Treatment of Refractory Infantile Spasms With Fenfluramine

Overview

This is a phase II clinical trial in which children with refractory infantile spasms (also called epileptic spasms or West syndrome) will be treated with fenfluramine, to evaluate efficacy, safety, and tolerability. Patients with infantile spasms that have not responded to treatment with vigabatrin and ACTH we will be invited to participate. Study participants will undergo baseline video-EEG, receive treatment with fenfluramine for 21 days, and then undergo repeat video-EEG to determine effectiveness. Patients with favorable response will have the opportunity to continue treatment for up to 6 months.

Full Title of Study: “A Phase II Study of Fenfluramine for Treatment of Refractory Infantile Spasms”

Study Type

  • Study Type: Interventional
  • Study Design
    • Allocation: Non-Randomized
    • Intervention Model: Single Group Assignment
    • Primary Purpose: Treatment
    • Masking: None (Open Label)
  • Study Primary Completion Date: December 31, 2021

Interventions

  • Drug: Fenfluramine
    • Open-label

Arms, Groups and Cohorts

  • Experimental: Fenfluramine treatment
    • Open label treatment with fenfluramine. Dosage will be titrated to 0.8 mg/kg/day, for an initial duration of 21 days. Patients with favorable response will have an option to continue treatment for up to 6 months.

Clinical Trial Outcome Measures

Primary Measures

  • Electroclinical response (Efficacy)
    • Time Frame: 12 months
    • Number of participants with resolution of epileptic spasms and hypsarrhythmia (if present at baseline) after 21 days of treatment, as determined by overnight video-electroencephalography (EEG) evaluation and caregiver seizure diary.

Secondary Measures

  • Computational electroencephalography response (Efficacy)
    • Time Frame: 12 months
    • Median and range of response quantified using the probability-weighted response index (PWRI), a novel computational electroencephalography measure of hypsarrhythmia burden.
  • Incidence of treatment emergent adverse events (Safety and tolerability)
    • Time Frame: 12 months
    • Detailed accounting of all treatment-emergent adverse events, including number of participants with clinically-significant valvulopathy and/or pulmonary hypertension, as determined by echocardiography.

Participating in This Clinical Trial

Inclusion Criteria

  • Children ages 3 to 36 months, inclusive – Clinical diagnosis of infantile spasms – Continued epileptic spasms despite adequate treatment with ACTH and vigabatrin. Exclusion Criteria:
  • Significant preexisting cardiovascular disease – Exposure to any cannabinoid product within 14 days of screening – Initiation or dose-titration of any second-line treatment for infantile spasms in the 14 days prior to screening. – Implantation of a vagal nerve simulator within 14 days of screening – Initiation and maintenance of the ketogenic diet within 3 months of screening
  • Gender Eligibility: All

    Minimum Age: 3 Months

    Maximum Age: 36 Months

    Are Healthy Volunteers Accepted: No

    Investigator Details

    • Lead Sponsor
      • University of California, Los Angeles
    • Provider of Information About this Clinical Study
      • Principal Investigator: Shaun Hussain, MD, Associate Professor – University of California, Los Angeles
    • Overall Official(s)
      • Shaun Hussain, MD, Principal Investigator, Univeristy of California, Los Angeles
    • Overall Contact(s)
      • Angela Martinez, 310-206-4037, angelamartinez@mednet.ucla.edu

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