A Study of Soticlestat as an Add-on Therapy in Children and Young Adults With Dravet Syndrome

IDENTIFIERS
Trial Protocol: TAK-935-3001
Clinicaltrials.gov: NCT04940624
JapicCTI: jRCT2051210074
PURPOSE

The main aim of the study is to learn if soticlestat, when given as an add-on therapy, reduces the number of convulsive seizures in children and young adults with Dravet Syndrome. Participants will receive their standard anti-seizure therapy, plus either a tablet of soticlestat or placebo for 16 weeks. A placebo looks just like soticlestat but will not have any medicine in it. Participants may continue treatment in an extension study, based on the extension study's entry criteria. Those that want to stop treatment will have a gradual dose reduction during 1 week and then be followed up for 2 weeks.

Recruiting
US
AU
BE
14+
CA
CN
FR
GR
HU
IT
JP
LV
NL
PL
RU
RS
ES
UA

Trial at a glance

What medical condition is being studied?   Epilepsy, Dravet Syndrome (DS)
What is the trial testing?   Soticlestat, Placebo
Are placebos part of the trial?   Yes
How many participants are being enrolled?   142
When is the trial being conducted?    - 
How long is participation in the trial?   Each participant will be in this study for about 25 weeks.

Key requirements

Sex   
All
Age   
2 - 21 Years
Accepts Healthy Volunteers?   
No

Trial Details / Participation Requirements

CLICK TO EXPANDCLICK TO COLLAPSE
Trial Phase:   Phase 3
Trial Type:   Interventional
Trial Arms/Groups or Cohorts:   
Soticlestat: Participants weighing <45 kg: Soticlestat, mini-tablets, at the dose of 40 mg to 200 mg, orally or via gastrostomy tube (G-tube) or percutaneous endoscopic gastrostomy (PEG) tube, twice daily (BID) based on the body weight up to 4 weeks in Titration Period. Participants will continue to receive the dose that they are on at the end of the Titration Period, for 12 weeks in the Maintenance Period. The total duration of the treatment will be up to 16 weeks (Treatment Period). The dose will be tapered down if participants decide 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 in Titration Period. Participants will continue to receive 300 mg BID for 12 weeks in the Maintenance Period. The total duration of the treatment will be up to 16 weeks (Treatment Period). The dose will be tapered down if participants decide to discontinue the treatment.
Placebo: Soticlestat placebo-matching mini-tablets or tablets, orally or via G-tube or PEG tube, BID, up to 4 weeks in the Titration Period. Participants will continue to receive the soticlestat placebo-matching mini-tablets or tablets for 12 weeks in the Maintenance Period. The total duration of the treatment will be up to 16 weeks. Soticlestat matching tapering will be done to maintain the blind if participants decide to discontinue the treatment.
Intervention:   Soticlestat, Placebo
Primary Outcome Measure(s):   
Percent Change From Baseline in Convulsive Seizure Frequency per 28 days During the Full Treatment Period [Time Frame: Baseline up to Week 16]
Percent Change From Baseline in Convulsive Seizure Frequency per 28 days During the Maintenance Period (EMA Region Specific) [Time Frame: Baseline up to Week 16]
Secondary Outcome Measure(s):   
Percentage of Responders During Maintenance Period [Time Frame: Baseline up to Week 16]
Percentage of Responders During the Full Treatment Period [Time Frame: Baseline up to Week 16]
Percent Change From Baseline in Frequency of all Seizures per 28 Days During the Maintenance Period [Time Frame: Baseline up to Week 16]
Percent Change From Baseline in Frequency of all Seizures per 28 Days During the Full Treatment Period [Time Frame: Baseline up to Week 16]
Percent Change From Baseline in Convulsive Seizure Frequency per 28 days During the Maintenance Period [Time Frame: Baseline up to Week 16]
Percentage of Participants with ≤0%, >0% to ≤25%, >25% to ≤50%, >50% to ≤75%, and >75% to ≤100% Reduction in Convulsive Seizures in a Cumulative Response Curve [Time Frame: Baseline up to Week 16]
Change from Baseline in Percentage of Convulsive Seizure-free Days [Time Frame: Baseline up to Week 16]
Longest Convulsive Seizure-free Interval. [Time Frame: Baseline up to Week 16]
Number of Days When Rescue Antiseizure Medication (ASM) is Used [Time Frame: Baseline up to Week 16]
Clinical Global Impression of Improvement (CGI-I) Score [Time Frame: Baseline up to Week 16]
Caregiver Global Impression of Improvement (Care GI-I) Score [Time Frame: Baseline up to Week 16]
CGI-I Seizure Intensity and Duration Score [Time Frame: Baseline up to Week 16]
CGI-I Nonseizure Symptoms Score [Time Frame: Baseline up to Week 16]
Change in Quality of Life Inventory-Disability (QI-Disability) Score [Time Frame: Baseline up to Week 16]
Entry Criteria:
- Boys, girls, men, and women can take part. - Must be from 2 to 21 years old. - Must have a diagnosis of Dravet Syndrome. - Must have had 4 or more convulsive seizures every month for the last 3 months. - Must weigh 10 kilograms (kg) or more. - Must be receiving anti-seizure therapy. Additional entry criteria will be discussed with the study doctor.

