NCT04462770 A Study of EPX-100 (Clemizole Hydrochloride) in Participants With Dravet Syndrome
| NCT ID | NCT04462770 |
| Status | Recruiting |
| Phase | Phase 3 |
| Sponsor | Epygenix |
| Condition | Dravet Syndrome |
| Study Type | INTERVENTIONAL |
| Enrollment | 150 participants |
| Start Date | 2020-09-15 |
| Primary Completion | 2027-04 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
Phase 3 trials are large pivotal studies comparing the treatment to current standard of care or placebo. Your participation directly contributes to the evidence needed for regulatory approval.
This trial targets 150 participants in total. It began in 2020-09-15 with a primary completion date of 2027-04.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief 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).
Eligibility Criteria
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 and 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 cortical malformation (not including mild atrophy associated with the natural progression of DS), * Genetic mutation of the SCN1A gene must be documented. Key Exclusion Criteria: 1. Known sensitivity, allergy, or previous exposure to clemizole HCl. 2. Exposure to any investigational drug or device \<90 days prior to screening or plans to participate in another drug or device trial at any time during the study. 3. Seizures secondary to illicit drug (this includes concomitant use of tetrahydrocannabinol \[THC\] and nonprescription cannabidiol preparations) or alcohol use, infection, neoplasm, demyelinating disease, degenerative neurological disease, or central nervous system disease deemed progressive, metabolic illness, or progressive degenerative disease. 4. Concurrent use of lorcaserin. Note: Prior use of lorcaserin is permitted if at least 30 days have passed since the last dose. 5. Concurrent use of fenfluramine. 6. Epilepsy surgery planned during the study or epilepsy surgery within 6 months prior to Screening.
Contact & Investigator
Amit Ray, MD
STUDY DIRECTOR
Harmony Biosciences Management, Inc.
Frequently Asked Questions
Who can join the NCT04462770 clinical trial?
This trial is open to participants of all sexes, aged 2 Years or older, studying Dravet Syndrome. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT04462770 trial and what does that mean for participants?
Phase 3 trials are large-scale studies comparing the new treatment to existing standards of care or a placebo. They provide the evidence needed for regulatory approval. This trial targets 150 participants.
Is NCT04462770 currently recruiting?
Yes, NCT04462770 is actively recruiting participants. Contact the research team at clinicaltrials@harmonybiosciences.com for enrollment information.
Where is the NCT04462770 trial being conducted?
This trial is being conducted at Birmingham, United States, Little Rock, United States, Los Angeles, United States, Orange, United States and 11 additional locations.
Who is sponsoring the NCT04462770 clinical trial?
NCT04462770 is sponsored by Epygenix. The principal investigator is Amit Ray, MD at Harmony Biosciences Management, Inc.. The trial plans to enroll 150 participants.