Using fenfluramine to treat young children with Dravet syndrome

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

This study is testing if fenfluramine can safely help young children with Dravet syndrome reduce their seizures.

Quick facts

• Phase: Phase 4
• Study type: Interventional
• Enrollment: 12 (estimated)
• Ages: 12 Months to 24 Months
• Sex: All
• Sponsor: University of Colorado, Denver (Academic / other)
• Locations: 3 sites (San Francisco, California and 2 other locations)
• Trial ID: NCT06598449 on ClinicalTrials.gov

What this trial studies

This clinical trial assesses the safety and efficacy of fenfluramine in children under 24 months diagnosed with Dravet syndrome, a severe form of epilepsy. The study aims to provide access to fenfluramine, which has shown promising results in older children, by evaluating its effects on seizure frequency and overall safety in a younger population. Eligible participants must have a confirmed diagnosis of Dravet syndrome and a history of prolonged seizures, with prior treatment failures on other anti-seizure medications. The trial will involve a thorough review of each patient's medical history and cardiac health before treatment initiation.

Who should consider this trial

Good fit: Ideal candidates are children aged 12 to 23 months with a confirmed diagnosis of Dravet syndrome and a history of prolonged seizures.

Not a fit: Patients with mild or greater mitral valve regurgitation may not benefit from this study due to exclusion criteria.

Why it matters

Potential benefit: If successful, this treatment could significantly reduce seizure frequency and improve the quality of life for young children with Dravet syndrome.

How similar studies have performed: Previous studies have shown success with fenfluramine in older children with Dravet syndrome, indicating potential effectiveness in younger patients as well.

Eligibility criteria

Inclusion Criteria:

Treating physicians must submit a clinical statement of potential benefit to the lead site for review of the multi-PIs, which includes patient's gender, age, diagnosis, genetic pathogenic variant, co-morbidities, seizure history, prior and current therapies, response to prior therapies and reason for request. Echocardiogram (ECHO) results must also be submitted to the lead site prior to final approval. Patients must be between 12 and 23 months old to be eligible. Each subject will be reviewed by the multi-PIs to ensure agreement that the subject has Dravet syndrome. Additional inclusion criteria:

1. SCN1A with a known or presumed pathogenic variant or VUS with a history of prolonged seizure or a clinical diagnosis of Dravet syndrome.
2. Failure of at least one anti-seizure medication that is not a sodium channel blocker (lamotrigine, oxcarbazepine, carbamazepine, eslicarbazepine)

Exclusion Criteria:

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. 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:

   1. Weight less than the 2nd percentile.
   2. Lack of weight gain that crosses two or more of the major percentile lines and is not congruent with length.

Inclusion of patients will be at the sole discretion of the multi-PIs based on a majority vote.

Where this trial is running

San Francisco, California and 2 other locations

• University of California San Francisco — San Francisco, California, United States (Not_yet_recruiting)
• Children's Hospital Colorado — Aurora, Colorado, United States (Recruiting)
• Mayo Clinic — Rochester, Minnesota, United States (Not_yet_recruiting)

Study contacts

• Principal investigator: Kelly Knupp, MD — University of Colorado, Denver
• Study coordinator: Rebecca Rochowiak

Conditions Dravet Syndrome; Children Under 2 Years; Fenfluramine; Children under 2 years