NCT03650452

Brief Summary

The purpose of this study is to investigate the effect on the frequency of all seizures (convulsive and drop) in participants treated with TAK-935 compared to placebo.

Trial Health

93
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
141

participants targeted

Target at P75+ for phase_2

Timeline
Completed

Started Aug 2018

Geographic Reach
8 countries

44 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

August 8, 2018

Completed
19 days until next milestone

First Submitted

Initial submission to the registry

August 27, 2018

Completed
1 day until next milestone

First Posted

Study publicly available on registry

August 28, 2018

Completed
1.8 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

June 9, 2020

Completed
1 month until next milestone

Study Completion

Last participant's last visit for all outcomes

July 20, 2020

Completed
7 months until next milestone

Results Posted

Study results publicly available

February 18, 2021

Completed
Last Updated

February 18, 2021

Status Verified

January 1, 2021

Enrollment Period

1.8 years

First QC Date

August 27, 2018

Results QC Date

January 19, 2021

Last Update Submit

February 9, 2021

Conditions

Keywords

Drug TherapyBrain DiseasesCentral Nervous System DiseasesTuberous SclerosisCDKL5 deficiency disorderDup15Q syndromeAnoxic brain injuryInfantile spamsWest syndromeCortical dysplasiaSCN1AOV-935Cholesterol 24S-hydroxylase inhibitorSeizureAnti-epileptic drugAnticonvulsantsNervous System DiseasesDrop seizureAtonic seizure

Outcome Measures

Primary Outcomes (1)

  • Percent Change From Baseline in Seizure Frequency Per 28 Days During the Maintenance Period

    Seizure frequency per 28 days is defined as total number of seizures (convulsive seizures for DS, drop seizures for LGS) reported during the period divided by number of days during the period seizures were assessed multiplied by 28. Percent change from Baseline is defined as (frequency of seizures per 28 days during maintenance period - frequency of seizures per 28 days at baseline) divided by frequency of seizures per 28 days at baseline multiplied by 100. Negative percent change from Baseline indicates improvement.

    Baseline; Maintenance Period: Weeks 9 to 20

Secondary Outcomes (10)

  • Percent Change From Baseline in Seizure Frequency Per 28 Days During the Treatment Period

    Baseline; Treatment Period: Weeks 0 to 20

  • Percent Change From Baseline in Convulsive Seizure Frequency Per 28 Days in Participants With Dravet Syndrome Stratum During the Maintenance Period

    Baseline; Maintenance Period: Weeks 9 to 20

  • Percent Change From Baseline in Drop Seizure Frequency Per 28 Days in Participants With the Lennox-Gastaut Syndrome (LGS) Stratum During the Maintenance Period

    Baseline; Maintenance Period: Weeks 9 to 20

  • Percentage of Participants With LGS Stratum Considered Treatment Responders Throughout the Maintenance Period

    Maintenance Period: Weeks 9 to 20

  • Percentage of Participants With Dravet Syndrome Stratum Considered Treatment Responders Throughout the Maintenance Period

    Maintenance Period: Weeks 9 to 20

  • +5 more secondary outcomes

Study Arms (2)

Placebo

PLACEBO COMPARATOR

TAK-935 placebo-matching tablets, orally or via gastrostomy tube (G-tube)/percutaneous endoscopic gastrostomy (PEG), twice a day (BID) up to Week 20.

Drug: Placebo

TAK-935

EXPERIMENTAL

TAK-935 tablets orally or via G-tube/PEG tube, BID. Participants weighing \<60 kg received total daily dose of study drug calculated based on body weight. Participants weighing ≥60 kg at Baseline, were administered with 200 mg/day followed by 400 mg/day, then 600 mg/day, up to Week 20.

Drug: TAK-935

Interventions

TAK-935 tablets or mini-tablets.

TAK-935

TAK-935 placebo-matching tablets or mini-tablets.

Placebo

Eligibility Criteria

Age2 Years - 17 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17)

You may qualify if:

  • Male and female participants aged greater than or equal to (\>=) 2 and less than or equal to (\<=) 17 years
  • Clinical diagnosis of DS or LGS
  • Weight of \>=10 kilogram (kg) at the Screening visit
  • Currently taking 1 to 4 anti-epileptic drugs (AEDs) at a stable dose
  • Failed to become and remain seizure free with trials of at least 2 AEDs

You may not qualify if:

  • Has been admitted to a medical facility and intubated for treatment of status epilepticus 2 or more times in the 3 months immediately prior to the screening visit
  • Non-epileptic events that cannot be reliably distinguished from epileptic seizures
  • Participation in a clinical study involving another study drug in the previous month

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (44)

