A Phase 2, Prospective, Interventional, Open-Label, Multisite, Extension Study To Assess the Long-Term Safety and Tolerability of Soticlestat (TAK935) as Adjunctive Therapy in Subjects With Developmental Epileptic Encephalopathies Including Dravet Syndrome, Lennox Gastaut Syndrome, CDKL5 Deficiency Disorder, and Chromosome 15 Duplication Syndrome (ENDYMION 1)
- Conditions
- Epileptic Encephalopathies: Dravet Syndrome, Lennox Gastaut Syndrome, CDKL5 Deficiency Disorder, and Chromosome 15 Duplication Syndrome.MedDRA version: 20.0Level: LLTClassification code 10073682Term: Dravet syndromeSystem Organ Class: 10010331 - Congenital, familial and genetic disordersMedDRA version: 20.1Level: PTClassification code 10048816Term: Lennox-Gastaut syndromeSystem Organ Class: 10029205 - Nervous system disordersMedDRA version: 22.1Level: PTClassification code 10083005Term: CDKL5 deficiency disorderSystem Organ Class: 10010331 - Congenital, familial and genetic disordersMedDRA version: 23.0Level: LLTClassification code 10083952Term: Dup15q syndromeSystem Organ Class: 10010331 - Congenital, familial and genetic disordersTherapeutic area: Diseases [C] - Nervous System Diseases [C10]
- Registration Number
- EUCTR2018-002485-39-PT
- Lead Sponsor
- Takeda Development Center Americas, Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 176
1. Subjects must have participated in a previous soticlestat study and
meet one of the following conditions:
-Successfully completed a Soticlestat clinical study.
-Received at least 10 weeks of treatment (combined Dose Optimization and Maintenance Period) with the study drug in an antecedent placebocontrolled blinded soticlestat clinical study and the subject 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 subject to continue receiving the study drug.
2. In the opinion of the investigator, the subject has the potential to
benefit from the administration of soticlestat (not applicable for Spain).
3. The subject provides written informed consent, or the subject's legal representative (ie, 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 legal
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 non-sterilized 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 post-vasectomy 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 th
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 soticlestat)
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 C-SSRS at Screening) or appearing suicidal per investigator judgment
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method