A Study of Soticlestat in Adults and Children With Rare Epilepsies

Epilepsy Clinical Trial

— Endymion 1  

Official title:

A Phase 2, Prospective, Interventional, Open-Label, Multi-Site, Extension Study to Assess the Long-Term Safety and Tolerability of Soticlestat (TAK-935) 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)

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT03635073
• Other study ID #: TAK-935-18-001
• Secondary ID: U1111-1218-55152
• Status: Active, not recruiting
• Phase: Phase 2
• Start date: July 19, 2018
• Est. completion date: May 22, 2026

Study information

• Verified date: February 2024
• Source: Takeda
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

The main aim is to assess the long-term safety and tolerability of soticlestat when used along with other anti-seizure treatment.

Participants will receive soticlestat twice a day. Participants will visit the study clinic every 2-6 months throughout the study.

Study treatments may continue as long as the participant is receiving benefit from it.

Description:

The drug being tested in this study is called soticlestat (TAK-935). This global, open-label extension (OLE) study will assess the long-term safety and tolerability of soticlestat in participants with developmental and epileptic encephalopathy (DEE) who participated in previous short-term efficacy/safety studies of soticlestat. All participants will receive Soticlestat treatment.

Participants who rollover from previous blinded study will undergo up to 2 weeks of Dose Optimization Period (depending on the previous study) followed by Maintenance Period. Participants who rollover from an open-label study will continue on their current dose until development is stopped by the sponsor, or the product is approved for marketing, or at any time at the discretion of the sponsor. There will be a 4-week Safety Follow-up Period after the last dose in Maintenance Period, including a 2-week dose Tapering Period.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 156
• Est. completion date: May 22, 2026
• Est. primary completion date: May 22, 2026
• Accepts healthy volunteers: No
• Gender: All
• Age group: 2 Years and older

Eligibility

Inclusion Criteria:

1. Participants 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 with the study drug in an antecedent placebo-controlled blinded soticlestat clinical study and the participant 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 participant to continue receiving the study drug.

2. In the opinion of the investigator, the participant has the potential to benefit from the administration of soticlestat

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 Soticlestat).

3. Participant 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.

Related Conditions & MeSH terms

• Epilepsies, Myoclonic
• Epilepsy
• Lennox Gastaut Syndrome
• Lennox-Gastaut Syndrome (LGS)
• Syndrome

Intervention

Drug:
• Soticlestat
Soticlestat tablets or mini-tablets.

