Epileptic Encephalopathies: Dravet Syndrome, Lennox Gastaut Syndrome, CDKL5 Deficiency Disorder, and Chromosome 15 Duplication Syndrome. MedDRA version: 20.0 Level: LLT Classification code 10073682 Term: Dravet syndrome System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.1 Level: PT Classification code 10048816 Term: Lennox-Gastaut syndrome System Organ Class: 10029205 - Nervous system disorders MedDRA version: 22.1 Level: PT Classification code 10083
Conditions
Interventions
Product Name: Soticlestat
Product Code: TAK-935
Pharmaceutical Form: Tablet
INN or Proposed INN: Soticlestat
CAS Number: 1429505-03-2
Current Sponsor code: TAK-935
Concentration unit: mg milligram(s)
Sponsors
Takeda Development Center Americas, Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1) Subjects must have participated in a previous TAK-935 study and meet one of the following conditions: • Successfully completed a TAK-935 clinical study. • Received at least 10 weeks of treatment (combined Dose Optimization and Maintenance Period) with the study drug in a TAK-935 clinical study and the subjects did not have a serious or severe AE that, in the investigator's or Sponsor's opinion, was related to the study drug and would make it unsafe for the patient to continue receiving the study drug. 2) In the opinion of the investigator, the subject has the potential to benefit from the administration of TAK935. 3) The subject provides written informed consent, or the subject's legal representative (parent or legal guardian) provides written informed consent and the subject provides assent, before any study procedures are performed. 4)The subject and subject's legal representative (ie, parent or guardian [as applicable]) are willing to comply with all study requirements. 5) From signing of informed consent, throughout the duration of the study, and for 30 days after last dose of study drug, female patients of childbearing potential* who are sexually active with a nonsterilized male partner** must agree to use a highly effective method of contraception (from the list below). In addition, they must not donate ova during this period. * Females NOT of childbearing potential are defined as those who are prior to first menarche or who have been surgically sterilized (hysterectomy, bilateral oophorectomy, or tubal ligation) or who are postmenopausal (eg, defined as =1 year since last regular menses with a follicle stimulating hormone level >40 IU/L or =5 years since last regular menses, confirmed before any study drug is administered). **Sterilized males should be =1 year postvasectomy and have confirmed that they have obtained documentation of the absence of sperm in the ejaculate. A highly effective method of contraception is defined as one that has no higher than a 1% failure rate per year when used consistently and correctly. In this study, the only acceptable methods of contraception are as follows: a. Combined (estrogen and progestogen containing) hormonal contraception associated with inhibition of ovulation: • Oral • Intravaginal • Transdermal b. Progestogen-only hormonal contraception associated with inhibition of ovulation: Oral • Injectable • Implantable c. Double-barrier methods (each time the patient has intercourse): • Sponge (plus spermicidal cream or jelly) PLUS male condom with or without spermicidal cream or jelly. • Cap (plus spermicidal cream or jelly) PLUS male condom with or without spermicidal cream or jelly. • Diaphragm (plus spermicidal cream or jelly) PLUS male condom with or without spermicidal cream or jelly. d. Intrauterine device (Copper T PLUS condom) e. Intrauterine hormone-releasing system f. Sterilization: • Bilateral tubal occlusion • Vasectomized partner (provided that the partner is the sole sexual partner of the patient and the absence of sperm in the ejaculate has been confirmed) g. Sexual abstinence, if it is the preferred and usual lifestyle of the patient, will be considered an acceptable method of contraception on a case-by-case basis upon prior approval by the medical monitor. Patients practicing abstinence as a method of contraception must refrain from heterosexual intercourse throughout the duration of the study and for 30 days after last dose of study drug. From signing of informed consent,
Exclusion criteria
Exclusion criteria: 1. Clinically significant disease, that, in the investigator’s opinion, precludes study participation 2. Enrollment in any other clinical trial involving an investigational drug, device, or treatment in the past 90 days (with the exception of an antecedent study involving TAK-935) 3. Subject is currently pregnant or breastfeeding or is planning to become pregnant during the study or within 30 days of the last study drug administration 4. Suicide attempt within the last year, at significant risk of suicide (either in the opinion of the investigator or defined as ‘yes’ to suicidal ideation question 4 or 5 on the Columbia- Suicide Severity Rating Scale [C-SSRS] at Screening) or appearing suicidal per investigator judgment
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: Assess the long-term safety and tolerability of soticlestat when administered as adjunctive therapy to at least one anti-seizure therapy (ie, antiepileptic drugs [AEDs], vagal nerve stimulator, ketogenic diet or modified Atkins diet) in subjects with rare epilepsies. ;Secondary Objective: The secondary objectives, in subjects receiving soticlestat as adjunctive therapy to at least one anti-seizure therapy, are the following: - To assess the effect of soticlestat on seizure frequency. - To assess the effect of soticlestat on the Clinical Global Impression of Severity (CGI-S) provided by the investigator. ;Primary end point(s): •?Incidence of AEs •?Change from Baseline in behavioral and adaptive functional measures using the Vineland Adaptive Behavior Scale (VABS) •?Change from Baseline in behavior measures using total scores and subscale scores of the Aberrant Behavior Checklist-Community Edition (ABC-C) for subjects =6 years of age •?Change from Baseline in the Columbia-Suicide Severity Rating Scale (CSSRS) categorization based onColumbia Classification Algorithm of Suicide Assessment categories 1,2,3,4, and 5 for subjects =6 years of age =6 years of age. •?Absolute values and change from Baseline in clinical laboratory assessments, vital sign measurements, body weight, and ECG parameters •?Incidence of potentially clinically significant clinical safety laboratory test values, vital signs, weight, height/length, and ECG evaluations ;Timepoint(s) of evaluation of this end point: Primary end points will be assessed throughout the study. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): •Percent change from Baseline in all seizure 28-day frequency • Percent change from Baseline in drop seizure 28-day frequency (LGS subjects) • Percent change from Baseline in convulsive seizure 28-day frequency (DS subjects) • Percent change from Baseline in motor seizure 28-day frequency • Change from Baseline in CGI-S ;Timepoint(s) of evaluation of this end point: At Weeks 12, 24, 36, 48, 64, 80, 104, and 108 | — |
Countries
Australia, Canada, China, Israel, Poland, Portugal, Spain, United States
Contacts
Public ContactMedical Director
Takeda Development Center Americas, Inc.
Outcome results
None listed