Neurological

Dravet Syndrome

Also known as Severe myoclonic epilepsy of infancy, SMEI, SCN1A epileptic encephalopathy

Dravet syndrome is a severe developmental and epileptic encephalopathy caused in approximately 80% of cases by de novo loss-of-function mutations in SCN1A, encoding the Nav1.

ORPHA:33069 ↗Gene SCN1APrevalence 1-9 per 100,000 (Orphanet)Onset InfantileAutosomal dominant genetic (de novo in most cases)

16

studies recruiting now

as of 7 Sept 2026

103

studies registered in total

as of 7 Sept 2026

23

countries with a recruiting site

as of 7 Sept 2026

15 Apr 2026

most recent study posted

among recruiting studies

Recruiting trials

Showing the 5 most recently updated recruiting studies, as recorded 7 Sept 2026. Live status on each study page.

See all 16 recruiting studies

Where recruiting studies are running

Countries with at least one recruiting site among the studies above, 7 Sept 2026. Tap a country to search trials there.

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Patient organisations

Dravet Syndrome FoundationPatient association
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Registry: Dravet Syndrome Patient Registry · Join ↗. Registries connect patients to researchers and often hear about trials first.

About Dravet Syndrome

Dravet syndrome is a severe developmental and epileptic encephalopathy caused in approximately 80% of cases by de novo loss-of-function mutations in SCN1A, encoding the Nav1.1 sodium channel subunit. Onset occurs in the first year of life with prolonged febrile and afebrile seizures, followed by developmental plateau and multiple seizure types that are refractory to conventional antiseizure medications. Sodium channel blockers (e.g., carbamazepine, phenytoin) worsen seizures and are contraindicated.

Common clinical features

Prolonged febrile seizuresMultiple seizure types (myoclonic, focal, absence)Developmental plateauAtaxiaSleep disturbanceHyperthermia-triggered seizuresBehavioral problems

From Orphanet’s phenotype annotations (CC BY 4.0). Not a complete list.

Treatments being studied

3 approved treatments and 7 in clinical development, from Open Targets (CC BY 4.0). Not medical advice.

Approved: Stiripentol (Diacomit)Approved: Cannabidiol (Epidiolex)Approved: Fenfluramine Hydrochloride (Fintepla)
Phase 3Zorevunersen
Phase 3Clobazam (Frisium)
Phase 3Fenfluramine
Phase 2Soticlestat
Phase 2Ataluren (Translarna)
Phase 2(S)-Verapamil
Phase 2Verapamil

Before you apply

Things trial teams commonly ask about for Dravet Syndrome. Not eligibility rules; those are set by each study.

  • SCN1A variant classification (pathogenic, likely pathogenic) and functional impact are required for gene therapy trial eligibility
  • Sodium channel blockers are contraindicated — ensure current antiseizure medication list is provided; trials screen for contraindicated drugs
  • Seizure frequency diary (minimum 3 months) is a standard inclusion criterion — document baseline convulsive seizure count
  • Fenfluramine (Fintepla) and cannabidiol (Epidiolex) are approved add-on therapies — prior treatment history affects trial stratification

Related conditions

Information, not medical advice. Trial listings are shown as recorded on ClinicalTrials.gov; whether any study is right for you is a decision for you and your clinicians, and eligibility is decided by each research team. Disease information from Orphanet (CC BY 4.0).