A Study of EPX-100 (Clemizole Hydrochloride) in Participants With Dravet Syndrome (ARGUS)

Epygenix

Status and phase: Enrolling; Phase 3

Conditions: Dravet Syndrome

Treatments: Drug: Placebo; Drug: Clemizole HCl

Study type: Interventional

Funder types: Industry

Identifiers

NCT04462770

EPX-100-001

2024-518628-57-00 (EU Trial (CTIS) Number)

Details and patient eligibility

About

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).

Full description

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.

Enrollment: 150 estimated patients

Sex: All

Ages: 2+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Key Inclusion Criteria:

1. Male and female participants 2 years and older at time of consent.

2. Participant or parent/legally authorized representative (LAR) willing and able to provide written informed consent, assent (if applicable) prior to initiation of any study related procedures.

3. Clinical diagnosis of DS. Participants must have seizures which are not completely controlled by AEDs with the following criteria:

   • Onset of seizures prior to 18 months of age,
   • Normal development at onset,
   • History of at least one type of countable motor seizure (CMS),
   • Brain MRI without cortical malformation (not including mild atrophy associated with the natural progression of DS),
   • Genetic mutation of the SCN1A gene must be documented.

Key Exclusion Criteria:

1. Known sensitivity, allergy, or previous exposure to clemizole HCl.
2. 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.
3. 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.
4. Concurrent use of lorcaserin. Note: Prior use of lorcaserin is permitted if at least 30 days have passed since the last dose.
5. Concurrent use of fenfluramine.
6. Epilepsy surgery planned during the study or epilepsy surgery within 6 months prior to Screening.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Triple Blind

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150 participants in 3 patient groups, including a placebo group

Double-blind clemizole HCl

Experimental group

Description:

Participants will receive their first dose of study drug following randomization.

Treatment:

Drug: Clemizole HCl

Placebo

Placebo Comparator group

Description:

Participants will receive their first dose of study drug following randomization.

Treatment:

Drug: Placebo

Open-label clemizole HCl

Experimental group

Description:

Eligible participants who complete the DB Period will have the option to continue in the OLE Period, during which they will receive clemizole HCL for up to 3 years.

Treatment:

Drug: Clemizole HCl

Drug: Placebo