Actively Recruiting
Assessment of Safety of the Use of Fenfluramine in Children With Dravet Syndrome Under 24 Months of Age
Led by University of Colorado, Denver · Updated on 2024-12-10
12
Participants Needed
3
Research Sites
140 weeks
Total Duration
On this page
Sponsors
U
University of Colorado, Denver
Lead Sponsor
U
UCB Pharma
Collaborating Sponsor
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
Assessment of Safety of the Use of Fenfluramine in Children With Dravet Syndrome Under 24 Months of Age
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Patient is aged between 12 and 23 months
- Clinical diagnosis of Dravet syndrome or known/presumed pathogenic SCN1A variant
- History of prolonged seizure
- Failure of at least one anti-seizure medication that is not a sodium channel blocker
- Submission of clinical statement including gender, age, diagnosis, genetic variant, co-morbidities, seizure history, therapies, and reasons for treatment request
- Echocardiogram results submitted before final approval
- Agreement by multiple principal investigators that patient has Dravet syndrome
You will not qualify if you...
- Presence of mild or greater mitral valve regurgitation or trace or greater aortic valve regurgitation
- Failure to thrive defined as weight less than 2nd percentile or lack of weight gain crossing two or more major percentiles not matching length
- Inclusion at discretion of multiple principal investigators by majority vote
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 3 locations
1
University of California San Francisco
San Francisco, California, United States, 94143
Not Yet Recruiting
2
Children's Hospital Colorado
Aurora, Colorado, United States, 80045
Actively Recruiting
3
Mayo Clinic
Rochester, Minnesota, United States, 55905
Not Yet Recruiting
Research Team
R
Rebecca Rochowiak
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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