Dravet Syndrome
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CLINICAL_TRIALS trial_result · 2026-08-07Clinical Trial Update: A Study to Evaluate Safety, Tolerability, and Pharmacokinetics of Fenfluramine (Hydrochloride) in Infants 1 Year to Less Than 2 Years of Age With Dravet Syndrome [Phase 3]
Phase 3 clinical trial evaluating safety, tolerability, and pharmacokinetics of fenfluramine in infants aged 1 to less than 2 years with Dra…
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CLINICAL_TRIALS trial_result · 2026-06-15Clinical Trial Update: A Study to Evaluate Safety, Tolerability, and Pharmacokinetics of Fenfluramine (Hydrochloride) in Infants 1 Year to Less Than 2 Years of Age With Dravet Syndrome [Phase 3]
A Phase 3 trial evaluating safety, tolerability, and pharmacokinetics of fenfluramine hydrochloride in infants aged 1 year to less than 2 ye…
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CLINICAL_TRIALS trial_result · 2026-06-09Clinical Trial Results: A Study of Soticlestat as an Add-on Therapy in Children and Adults With Dravet Syndrome or Lennox-Gastaut Syndrome [Phase 3]
Phase 3 trial evaluating soticlestat as add-on therapy in children and adults with Dravet syndrome or Lennox-Gastaut syndrome has terminated…
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CLINICAL_TRIALS safety_alert · 2026-04-22Clinical Trial Update: Assessment of Potential for Chronic Liver Injury in Participants Treated With Epidiolex (Cannabidiol) Oral Solution [Phase 4]
This Phase 4 clinical trial update describes ongoing assessment of the potential for chronic liver injury in participants treated with Epidi…
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CLINICAL_TRIALS research · 2026-04-21Clinical Trial Update: ASCEND: Safety and Tolerability of ION337 for the Treatment of Dravet Syndrome [Phase 1, Phase 2]
Ongoing Phase 1/2 ASCEND trial evaluating the safety and tolerability of Ionis' antisense therapy ION337 for Dravet syndrome; trial status i…
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CLINICAL_TRIALS approval · 2026-04-20Clinical Trial Update: Treatment of Dravet Syndrome With Fenfluramine (Expanded Access Protocol)
The document notes that fenfluramine (Fintepla) has been approved for marketing for the treatment of Dravet Syndrome, including its use unde…
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CLINICAL_TRIALS research · 2026-04-16Clinical Trial Update: A Clinical Study to Evaluate the Safety and Efficacy of ETX101 in Infants and Children With SCN1A-Positive Dravet Syndrome [Phase 1, Phase 2]
A Phase 1/2 clinical study is evaluating the safety and efficacy of ETX101 in infants and children with SCN1A-positive Dravet syndrome, and …
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CLINICAL_TRIALS research · 2026-04-15Clinical Trial Update: A Phase 3, Placebo-Controlled Study to Investigate LP352 in Children and Adults With Dravet Syndrome (DS) [Phase 3]
A Phase 3, placebo-controlled study evaluating LP352 in children and adults with Dravet syndrome is currently recruiting.
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CLINICAL_TRIALS research · 2026-04-13Clinical Trial Update: A Double-blind Study Evaluating the Efficacy, Safety, and Tolerability of Zorevunersen in Patients With Dravet Syndrome [Phase 3]
Phase 3 double-blind trial evaluating Zorevunersen for Dravet syndrome is currently recruiting to assess efficacy, safety, and tolerability.
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CLINICAL_TRIALS research · 2026-04-03Clinical Trial Update: A Study to Evaluate Safety, Tolerability, and Pharmacokinetics of Fenfluramine (Hydrochloride) in Infants 1 Year to Less Than 2 Years of Age With Dravet Syndrome [Phase 3]
This Phase 3 clinical study is evaluating the safety, tolerability, and pharmacokinetics of fenfluramine hydrochloride in infants aged 1 yea…
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CLINICAL_TRIALS research · 2026-04-03Clinical Trial Update: The FINTEPLA as an Anti-SUDEP Therapy in Dravet Syndrome Project [Phase 4]
Phase 4 clinical trial update for FINTEPLA (fenfluramine) as an anti-SUDEP therapy in Dravet syndrome. The study is not yet recruiting.
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CLINICAL_TRIALS trial_result · 2026-03-19Clinical Trial Update: A Study of Soticlestat in Adults and Children With Rare Epilepsies [Phase 2]
Takeda reports termination of a Phase 2 study of Soticlestat in adults and children with rare epilepsies, with results posted.