Trial Locations

Any
Not yet recruiting
Recruiting
Any
RESULTS
Osaka City General Hospital

2-chōme-13-22 Miyakojimahondōri, Miyakojima Ward, Osaka, 534-0021, Japan

Recruiting
5,479 miles (8,817 km)  awayfrom
berlin, germany
View on Map
Phoenix Childrens Hospital

1919 E Thomas Rd, Phoenix, AZ 85016, USA

Will be Recruiting
5,648 miles (9,089 km)  awayfrom
berlin, germany
View on Map
University of California Benioff Children's Hospital

1975 4th St, San Francisco, CA 94158, USA

Will be Recruiting
5,657 miles (9,104 km)  awayfrom
berlin, germany
View on Map
Pediatric Neurology PA

1245 W Fairbanks Ave Suite 305, Winter Park, FL 32789, USA

Will be Recruiting
4,864 miles (7,827 km)  awayfrom
berlin, germany
View on Map
Clinical Integrative Research Center of Atlanta

5887 Glenridge Dr #140, Sandy Springs, GA 30328, USA

Will be Recruiting
4,698 miles (7,560 km)  awayfrom
berlin, germany
View on Map
University of Iowa Hospitals & Clinics - (CRS)

200 Hawkins Dr, Iowa City, IA 52242, USA

Will be Recruiting
4,544 miles (7,314 km)  awayfrom
berlin, germany
View on Map
NYU Comprehensive Epilepsy Center

223 E 34th St, New York, NY 10016, USA

Will be Recruiting
3,964 miles (6,379 km)  awayfrom
berlin, germany
View on Map
Medical University of South Carolina

171 Ashley Ave Suite 419, MSC, # 403, Charleston, SC 29425, USA

Will be Recruiting
4,589 miles (7,386 km)  awayfrom
berlin, germany
View on Map
Cook Children's Medical Center - Jane and John Justin Neurosciences Center

1500 Cooper St 4th Floor, Fort Worth, TX 76104, USA

Will be Recruiting
5,226 miles (8,411 km)  awayfrom
berlin, germany
View on Map
Seattle Children's Hospital

4800 Sand Point Way NE, Seattle, WA 98105, USA

Will be Recruiting
5,039 miles (8,110 km)  awayfrom
berlin, germany
View on Map
Sydney Children's Hospital

High St, Randwick NSW 2031, Australia

Will be Recruiting
10,005 miles (16,101 km)  awayfrom
berlin, germany
View on Map
Austin Hospital

145 Studley Rd, Heidelberg VIC 3084, Australia

Will be Recruiting
9,927 miles (15,976 km)  awayfrom
berlin, germany
View on Map
Alfred Hospital

Alfred Health, 55 Commercial Rd, Melbourne VIC 3004, Australia

Will be Recruiting
9,928 miles (15,978 km)  awayfrom
berlin, germany
View on Map
UZ Antwerpen

Drie Eikenstraat 655, 2650 Edegem, Belgium

Will be Recruiting
394 miles (634 km)  awayfrom
berlin, germany
View on Map
Hopital Universitaire des Enfants Reine Fabiola

Av. Jean Joseph Crocq 15, 1020 Bruxelles, Belgium

Will be Recruiting
403 miles (649 km)  awayfrom
berlin, germany
View on Map
Child and Family Research Institute

938 W 28th Ave, Vancouver, BC V5Z 4H4, Canada

Will be Recruiting
4,958 miles (7,980 km)  awayfrom
berlin, germany
View on Map
Hospital For Sick Children

555 University Ave, Toronto, ON M5G 1X8, Canada

Will be Recruiting
4,023 miles (6,474 km)  awayfrom
berlin, germany
View on Map
Peking University First Hospital

