NCT06598449 · University of Colorado, Denver
Assessment of Safety of the Use of Fenfluramine in Children With Dravet Syndrome Under 24 Months of Age
What this study is about
Dravet syndrome is a genetic epilepsy associated with pathogenic variants in SCN1A that codes for Nav1.1, a protein necessary for sodium channels. Children with Dravet syndrome classically present in the first year of life with prolonged seizures, often hemiclonic and in the setting of fever or temperature changes such as getting in or out of bath water.
View original scientific description
Dravet syndrome is a genetic epilepsy associated with pathogenic variants in SCN1A that codes for Nav1.1, a protein necessary for sodium channels. Children with Dravet syndrome classically present in the first year of life with prolonged seizures, often hemiclonic and in the setting of fever or temperature changes such as getting in or out of bath water. Many anti-seizure medications are sodium channel blockers and exacerbate seizures in this patient population.
Interventions
DRUG
fenfluramine
Clear, cherry flavored oral solution
Primary outcome measures
Percentage of participants that have at least one adverse event
Time frame: 12 months
A description of the percentage of participants that have at least one adverse event.
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
Inclusion criteria
- Treating physicians must submit a clinical statement of potential benefit to the lead site for review of the multi-PIs, which includes patient's gender, age, diagnosis, genetic pathogenic variant, co-morbidities, seizure history, prior and current therapies, response to prior therapies and reason for request. Echocardiogram (ECHO) results must also be submitted to the lead site prior to final approval. Patients must be between 12 and 23 months old to be eligible. Each subject will be reviewed by the multi-PIs to ensure agreement that the subject has Dravet syndrome. Additional inclusion criteria: 1. SCN1A with a known or presumed pathogenic variant or VUS with a history of prolonged seizure or a clinical diagnosis of Dravet syndrome. 2. Failure of at least one anti-seizure medication that is not a sodium channel blocker (lamotrigine, oxcarbazepine, carbamazepine, eslicarbazepine)
Exclusion criteria
- Patients with mild or greater mitral valve regurgitation and
Where
- Aurora, Colorado
Related conditions & keywords
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Data: ClinicalTrials.gov · synced May 15, 2026 · Source of record for eligibility and locations