Phoenix Children's Hospital

Phoenix, Arizona, 85016, United States

Location

Children's Hospital Los Angeles

Los Angeles, California, 90027, United States

Location

Colorado Children's Hospital

Aurora, Colorado, 80045, United States

Location

Nicklaus Children's Hospital

Miami, Florida, 33155, United States

Location

Pediatric Neurology PA

Orlando, Florida, 32819, United States

Location

Rare Disease Research, LLC

Atlanta, Georgia, 30318, United States

Location

Center for Rare Neurological Diseases

Norcross, Georgia, 30093, United States

Location

Ann and Robert H Lurie Childrens Hospital of Chicago

Chicago, Illinois, 60611, United States

Location

Mayo Clinic - PPDS

Rochester, Minnesota, 55905, United States

Location

Northeast Regional Epilepsy Group

Hackensack, New Jersey, 07601, United States

Location

Children's Hospital at Saint Peter's University Hospital

New Brunswick, New Jersey, 08901, United States

Location

Columbia University Medical Center

New York, New York, 10032, United States

Location

Wake Forest Baptist Medical Center

Winston-Salem, North Carolina, 27157, United States

Location

Medical University of South Carolina

Charleston, South Carolina, 29425, United States

Location

Cook Children's Medical Center

Fort Worth, Texas, 76104, United States

Location

Monash Children's Hospital

Clayton, Victoria, 3168, Australia

Location

Austin Hospital

Heidelberg West, Victoria, 3081, Australia

Location

Hospital For Sick Children

Toronto, Ontario, M5G 1X8, Canada

Location

Peking University First Hospital

Beijing, 100034, China

Location

Capital Medical University (CMU) - Beijing Children's Hospital

Beijing, 100045, China

Location

Beijing Children's Hospital,Capital Medical University

Beijing, 100069, China

Location

Xiangya Hospital Central South University

Changsha, 410078, China

Location

Children's Hospital of Fudan University

Shanghai, 201102, China

Location

Shenzhen Children's Hospital

Shenzhen, 518026, China

Location

Sheba Medical Center-PPDS

Tel Litwinsky, Ramat Gan, 52621, Israel

Location

Soroka University Medical Centre

Bear Sheva, 84101, Israel

Location

Bnai Zion Medical Center

Haifa, 31048, Israel

Location

Edith Wolfson Medical Center

Holon, 58100, Israel

Location

Hadassah Medical Center

Jerusalem, 91120, Israel

Location

Schneider Childrens Medical Center of Israel

Petah Tikva, 49202, Israel

Location

Tel Aviv Sourasky Medical Center

Tel Aviv, 64239, Israel

Location

Szpital Kliniczny im. H.Swiecickiego Uniwersytetu Medycznego im. Karola Marcinkowskiego w Poznaniu

Poznan, Greater Poland Voivodeship, 60-355, Poland

Location

Uniwersyteckie Centrum Kliniczne - PPDS

Gdansk, Pomeranian Voivodeship, 80-211, Poland

Location

Centrum Medyczne Plejady

Krakow, 30-363, Poland

Location

Samodzielny Publiczny Dzieciecy Szpital Kliniczny w Warszawie

Warsaw, 02-091, Poland

Location

Instytut Pomnik Centrum Zdrowia Dziecka

Warsaw, 04-730, Poland

Location

NZOZ Centrum Neurologii Dzieciecej i Leczenia Padaczki

Kielce, Świętokrzyskie Voivodeship, 25-316, Poland

Location

Centro Hospitalar Lisboa Central- Hospital Dona Estefania

Lisbon, 1169-045, Portugal

Location

Centro Hospitalar Lisboa Norte, E.P.E. Hospital de Santa Maria

Lisbon, 1649-035, Portugal

Location

Largo da Maternidade de Julio DinisCentro Materno Infantil do Norte

Porto, 4050-651, Portugal

Location

Clinica Universidad Navarra

Pamplona, Navarre, 31008, Spain

Location

Hospital Vithas La Salud

Granada, 18008, Spain

Location

Hospital Ruber Internacional

Madrid, 28034, Spain

Location

Hospital Universitari i Politecnic La Fe de Valencia

Valencia, 46026, Spain

Location

Related Publications (1)

  • Hahn CD, Jiang Y, Villanueva V, Zolnowska M, Arkilo D, Hsiao S, Asgharnejad M, Dlugos D. A phase 2, randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of soticlestat as adjunctive therapy in pediatric patients with Dravet syndrome or Lennox-Gastaut syndrome (ELEKTRA). Epilepsia. 2022 Oct;63(10):2671-2683. doi: 10.1111/epi.17367. Epub 2022 Aug 4.

MeSH Terms

Conditions

EpilepsyEpilepsies, MyoclonicLennox Gastaut SyndromeBrain DiseasesCentral Nervous System DiseasesTuberous SclerosisCDKL5 deficiency disorderHypoxia-Ischemia, BrainSpasms, InfantileMalformations of Cortical DevelopmentSeizuresNervous System Diseases

Condition Hierarchy (Ancestors)

Epilepsy, GeneralizedEpileptic SyndromesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesHamartomaNeoplasmsNeoplasms, Multiple PrimaryNeoplastic Syndromes, HereditaryMalformations of Cortical Development, Group INervous System MalformationsNeurocutaneous SyndromesHeredodegenerative Disorders, Nervous SystemNeurodegenerative DiseasesCongenital AbnormalitiesBrain IschemiaCerebrovascular DisordersHypoxia, BrainVascular DiseasesCardiovascular DiseasesHypoxiaSigns and Symptoms, RespiratorySigns and SymptomsPathological Conditions, Signs and SymptomsNeurologic Manifestations

Results Point of Contact

Title
Medical Director
Organization
Takeda

Study Officials

  • Medical Director Clinical Science

    Takeda

    STUDY DIRECTOR

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
OTHER
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

August 27, 2018

First Posted

August 28, 2018

Study Start

August 8, 2018

Primary Completion

June 9, 2020

Study Completion

July 20, 2020

Last Updated

February 18, 2021

Results First Posted

February 18, 2021

Record last verified: 2021-01

Data Sharing

IPD Sharing
Will share

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.

Shared Documents
STUDY PROTOCOL, SAP, ICF, CSR
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.
More information

Locations