8 Xishiku St, Xicheng Qu, Beijing Shi, China

Will be Recruiting
4,570 miles (7,355 km)  awayfrom
berlin, germany
View on Map
Beijing Children's Hospital,Capital Medical University

Fengtai District, China

Will be Recruiting
4,573 miles (7,359 km)  awayfrom
berlin, germany
View on Map
Children's Hospital of Chongqing Medical University

136 Zhongshan 2nd Rd, Liang Lu Kou, Yuzhong Qu, Chongqing Shi, China

Will be Recruiting
4,751 miles (7,646 km)  awayfrom
berlin, germany
View on Map
The Second Affiliated Hospital of Guangzhou Medical Univeristy

Haizhu District, Guangzhou, Guangdong Province, China

Will be Recruiting
5,360 miles (8,627 km)  awayfrom
berlin, germany
View on Map
Guangzhou Women And Children's Medical Center

Guangzhou Women and Children's Medical Center station, Tianhe Qu, Guangzhou Shi, China

Will be Recruiting
5,360 miles (8,627 km)  awayfrom
berlin, germany
View on Map
Shenzhen Children's Hospital

Yitian Rd, Fu Tian C B D, Futian Qu, Shenzhen Shi, Guangdong Sheng, China

Will be Recruiting
5,421 miles (8,725 km)  awayfrom
berlin, germany
View on Map
Wuhan Childrens hospital

Huiji Rd, Jiangan Qu, Wuhan Shi, Hubei Sheng, China

Will be Recruiting
4,992 miles (8,033 km)  awayfrom
berlin, germany
View on Map
Xiangya Hospital Central South University

Wujialing Shangquan, Kaifu District, Changsha, Changsha, Hunan, China

Will be Recruiting
5,071 miles (8,161 km)  awayfrom
berlin, germany
View on Map
The First Hospital of Jilin University

China, Jilin Sheng, Changchun Shi, Chaoyang Qu, Mingde Rd, 明德街908 邮政编码: 130021

Will be Recruiting
4,649 miles (7,482 km)  awayfrom
berlin, germany
View on Map
Children's Hospital of Shanghai

Jing'an District, Shanghai, China

Will be Recruiting
5,218 miles (8,397 km)  awayfrom
berlin, germany
View on Map
Children's Hospital of Fudan University

399 Wanyuan Rd, Minhang Qu, Shanghai Shi, China

Will be Recruiting
5,221 miles (8,402 km)  awayfrom
berlin, germany
View on Map
Hopital Roger Salengro

Avenue du Professeur Emile Laine, 59037 Lille, France

Will be Recruiting
463 miles (746 km)  awayfrom
berlin, germany
View on Map
Hopital Necker - Enfants Malades

149 Rue de Sèvres, 75015 Paris, France

Will be Recruiting
546 miles (879 km)  awayfrom
berlin, germany
View on Map
Hopital Robert Debre

Hôpital Robert Debré, Bd Sérurier, 75019 Paris, France

Will be Recruiting
542 miles (872 km)  awayfrom
berlin, germany
View on Map
Childrens' Hospital of Athens 'P. and A. Kyriakou'

Levadias, Athina 115 27, Greece

Will be Recruiting
1,121 miles (1,804 km)  awayfrom
berlin, germany
View on Map
Attikon University General Hospital

Rimini 1, Chaidari 124 62, Greece

Will be Recruiting
1,117 miles (1,798 km)  awayfrom
berlin, germany
View on Map
University General Hospital of Larissa

Tsakalof 1, Larisa 412 21, Greece

Will be Recruiting
988 miles (1,589 km)  awayfrom
berlin, germany
View on Map
Hippokration Hospital

Konstantinoupoleos 49, Thessaloniki 546 42, Greece

Will be Recruiting
938 miles (1,510 km)  awayfrom
berlin, germany
View on Map
Orszagos Mentalis, Ideggyogyaszati es Idegsebeszeti Intezet

Budapest, Laky Adolf u. 44, 1145 Hungary

Will be Recruiting
428 miles (689 km)  awayfrom
berlin, germany
View on Map
ASST di Mantova - Azienda Ospedaliera Carlo Poma

Str. Lago Paiolo, 10, 46100 Mantova MN, Italy

Will be Recruiting
523 miles (841 km)  awayfrom
berlin, germany
View on Map
ASST di Pavia - Fondazione Istituto Neurologico Mondino IRCCS

