An Efficacy and Safety Study of Clemizole HCl in Patients... | NCT05066217

Status Module

Record Verification Date

Jul 2026

Overall Recruitment Status or Expanded Access Status

Recruiting

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Apr 9, 2025Actual

Primary Completion Date

Nov 1, 2026Estimated

Completion Date

Nov 1, 2029Estimated

First Submitted Date

Sep 23, 2021

First Submission Date that Met QC Criteria

Sep 23, 2021

First Posted Date

Oct 4, 2021Actual

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Jul 1, 2026

Last Update Posted Date

Jul 2, 2026Actual

Name	Class
Harmony Biosciences Management, Inc.	INDUSTRY

Oversight Module

Has Data Monitoring Committee (DMC)

Yes

Is FDA Regulated Drug

Yes

Is FDA Regulated Device

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

Brief Summary

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

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.

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Phase 3

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

260Estimated

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
Placebo	Placebo Comparator	Participants will receive their first dose of study drug following randomization.	Drug: Placebo
Double-blind clemizole HCl	Experimental	Participants will receive their first dose of study drug following randomization.	Drug: Clemizole HCl
Open-label clemizole HCl	Experimental	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.	Drug: Clemizole HCl

Interventions

Name	Type	Description	Arm Group Labels	Other Names
Clemizole HCl	Drug	Clemizole HCl will be administered as an oral solution.	Double-blind clemizole HCl Open-label clemizole HCl

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Percent Change in CMMS-28	Percent change in CMMS-28 from the Baseline Period through the end of the DB Period	From Baseline Period up to 16 weeks

Secondary Outcomes

Measure	Description	Time Frame
Proportion of Participants with ≥50% Reduction in CMMS-28	Proportion of participants with ≥50% reduction in CMMS-28 from the Baseline Period through the end of the DB Period	From Baseline Period up to 16 weeks
Percent Change in CMMS-28 Seizure-free Days

Other Outcomes

Not provided

Eligibility Module

Eligibility Criteria

Key Inclusion Criteria:

Males or females, ages ≥2 to ≤55 years, at the time of Screening.
Participant/parent/legal authorized representative (LAR) willing and able to give written informed consent/assent.
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:

Known sensitivity, allergy, or previous exposure to clemizole HCl.
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).
Family history of sudden cardiac death, unexplained death, or death from a primary dysrhythmia potentially associated with QT prolongation in any family member.
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.
Epilepsy surgery planned during the study or epilepsy surgery within 6 months prior to Screening.
Concomitant use of fenfluramine.
Prior or concomitant use of lorcaserin.

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

2 Years

Maximum Age

55 Years

Standard Ages

ChildAdult

Study Population

Not provided

Sampling Method

Not provided

Contacts/Locations Module

Central Contacts

Name	Role	Phone	Extension	Email
Juby Philip	Contact	(302) 559-4320		clinicaltrials@harmonybiosciences.com
Cindy Sandy	Contact	(317) 258-7262		clinicaltrials@harmonybiosciences.com

Overall Officials

Name	Affiliation	Role
Amit Ray, MD	Harmony Biosciences Management, Inc.	Study Director

Locations

Facility	Status	City	State	ZIP	Country	Contacts
Arkansas Children's Hospital	Active, not recruiting	Little Rock	Arkansas	72202	United States

ID	Term
D065768	Lennox Gastaut Syndrome
D012640	Seizures

ID	Term
D000073376	Epileptic Syndromes
D004827	Epilepsy
D001927	Brain Diseases
D002493	Central Nervous System Diseases
D009422	Nervous System Diseases
D030342	Genetic Diseases, Inborn
D009358	Congenital, Hereditary, and Neonatal Diseases and Abnormalities
D009461	Neurologic Manifestations
D012816	Signs and Symptoms
D013568	Pathological Conditions, Signs and Symptoms