A Study of EPX-100 (Clemizole Hydrochloride) in Participants With Dravet Syndrome
ConditionDravet Syndrome
Trial statusRecruiting
Trial phasePhase 3
Trial typeInterventional
Biological sexAll
Age2+
SponsorEpygenix
About this trial
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).
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Male and female participants 2 years and older at time of consent.
Participant or parent/legally authorized representative (LAR) willing and able to provide written informed consent and assent (if applicable) prior to initiation of any study related procedures.
Onset of seizures prior to 18 months of age,
Normal development at onset,
Disqualifiers
Known sensitivity, allergy, or previous exposure to clemizole HCl.
Exposure to any investigational drug or device <90 days prior to screening or plans to participate in another drug or device trial at any time during the study.
Seizures secondary to illicit drug (this includes concomitant use of tetrahydrocannabinol [THC] and nonprescription cannabidiol preparations) or alcohol use, infection, neoplasm, demyelinating disease, degenerative neurological disease, or central nervous system disease deemed progressive, metabolic illness, or progressive degenerative disease.
Concurrent use of lorcaserin. Note: Prior use of lorcaserin is permitted if at least 30 days have passed since the last dose.
Trial design
Design model
Parallel
Treatments tested in this trial
Clemizole HCl
DrugClemizole HCl will be administered as an oral solution.
Placebo
DrugPlacebo will be administered as an oral solution.
Treatment groups
150 Participants
are divided into 3 treatment groupsGroup A: Double-blind clemizole HClExperimental treatment 1 intervention
Group B: PlaceboPlacebo comparator 1 intervention
Group C: Open-label clemizole HClExperimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
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 weeks2
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 85Secondary outcomes
1
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 weeks2
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 853
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 weeks4
European Union: Number of Countable Motor Seizure-free Days in the Maintenance Period Relative to Baseline
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 85Sponsors and contacts
Click on the lead sponsor to view all of their trials.
Epygenix
Lead sponsor
Harmony Biosciences Management, Inc.
Collaborator
Source ClinicalTrials.gov