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EPX-100 for Dravet Syndrome (ARGUS Trial)

Phase 2
Recruiting
Research Sponsored by Epygenix
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Clinical diagnosis of Dravet Syndrome with specific seizure criteria
Sexually active women of child-bearing potential (WCBP) must be using a medically acceptable method of birth control and have a negative serum or urine pregnancy test at the screening (Visit 1) and Randomization (Visit 2)
Must not have
Seizures secondary to specific causes
Prior or concurrent use of lorcaserin
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 20 weeks

Summary

This trial will test if EPX-100 is a safe and effective treatment for Dravet Syndrome.

Who is the study for?
This trial is for children and adults aged 2 years and older with Dravet Syndrome who experience frequent convulsive seizures. Participants must be able to maintain a seizure diary, use birth control if applicable, and have not been exposed to the study drug or similar drugs before. They should not have any significant medical conditions that could interfere with the trial.
What is being tested?
The study tests EPX-100 (Clemizole HCl) as an additional treatment alongside standard anti-epileptic drugs in patients with Dravet Syndrome. It aims to see if it's safe and effective at controlling seizures compared to a placebo (a substance with no active drug).
What are the potential side effects?
While specific side effects of EPX-100 are not listed here, common side effects may include allergic reactions, potential interactions with other medications, or exacerbation of existing health issues. The safety profile will be closely monitored throughout the trial.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I have been diagnosed with Dravet Syndrome and meet the specific seizure criteria.
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I am using birth control and my pregnancy tests are negative.
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I've had 4 or more seizures in the last 28 days.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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My seizures are caused by a specific condition.
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I have used lorcaserin before or am currently using it.
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I am taking fenfluramine without an echocardiogram report.
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I am not taking any medications that interfere with EPX-100.
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I am allergic or sensitive to EPX-100 (clemizole HCl).

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~20 weeks
This trial's timeline: 3 weeks for screening, Varies for treatment, and 20 weeks for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
The mean percent change in countable convulsive seizure frequency (CCSF) in the Titration and Maintenance (T+M) periods relative to baseline.
Secondary study objectives
The change in the Sleep Disturbance Scale for Children (SDSC) between EPX-100 and placebo by visit
The difference between EPX-100 vs placebo in proportion of participants with >25% reduction in the mean CCSF in the T+M periods relative to baseline.
The difference between EPX-100 vs placebo in proportion of participants with >50% reduction in the mean CCSF in the T+M periods relative to baseline.
+9 more

Trial Design

2Treatment groups
Experimental Treatment
Placebo Group
Group I: EPX-100 (clemizole HCl)Experimental Treatment1 Intervention
Group II: Placebo armPlacebo Group1 Intervention
Color- and taste-matched placebo oral solution dosed to match the active arm.

Find a Location

Who is running the clinical trial?

Liberyx TherapeuticsUNKNOWN
Forest Hills LabIndustry Sponsor
2 Previous Clinical Trials
72 Total Patients Enrolled
Harmony Biosciences, LLCIndustry Sponsor
8 Previous Clinical Trials
1,918 Total Patients Enrolled
EpygenixLead Sponsor
2 Previous Clinical Trials
48 Total Patients Enrolled
George Nomikos, MD, PhDStudy DirectorHarmony Biosciences, LLC
Hahn-Jun Lee, M.Sc, Ph.D.Study DirectorEpygenix Therapeutics, Inc.
Lorianne Masouka, MDStudy DirectorEpygenix Therapeutics, Inc.

Media Library

EPX-100 (clemizole HCl) Clinical Trial Eligibility Overview. Trial Name: NCT04462770 — Phase 2
Dravet Syndrome Research Study Groups: EPX-100 (clemizole HCl), Placebo arm
Dravet Syndrome Clinical Trial 2023: EPX-100 (clemizole HCl) Highlights & Side Effects. Trial Name: NCT04462770 — Phase 2
EPX-100 (clemizole HCl) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04462770 — Phase 2
~23 spots leftby Apr 2026