Clinical trial NCT05066217: An Efficacy and Safety Study of Clemizole HCl in Patients With Lennox-Gastaut Syndrome
Official title: Multicenter, Randomized, Double-blind, Placebo-controlled Trial of Clemizole HCl as Adjunctive Therapy in Patients With Lennox-Gastaut Syndrome
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE3
Conditions: Lennox Gastaut Syndrome
Keywords: Lennox Gastaut Syndrome
Lennox-Gastaut Syndrome
LGS
Clemizole HCl
Seizure
Lead sponsor: Epygenix
Sponsor class: INDUSTRY
Collaborators: name: Harmony Biosciences Management, Inc.
First posted: 2021-10-04
Enrollment: 260
Sex: ALL
Minimum age (years): 2
Maximum age (years): 55
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention model description: Patients are randomized 1:1 to clemizole HCl (EPX-100) or placebo.
Primary purpose: TREATMENT

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.

Eligibility 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.

Primary outcomes:
measure: Percent Change in CMMS-28
timeFrame: From Baseline Period up to 16 weeks
Secondary outcomes:
measure: Proportion of Participants with ≥50% Reduction in CMMS-28
timeFrame: From Baseline Period up to 16 weeks
measure: Percent Change in CMMS-28 Seizure-free Days
timeFrame: From Baseline Period up to 16 weeks
measure: Clinical Global Impression of Change (CGI-C) Score
timeFrame: Week 16
measure: Caregiver Global Impression of Change (CaGI-C) Score
timeFrame: Week 16
measure: Caregiver Global Impression of Change in Seizure Intensity/Duration (CaGI-CSID) Score
timeFrame: Week 16
measure: Change in Quality of Life Inventory (QI)-Disability Score
timeFrame: From Baseline Period up to 16 weeks
measure: Percent Change per 28 Days in the Number of Seizure Free Days
timeFrame: From Baseline Period up to 16 weeks
measure: Percent Change in CMMS-28
timeFrame: From Baseline Period up to 12 weeks
measure: Proportion of Participants with ≥50% Reduction in CMMS-28
timeFrame: From Baseline Period up to 12 weeks
measure: Incidence of Treatment-Emergent Adverse Events (TEAEs)
timeFrame: From the first dose administration of study drug up to end of the study, approximately up to 172 weeks
Other outcomes:
Locations:
city: Kissimmee
country: United States
facility: Rare Disease Research
state: Florida
city: Raleigh
country: United States
facility: On-Site Clinical Solution
state: North Carolina
Study officials:
affiliation: Harmony Biosciences Management, Inc.
name: Amit Ray, MD
role: STUDY_DIRECTOR
References: