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Home › Conditions › Dravet syndrome

Dravet syndrome

Part of Epilepsy

3 published articles · Updated continuously

Clinical Trial Landscape

Clinical Trials for Dravet syndrome

10 trials tracked for Dravet syndrome: 7 in phase 3 or 4 and 6 with published results. The most-cited published study has 1661 citations.

10Trials tracked
7Phase 3 & 4
0Recruiting
6With published results
Phase distribution
Phase 3 7 Phase 2 3
  1. Phase 3 Antiepileptic Efficacy Study of GWP42003-P in Children and Young Adults With Dravet Syndrome (GWPCARE1) Completed · 1,661 cited
  2. Phase 3 A Trial of Two Fixed Doses of ZX008 (Fenfluramine HCl) in Children and Young Adults With Dravet Syndrome Completed · 369 cited
  3. Phase 3 GWPCARE2 A Study to Investigate the Efficacy and Safety of Cannabidiol (GWP42003-P) in Children and Young Adults With Dravet Syndrome Completed · 263 cited
  4. Phase 3 A 2-Part Study to Investigate the Dose-Ranging Safety and Pharmacokinetics, Followed by the Efficacy and Safety of ZX008 (Fenfluramine Hydrochloride) Oral Solution as an Adjunctive Therapy in Children ≥ 2 Years Old and Young Adults With Dravet Syndrome Completed · 243 cited
  5. Phase 3 An Open Label Extension Study of Cannabidiol (GWP42003-P) in Children and Adults With Dravet or Lennox-Gastaut Syndromes Completed · 112 cited
  6. Phase 3 An Open-Label Extension Trial to Assess the Long-Term Safety of ZX008 (Fenfluramine Hydrochloride HCl) Oral Solution in Children and Young Adults With Dravet Syndrome Completed · 81 cited
Show 4 more trials
  1. Phase 3 A Study to Investigate the Long-Term Safety of ZX008 (Fenfluramine Hydrochloride) Oral Solution in Children and Adults With Epileptic Encephalopathy Including Dravet Syndrome and Lennox-Gastaut Syndrome Completed
  2. Phase 2 A Dose-ranging Pharmacokinetics and Safety Study of GWP42003-P in Children With Dravet Syndrome (GWPCARE1) Completed
  3. Phase 2 Verapamil as Therapy for Children and Young Adults With Dravet Syndrome Completed
  4. Phase 2 A Phase 2, Multicenter, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy, Safety, and Tolerability of TAK-935 (OV935) as an Adjunctive Therapy in Pediatric Participants With Developmental and/or Epileptic Encephalopathies Completed

Showing the 10 most-cited and recently-updated of 10 trials. Browse the full registry →

Trial data sourced from ClinicalTrials.gov. Counts describe the research landscape and are not a treatment recommendation. Informational only — not medical advice.

What the trials found For clinicians

Dravet syndrome: what the trials found

GWP42003-P has demonstrated significant reductions in convulsive seizure frequency (p=0.0123) and non-convulsive seizure frequency 1. Clinical trials showed a reduction of 48.7 vs 26.9 for convulsive seizures (p=0.0299) and 56.4 vs 29.7 for total seizures (p=0.0255), with a significant proportion of patients achieving a ≥50% reduction in convulsive seizure frequency 3.

ZX008 (Fenfluramine Hydrochloride) demonstrated statistically significant improvements in convulsive seizure frequency compared to placebo (p=<0.001) and resulted in longer mean convulsive seizure-free intervals (22.0 vs 13.0 days, p=0.004) 4. In larger cohorts, ZX008 showed a higher percentage of participants achieving ≥25% reduction in convulsive seizure frequency compared to placebo across different dosage arms 2.

Safety data for ZX008 indicates that 75.5% of participants experienced treatment-emergent adverse events (TEAEs) 7, with 26.5% experiencing serious TEAEs during the open-label extension period 6. Only 3.5% of patients withdrew from the investigational medicinal product due to TEAEs during this period 6.

Recent results — preliminary, needs further review

  • TAK-935 showed a significant reduction in convulsive seizure frequency specifically within the Dravet Syndrome stratum (p=0.0001) and overall treatment periods (p=0.0024).
  • Verapamil was evaluated in a small cohort, though results are not yet corroborated.

For the clinician treating this condition

  • GWP42003-P is associated with statistically significant reductions in both convulsive and non-convulsive seizure frequencies.
  • ZX008 (Fenfluramine Hydrochloride) significantly reduces convulsive seizure frequency and extends the duration of seizure-free intervals compared to placebo.
  • While ZX008 shows efficacy, it is associated with a high incidence of treatment-emergent adverse events (75.5%) and serious TEAEs (26.5%).

AI synthesis of 6 cited trials, updated Jun 22, 2026. Informational only — not medical advice; trial data sourced from ClinicalTrials.gov. How we use AI.

HCP Mode — summaries include clinical detail, trial data, and statistical outcomes.
Patient Mode — summaries use plain language, avoiding clinical jargon.
Cannabidiol is associated with 49.9% seizure reduction in patients with Developmental and Epileptic Encephalopathies
Pediatrics Meta-analysis
Cannabidiol is associated with 49.9% seizure reduction in patients with Developmental and Epileptic Encephalopathies Cannabidiol shows significant seizure reduction in some DEE patients
This meta-analysis of 2592 individuals with Developmental and Epileptic Encephalopathies (DEEs) found that pharmaceutical-grade cannabidiol …
Pharmaceutical-grade cannabidiol may help nearly half of people with certain severe epilepsy syndromes reduce their seizures by at least 50 …
Jul 2, 2026
Systemic inflammatory indices are elevated in pediatric drug-resistant epilepsy versus self-limited epilepsy with centrotemporal spikes
Neurology Cohort
Systemic inflammatory indices are elevated in pediatric drug-resistant epilepsy versus self-limited epilepsy with centrotemporal spikes Are inflammation markers higher in children with hard-to-treat epilepsy than those with self-limited cases?
This real-world cohort study of 140 pediatric patients compared systemic inflammatory indices (NLR, SII, CRP) between those with drug-resist…
Children with hard-to-treat epilepsy show higher blood inflammation markers than those with self-limited cases, though the exact link to bra…
Frontiers Apr 15, 2026
Genetic mutations in febrile sensitivity epilepsy correlate with earlier onset and specific diagnoses in children
Neurology Cohort
Genetic mutations in febrile sensitivity epilepsy correlate with earlier onset and specific diagnoses in children Fever-Triggered Seizures in Children Trace Back to Specific Genes
This retrospective cohort study examined 30 gene-positive cases and 31 gene-negative controls at Wuxi Children's Hospital.
Half of children with fever-triggered seizures carry specific gene mutations that help doctors predict which treatments will work and which …
Frontiers Apr 14, 2026