A Multicenter, Randomized, Double-Blind, Placebo-Controlled, Parallel Group Study to Evaluate the Efficacy, Safety, and Tolerability of Soticlestat as Adjunctive Therapy in Pediatric and Adult Subjects With Lennox-Gastaut Syndrome (LGS)

Who is this study for? Patients with Lennox Gastaut Syndrome (LGS)
What treatments are being studied? Soticlestat
Status: Completed
Location: See all (98) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 3
SUMMARY

The aims of the study are: * to learn if soticlestat, when given as add-on therapy, reduces the number of major motor drop seizures in children, teenagers, and adults with Lennox-Gastaut Syndrome. * to assess the safety profile of soticlestat when given in combination with other therapies. Participants will receive their standard antiseizure therapy, plus either tablets of soticlestat or placebo. A placebo looks just like soticlestat but will not have any medicine in it. Participants will take soticlestat or placebo for 16 weeks, followed by a gradual dose reduction for 1 week. Then, participants will be followed up for 2 weeks.

Eligibility
Participation Requirements
Sex: All
Minimum Age: 2
Maximum Age: 55
Healthy Volunteers: f
View:

• Has documented clinical diagnosis of LGS.

• Has had ≥8 MMD seizures each month in the 3 months prior to Screening based on the historical information and has had ≥8 MMD seizures per 28 days during the 4 to 6 week prospective Baseline Period.

• Weighs ≥10 kg at the Screening Visit (Visit 1).

• Failure to control seizures despite appropriate trials of at least 1 ASM based on historical information, and is currently on an antiseizure therapy or other treatment options considered as standard of care (SOC).

• Artisanal cannabidiols are allowed at a stable dose for at least 4 weeks before the screening visit (Visit 1); the dosing regimen and manufacturer should remain constant throughout the study. (Artisanal cannabidiols will not be counted as ASMs.)

• Currently taking 0 to 3 ASMs at stable doses for at least 4 weeks before the Screening Visit (Visit 1); Fenfluramine and cannabidiol (Epidiolex) are allowed where available and counted as an ASM. ASM dosing regimen must remain constant throughout the study.

