An Efficacy and Safety Study of Clemizole HCl in Patients With Lennox-Gastaut Syndrome
Purpose
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).
Condition
- Lennox Gastaut Syndrome
Eligibility
- Eligible Ages
- Between 2 Years and 55 Years
- Eligible Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- 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
- 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.
Study Design
- Phase
- Phase 3
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel Assignment
- Intervention Model Description
- Patients are randomized 1:1 to clemizole HCl (EPX-100) or placebo.
- Primary Purpose
- Treatment
- Masking
- Triple (Participant, Care Provider, Investigator)
Arm Groups
| Arm | Description | Assigned Intervention |
|---|---|---|
|
Placebo Comparator Placebo |
Participants will receive their first dose of study drug following randomization. |
|
|
Experimental Double-blind clemizole HCl |
Participants will receive their first dose of study drug following randomization. |
|
|
Experimental 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. |
|
Recruiting Locations
UCI Center for Innovative Health Therapies
Orange, California 92868
Orange, California 92868
Contact:
David King-Stephens, MD
714-456-7720
David King-Stephens, MD
714-456-7720
University of Miami Miller School of Medicine
Miami, Florida 33136
Miami, Florida 33136
AdventHealth Innovation Tower
Orlando, Florida 32804
Orlando, Florida 32804
Re:Cognition Health
Chicago, Illinois 60611
Chicago, Illinois 60611
Neurology Center for Epilepsy and Seizures
Marlboro, New Jersey 07746
Marlboro, New Jersey 07746
Contact:
732-433-3431
732-433-3431
Tekton Research
Marlboro, New Jersey 07746
Marlboro, New Jersey 07746
NYU Comprehensive Epilepsy Center - BRANY - PPDS
New York, New York 10016-4852
New York, New York 10016-4852
Weill Cornell Medicine/New York Presbyterian Hospital
New York, New York 10021
New York, New York 10021
Duke University Medical Center
Durham, North Carolina 27705
Durham, North Carolina 27705
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania 19104
Philadelphia, Pennsylvania 19104
Clinical Neurosciences Center
Salt Lake City, Utah 84132
Salt Lake City, Utah 84132
More Details
- Status
- Recruiting
- Sponsor
- Epygenix
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.