search
Back to results

A Study of Soticlestat in Adults and Children With Rare Epilepsies (Endymion 1)

Primary Purpose

Epilepsy, Dravet Syndrome (DS), Lennox-Gastaut Syndrome (LGS)

Status
Active
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
Soticlestat
Sponsored by
Takeda
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Epilepsy focused on measuring Soticlestat, Drug Therapy

Eligibility Criteria

2 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

  • Xenosciences Inc
  • David Geffen School of Medicine at UCLA
  • David Geffen School of Medicine at UCLA
  • Colorado Children's Hospital
  • Colorado Children's Hospital
  • Nicklaus Children's Hospital
  • Nicklaus Children's Hospital
  • Medsol Clinical Research Center Inc
  • Medsol Clinical Research Center Inc
  • University of South Florida
  • Pediatric Neurology PA
  • Rare Disease Research, LLC
  • Rare Disease Research, LLC
  • Center for Rare Neurological Diseases
  • Ann and Robert H Lurie Childrens Hospital of Chicago
  • Bluegrass Epilepsy Research LLC
  • Boston Children's Hospital
  • Mayo Clinic - PIN
  • Minnesota Epilepsy Group PA
  • Max Benzaquen, M.D., PC
  • Northeast Regional Epilepsy Group
  • Children's Hospital at Saint Peter's University Hospital
  • NYU - Ambulatory Care Center (ACC)
  • Columbia University Medical Center - PIN
  • Wake Forest Baptist Medical Center - PPDS
  • Medical University of South Carolina Childrens Hospital - PIN
  • Cook Children's Medical Center - Jane and John Justin Neurosciences Center
  • Monash Children's Hospital
  • Austin Hospital
  • Austin Hospital
  • Hospital for Sick Children
  • Shenzhen Children's Hospital
  • Children's Hospital of Fudan University
  • Peking University First Hospital
  • Beijing Children's Hospital,Capital Medical University
  • Beijing Children's Hospital,Capital Medical University
  • Xiangya Hospital Central South University
  • Xiangya Hospital of Central South University
  • Tel Aviv Sourasky Medical Center PPDS
  • Soroka University Medical Centre
  • Soroka University Medical Centre
  • Bnai Zion Medical Center
  • Edith Wolfson Medical Center
  • Schneider Children's Medical Center of Israel - Petah Tikvah - PIN
  • Schneider Children's Medical Center of Israel - Petah Tikvah - PIN
  • Sheba Medical Center - PPDS
  • Sheba Medical Center - PPDS
  • Tel Aviv Sourasky Medical Center PPDS
  • Uniwersyteckie Centrum Kliniczne
  • NZOZ Centrum Neurologii Dzieciecej i Leczenia Padaczki
  • Szpital Kliniczny im. H.Swiecickiego Uniwersytetu Medycznego im. Karola Marcinkowskiego w Poznaniu
  • Centrum Medyczne Plejady
  • Samodzielny Publiczny Dzieciecy Szpital Kliniczny w Warszawie
  • Samodzielny Publiczny Dzieciecy Szpital Kliniczny w Warszawie
  • Centro Hospitalar Lisboa Central- Hospital Dona Estefania
  • Centro Hospitalar Lisboa Norte, E.P.E. Hospital de Santa Maria
  • Centro Hospitalar Lisboa Norte, E.P.E. Hospital de Santa Maria
  • Hospital Ruber Internacional (Grupo Quironsalud)
  • Clinica Universidad Navarra
  • Hospital Vithas La Salud
  • Hospital Ruber Internacional (Grupo Quironsalud)
  • Hospital Universitari i Politecnic La Fe de Valencia

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Soticlestat

Arm Description

Treatment: Soticlestat, tablets orally twice daily at optimized dose, titrated in up to 2 weeks of Dose Optimization Period, followed by Maintenance Period, which lasts until development is stopped by the sponsor, or the product is approved for marketing, or at any time at the discretion of the sponsor.

Outcomes

Primary Outcome Measures

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.
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.
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
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

Secondary Outcome Measures

Percent Change from Baseline in all Seizure 28-day Frequency
Percent Change from Baseline in Drop Seizure 28-day Frequency (Lennox-Gastaut syndrome [LGS] Participants)
Percent Change from Baseline in Convulsive Seizure 28-day Frequency (Dravet syndrome [DS] Participants)
Percent Change from Baseline in Motor Seizure 28-day Frequency
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).

