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

• Register: ClinicalTrials.gov
• Last refreshed on: 21 April 2025
• Main ID: NCT05066217
• Date of registration: 23/09/2021
• Prospective Registration: Yes
• Primary sponsor: Epygenix
• Public title: An Efficacy and Safety Study of Clemizole HCl in Patients With Lennox-Gastaut Syndrome
• Scientific title: Multicenter, Randomized, Double-blind, Placebo-controlled Trial of Clemizole HCl as Adjunctive Therapy in Patients With Lennox-Gastaut Syndrome
• Date of first enrolment: April 8, 2025
• Target sample size: 260
• Recruitment status: Recruiting
• URL: https://clinicaltrials.gov/ct2/show/NCT05066217
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Triple (Participant, Care Provider, Investigator).
• Phase: Phase 3

Countries of recruitment

United States

Contacts

• Name: Amit Ray, MD
• Affiliation: Harmony Biosciences Management, Inc.

• Name: Juby Philip

Key inclusion & 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.


Age minimum: 2 Years
Age maximum: 55 Years
Gender: All

Health Condition(s) or Problem(s) studied

Lennox Gastaut Syndrome

Intervention(s)

Drug: Placebo

Drug: Clemizole HCl

Primary Outcome(s)

Percent Change in CMMS-28 [Time Frame: From Baseline Period up to 16 weeks]

Secondary Outcome(s)

Clinical Global Impression of Change (CGI-C) Score [Time Frame: Week 16]

Proportion of Participants with =50% Reduction in CMMS-28 [Time Frame: From Baseline Period up to 16 weeks]

Proportion of Participants with =50% Reduction in CMMS-28 [Time Frame: From Baseline Period up to 12 weeks]

Caregiver Global Impression of Change (CaGI-C) Score [Time Frame: Week 16]

Caregiver Global Impression of Change in Seizure Intensity/Duration (CaGI-CSID) Score [Time Frame: Week 16]

Change in Quality of Life Inventory (QI)-Disability Score [Time Frame: From Baseline Period up to 16 weeks]

Incidence of Treatment-Emergent Adverse Events (TEAEs) [Time Frame: From the first dose administration of study drug up to end of the study, approximately up to 172 weeks]

Percent Change in CMMS-28 Seizure-free Days [Time Frame: From Baseline Period up to 16 weeks]

Percent Change in CMMS-28 [Time Frame: From Baseline Period up to 12 weeks]

Percent Change per 28 Days in the Number of Seizure Free Days [Time Frame: From Baseline Period up to 16 weeks]

Secondary ID(s)

EPX-100-003

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Harmony Biosciences Management, Inc.