The FINTEPLA as an Anti-SUDEP Therapy in Dravet Syndrome Project

Phase 4Not Yet RecruitingNCT07112365

The University of Texas Health Science Center, Houston25 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

Purpose

PREVENTION

Masking

NONE

Enrollment

Conditions

Dravet Syndrome

Interventions

Fenfluramine treatment (Fintepla)DRUG

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.

Hypercapnia Challenge using the device (RespirAct)DEVICE

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.

Eligibility

Sex: ALLMin age: 16 Years

Medical Language ↔ Plain English

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 Tricyclic antidepressants (TCAs)), St. John's Wort, Tryptophan, and Dextromethorphan

Locations (1)

The University of Texas Health Science Center at Houston

Houston, Texas, United States

Outcomes

Primary Outcomes

Change in blood oxygenation level in response to CO₂

Measures how much blood oxygenation level dependent (BOLD) signal changes in response to CO₂, using fMRI.

Time frame: Baseline, Day ~60

Change in speed of cerebrovascular response to CO₂

Measures how quickly the brain responds to CO₂ exposure during fMRI.

Time frame: Baseline, Day ~60

Secondary Outcomes

Change in breathing response to CO₂

Assesses how the participant's breathing rate changes when CO₂ levels are increased.

Time frame: Baseline, Day ~60

Central Contacts

Samden Lhatoo, MD, FRCP

CONTACT

(713) 500-7106 Samden.D.Lhatoo@uth.tmc.edu

Sandhya Rani

CONTACT

(713) 500-7499 M.R.Sandhya.Rani@uth.tmc.edu

Data from ClinicalTrials.gov

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