Full Information

First Posted
August 6, 2018
Last Updated
May 18, 2023
Sponsor
Takeda
search

1. Study Identification

Unique Protocol Identification Number
NCT03635073
Brief Title
A Study of Soticlestat in Adults and Children With Rare Epilepsies
Acronym
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)
Study Type
Interventional

2. Study Status

Record Verification Date
May 2023
Overall Recruitment Status
Active, not recruiting
Study Start Date
July 19, 2018 (Actual)
Primary Completion Date
May 22, 2026 (Anticipated)
Study Completion Date
May 22, 2026 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Takeda

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Product Manufactured in and Exported from the U.S.
No
Data Monitoring Committee
No

5. Study Description

Brief 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.
Detailed 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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Epilepsy, Dravet Syndrome (DS), Lennox-Gastaut Syndrome (LGS)
Keywords
Soticlestat, Drug Therapy

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
156 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Soticlestat
Arm Type
Experimental
Arm Description
Treatment: Soticlestat, tablets orally twice daily at optimized dose, titrated in up to 2 weeks of Dose Optimization Period, followed by Maintenance Period, which lasts until development is stopped by the sponsor, or the product is approved for marketing, or at any time at the discretion of the sponsor.
Intervention Type
Drug
Intervention Name(s)
Soticlestat
Other Intervention Name(s)
TAK-935
Intervention Description
Soticlestat tablets or mini-tablets.
Primary Outcome Measure Information:
Title
Percentage of Participants Who Experience At least one Adverse Event (AE)
Description
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.
Time Frame
Up to 6 years
Title
Change from Baseline in Behavioral and Adaptive Functional Measures Using the Vineland Adaptive Behavior Scale (VABS)
Description
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.
Time Frame
Up to 6 years
Title
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
Time Frame
Up to 6 years
Title
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
Time Frame
Up to 6 years
Secondary Outcome Measure Information:
Title
Percent Change from Baseline in all Seizure 28-day Frequency
Time Frame
Up to 6 years
Title
Percent Change from Baseline in Drop Seizure 28-day Frequency (Lennox-Gastaut syndrome [LGS] Participants)
Time Frame
Up to 6 years
Title
Percent Change from Baseline in Convulsive Seizure 28-day Frequency (Dravet syndrome [DS] Participants)
Time Frame
Up to 6 years
Title
Percent Change from Baseline in Motor Seizure 28-day Frequency
Time Frame
Up to 6 years
Title
Change from Baseline in Clinician's Clinical Global Impression of Severity (CGI-S)
Description
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).
Time Frame
Up to 6 years

