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Phase 2 N=141 Randomized Quadruple-blind Treatment

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

Epilepsy · Dravet Syndrome · Lennox-Gastaut Syndrome

Enrolled (actual)
141
Serious AEs
17.0%
Results posted
Feb 2021
Primary outcome: Primary: Percent Change From Baseline in Seizure Frequency Per 28 Days During the Maintenance Period — 3.11; -27.76 percent change — p=0.0007

Study Design & Population

Study type
Interventional
Phase
Phase 2
Interventions
TAK-935 (Drug); Placebo (Drug)
Age
Pediatric · 2+ yrs
Sex
All
Sponsor
Takeda
Primary completion
Jun 2020

Outcome Measures

OutcomeResultp-value
PRIMARY
Percent Change From Baseline in Seizure Frequency Per 28 Days During the Maintenance Period
3.11; -27.76 0.0007 sig
SECONDARY
Percent Change From Baseline in Seizure Frequency Per 28 Days During the Treatment Period
0.75; -30.05 0.0024 sig
SECONDARY
Percent Change From Baseline in Convulsive Seizure Frequency Per 28 Days in Participants With Dravet Syndrome Stratum During the Maintenance Period
9.38; -36.50 0.0001 sig
SECONDARY
Percent Change From Baseline in Drop Seizure Frequency Per 28 Days in Participants With the Lennox-Gastaut Syndrome (LGS) Stratum During the Maintenance Period
-1.90; -18.46 0.1470
SECONDARY
Percentage of Participants With LGS Stratum Considered Treatment Responders Throughout the Maintenance Period
29.7; 42.5; 16.2; 27.5; 2.7; 10.0
SECONDARY
Percentage of Participants With Dravet Syndrome Stratum Considered Treatment Responders Throughout the Maintenance Period
13.6; 66.7; 0; 41.7; 0; 20.8
SECONDARY
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
-0.3; -0.2 0.6829
SECONDARY
Percentage of Participants With Clinical Global Impression of Change (CGI-C) Responses as Per the Investigator Reported Impression of Efficacy and Tolerability TAK-935
12.2; 17.2; 2.0; 15.5; 85.7; 65.5
SECONDARY
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
2.0; 13.8; 12.2; 10.3; 18.4; 32.8
SECONDARY
Change From Baseline in Plasma 24S-Hydroxycholesterol (24HC) Levels in Participants Treated With TAK-935 as an Adjunctive Therapy
102.77; 102.20; -0.10
SECONDARY
Change From Baseline in Seizure Frequency in Participants Treated With TAK-935 as an Adjunctive Therapy
31.00; 32.12; 0.30; -6.29

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.

Eligibility Criteria

Inclusion Criteria

  • Male and female participants aged greater than or equal to (>=) 2 and less than or equal to ( =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
View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT03650452). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.

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