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

NCT06598449 · Status: RECRUITING · Phase: PHASE4 · Type: INTERVENTIONAL · Enrollment: 5

Last updated 2026-05-15

No results posted yet for this study

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 an intermediate size patient population expanded access protocol to allow access to fenfluramine for children under 24 months of age.

Conditions

  • Dravet Syndrome (DS)
  • Children Under 2 Years

Interventions

DRUG

fenfluramine

Clear, cherry flavored oral solution

Sponsors & Collaborators

  • University of Colorado, Denver

    lead OTHER

Principal Investigators

  • Kelly Knupp, MD · University of Colorado, Denver

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
12 Months
Max Age
24 Months
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-10-22
Primary Completion
2026-12-31
Completion
2027-07-31
FDA Drug
Yes

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06598449 on ClinicalTrials.gov