Summary

Eligibility
for people ages 12 months to 24 months (full criteria)
Location
at UCSF
Dates
study started
study ends around
Principal Investigator
by Joseph Sullivan, MD (ucsf)
Headshot of Joseph Sullivan
Joseph Sullivan

Description

Summary

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.

Official Title

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

Keywords

Dravet Syndrome (DS), Children Under 2 Years, Fenfluramine, Dravet Syndrome, Myoclonic Epilepsies, Syndrome

Eligibility

Locations

  • University of California San Francisco not yet accepting patients
    San Francisco California 94143 United States
  • Children's Hospital Colorado accepting new patients
    Aurora Colorado 80045 United States
  • Mayo Clinic not yet accepting patients
    Rochester Minnesota 55905 United States

Lead Scientist at University of California Health

  • Joseph Sullivan, MD (ucsf)
    Professor, Neurology, School of Medicine. Authored (or co-authored) 65 research publications

Details

Status
accepting new patients at some sites,
but this study is not currently recruiting here
Start Date
Completion Date
(estimated)
Sponsor
University of Colorado, Denver
ID
NCT06598449
Phase
Phase 4 research study
Study Type
Interventional
Participants
Expecting 12 study participants
Last Updated