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. This creates some limitations in medication choices for this patient population. Recently fenfluramine was approved for use in Dravet syndrome for people 2 years and older. Randomized studies demonstrated a 74.9% reduction of convulsive motor seizures compared to 19.2% in the placebo group. Additionally, 16% of children treated with fenfluramine were seizure free. Fenfluramine is likely to be as effective in children under the age of 2 years. The current study has proposed a treatment protocol to allow access to fenfluramine for children under 24 months of age.
Dravet Syndrome (DS), Children Under 2 Years
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. This creates some limitations in medication choices for this patient population. Recently fenfluramine was approved for use in Dravet syndrome for people 2 years and older. Randomized studies demonstrated a 74.9% reduction of convulsive motor seizures compared to 19.2% in the placebo group. Additionally, 16% of children treated with fenfluramine were seizure free. Fenfluramine is likely to be as effective in children under the age of 2 years. The current study has proposed a treatment protocol to allow access to fenfluramine for children under 24 months of age.
Assessment of Safety of the Use of Fenfluramine in Children With Dravet Syndrome Under 24 Months of Age
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University of California San Francisco, San Francisco, California, United States, 94143
Children's Hospital Colorado, Aurora, Colorado, United States, 80045
Mayo Clinic, Rochester, Minnesota, United States, 55905
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
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12 Months to 24 Months
ALL
No
University of Colorado, Denver,
Kelly Knupp, MD, PRINCIPAL_INVESTIGATOR, University of Colorado - Anschutz Medical Campus
Joseph Sullivan, MD, PRINCIPAL_INVESTIGATOR, University of California, San Francisco
Elaine Wirrel, MD, PRINCIPAL_INVESTIGATOR, Mayo Clinic
2027-07