A 20-Week Multicenter, Randomized, Double-Blind, Placebo-Controlled Trial of EPX-100 (Clemizole Hydrochloride) as Adjunctive Therapy in Children and Adult Participants With Dravet Syndrome (ARGUS Trial)
Epygenix
Summary
This is a multicenter, Phase 3, randomized, double-blind, placebo-controlled study designed to evaluate the efficacy and safety of clemizole hydrochloride (EPX-100) as adjunctive therapy in children and adult participants with Dravet syndrome (DS).
Description
This is a global, multicenter, randomized, double-blind, placebo-controlled study to evaluate the safety and efficacy of clemizole hydrochloride as adjunctive therapy in children and adult participants with DS. The study consists of a 4-week Observational Period, a 16-week Double-Blind (DB) Period and an Open-Label Extension (OLE) Period for up to 156 weeks.
Eligibility
- Age range
- 2+ years
- Sex
- All
- Healthy volunteers
- No
Key Inclusion Criteria: 1. Male and female participants 2 years and older at time of consent. 2. Participant or parent/legally authorized representative (LAR) willing and able to provide written informed consent, assent (if applicable) prior to initiation of any study related procedures. 3. Clinical diagnosis of DS. Participants must have seizures which are not completely controlled by AEDs with the following criteria: * Onset of seizures prior to 18 months of age, * Normal development at onset, * History of at least one type of countable motor seizure (CMS), * Brain MRI without…
Interventions
- DrugClemizole HCl
Clemizole HCl will be administered as an oral solution.
- DrugPlacebo
Placebo will be administered as an oral solution.
Locations (46)
- Children's Hospital of Los AngelesLos Angeles, California
- University of California IrvineOrange, California
- UCSF Medical CenterSan Francisco, California
- Yale University School of MedicineNew Haven, Connecticut
- The Nemours FoundationWilmington, Delaware
- Rare Disease Research FLKissimmee, Florida