Locations
United States
Arizona
Phoenix Childrens Hospital
Phoenix
Center For Neurosciences
Tucson
California
David Geffen School of Medicine at UCLA
Los Angeles
University of California Benioff Children's Hospital
San Francisco
Colorado
Children's Hospital Colorado.
Denver
Florida
Pediatric Neurology PA
Winter Park
Georgia
Clinical Integrative Research Center of Atlanta
Atlanta
Iowa
University of Iowa Hospitals & Clinics - (CRS)
Iowa City
Maryland
Midatlantic Epilepsy and Sleep Center
Bethesda
Minnesota
Minnesota Epilepsy Group PA
Roseville
New Jersey
Institute of Neurology and Neurosurgery at Saint Barnabas, LLC
Livingston
New York
Northwell Health Physician Partners - Neurology at Lenox Hill
New York
NYU Comprehensive Epilepsy Center
New York
Premier Healthcare Inc.
New York
Oregon
Oregon Health and Science University
Portland
Pennsylvania
Children's Hospital of Philadelphia
Philadelphia
St. Christopher's Hospital for Children
Philadelphia
Thomas Jefferson University
Philadelphia
WellSpan Oncology Research
York
South Carolina
Medical University of South Carolina Children Hospital - PIN
Charleston
Texas
Cook Children's Medical Center - Jane and John Justin Neurosciences Center
Fort Worth
Utah
University of Utah - Primary Children's Hospital - PPDS
Salt Lake City
Washington
Seattle Children's Hospital
Seattle
MultiCare Institute for Research & Innovation (Tacoma)
Tacoma
Other Locations
Australia
Austin Hospital
Heidelberg
Alfred Hospital
Melbourne
Sydney Children's Hospital
Randwick
Queensland Childrens Hospital
South Brisbane
Belgium
Hopital Universitaire des Enfants Reine Fabiola
Brussels
UZ Antwerpen PIN
Edegem
Centre Neurologique William Lennox
Ottignies-louvain-la-neuve
Canada
Alberta Childrens Hospital
Calgary
Hospital For Sick Children
Toronto
Child and Family Research Institute
Vancouver
China
Beijing Children's Hospital,Capital Medical University
Beijing
Peking University First Hospital
Beijing
The First Hospital of Jilin University
Changchun
Xiangya Hospital Central South University
Changsha
Children's Hospital of Chongqing Medical University
Chongqing
Guangzhou Women And Children's Medical Center
Guangzhou
The Second Affiliated Hospital of Guangzhou Medical University
Guangzhou
Jiangxi Provincial Children's Hospital
Nanchang
Children's Hospital of Fudan University
Shanghai
Children's Hospital of Shanghai
Shanghai
Shenzhen Children's Hospital
Shenzhen
Wuhan Childrens hospital
Wuhan
France
CHRU Dijon Hopital General
Dijon
Hopital Roger Salengro
Lille
Hopitaux de La Timone
Marseille
Hopital Necker - Enfants Malades
Paris
Hopital Robert Debre
Paris
Germany
Krankenhaus Mara gGmbH - Epilepsiezentrum Bethel
Bielefeld
Klinikum der Johann-Wolfgang Goethe-Universitat
Frankfurt Am Main
Kleinwachau Sachsisches Epilepsiezentrum Radeberg Gemeinnutzige Gmbh
Radeberg
Schon Klinik Vogtareuth
Vogtareuth
Greece
Attikon University General Hospital
Chaidari
University General Hospital of Larissa
Larisa
Hippokration Hospital
Thessaloniki
Hungary
Bethesda Gyermekkorhaz
Budapest
Orszagos Mentalis, Ideggyogyaszati es Idegsebeszeti Intezet
Budapest
Szent Janos Korhaz es Eszak-Budai Egyesitett Korhazak
Budapest
Pecsi Tudomanyegyetem
Pecs
Italy
Ospedale Bellaria
Bologna
Azienda Ospedaliero Universitaria A Meyer - INCIPIT - PIN
Firenze
ASST di Pavia - Fondazione Istituto Neurologico Mondino IRCCS
Pavia
Fondazione Policlinico Universitario A Gemelli
Roma
Japan
Fukuoka Children's Hospital
Fukuoka-shi
National Center of Neurology and Psychiatry
Kodaira-shi
Kumamoto-Ezuko Medical Center for The Severely Disabled
Kumamoto-shi
Aichi Medical University Hospital
Nagakute-shi
Yasuhara Childrens Clinic
Neyagawa-shi
National Hospital Organization Nishi-Niigata Chuo National Hospital
Niigata-shi
Okayama University Hospital
Okayama-city
National Hospital Organization Nagasaki Medical Center
Omura-shi
Osaka City General Hospital
Osaka-shi
National Hospital Organization Shizuoka Institute of Epilepsy and Neurological Disorders
Shizuoka-shi
Osaka University Hospital
Suita-shi
Latvia
Childrens University Hospital
Riga
Netherlands
Kempenhaeghe - PPDS
Heeze
Stichting Epilepsie Instellingen Nederland
Zwolle
Poland
Uniwersyteckie Centrum Kliniczne
Gdansk
Centrum Medyczne Plejady
Krakow
Szpital Kliniczny im. H.Swiecickiego Uniwersytetu Medycznego im. Karola Marcinkowskiego w Poznaniu
Poznan
Russian Federation
UGMK-Zdorojie, LLC
Ekaterinburg
Serbia
Clinic for Neurology and Psychiatry for Children and Youth
Belgrade
Mother and Child Health Care Institute of Serbia Dr Vukan Cupic
Belgrade
University Clinical Center Nis
Nis
Children and Youth Health Care Institute of Vojvodina
Novi Sad
Spain
Hospital Universitario Vall d'Hebron - PPDS
Barcelona
Hospital Regional Universitario de Malaga Hospital General
Malaga
Clinica Universidad Navarra
Pamplona
Centro de Neurologia Avanzada
Sevilla
Hospital Universitari i Politecnic La Fe de Valencia
Valencia
Ukraine
Communal Non-profit Enterprise City Childrens Clinical Hospital #6 of DCC
Dnipro
Municipal Institution Dnipropetrovsk Regional Children Clinical Hospital of DRC
Dnipro
Communal Non-commercial Enterprise Iv-Frank Regional Childrens Clinical Hosp of Iv-Frank RC
Ivano-frankivsk
CNPE Clinical Hospital Psychiatry of the Executive Body of the Kyiv City Council KCSA
Kyiv
SI Ukr. Med. Rehabilitation Center For Children With Organic Injury of Nervous System of MoH of Ukr
Kyiv
Time Frame
Start Date: 2021-11-08
Completion Date: 2024-01-25
Participants
Target number of participants: 270
Treatments
Placebo_comparator: Placebo
Soticlestat placebo-matching mini-tablets or tablets, orally or via G-tube or MIC-KEY button or J-tube, BID, up to 4 weeks during titration. Participants continued to receive soticlestat placebo-matching mini-tablets or tablets for 12 weeks during maintenance. The total duration of the treatment was up to 16 weeks (Full Treatment Period). Soticlestat matching tapering was done to maintain the blind if participants decided to discontinue the treatment.
Experimental: Soticlestat
Participants weighing \<45 kg: Soticlestat, mini-tablets, at the dose of 40 mg to 200 mg, orally or via G-tube or MIC-KEY button or J-tube, BID based on body weight up to 4 weeks during titration. Participants continued to receive the dose that they were on at the end of the titration, for 12 weeks during maintenance. The total duration of the treatment was up to 16 weeks (Full Treatment Period) with the dose tapered down if participants decided to discontinue the treatment.~Participants weighing ≥45 kg: Soticlestat mini-tablets or tablets with a starting dose of 100 mg BID followed by 200 mg BID and, then 300 mg BID, up to 4 weeks during titration. Participants continued to receive 300 mg BID for 12 weeks during maintenance. The total duration of the treatment was up to 16 weeks (Full Treatment Period) with the dose tapered down if participants decided to discontinue the treatment.
Related Therapeutic Areas
Sponsors
Leads: Takeda

This content was sourced from clinicaltrials.gov

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