Treatment of Dravet Syndrome With Fenfluramine (Expanded Access Protocol)

Overview

About this study

The purpose of this study is to provide access to fenfluramine for patients with intractable epilepsy associated with Dravet syndrome, and to evaluate the safety of fenfluramine.

 

Participation eligibility

Participant eligibility includes age, gender, type and stage of disease, and previous treatments or health concerns. Guidelines differ from study to study, and identify who can or cannot participate. There is no guarantee that every individual who qualifies and wants to participate in a trial will be enrolled. Contact the study team to discuss study eligibility and potential participation.

Inclusion Criteria:

  • Patients must be between 3 and 24 months old.
  • Each subject will be reviewed by the multi-PIs to ensure agreement that the subject has Dravet syndrome.
  • Genetic diagnosis of Dravet syndrome with intractable seizures despite failing all available medications for seizures.
  • SCN1A with a known or presumed pathogenic variant or VUS with a history of prolonged seizure.
  • Failure of at least one anti-seizure medication that is not a sodium channel blocker (lamotrigine, oxcarbazepine, carbamazepine, eslicarbazepine).
  • Inclusion of patients will be at the sole discretion of the multi-PIs.

Exclusion Criteria:

  • Patients with mild or greater mitral valve regurgitation and/or trace or greater aortic valve regurgitation.
  • 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.

Eligibility last updated 2/7/22. Questions regarding updates should be directed to the study team contact.

 

 

Participating Mayo Clinic locations

Study statuses change often. Please contact the study team for the most up-to-date information regarding possible participation.

Mayo Clinic Location Status Contact

Rochester, Minn.

Mayo Clinic principal investigator

Elaine Wirrell, M.D.

Contact us for the latest status

Contact information:

Precylla Ruiz

(507) 538-6606

Ruiz.Precylla@mayo.edu

More information

Publications

Publications are currently not available