Trial reportEpilepsia2026
Soticlestat as an adjunctive therapy in children and young adults with Dravet syndrome.
Trial report in Epilepsia, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT04940624 (A Multicenter, Randomized, Double-Blind, Placebo-Controlled, Parallel Group Study to Evaluate the Efficacy, Safety, and Tolerability of Soticlestat as Adjunctive Therapy in Pediatric and Young Adult Subjects With Dravet Syndrome), which is not on this map. Cited by 4 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
A Multicenter, Randomized, Double-Blind, Placebo-Controlled, Parallel Group Study to Evaluate the Efficacy, Safety, and Tolerability of Soticlestat as Adjunctive Therapy in Pediatric and Young Adult Subjects With Dravet Syndrome (DS)
Who cites it
4 citing papers in PubMed.
- Listening across frequencies: Capturing seizures of all patients to advance antiseizure medication development.Epilepsia · 2026Article
- Review
- Clinical Failure Validates a Fifth Predictive Preclinical Antiseizure Medicine Discovery Model.Epilepsy currents · 2026Article
- Virtual screening and zebrafish phenotype-based evaluation argues against repurposing 4-phenylbutyrate for STXBP1-related disorders.bioRxiv : the preprint server for biology · 2026Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
15 authors.
Funding
Abstract
objectiveThis study evaluated the efficacy, safety, and tolerability of soticlestat as adjunctive therapy in children and young adults with Dravet syndrome (DS).
methodsSKYLINE (NCT04940624) was a multicenter, randomized, double-blind, placebo-controlled, phase 3 trial that enrolled patients with DS aged 2-21 years with uncontrolled convulsive seizures (≥4/month despite adequate treatment). Participants received oral soticlestat 300 mg (weight adjusted) or matching placebo twice daily. The total study duration was 16 weeks, comprising 4-week dose titration and 12-week maintenance treatment periods. The primary endpoint was a comparison of monthly convulsive seizure frequency between baseline and the titration/maintenance periods. Key secondary endpoints included several modified Caregiver and Clinical Global Impression of Improvement (GI-I) scales for DS.
resultsOne hundred forty-four participants were randomized (71 placebo, 73 soticlestat) with a mean (SD) age of 10.3 (5.0) years; 72 (50%) were male, and 117 (81.3%) were receiving ≥3 antiseizure medications. Median change from baseline in convulsive seizure frequency over the full treatment period was -8.64% with placebo (n = 71) and -22.16% with soticlestat (n = 73), a difference of -15.64% (p = .061); in the maintenance treatment period, these changes were -11.99% with placebo and -23.29% with soticlestat, a difference of -14.29% (p = .089). The proportion of participants with ≥50% reduction in convulsive seizures was 9.9% with placebo and 27.4% with soticlestat (nominal p = .008). Soticlestat showed clinically meaningful results in the Caregiver and Clinical GI-I, and Clinical GI-I Seizure Intensity and Duration scales over the 16-week treatment period (all nominal p-values ≤ .004). The most commonly reported treatment-emergent adverse events related to study drug were somnolence, change in seizure presentation, decreased appetite, and insomnia. SIGNIFICANCE: Although statistical significance was narrowly missed, soticlestat showed a numerical benefit over placebo for convulsive seizure decrease. Clinically meaningful benefits across multiple secondary endpoints were observed. No new safety concerns emerged.
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