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

Launched by UNIVERSITY OF COLORADO, DENVER · Sep 13, 2024

Keywords

ClinConnect Summary

This clinical trial is looking at the safety of a medication called fenfluramine for children under 2 years old who have Dravet syndrome, a type of epilepsy that causes prolonged seizures. Fenfluramine has already shown promise in older children by significantly reducing seizures, and researchers want to see if it can help younger children as well. To participate in this study, children must be between 12 and 23 months old and have a diagnosis of Dravet syndrome, which can be confirmed by a genetic test. They should also have tried at least one other anti-seizure medication without success.

If your child qualifies and joins the trial, they will be monitored closely by doctors to assess how well fenfluramine works and to ensure their safety. It’s important to note that some children may experience appetite loss while taking this medication, so careful assessments will be made to ensure the child is maintaining a healthy weight. This study is currently recruiting participants, and all decisions about inclusion will be made by a team of doctors to best support each child's needs.

Gender

ALL

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.