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Purpose

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.

Condition

Eligibility

Eligible Ages
Over 16 Years
Eligible Sex
All
Accepts 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 Tricyclic antidepressants (TCAs)), St. John's Wort, Tryptophan, and Dextromethorphan

Study Design

Phase
Phase 4
Study Type
Interventional
Allocation
N/A
Intervention Model
Single Group Assignment
Primary Purpose
Prevention
Masking
None (Open Label)

Arm Groups

ArmDescriptionAssigned Intervention
Experimental
Fintepla plus Hypercapnia Challenge using the Respiract Device
  • Drug: Fenfluramine 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.
    Other names:
    • Fenfluramine
  • Device: Hypercapnia 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.
    Other names:
    • RespirAct

Recruiting Locations

The University of Texas Health Science Center at Houston
Houston, Texas 77030
Contact:
Samden Lhatoo, MD, FRCP
(713) 500-7106
Samden.D.Lhatoo@uth.tmc.edu

More Details

Status
Recruiting
Sponsor
The University of Texas Health Science Center, Houston

Study Contact

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

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.