10. Eligibility

Sex
All
Minimum Age & Unit of Time
2 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: 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. In the opinion of the investigator, the participant has the potential to benefit from the administration of soticlestat Exclusion Criteria: Clinically significant disease, that, in the investigator's opinion, precludes study participation. 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). 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. 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.
Facility Information:
Facility Name
Xenosciences Inc
City
Phoenix
State/Province
Arizona
ZIP/Postal Code
85004
Country
United States
Facility Name
David Geffen School of Medicine at UCLA
City
Los Angeles
State/Province
California
ZIP/Postal Code
900095-1752
Country
United States
Facility Name
David Geffen School of Medicine at UCLA
City
Los Angeles
State/Province
California
ZIP/Postal Code
90095-8358
Country
United States
Facility Name
Colorado Children's Hospital
City
Aurora
State/Province
Colorado
ZIP/Postal Code
80045-7106
Country
United States
Facility Name
Colorado Children's Hospital
City
Aurora
State/Province
Colorado
ZIP/Postal Code
80045
Country
United States
Facility Name
Nicklaus Children's Hospital
City
Miami
State/Province
Florida
ZIP/Postal Code
33155-3009
Country
United States
Facility Name
Nicklaus Children's Hospital
City
Miami
State/Province
Florida
ZIP/Postal Code
33155
Country
United States
Facility Name
Medsol Clinical Research Center Inc
City
Port Charlotte
State/Province
Florida
ZIP/Postal Code
33952
Country
United States
Facility Name
Medsol Clinical Research Center Inc
City
Port Charlotte
State/Province
Florida
ZIP/Postal Code
33980
Country
United States
Facility Name
University of South Florida
City
Tampa
State/Province
Florida
ZIP/Postal Code
33606
Country
United States
Facility Name
Pediatric Neurology PA
City
Winter Park
State/Province
Florida
ZIP/Postal Code
32789
Country
United States
Facility Name
Rare Disease Research, LLC
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30318
Country
United States
Facility Name
Rare Disease Research, LLC
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30322
Country
United States
Facility Name
Center for Rare Neurological Diseases
City
Norcross
State/Province
Georgia
ZIP/Postal Code
30093
Country
United States
Facility Name
Ann and Robert H Lurie Childrens Hospital of Chicago
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60611
Country
United States
Facility Name
Bluegrass Epilepsy Research LLC
City
Lexington
State/Province
Kentucky
ZIP/Postal Code
40504
Country
United States
Facility Name
Boston Children's Hospital
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02115
Country
United States
Facility Name
Mayo Clinic - PIN
City
Rochester
State/Province
Minnesota
ZIP/Postal Code
55905
Country
United States
Facility Name
Minnesota Epilepsy Group PA
City
Saint Paul
State/Province
Minnesota
ZIP/Postal Code
55102
Country
United States
Facility Name
Max Benzaquen, M.D., PC
City
Chesterfield
State/Province
Missouri
ZIP/Postal Code
63017
Country
United States
Facility Name
Northeast Regional Epilepsy Group
City
Hackensack
State/Province
New Jersey
ZIP/Postal Code
07601-1974
Country
United States
Facility Name
Children's Hospital at Saint Peter's University Hospital
City
New Brunswick
State/Province
New Jersey
ZIP/Postal Code
08901
Country
United States
Facility Name
NYU - Ambulatory Care Center (ACC)
City
New York
State/Province
New York
ZIP/Postal Code
10016
Country
United States
Facility Name
Columbia University Medical Center - PIN
City
New York
State/Province
New York
ZIP/Postal Code
10032
Country
United States
Facility Name
Wake Forest Baptist Medical Center - PPDS
City
Winston-Salem
State/Province
North Carolina
ZIP/Postal Code
27157
Country
United States
Facility Name
Medical University of South Carolina Childrens Hospital - PIN
City
Charleston
State/Province
South Carolina
ZIP/Postal Code
29425
Country
United States
Facility Name
Cook Children's Medical Center - Jane and John Justin Neurosciences Center
City
Fort Worth
State/Province
Texas
ZIP/Postal Code
76104
Country
United States
Facility Name
Monash Children's Hospital
City
Clayton
State/Province
Victoria
ZIP/Postal Code
3168
Country
Australia
Facility Name
Austin Hospital
City
Heidelberg West
State/Province
Victoria
ZIP/Postal Code
3081
Country
Australia
Facility Name
