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Completed

NCT Number: NCT03650452

A Phase 2, Multicenter, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy, Safety, and Tolerability of TAK-935 (OV935) as an Adjunctive Therapy in Pediatric Participants With Developmental and/or Epileptic Encephalopathies

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.

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Key information

Conditions

Epilepsy Brain Diseases Brain Ischemia CDKL5 deficiency disorder Cardiovascular Diseases Central Nervous System Diseases Cerebrovascular Disorders Congenital Abnormalities Congenital, Hereditary, and Neonatal Diseases and Abnormalities Dravet Syndrome Epilepsies, Myoclonic Epilepsy, Generalized Epileptic Syndromes Genetic Diseases, Inborn Hamartoma Heredodegenerative Disorders, Nervous System Hypoxia Hypoxia, Brain Hypoxia-Ischemia, Brain Lennox Gastaut Syndrome Lennox-Gastaut Syndrome Malformations of Cortical Development Malformations of Cortical Development, Group I Neoplasms Neoplasms, Multiple Primary Neoplastic Syndromes, Hereditary Nervous System Diseases Nervous System Malformations Neurocutaneous Syndromes Neurodegenerative Diseases Neurologic Manifestations Pathological Conditions, Signs and Symptoms Seizures Signs and Symptoms Signs and Symptoms, Respiratory Spasms, Infantile Tuberous Sclerosis Vascular Diseases

Age range

2 year–17 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Monash Children's Hospital, Clayton, Victoria, Australia

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About this study

The drug being tested in this study is called TAK-935 (OV935). This randomized, double-blind study will assess the effects of TAK-935 (OV935), compared to placebo, on efficacy, safety, and tolerability in pediatric participants with Dravet syndrome (DS) or Lennox Gastaut syndrome (LGS). This multi-center trial will be conducted worldwide and will enroll approximately 126 participants.

Participants will be randomized based on their diagnosis in 2 categories; DS or LGS. The study will consist of 2 periods: Screening Period and Treatment Period. The overall duration of Treatment Period is up to 20 weeks including 8-week Dose Optimization Period and 12-week Maintenance Period. The overall time to participants in this study is approximately 30 weeks.

Participants completing this study will have an option to enroll in the open-label extension study, under a separate protocol.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • 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

Exclusion criteria

  • 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

Treatment and study plan

TAK-935

Drug

TAK-935 tablets or mini-tablets.

Placebo

Drug

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

Primary outcomes

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

    Time frame: Baseline; Maintenance Period: Weeks 9 to 20

    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.

Secondary outcomes

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

    Time frame: Baseline; Treatment Period: Weeks 0 to 20

    Seizure Frequency per 28 days is defined as total number of Seizures reported (convulsive seizures for DS, drop seizures for LGS) 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 treatment 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.

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

    Time frame: Baseline; Maintenance Period: Weeks 9 to 20

    Convulsive seizure frequency per 28 days is defined as total number of convulsive seizures 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 [(Maintenance Period Convulsive Seizure Frequency - Baseline Period Convulsive Seizure Frequency) divided by Baseline Convulsive Seizure Frequency] multiplied by 100. Negative percent change from Baseline indicates improvement.

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

    Time frame: Baseline; Maintenance Period: Weeks 9 to 20

    Drop seizure frequency per 28 days is defined as total number of drop seizures 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 [(Maintenance Period Drop Seizure Frequency - Baseline Period Drop Seizure Frequency) divided by Baseline Drop Seizure Frequency] multiplied by 100. Negative percent change from Baseline indicates improvement.

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

    Time frame: Maintenance Period: Weeks 9 to 20

    Responders are defined as having over 50% drop seizure reduction compared to Baseline. Percent Reduction from Baseline (%) is defined as [(Maintenance Period Drop Seizure Frequency - Baseline Period Drop Seizure Frequency) divided by Baseline Drop Seizure Frequency] multiplied by 100. Data is reported as reduction of 25%, 50%, 75% and 100% or more in drop seizures from Baseline.

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

    Time frame: Maintenance Period: Weeks 9 to 20

    Responders are defined as having over 50% convulsive seizure reduction compared to Baseline. Percent Reduction from Baseline (%) is defined as [(Maintenance Period Convulsive Seizure Frequency - Baseline Period Convulsive Seizure Frequency) divided by Baseline Convulsive Seizure Frequency] multiplied by 100. Data is reported as reduction of 25%, 50%, 75% and 100% or more in drop seizures from Baseline.

  6. Change From Baseline in Clinician's Clinical Global Impression of Severity (CGI-S) Responses of Investigator Reported Impression of Efficacy and Tolerability of Study Drug

    Time frame: Baseline and Week 20

    The CGI-Severity (CGI-S) focuses on clinicians' observations of the participant's cognitive, functional, and behavioral performance since the beginning of the study. 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 (amongst the most severely ill participants). A negative change from Baseline indicates improvement.

  7. Percentage of Participants With Clinical Global Impression of Change (CGI-C) Responses as Per the Investigator Reported Impression of Efficacy and Tolerability TAK-935

    Time frame: Week 20

    CGI-Change (CGI-C) treatment response ratings should take account of both therapeutic efficacy and treatment-related AEs. Each component of the CGI is rated separately; the instrument does not yield a global score. The CGI-C is rated on a 7-point scale, where, 0 = Marked improvement and no side-effects, 1 = Marked improvement and minimal side-effects, 2 = No Change, 3 = Minimal improvement and marked side-effects and 4 = Unchanged or worse and side-effects outweigh the therapeutic effect. Lower scores indicated improvement.

  8. Percentage of Participants With Caregiver Global Impression of Change (Care GI-C) Responses as Per the Parent/Family Reported Impression of Efficacy and Tolerability of TAK-935

    Time frame: Week 20

    The Care GI-C is rated on a 7-point scale, with the severity of illness scale where, 1 = Very much improved, 2 = Much improved, 3 = Slightly improved, 4 = No change, 5 = Slightly worse, 6 = Much worse and 7 = Very much worse. Lower scores indicated improvement.

  9. Change From Baseline in Plasma 24S-Hydroxycholesterol (24HC) Levels in Participants Treated With TAK-935 as an Adjunctive Therapy

    Time frame: Baseline and Week 24

    A negative change from Baseline indicates improvement.

  10. Change From Baseline in Seizure Frequency in Participants Treated With TAK-935 as an Adjunctive Therapy

    Time frame: Baseline and Week 20

    Seizure frequency was based on convulsive seizures for the participants in the Dravet Syndrome Indication and Drop Seizures for the participants in the LGS Indication. Seizure frequency per 28 days = (total number of seizures reported during the period) / (number of days during the period seizures were assessed) * 28. A negative change from Baseline indicates improvement.

Sponsors and collaborators

Lead sponsor

Takeda

Industry

Collaborators

  • Healx AI

Registry information

Official study title

A Phase 2, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy, Safety, and Tolerability of TAK-935 (OV935) as an Adjunctive Therapy in Pediatric Patients With Developmental and/or Epileptic Encephalopathies

Acronym: ELEKTRA

Important dates

Study start
2018
Primary completion
2020
Study completion
2020
First posted
Aug 28, 2018
Registry last updated
Feb 18, 2021

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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