Via Mondino, 2, 27100 Pavia PV, Italy

Will be Recruiting
541 miles (870 km)  awayfrom
berlin, germany
View on Map
Azienda Ospedaliero Universitaria A Meyer - INCIPIT - PIN

Viale Gaetano Pieraccini, 24, 50139 Firenze FI, Italy

Will be Recruiting
609 miles (981 km)  awayfrom
berlin, germany
View on Map
Fukuoka Children's Hospital

5-chōme-1-1 Kashiiteriha, Higashi Ward, Fukuoka, 813-0017, Japan

Will be Recruiting
5,382 miles (8,662 km)  awayfrom
berlin, germany
View on Map
Kumamoto-Ezuko Medical Center for The Severely Disabled

862-0947, Japan

Will be Recruiting
5,444 miles (8,761 km)  awayfrom
berlin, germany
View on Map
National Hospital Organization Nagasaki Medical Center

2-chōme-1001-1 Kubara, Ōmura, Nagasaki 856-0835, Japan

Will be Recruiting
5,412 miles (8,709 km)  awayfrom
berlin, germany
View on Map
National Hospital Organization Nishi-Niigata Chuo National Hospital

1-chōme-14-1 Masago, Nishi Ward, Niigata, 950-2085, Japan

Will be Recruiting
5,390 miles (8,674 km)  awayfrom
berlin, germany
View on Map
Okayama University Hospital

2-chōme-5-1 Shikatachō, Kita Ward, Okayama, 700-0914, Japan

Will be Recruiting
5,434 miles (8,745 km)  awayfrom
berlin, germany
View on Map
Yasuhara Childrens Clinic

Japan, 〒572-0085 Osaka, Neyagawa, Kōrishinmachi, 22−6 メディカルプラザ香里新町 3F

Will be Recruiting
5,477 miles (8,815 km)  awayfrom
berlin, germany
View on Map
Osaka University Hospital

2-15 Yamadaoka, Suita, Osaka 565-0871, Japan

Will be Recruiting
5,472 miles (8,807 km)  awayfrom
berlin, germany
View on Map
National Hospital Organization Shizuoka Institute of Epilepsy and Neurological Disorders

886 Urushiyama, Aoi Ward, Shizuoka, 420-8688, Japan

Will be Recruiting
5,544 miles (8,923 km)  awayfrom
berlin, germany
View on Map
National Center of Neurology and Psychiatry

4-chōme-1-1 Ogawahigashichō, Kodaira, Tokyo 187-8551, Japan

Will be Recruiting
5,532 miles (8,903 km)  awayfrom
berlin, germany
View on Map
Aichi Medical University Hospital

Japan, 〒480-1195 Aichi, Nagakute, Yazako, Karimata−1−1

Will be Recruiting
5,495 miles (8,843 km)  awayfrom
berlin, germany
View on Map
Childrens University Hospital

Vienības gat. 45, Zemgales priekšpilsēta, Rīga, LV-1004, Latvia

Will be Recruiting
524 miles (843 km)  awayfrom
berlin, germany
View on Map
Kempenhaeghe - PPDS

Sterkselseweg 65, 5591 VE Heeze, Netherlands

Will be Recruiting
341 miles (549 km)  awayfrom
berlin, germany
View on Map
Stichting Epilepsie Instellingen Nederland

Dokter Denekampweg 20, 8025 BV Zwolle, Netherlands

Will be Recruiting
305 miles (491 km)  awayfrom
berlin, germany
View on Map
Centrum Medyczne Plejady

Tadeusza Szafrana 5d, 30-363 Kraków, Poland

Will be Recruiting
331 miles (532 km)  awayfrom
berlin, germany
View on Map
Dzieciecy Szpital Kliniczny im. Jozefa Polikarpa Brudzinskiego w Warszawie

Żwirki i Wigury 63A, 02-091 Warszawa, Poland

Will be Recruiting
321 miles (517 km)  awayfrom
berlin, germany
View on Map
Uniwersyteckie Centrum Kliniczne

Dębinki 7, 80-952 Gdańsk, Poland

Will be Recruiting
251 miles (403 km)  awayfrom
berlin, germany
View on Map
Szpital Kliniczny im. H.Swiecickiego Uniwersytetu Medycznego im. Karola Marcinkowskiego w Poznaniu

Przybyszewskiego 49, 60-355 Poznań, Poland

Will be Recruiting
148 miles (238 km)  awayfrom
berlin, germany
View on Map
Russian National Research Medical University n.a. N.I.Pirogov

