency of seizures per 28 days at Baseline multiplied by 100. Positive percent change from Baseline indicates seizure increase and negative percent change from Baseline indicates seizure decrease.
Time frame: Treatment Period: Weeks 0 to 20
Percentage of Participants Considered as Treatment Responders During the Maintenance Period
Responders are defined as having over 50% motor seizure reduction compared to Baseline. Percent reduction from Baseline (%) is defined as \[(Maintenance Period motor Seizure Frequency - Baseline Period motor Seizure Frequency) divided by Baseline motor Seizure Frequency\] multiplied by 100. Data is reported as reduction of 25%, 50%, 75% and 100% or more in motor seizures from Baseline.
Time frame: Maintenance Period: Weeks 9 to 20
Percent Change From Baseline in Frequency of Motor Seizures Longer Than 5 Minutes in Participants With CDD
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Boston, Massachusetts, United States
Boston Children's Hospital Translational Neuroscience Center
Boston, Massachusetts, United States
Minnesota Epilepsy Group, P.A.
Saint Paul, Minnesota, United States
New York University (NYU)
New York, New York, United States
Columbia University Medical Center
New York, New York, United States
Seizure frequency is defined as total number of Seizures reported during the period divided by number of days with no missing seizure count during the period. Percent Change from Baseline is defined as (frequency of seizures during Treatment period - frequency of seizures at Baseline) divided by frequency of seizures at Baseline multiplied by 100. Positive percent change from Baseline indicates seizure increase and negative percent change from Baseline indicates seizure decrease. The data is reported only for CDD participants.
Time frame: Treatment Period: Weeks 0 to 20
Proportion of Motor Seizure-free Days in Participants During the Maintenance Period
Seizure-free days is defined as number of days with zero motor seizure during the period the Maintenance Period divided by number of days participant was in the Maintenance Period.
Time frame: Maintenance Period: Weeks 9 to 20
Change From Baseline in Clinician's Global Impression of Severity (CGI-S) Responses of Investigator
The CGI-S focuses on clinician's observations of the participant's cognitive, functional, and behavioral performance since the beginning of the study. The CGI-S is rated on a 7-point scale, with the severity of illness scale using a range of responses where, 1= normal, not at all ill, 2= borderline mentally ill, 3= mildly ill, 4= moderately ill, 5= markedly ill, 6= severely ill and 7=amongst the most extremely ill participants. Negative change from Baseline indicates improvement.
Time frame: Baseline to Week 20
Percentage of Participants With Clinical Global Impression of Change (CGI-C) Responses as Per the Investigator Reported Impression
CGI-Change (CGI-C) treatment response ratings should take account of both therapeutic efficacy and treatment-related AEs. Each component of the CGI is rated separately; the instrument does not yield a global score. The CGI-C is rated on a 5-point scale, where, 0 = marked improvement and no side-effects, 1 = marked improvement and minimal side-effects, 2 = no change, 3 = minimal improvement and marked side-effects and 4 = unchanged or worse and side-effects outweigh the therapeutic effect. Lower scores indicated improvement.
Time frame: Week 20
Percentage of Participants With Care Clinical Global Impression of Change (CGI-C) Responses of Parent/Family
CGI-Change (CGI-C) treatment response ratings should take account of both therapeutic efficacy and treatment-related AEs. Each component of the CGI is rated separately; the instrument does not yield a global score. The CGI-C is rated on a 7-point scale where, 1 = very much improved, 2 = much improved, 3 = slightly improved, 4= no change, 5= slightly worse, 6= much worse and 7= very much worse and marked side-effects. Lower scores indicated improvement.
Time frame: Week 20
Change From Baseline of Plasma 24S-hydroxycholesterol (24HC) Levels
Time frame: Baseline to Week 20
Change From Baseline in Seizure Frequency in Participants Treated With TAK-935 as an Adjunctive Therapy
Seizure Frequency per 28 days is defined as total number of Seizures reported during the period divided by number of days with no missing seizure during the period seizures were assessed multiplied by 28. Positive change from Baseline indicates seizure increase and negative change from Baseline indicates seizure decrease.
Time frame: Baseline to Week 20