Locations

Country	Name	City	State
Australia	Monash Children's Hospital	Clayton	Victoria
Australia	Austin Hospital	Heidelberg	Victoria
Australia	Austin Hospital	Heidelberg West	Victoria
Canada	Hospital for Sick Children	Toronto	Ontario
China	Beijing Children's Hospital,Capital Medical University	Beijing
China	Beijing Children's Hospital,Capital Medical University	Beijing
China	Peking University First Hospital	Beijing
China	Xiangya Hospital Central South University	Changsha
China	Xiangya Hospital of Central South University	Changsha
China	Children's Hospital of Fudan University	Shanghai	Shanghai
China	Shenzhen Children's Hospital	Shenzhen	Guangdong
Israel	Soroka University Medical Centre	Be'er Sheva
Israel	Soroka University Medical Centre	Beer Sheva
Israel	Bnai Zion Medical Center	Haifa
Israel	Edith Wolfson Medical Center	Holon
Israel	Schneider Children's Medical Center of Israel - Petah Tikvah - PIN	Petach Tikva
Israel	Schneider Children's Medical Center of Israel - Petah Tikvah - PIN	Petah Tikva
Israel	Sheba Medical Center - PPDS	Ramat-Gan
Israel	Sheba Medical Center - PPDS	Ramat-Gan
Israel	Tel Aviv Sourasky Medical Center PPDS	Tel Aviv
Israel	Tel Aviv Sourasky Medical Center PPDS	Tel Aviv	Tel-Aviv
Poland	Uniwersyteckie Centrum Kliniczne	Gdansk	Pomorskie
Poland	NZOZ Centrum Neurologii Dzieciecej i Leczenia Padaczki	Kielce	Swietokrzyskie
Poland	Centrum Medyczne Plejady	Krakow
Poland	Szpital Kliniczny im. H.Swiecickiego Uniwersytetu Medycznego im. Karola Marcinkowskiego w Poznaniu	Poznan	Wielkopolskie
Poland	Samodzielny Publiczny Dzieciecy Szpital Kliniczny w Warszawie	Warsaw
Poland	Samodzielny Publiczny Dzieciecy Szpital Kliniczny w Warszawie	Warszawa
Portugal	Centro Hospitalar Lisboa Central- Hospital Dona Estefania	Lisboa
Portugal	Centro Hospitalar Lisboa Norte, E.P.E. Hospital de Santa Maria	Lisboa
Portugal	Centro Hospitalar Lisboa Norte, E.P.E. Hospital de Santa Maria	Lisboa
Spain	Hospital Vithas La Salud	Granada
Spain	Hospital Ruber Internacional (Grupo Quironsalud)	Madrid
Spain	Hospital Ruber Internacional (Grupo Quironsalud)	Madrid	Madrid, Communidad Delaware
Spain	Clinica Universidad Navarra	Pamplona	Navarra
Spain	Hospital Universitari i Politecnic La Fe de Valencia	Valencia
United States	Rare Disease Research, LLC	Atlanta	Georgia
United States	Rare Disease Research, LLC	Atlanta	Georgia
United States	Colorado Children's Hospital	Aurora	Colorado
United States	Colorado Children's Hospital	Aurora	Colorado
United States	Boston Children's Hospital	Boston	Massachusetts
United States	Medical University of South Carolina Childrens Hospital - PIN	Charleston	South Carolina
United States	Max Benzaquen, M.D., PC	Chesterfield	Missouri
United States	Ann and Robert H Lurie Childrens Hospital of Chicago	Chicago	Illinois
United States	Cook Children's Medical Center - Jane and John Justin Neurosciences Center	Fort Worth	Texas
United States	Northeast Regional Epilepsy Group	Hackensack	New Jersey
United States	Bluegrass Epilepsy Research LLC	Lexington	Kentucky
United States	David Geffen School of Medicine at UCLA	Los Angeles	California
United States	David Geffen School of Medicine at UCLA	Los Angeles	California
United States	Nicklaus Children's Hospital	Miami	Florida
United States	Nicklaus Children's Hospital	Miami	Florida
United States	Children's Hospital at Saint Peter's University Hospital	New Brunswick	New Jersey
United States	Columbia University Medical Center - PIN	New York	New York
United States	NYU - Ambulatory Care Center (ACC)	New York	New York
United States	Center for Rare Neurological Diseases	Norcross	Georgia
United States	Xenosciences Inc	Phoenix	Arizona
United States	Medsol Clinical Research Center Inc	Port Charlotte	Florida
United States	Medsol Clinical Research Center Inc	Port Charlotte	Florida
United States	Mayo Clinic - PIN	Rochester	Minnesota
United States	Minnesota Epilepsy Group PA	Saint Paul	Minnesota
United States	University of South Florida	Tampa	Florida
United States	Wake Forest Baptist Medical Center - PPDS	Winston-Salem	North Carolina
United States	Pediatric Neurology PA	Winter Park	Florida

Sponsors (1)

Lead Sponsor	Collaborator
Takeda

Countries where clinical trial is conducted

United States,  Australia,  Canada,  China,  Israel,  Poland,  Portugal,  Spain, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Percentage of Participants Who Experience At least one Adverse Event (AE)	An Adverse Event (AE) is defined any untoward medical occurrence in a subject or clinical study subject administered a medicinal product and which does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable or unintended sign (for example, an abnormal laboratory finding, vital sign or ECG evaluation), symptom, or disease temporally associated with the use of a medicinal product, whether considered related to this medicinal product.	Up to 6 years
Primary	Change from Baseline in Behavioral and Adaptive Functional Measures Using the Vineland Adaptive Behavior Scale (VABS)	The VABS, 3rd edition, Parent Caregiver Form (VABS-3 Parent Caregiver Form), is a parent-report questionnaire of adaptive functioning or how an individual behaves in their day-to-day life at home and in the community.	Up to 6 years
Primary	Change from Baseline in Behavior Measures Using Total Scores and Subscale Scores of the Aberrant Behavior Checklist-Community Edition (ABC-C) for Participants =6 Years of age		Up to 6 years
Primary	Change from Baseline in the Columbia-Suicide Severity Rating Scale (C-SSRS) Categorization Based on Columbia Classification Algorithm of Suicide Assessment Categories 1,2,3,4, and 5 for Participants =6 Years of age		Up to 6 years
Secondary	Percent Change from Baseline in all Seizure 28-day Frequency		Up to 6 years
Secondary	Percent Change from Baseline in Drop Seizure 28-day Frequency (Lennox-Gastaut syndrome [LGS] Participants)		Up to 6 years
Secondary	Percent Change from Baseline in Convulsive Seizure 28-day Frequency (Dravet syndrome [DS] Participants)		Up to 6 years
Secondary	Percent Change from Baseline in Motor Seizure 28-day Frequency		Up to 6 years
Secondary	Change from Baseline in Clinician's Clinical Global Impression of Severity (CGI-S)	CGI-S is used to obtain an assessment of symptom severity, focusing on clinicians' observations of the subject's current cognitive, functional, and behavioral performance. The CGI-S is rated on a 7-point scale, with the severity of illness scale using a range of responses from 1 (normal) through to 7 (among the most severely ill participants).	Up to 6 years

External Links

•   To obtain more information on the study, click here/on this link