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

Part of paid clinical trials in Aurora, Colorado.

Sponsor
University of Colorado, Denver
Study ID
NCT06598449
Phase
PHASE4
Status
Recruiting
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Conditions

  • Children Under 2 Years
  • Dravet Syndrome (DS)

Eligibility Criteria

Sex
ALL
Age
12 Months - 24 Months
Healthy Volunteers
Not accepted

Interventions

  • fenfluramine — DRUG
    Clear, cherry flavored oral solution

Study Details

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.

Key Dates

Start date
Oct 22, 2024
Status verified
May 2026
Primary completion
Dec 31, 2026
Completion
Jul 31, 2027

Study Design

Enrollment
5 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: Fenfluramine
    The dose of FENFLURAMINE for the duration of the Treatment Period will range from 0.2 mg/kg/day to a maximum of 0.7 mg/kg/day, not to exceed a total daily dose of 26 mg/day; for patients concurrently being prescribed stiripentol, the maximum will be 0.4 mg/kg/day, not to exceed a total daily dose of 17 mg/day. Under this treatment plan, FENFLURAMINE will be administered twice daily in equally divided doses, with at least 8 hours and no more than 12 hours between doses in a single day.

Primary Outcome Measure

Percentage of participants that have at least one adverse event [ Time Frame: 12 months ]

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
Children's Hospital ColoradoAuroraColorado80045
Rebecca Rochowiak
[email protected]
720-777-0709
Kelly Knupp, MD (PRINCIPAL_INVESTIGATOR)

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By research site
Children's Hospital Colorado· Aurora, CO

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