Lennox Gastaut Syndrome
Conditions
Keywords
Lennox Gastaut Syndrome, Lennox-Gastaut Syndrome, LGS, Clemizole HCl, Seizure
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.
Interventions
DRUGClemizole HCl
Clemizole HCl will be administered as an oral solution.
DRUGPlacebo
Placebo will be administered as an oral solution.
Sponsors
Epygenix
Harmony Biosciences Management, Inc.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
TRIPLE (Subject, Caregiver, Investigator)
Intervention model description
Patients are randomized 1:1 to clemizole HCl (EPX-100) or placebo.
Eligibility
Sex/Gender
ALL
Age
2 Years to 55 Years
Healthy volunteers
No
Inclusion 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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Percent Change in CMMS-28 | From Baseline Period up to 16 weeks | Percent change in CMMS-28 from the Baseline Period through the end of the DB Period |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Proportion of Participants with ≥50% Reduction in CMMS-28 | From Baseline Period up to 16 weeks | Proportion of participants with ≥50% reduction in CMMS-28 from the Baseline Period through the end of the DB Period |
| Percent Change in CMMS-28 Seizure-free Days | From Baseline Period up to 16 weeks | Percent change in CMMS-28 seizure-free days from the Baseline Period through the end of the DB Period |
| Clinical Global Impression of Change (CGI-C) Score | Week 16 | CGI-C score at the end of the DB Period |
| Caregiver Global Impression of Change (CaGI-C) Score | Week 16 | CaGI-C score at the end of the DB Period |
| Caregiver Global Impression of Change in Seizure Intensity/Duration (CaGI-CSID) Score | Week 16 | CaGI-CSID score at the end of the DB Period |
| Change in Quality of Life Inventory (QI)-Disability Score | From Baseline Period up to 16 weeks | Change in QI-disability score from Baseline to the end of the DB Period |
| Percent Change per 28 Days in the Number of Seizure Free Days | From Baseline Period up to 16 weeks | Percent change per 28 days in the number of seizure-free days (based on all seizure types) from the Baseline Period through the end of the DB Period |
| Percent Change in CMMS-28 | From Baseline Period up to 12 weeks | Percent change in CMMS-28 from the Baseline Period through the end of the DB Maintenance Phase only |
| Incidence of Treatment-Emergent Adverse Events (TEAEs) | From the first dose administration of study drug up to end of the study, approximately up to 172 weeks | Incidence of TEAEs will be compared among the treatment groups |
Countries
China, Italy, Poland, Romania, Serbia, Spain, United States
Contacts
CONTACTJuby Philip
CONTACTCindy Sandy
STUDY_DIRECTORAmit Ray, MD
Harmony Biosciences Management, Inc.
Outcome results
None listed