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CLINICAL_TRIALS research · 2026-03-17Clinical Trial Update: A Study to Investigate the Transition of Children From 'Artisanal" Cannabidiol (CBD) to Epidiolex [Phase 4]
A Phase 4 study is evaluating how children with Dravet syndrome or Lennox-Gastaut syndrome transition from artisanal cannabidiol to Epidiole…
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CLINICAL_TRIALS research · 2026-02-27Clinical Trial Update: A PET-MRI Study of Serotoninergic Brainstem Pathway in Patients With Dravet Syndrome [NA]
This registry entry outlines a PET-MRI study of serotoninergic brainstem pathways in patients with Dravet syndrome and epilepsy. The trial s…
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CLINICAL_TRIALS research · 2026-02-24Clinical Trial Update: Multicentre Real-life Follow-up Study of Rare Epileptic Syndromes in Children and Adolescents
This multicentre real-life follow-up study aims to characterize rare epileptic syndromes in children and adolescents, including West syndrom…
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CLINICAL_TRIALS research · 2026-02-17Clinical Trial Update: Epidyolex® in Lennox Gastaut, Dravet Syndrome and Tuberous Sclerosis Complex: an Observational Study in ITALY
An observational study of Epidyolex (cannabidiol) in Lennox-Gastaut syndrome, Dravet syndrome, and tuberous sclerosis complex is currently r…
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CLINICAL_TRIALS research · 2026-02-04Clinical Trial Update: An Open-Label Extension Study of STK-001 for Patients With Dravet Syndrome [Phase 2]
Open-label extension update for STK-001 in Dravet syndrome within a Phase 2 trial; the study is active but not recruiting.
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CLINICAL_TRIALS research · 2026-02-03Clinical Trial Update: Longitudinal Study of Phenotypic and Developmental Severity in Patients With Dravet Syndrome With SCN1A Gene Mutation
A longitudinal observational study in Dravet syndrome patients with SCN1A mutation is currently recruiting to characterize phenotypic and de…
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CLINICAL_TRIALS approval · 2026-01-23Clinical Trial Update: Treatment Plan to Provide Expanded Access to Stiripentol for Patients With Dravet Syndrome
The document indicates that stiripentol has been approved for marketing and a plan to provide expanded-access treatment to patients with Dra…
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CLINICAL_TRIALS other · 2026-01-14Clinical Trial Update: Neurodevelopmental Impact of Epilepsy on Autonomic Function in Dravet Syndrome [NA]
Clinical trial update describing a study on how epilepsy-related neurodevelopmental factors affect autonomic function in Dravet Syndrome. Th…
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CLINICAL_TRIALS research · 2025-12-12Clinical Trial Update: A Clinical Study to Evaluate the Safety and Efficacy of ETX101, an AAV9-Delivered Gene Therapy in Children With SCN1A-positive Dravet Syndrome [Phase 1, Phase 2]
A clinical study evaluating the safety and efficacy of ETX101, an AAV9-delivered gene therapy, in children with SCN1A-positive Dravet syndro…
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CLINICAL_TRIALS trial_result · 2025-12-03Clinical Trial Update: Open-label, Long-term Safety Study of LP352 in Subjects With Developmental and Epileptic Encephalopathy [Phase 2]
This report covers a long-term, open-label Phase 2 safety study of LP352 in developmental and epileptic encephalopathy, including Dravet and…
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CLINICAL_TRIALS trial_result · 2025-11-17Clinical Trial Update: 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 [Phase 3]
Phase 3 study update reporting long-term safety data for ZX008 (fenfluramine hydrochloride) oral solution in children and adults with epilep…
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CLINICAL_TRIALS research · 2025-11-14Clinical Trial Update: A Clinical Study to Evaluate the Safety and Efficacy of ETX101, an AAV9-Delivered Gene Therapy in Children With SCN1A-positive Dravet Syndrome (Australia Only) [Phase 1, Phase 2]
Clinical trial update for ETX101, an AAV9-delivered gene therapy for SCN1A-positive Dravet syndrome, currently in Phase 1/Phase 2 in Austral…
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CLINICAL_TRIALS research · 2025-11-06Clinical Trial Update: Clinical Utility of Reduced EEG Home Monitoring in Fenfluramine Titration for Dravet and LGS [NA]
This clinical trial update describes a study on reduced EEG home monitoring during fenfluramine titration for Dravet syndrome and Lennox-Gas…