Not medical advice. This website is not medical advice, a diagnosis, a match to a trial, or a recommendation to join a study. Listings are plain-English rewrites of public ClinicalTrials.gov records. Whether a site is still enrolling, and whether you qualify, can only be confirmed by the study team and your clinician. Source registry: ClinicalTrials.gov. Editorial rules: methodology.
RecruitingPhase 3interventionalRules rewrite

Phase 3: testing Clemizole HCl for Dravet Syndrome

This is a multicenter, Phase 3, randomly assigned, neither you nor the study doctors know who gets the real treatment, compared with a dummy treatment study designed to measure the how well it works and safety of clemizole hydrochloride (EPX-100) as added on top of your current treatment therapy in children and adult participants with Dravet syndrome (DS).

Official registry: https://clinicaltrials.gov/study/NCT04462770 · Epygenix · enrollment listed as 150

What this phase means

Phase 3: this treatment already looked useful in smaller studies. Now it is tested in a large group, often against standard care or a dummy treatment, to see if it should be approved.

Phase labels are FDA/ICH categories applied to the registry Phase field. They are not a finding that this study succeeded.

What the registry says happens

If you join, the listed intervention(s) are Clemizole HCl, or Placebo. The registry lists allocation as randomized. This is a multicenter, Phase 3, randomly assigned, neither you nor the study doctors know who gets the real treatment, compared with a dummy treatment study designed to measure the how well it works and safety of clemizole hydrochloride (EPX-100) as added on top of your current treatment therapy in children and adult participants with Dravet syndrome (DS).

They plan to enroll about 150 people at 86 sites. Taking part typically means taking a study drug (or dummy pill) plus check-ins.

Official title (unrewritten)

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)

Interventions: Clemizole HCl, Placebo

Primary outcome (registry wording): Percent Change in Countable Motor Seizures Per 28 Days (CMS-28) in the Titration Plus Maintenance Periods Relative to Baseline

Who it is for (from inclusion criteria)

  • Ages 2 Years to any
  • Male and female participants 2 years and older at time of consent
  • 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

Public study contact (from the registry)

Krystle Rapchak

+1 (312) 847-1289

clinicaltrials@harmonybiosciences.com

Published on ClinicalTrials.gov. Contact them as a possible participant. They decide eligibility.

Sites listed on ClinicalTrials.gov

86 listed

+ 55 sites outside the US on the same record

Related recruiting trials in this snapshot

RecruitingNot a traditional drug phaseBethesda, Maryland
Surgery as a Treatment for Medically Intractable Epilepsy

Background: \- Drug resistant epilepsy is the term used to describe epilepsy that cannot be controlled by medication. Many people whose seizures do not respond to medication will respond to surgical treatment, relieving seizures completely or almost completely in one-half to two-thirds of patients who qualify for surgery.

NCT01273129 · Official ClinicalTrials.gov record · National Institute of Neurological Disorders and Stroke (NINDS)

RecruitingNot a traditional drug phaseSioux Falls, South Dakota
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford

The Coordination of Rare Diseases at Sanford (CoRDS) is an international patient registry that serves as a centralized resource for rare disease research. Individuals affected by rare diseases, undiagnosed conditions, and disorders of unknown prevalence may voluntarily contribute health information to support natural history studies, participant recruitment, and other research efforts.

NCT01793168 · Official ClinicalTrials.gov record · Sanford Health

FAQ about this NCT record

Is NCT04462770 still recruiting?

The sponsor posted status “Recruiting” on ClinicalTrials.gov at last ingest. Sites can pause independently. Confirm with the official record and the listed contact.

What phase is NCT04462770?

Phase 3. Phase labels are FDA/ICH categories applied to the registry Phase field. They are not a finding that this study succeeded.

Can PlainTrials tell me if I qualify for NCT04462770?

No. The inclusion and exclusion lists on this page are readable extracts of the public eligibility block. Only the study team can screen you.

How to read a ClinicalTrials.gov record · Questions to ask the coordinator · Glossary