Ulitsa Ostrovityanova, 1, Moskva, Russia, 117997

Will be Recruiting
995 miles (1,602 km)  awayfrom
berlin, germany
View on Map
Russian National Research Medical University n.a. N.I.Pirogov

Ulitsa Ostrovityanova, 1, Moskva, Russia, 117997

Will be Recruiting
995 miles (1,602 km)  awayfrom
berlin, germany
View on Map
UGMK-Zdorojie, LLC

Ulitsa Sheynkmana, 113, Yekaterinburg, Sverdlovskaya oblast', Russia, 620144

Will be Recruiting
1,871 miles (3,011 km)  awayfrom
berlin, germany
View on Map
Krasnoyarsk State Medical University n.a. V.F. Voyno-Ysenetskiy

Partizana Zheleznyaka St, 1, Krasnoyarsk, Krasnoyarskiy kray, Russia, 660022

Will be Recruiting
3,037 miles (4,887 km)  awayfrom
berlin, germany
View on Map
Tyumen State Medical Academy

Одесская улица, 50, Tyumen, Tyumenskaya oblast', Russia, 625023

Will be Recruiting
2,049 miles (3,298 km)  awayfrom
berlin, germany
View on Map
Clinic for Neurology and Psychiatry for Children and Youth

Belgrade, Serbia

Will be Recruiting
622 miles (1,001 km)  awayfrom
berlin, germany
View on Map
Mother and Child Health Care Institute of Serbia Dr Vukan Cupic

Radoja Dakića 6-8, Beograd 11070, Serbia

Will be Recruiting
619 miles (997 km)  awayfrom
berlin, germany
View on Map
University Clinical Center Nis

Bulevar Dr Zorana Đinđića 48, Niš 18000, Serbia

Will be Recruiting
747 miles (1,203 km)  awayfrom
berlin, germany
View on Map
Children and Youth Health Care Institute of Vojvodina

Novi Sad, Serbia

Will be Recruiting
576 miles (927 km)  awayfrom
berlin, germany
View on Map
Clinica Universidad Navarra

Av. de Pío XII, 36, 31008 Pamplona, Navarra, Spain

Will be Recruiting
966 miles (1,555 km)  awayfrom
berlin, germany
View on Map
Hospital Universitario Vall d'Hebron - PPDS

Passeig de la Vall d'Hebron, 119, 08035 Barcelona, Spain

Will be Recruiting
929 miles (1,495 km)  awayfrom
berlin, germany
View on Map
Hospital Vithas La Salud

Hospital Vithas La Salud, Av. Santa María de la Alhambra, 6, 18008 Granada, Spain

Will be Recruiting
1,340 miles (2,157 km)  awayfrom
berlin, germany
View on Map
Hospital Universitari i Politecnic La Fe de Valencia

Avinguda de Fernando Abril Martorell, 106, 46026 València, Valencia, Spain

Will be Recruiting
1,115 miles (1,794 km)  awayfrom
berlin, germany
View on Map
Municipal Institution Dnipropetrovsk Regional Children Clinical Hospital of DRC

Karavajeva St, 68, Dnipropetrovs'k, Dnipropetrovs'ka oblast, Ukraine, 49000

Will be Recruiting
985 miles (1,586 km)  awayfrom
berlin, germany
View on Map
Communal Non-profit Enterprise City Childrens Clinical Hospital #5 of DCC

Dnipro, Dnipropetrovsk Oblast, Ukraine, 49000

Will be Recruiting
988 miles (1,590 km)  awayfrom
berlin, germany
View on Map
Communal Non-commercial Enterprise Iv-Frank Regional Childrens Clinical Hosp of Iv-Frank RC

Fed'kovycha St, 91, Ivano-Frankivs'k, Ivano-Frankivs'ka oblast, Ukraine, 76000

Will be Recruiting
552 miles (888 km)  awayfrom
berlin, germany
View on Map
CNPE Clinical Hospital Psychiatry of the Executive Body of the Kyiv City Council KCSA

Kyiv, Ukraine, 02000

Will be Recruiting
749 miles (1,206 km)  awayfrom
berlin, germany
View on Map
SI Ukr. Med. Rehabilitation Center For Children With Organic Injury of Nervous System of MoH of Ukr

Kyiv, Ukraine, 02000

Will be Recruiting
749 miles (1,206 km)  awayfrom
berlin, germany
View on Map
View all (74)
Trial Protocol ID: TAK-935-3001
Back to Trial Summary