Disease

Genetic developmental and epileptic encephalopathy

Late-stage therapeutic development

Also known as hereditary developmental and epileptic encephalopathy, developmental and epileptic encephalopathy.

Clinical trials

Associated genes

Related proteins

Latest activity

beta

Recent clinical, regulatory, research and industry developments relating to this disease.

A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Investigate the Efficacy, Safety, and Tolerability of LP352 in the Treatment of Seizures in Children and Adults With Developmental and Epileptic Encephalopathies

Clinical trial2026-08-12Status changed to Active, not recruiting · ClinicalTrials.gov

What's happening now

An analyst briefing on current research, clinical, regulatory and industry activity surrounding this disease.

Executive briefingUpdating summary…Momentum: Moderate

Key developments

6 clinical trials expected to report results, the earliest in Q4 2026.

Upcoming Milestones

Major developments

Upcoming trial readoutHigh impact

Phase III Trial of Neoadjuvant Durvalumab (NSC 778709) Plus Chemotherapy Versus Chemotherapy Alone for Adults With MammaPrint High 2 Risk (MP2) Hormone Receptor (HR) Positive / Human Epidermal Growth Factor Receptor (HER2) Negative Stage II-III Breast Cancer

Results expected Q2 20322026-09-18

Upcoming trial readoutHigh impact

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)

Results expected Q2 20272026-09-15

Clinical Milestones8View

Results posted 1

2025-11-17An Open-Label Extension Trial to Assess the Long-Term Safety of ZX008 (Fenfluramine Hydrochloride) Oral Solution as an Adjunctive Therapy for Seizures in Patients With Rare Seizure Disorders Such as Epileptic Encephalopathies Including Dravet Syndrome and Lennox-Gastaut SyndromeResults posted

Results expected 5

Other updates 1

2025-11-12A Prospective, Long-Term, Interventional, Active Extension Study to Evaluate the Safety and Tolerability of NBI-921352 as Adjunctive Therapy in Subjects With SCN8A Developmental and Epileptic Encephalopathy Syndrome (SCN8A-DEE)Terminated

Activity timeline8

2026-09-18ClinicalPhase III Trial of Neoadjuvant Durvalumab (NSC 778709) Plus Chemotherapy Versus Chemotherapy Alone for Adults With MammaPrint High 2 Risk (MP2) Hormone Receptor (HR) Positive / Human Epidermal Growth Factor Receptor (HER2) Negative Stage II-III Breast CancerResults expected Q2 2032
2026-09-15ClinicalA 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)Results expected Q2 2027
2026-08-24ClinicalEMPEROR: A Multicenter, Randomized, Double-blind, Sham-controlled, Parallel Group, Phase 3 Study Evaluating the Efficacy, Safety, and Tolerability of Zorevunersen (STK-001) in Patients With Dravet SyndromeResults expected Q1 2027
2026-08-04ClinicalA Phase 3, Randomized, Multi-Center, Double-Blind, Placebo-Controlled Clinical Trial to Evaluate the Efficacy, Safety, Tolerability, and Pharmacokinetics of Relutrigine in Participants With DEE Followed by an Open-Label ExtensionResults expected Q4 2026
2026-05-15ClinicalAssessment of Safety of the Use of Fenfluramine in Children With Dravet Syndrome Under the Age of 24 MonthsResults expected Q4 2026
2026-05-07ClinicalA Prospective, Case-control Evaluation of Ketogenic Dietary Therapy for New-onset Childhood Absence EpilepsyResults expected Q2 2028
2025-11-17ClinicalAn Open-Label Extension Trial to Assess the Long-Term Safety of ZX008 (Fenfluramine Hydrochloride) Oral Solution as an Adjunctive Therapy for Seizures in Patients With Rare Seizure Disorders Such as Epileptic Encephalopathies Including Dravet Syndrome and Lennox-Gastaut SyndromeResults posted
2025-11-12ClinicalA Prospective, Long-Term, Interventional, Active Extension Study to Evaluate the Safety and Tolerability of NBI-921352 as Adjunctive Therapy in Subjects With SCN8A Developmental and Epileptic Encephalopathy Syndrome (SCN8A-DEE)Terminated

Clinical trials

16 sponsors · 0 new · 0 completed in the last 12 months (net -1)

The current development programme across all trial phases.

