RECRUITING

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

Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

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. 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

Quick Facts

Study Start:2024-10-22
Study Completion:2027-07
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT06598449

Participation Criteria

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.

Ages Eligible for Study:12 Months to 24 Months
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:CHILD
Inclusion CriteriaExclusion Criteria
  1. Age 18 years or older
  2. Willing and able to provide informed consent
  3. Able to understand and follow study procedures
  4. Stable medical condition
  1. 1. Patients with mild or greater mitral valve regurgitation and/or trace or greater aortic valve regurgitation will not be eligible for participation. The clinical statement can be submitted first for initial, conditional approval and then ECHO results can be submitted at a later date for final approval.
  2. 2. Patients with failure to thrive will not be eligible for participation as fenfluramine can suppress appetite and has a risk for weight loss. Failure to thrive will be evaluated on the following criteria:
  3. 1. Weight less than the 2nd percentile.
  4. 2. Lack of weight gain that crosses two or more of the major percentile lines and is not congruent with length.

Contacts and Locations

Study Contact

Rebecca Rochowiak
CONTACT
720-777-0717
rebecca.rochowiak@childrenscolorado.org

Principal Investigator

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

Study Locations (Sites)

University of California San Francisco
San Francisco, California, 94143
United States
Children's Hospital Colorado
Aurora, Colorado, 80045
United States
Mayo Clinic
Rochester, Minnesota, 55905
United States

Collaborators and Investigators

Sponsor: 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

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2024-10-22
Study Completion Date2027-07

Study Record Updates

Study Start Date2024-10-22
Study Completion Date2027-07

Terms related to this study

Keywords Provided by Researchers

  • Fenfluramine
  • Dravet Syndrome
  • Children under 2 years

Additional Relevant MeSH Terms

  • Dravet Syndrome (DS)
  • Children Under 2 Years