Austin Hospital
City
Heidelberg
State/Province
Victoria
ZIP/Postal Code
3084
Country
Australia
Facility Name
Hospital for Sick Children
City
Toronto
State/Province
Ontario
ZIP/Postal Code
M5G 1X8
Country
Canada
Facility Name
Shenzhen Children's Hospital
City
Shenzhen
State/Province
Guangdong
ZIP/Postal Code
518026
Country
China
Facility Name
Children's Hospital of Fudan University
City
Shanghai
State/Province
Shanghai
ZIP/Postal Code
201102
Country
China
Facility Name
Peking University First Hospital
City
Beijing
ZIP/Postal Code
100034
Country
China
Facility Name
Beijing Children's Hospital,Capital Medical University
City
Beijing
ZIP/Postal Code
100045
Country
China
Facility Name
Beijing Children's Hospital,Capital Medical University
City
Beijing
ZIP/Postal Code
100069
Country
China
Facility Name
Xiangya Hospital Central South University
City
Changsha Shi
ZIP/Postal Code
410008
Country
China
Facility Name
Xiangya Hospital of Central South University
City
Changsha
ZIP/Postal Code
410008
Country
China
Facility Name
Tel Aviv Sourasky Medical Center PPDS
City
Tel Aviv
State/Province
Tel-Aviv
ZIP/Postal Code
64239
Country
Israel
Facility Name
Soroka University Medical Centre
City
Be'er Sheva
ZIP/Postal Code
84101
Country
Israel
Facility Name
Soroka University Medical Centre
City
Beer Sheva
ZIP/Postal Code
84101
Country
Israel
Facility Name
Bnai Zion Medical Center
City
Haifa
ZIP/Postal Code
31048
Country
Israel
Facility Name
Edith Wolfson Medical Center
City
Holon
ZIP/Postal Code
58100
Country
Israel
Facility Name
Schneider Children's Medical Center of Israel - Petah Tikvah - PIN
City
Petach Tikva
ZIP/Postal Code
49100
Country
Israel
Facility Name
Schneider Children's Medical Center of Israel - Petah Tikvah - PIN
City
Petah Tikva
ZIP/Postal Code
49100
Country
Israel
Facility Name
Sheba Medical Center - PPDS
City
Ramat-Gan
ZIP/Postal Code
52621
Country
Israel
Facility Name
Sheba Medical Center - PPDS
City
Ramat-Gan
ZIP/Postal Code
5265601
Country
Israel
Facility Name
Tel Aviv Sourasky Medical Center PPDS
City
Tel Aviv
ZIP/Postal Code
64239
Country
Israel
Facility Name
Uniwersyteckie Centrum Kliniczne
City
Gdansk
State/Province
Pomorskie
ZIP/Postal Code
80-952
Country
Poland
Facility Name
NZOZ Centrum Neurologii Dzieciecej i Leczenia Padaczki
City
Kielce
State/Province
Swietokrzyskie
ZIP/Postal Code
25-316
Country
Poland
Facility Name
Szpital Kliniczny im. H.Swiecickiego Uniwersytetu Medycznego im. Karola Marcinkowskiego w Poznaniu
City
Poznan
State/Province
Wielkopolskie
ZIP/Postal Code
60-355
Country
Poland
Facility Name
Centrum Medyczne Plejady
City
Krakow
ZIP/Postal Code
30-363
Country
Poland
Facility Name
Samodzielny Publiczny Dzieciecy Szpital Kliniczny w Warszawie
City
Warsaw
ZIP/Postal Code
02-091
Country
Poland
Facility Name
Samodzielny Publiczny Dzieciecy Szpital Kliniczny w Warszawie
City
Warszawa
ZIP/Postal Code
02-091
Country
Poland
Facility Name
Centro Hospitalar Lisboa Central- Hospital Dona Estefania
City
Lisboa
ZIP/Postal Code
1169-045
Country
Portugal
Facility Name
Centro Hospitalar Lisboa Norte, E.P.E. Hospital de Santa Maria
City
Lisboa
ZIP/Postal Code
1600-035
Country
Portugal
Facility Name
Centro Hospitalar Lisboa Norte, E.P.E. Hospital de Santa Maria
City
Lisboa
ZIP/Postal Code
1649-035
Country
Portugal
Facility Name
Hospital Ruber Internacional (Grupo Quironsalud)
City
Madrid
State/Province
Madrid, Communidad Delaware
ZIP/Postal Code
28034
Country
Spain
Facility Name
Clinica Universidad Navarra
City
Pamplona
State/Province
Navarra
ZIP/Postal Code
31008
Country
Spain
Facility Name
Hospital Vithas La Salud
City
Granada
ZIP/Postal Code
18008
Country
Spain
Facility Name
Hospital Ruber Internacional (Grupo Quironsalud)
City
Madrid
ZIP/Postal Code
28034
Country
Spain
Facility Name
Hospital Universitari i Politecnic La Fe de Valencia
City
Valencia
ZIP/Postal Code
46026
Country
Spain

12. IPD Sharing Statement

Plan to Share IPD
Yes
IPD Sharing Plan Description
Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.
IPD Sharing Access Criteria
IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.
IPD Sharing URL
https://vivli.org/ourmember/takeda/
Links:
URL
https://clinicaltrials.takeda.com/study-detail/517cf1e47a6a477f?idFilter=%5B%22TAK-935-18-001%22%5D
Description
To obtain more information on the study, click here/on this link

Learn more about this trial

A Study of Soticlestat in Adults and Children With Rare Epilepsies

We'll reach out to this number within 24 hrs