Clinical programme

All trials

Active

Late-stage

Completed

Late-stage studies

Phase 3 · Recruiting · National Cancer Institute (NCI)

Phase 3 · Recruiting · Epygenix

A Prospective, Case-control Evaluation of Ketogenic Dietary Therapy for New-onset Childhood Absence Epilepsy

Phase 3 · Recruiting · Johns Hopkins University

A Phase 3, Randomized, Multi-Center, Double-Blind, Placebo-Controlled Clinical Trial to Evaluate the Efficacy, Safety, Tolerability, and Pharmacokinetics of Relutrigine in Participants With DEE Followed by an Open-Label Extension

Phase 3 · Active, not recruiting · Praxis Precision Medicines

Assessment of Safety of the Use of Fenfluramine in Children With Dravet Syndrome Under the Age of 24 Months

Phase 4 · Recruiting · University of Colorado, Denver

EMPEROR: A Multicenter, Randomized, Double-blind, Sham-controlled, Parallel Group, Phase 3 Study Evaluating the Efficacy, Safety, and Tolerability of Zorevunersen (STK-001) in Patients With Dravet Syndrome

Phase 3 · Recruiting · Stoke Therapeutics, Inc

Recruiting

Phase 3 · Recruiting · National Cancer Institute (NCI)

Phase 3 · Recruiting · Epygenix

A Prospective, Case-control Evaluation of Ketogenic Dietary Therapy for New-onset Childhood Absence Epilepsy

Phase 3 · Recruiting · Johns Hopkins University

Assessment of Safety of the Use of Fenfluramine in Children With Dravet Syndrome Under the Age of 24 Months

Phase 4 · Recruiting · University of Colorado, Denver

Phase 3 · Recruiting · Stoke Therapeutics, Inc

Recently completed

An Open-Label Extension Trial to Assess the Long-Term Safety of ZX008 (Fenfluramine Hydrochloride) Oral Solution as an Adjunctive Therapy for Seizures in Patients With Rare Seizure Disorders Such as Epileptic Encephalopathies Including Dravet Syndrome and Lennox-Gastaut Syndrome

Phase 3 · Completed · Zogenix, Inc.

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)

Phase 3 · Completed · Takeda

A Pilot Study of Dexmedetomidine-Propofol in Children Undergoing Magnetic Resonance Imaging

Phase 4 · Completed · Joseph Cravero

Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy, Safety and Tolerability of AD04 (Ondansetron) in Adults With Alcohol Use Disorder (AUD) and Selected Polymorphisms in the Serotonin Transporter and Receptor Genes

Phase 3 · Completed · Adial Pharmaceuticals

Randomized Double-blind Placebo-controlled Trial of Memantine Hydrochloride for the Treatment of Childhood-onset Epileptic Encephalopathies

Phase 4 · Completed · Kenneth Myers, MD

Neuroinflammation in Children With Infantile Spasms Measured With 11C-PK11195 Positron Emission Tomography: Response to ACTH

Phase 4 · Completed · Wayne State University

Associated genes

12 matches

Genes associated with this disease in the canonical knowledge graph (Open Targets evidence). Number shows the association score.

GABRB20.78 SCN1A0.77 DOCK70.74 ATP6V1A0.74 KCNQ20.73 FRRS1L0.72 SMC1A0.72 SYNJ10.72 CACNA1E0.71 PIGQ0.71 SLC25A220.71 RHOBTB20.70

Disease biology

12 matches

Proteins whose encoding gene is associated with this disease, reached through the canonical gene→disease graph. Number shows the gene's association score.

Gamma-aminobutyric acid receptor subunit beta-2via GABRB20.78 Sodium channel protein type 1 subunit alphavia SCN1A0.77 Potassium voltage-gated channel subfamily KQT member 2via KCNQ20.73 Voltage-dependent R-type calcium channel subunit alpha-1Evia CACNA1E0.71 Gamma-aminobutyric acid receptor subunit alpha-2via GABRA20.66 Potassium voltage-gated channel subfamily A member 2via KCNA20.64 Gamma-aminobutyric acid receptor subunit alpha-5via GABRA50.63 BDNF/NT-3 growth factors receptorvia NTRK20.61 Voltage-dependent calcium channel subunit alpha-2/delta-2via CACNA2D20.58 Potassium voltage-gated channel subfamily B member 1via KCNB10.52 Voltage-gated delayed rectifier potassium channel KCNH5via KCNH50.51 Voltage-gated potassium channel KCNC2via KCNC20.39

Reference

Authoritative identity, definition & identifiers.

Synonyms

hereditary developmental and epileptic encephalopathy, developmental and epileptic encephalopathy

References & data sources

Disease identity & definition — NLM Medical Subject Headings (MeSH), public domain
Clinical trials — ClinicalTrials.gov (U.S. National Library of Medicine)
Related entities are derived from literature co-mention (studied together) — associative, not causal.