This is a multicenter, Phase 3, randomized, double-blind, placebo-controlled study designed to evaluate the efficacy and safety of clemizole hydrochloride (EPX-100) as adjunctive therapy in children and adult participants with Dravet syndrome (DS).
This is a global, multicenter, randomized, double-blind, placebo-controlled study to evaluate the safety and efficacy of clemizole hydrochloride as adjunctive therapy in children and adult participants with DS. The study consists of a 4-week Observational Period, a 16-week Double-Blind (DB) Period and an Open-Label Extension (OLE) Period for up to 156 weeks.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
TRIPLE
Enrollment
150
Clemizole HCl will be administered as an oral solution.
Placebo will be administered as an oral solution.
Percent Change in Countable Motor Seizures Per 28 Days (CMS-28) in the Titration Plus Maintenance Periods Relative to Baseline
Percent change in CMS-28 from the Baseline Period through the end of the DB period.
Time frame: From Baseline Period (Day 1) up to 16 weeks
European Union: Percent Change in Countable Motor Seizures Per 28 Days in the Maintenance Period Relative to Baseline
Percent change in CMS-28 from the Baseline Period through the end of the maintenance period.
Time frame: From maintenance period Baseline (Day 29) up to Day 85
Proportion of Participants with >=50% reduction in Countable Motor Seizures Per 28 Days in the Titration Plus Maintenance Periods Relative to Baseline
Proportion of participants with \>=50% reduction in CMS-28 from the Baseline Period through the end of the DB Period.
Time frame: From Baseline Period (Day 1) up to 16 weeks
European Union: Proportion of Participants with >=50% reduction in Countable Motor Seizures Per 28 Days in the Maintenance Period Relative to Baseline
Proportion of participants with \>=50% reduction in CMS-28 from the Baseline Period through the end of the maintenance period.
Time frame: From maintenance period Baseline (Day 29) up to Day 85
Number of Countable Motor Seizure-free Days in the Titration Plus Maintenance Periods Relative to Baseline
Number of countable motor seizure-free days from the Baseline Period through the end of the DB Period.
Time frame: From Baseline Period (Day 1) up to 16 weeks
European Union: Number of Countable Motor Seizure-free Days in the Maintenance Period Relative to Baseline
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Children's Hospital of Los Angeles
Los Angeles, California, United States
TERMINATEDUniversity of California Irvine
Orange, California, United States
RECRUITINGUCSF Medical Center
San Francisco, California, United States
RECRUITINGYale University School of Medicine
New Haven, Connecticut, United States
NOT_YET_RECRUITINGThe Nemours Foundation
Wilmington, Delaware, United States
RECRUITINGRare Disease Research FL
Kissimmee, Florida, United States
NOT_YET_RECRUITINGPediatric Neurology and Epilepsy Specialists
Winter Park, Florida, United States
NOT_YET_RECRUITINGClinical Integrative Research Center of Atlanta (CIRCA)
Atlanta, Georgia, United States
NOT_YET_RECRUITINGAnn & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States
WITHDRAWNNorton Children's Research Institute
Louisville, Kentucky, United States
NOT_YET_RECRUITING...and 36 more locations
Number of countable motor seizure-free days from the Baseline Period through the end of the maintenance period.
Time frame: From maintenance period Baseline (Day 29) up to Day 85
Clinical Global Impression of Improvement - Clinician (CGII-C) Score
CGII-C score at the end of the maintenance Period. This 1-item scale asks the clinician to rate how the participant's symptoms have improved or worsened relative to baseline.
Time frame: Day 85
Clinical Global Impression of Improvement - Participant/Caregiver (CGII-P) Score
CGII-C score at the end of the maintenance Period. This 1-item scale asks the clinician to rate how the participant's symptoms have improved or worsened relative to baseline.
Time frame: Day 85
Percent Change in All Seizures in the Titration Plus Maintenance Periods Relative to Baseline
Percent change in all seizures at the end of the DB Period.
Time frame: From Baseline Period (Day 1) up to 16 weeks
Percent Change in All Seizures in the Maintenance Period Relative to Baseline
Percent change in all seizures at the end of the maintenance period.
Time frame: From maintenance period Baseline (Day 29) up to Day 85
Incidence of Rescue Anti-epileptic Drug (AED) Use in the Titration Plus Maintenance Periods Relative to Baseline
Incidence of rescue AED use as measured by the number of days on rescue AEDs from the Baseline Period through the end of the DB Period.
Time frame: From Baseline Period (Day 1) up to 16 weeks
Incidence of Rescue Anti-epileptic Drug Use in the Maintenance Period Relative to Baseline
Incidence of rescue AED use as measured by the number of days on rescue AEDs from the Baseline Period through the end of the maintenance period.
Time frame: From maintenance period Baseline (Day 29) up to Day 85
United States FDA: Proportion of Participants with >=50% Reduction in the Countable Motor Seizures Per 28 Days in the Maintenance Period Relative to Baseline
Proportion of participants with ≥50% reduction in CMS-28 from the Baseline Period through the end of the DB Maintenance Phase only.
Time frame: From maintenance period Baseline (Day 29) up to Day 85
Incidence of Treatment-Emergent Adverse Events (TEAEs)
Incidence of TEAEs will be compared among the treatment groups.
Time frame: From the first dose administration of study drug up to end of the study, approximately up to 172 weeks