An Efficacy and Safety Study of Clemizole HCl in Patients With Lennox-Gastaut Syndrome

Epygenix

Status and phase: Enrolling; Phase 3

Conditions: Lennox Gastaut Syndrome

Treatments: Drug: Clemizole HCl; Drug: Placebo

Study type: Interventional

Funder types: Industry

Identifiers

NCT05066217

EPX-100-003

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 HCL (EPX-100) as adjunctive therapy in children and adult participants with Lennox-Gastaut syndrome (LGS).

Full description

This is a multicenter, Phase 3, randomized, double-blind, placebo-controlled study designed to evaluate the efficacy and safety of clemizole HCl as adjunctive therapy in children and adult participants with LGS.

The study will consist of an Observational Period, a Double-Blind (DB) Period, and an optional Open-Label Extension (OLE) Period.

Enrollment: 260 estimated patients

Sex: All

Ages: 2 to 55 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Key Inclusion Criteria:

1. Males or females, ages ≥2 to ≤55 years, at the time of Screening.

2. Participant/parent/legal authorized representative (LAR) willing and able to give written informed consent/assent.

3. Diagnosis of LGS, including:

   • Evidence of at least one type of countable major motor seizure.
   • History of electroencephalogram (EEG) consistent with LGS (abnormal background activity, and one of the following: 1) slow spike-wave discharges [<2.5 Hz], or 2) paroxysmal fast activity during sleep).
   • Abnormal cognitive development.
   • Onset of seizures at 11 years of age or younger.

Key Exclusion Criteria:

1. Known sensitivity, allergy, or previous exposure to clemizole HCl.
2. Known history of long QT syndrome or any significant history of a serious abnormality of the electrocardiogram (ECG) (e.g., recent myocardial infarction, clinically significant arrhythmia).
3. Family history of sudden cardiac death, unexplained death, or death from a primary dysrhythmia potentially associated with QT prolongation in any family member.
4. Seizures secondary to illicit drug or alcohol use, infection, neoplasm, demyelinating disease, degenerative neurological disease, or progressive central nervous system disease, metabolic illness, recent anoxic episode within the last 6 months requiring resuscitation, or progressive degenerative disease or any other condition, which in the opinion of the investigator, could affect seizure control.
5. Epilepsy surgery planned during the study or epilepsy surgery within 6 months prior to Screening.
6. Concomitant use of fenfluramine.
7. Prior or concomitant use of lorcaserin.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Triple Blind

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260 participants in 3 patient groups, including a placebo group

Placebo

Placebo Comparator group

Description:

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

Treatment:

Drug: Placebo

Double-blind clemizole HCl

Experimental group

Description:

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

Treatment:

Drug: Clemizole HCl

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: Placebo

Drug: Clemizole HCl