Using fenfluramine to treat young children with Dravet syndrome

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

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.

Why it matters

Who can join: the full rules

Inclusion Criteria:

Treating physicians must submit a clinical statement of potential benefit, 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. 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.

Where it's running

• Children's Hospital Colorado
  Aurora, Colorado, United States Recruiting

Study contacts

• Principal investigator: Kelly Knupp, MD — University of Colorado, Denver
• Study coordinator: Rebecca Rochowiak
• Email: rebecca.rochowiak@childrenscolorado.org
• Phone: 720-777-0717

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.