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

NCT ID: NCT05066217

Last Updated: 2025-12-09

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE3

Total Enrollment

260 participants

Study Classification

INTERVENTIONAL

Study Start Date

2025-04-09

Study Completion Date

2029-11-01

Brief Summary

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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).

Detailed Description

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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.

Conditions

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Lennox Gastaut Syndrome

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Patients are randomized 1:1 to clemizole HCl (EPX-100) or placebo.
Primary Study Purpose

TREATMENT

Blinding Strategy

TRIPLE

Participants Caregivers Investigators

Study Groups

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Placebo

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

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Placebo will be administered as an oral solution.

Double-blind clemizole HCl

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

Group Type EXPERIMENTAL

Clemizole HCl

Intervention Type DRUG

Clemizole HCl will be administered as an oral solution.

Open-label clemizole HCl

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.

Group Type EXPERIMENTAL

Clemizole HCl

Intervention Type DRUG

Clemizole HCl will be administered as an oral solution.

Placebo

Intervention Type DRUG

Placebo will be administered as an oral solution.

Interventions

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Clemizole HCl

Clemizole HCl will be administered as an oral solution.

Intervention Type DRUG

Placebo

Placebo will be administered as an oral solution.

Intervention Type DRUG

Other Intervention Names

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EPX-100

Eligibility Criteria

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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.

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.
Minimum Eligible Age

2 Years

Maximum Eligible Age

55 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Harmony Biosciences Management, Inc.

INDUSTRY

Sponsor Role collaborator

Epygenix

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Amit Ray, MD

Role: STUDY_DIRECTOR

Harmony Biosciences Management, Inc.

Locations

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Rare Disease Research

Kissimmee, Florida, United States

Site Status RECRUITING

Minnesota Epilepsy Group, P.A.

Roseville, Minnesota, United States

Site Status RECRUITING

Neurology Center for Epilepsy and Seizures

Marlboro, New Jersey, United States

Site Status RECRUITING

On-Site Clinical Solution

Charlotte, North Carolina, United States

Site Status RECRUITING

Countries

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United States

Central Contacts

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Juby Philip

Role: CONTACT

(302) 559-4320

Cindy Sandy

Role: CONTACT

(317) 258-7262

Facility Contacts

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Role: primary

407-545-7610

Role: primary

651-377-8335

Role: primary

732-433-3431

Role: primary

(800) 785-3150 ext. 198

Other Identifiers

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EPX-100-003

Identifier Type: -

Identifier Source: org_study_id

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