The FINTEPLA as an Anti-SUDEP Therapy in Dravet Syndrome (FAST-DS) Project.
The University of Texas Health Science Center, Houston
Summary
This study investigates cerebrovascular reactivity (CVR) and functional brain connectivity in Dravet Syndrome (DS) patients with convulsive seizures. Using functional MRI (fMRI), we will define differences in brain responses to CO₂ changes before administration of the drug Fintepla (Baseline), with a library of healthy controls and with those obtained after administration of Fintepla (Day \~60). Changes in CVR and their relation to ventilatory responses will also be assessed during fMRI.
Eligibility
- Age range
- 16+ years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: * DS patients (with or without SCN1A pathogenic mutations) * Generalized convulsive seizures Exclusion Criteria: * known cardiorespiratory, hepatic or renal disease, and/or * allergic reactions or other contraindications to fenfluramine and/or * on Stiripentol treatment, and/or * on serotonergic medications, and/or * contraindications to Midazolam anesthesia * taken the following drugs within 14 days: monoamine oxidase inhibitors (MAOIs), anti-depressants (Selective Serotonin Reuptake Inhibitors (SSRIs), Serotonin and norepinephrine reuptake inhibitors (SNRIs), and Tricy…
Interventions
- DrugFenfluramine treatment (Fintepla)
Participants will receive Fintepla starting at 0.2 mg/kg/day, up to a maximum of 0.6 mg/kg/day (or 5.9 mL/day, whichever is lower), per FDA guidelines. Dosing will be adjusted as tolerated. After Day \~60, the dose will be gradually tapered.
- DeviceHypercapnia Challenge using the device (RespirAct)
Participants will undergo a hypercapnia challenge using the RespirAct device during fMRI. The protocol includes alternating one minute blocks of controlled CO₂ increases and normocapnia under normoxic conditions.
Location
- The University of Texas Health Science Center at HoustonHouston, Texas