{
  "abstract": "Objectives Fenfluramine (FFA) efficacy trajectories in patients with Lennox-Gastaut syndrome (LGS; a developmental and epileptic encephalopathy) randomized to placebo/FFA during a clinical trial RCT and transitioned to FFA in the OLE are described.Methods Efficacy outcomes, including median percentage change from baseline in frequency of seizures associated with a fall (SF), number of days without SF, SF responder rates (≥50%, ≥75%), and Clinical Global Impression—Improvement scale scores, were assessed in patients who transitioned to FFA 0.2 mg/kg/d following the 14-week RCT.Results The RCT-placebo group (n=87) maintained a consistent median SF percentage change from baseline (-12.6% to -7.2%). Within Month 1 of the OLE, median SF percentage change from baseline in RCT-placebo (n=85, -29.0%) became numerically comparable to RCT-FFA (n=156, -25.4%). In all other assessed outcomes in the OLE, RCT-placebo and RCT-FFA were numerically comparable in efficacy.Conclusions Patients previously randomized to placebo exhibited numerical improvements in efficacy following transition to FFA treatment. Regression to the mean was not observed, suggesting that changes could be attributed to FFA treatment. Median SF percentage change in RCT-placebo occurred during OLE Month 1, confirming rapid onset of efficacy. Analysis of time to efficacy in a larger population of patients with LGS would be beneficial.scott.bergfeld@medvalsci.com",
  "authors": [
    {
      "affiliations": [
        "UCB, Slough, UK"
      ],
      "name": "Whelton Michelle"
    },
    {
      "affiliations": [
        "Member, EpiCARE",
        "Necker Enfants Malades Hospital, Institut Imagine, Université Paris Cité, Paris, FR"
      ],
      "name": "Nabbout Rima"
    },
    {
      "affiliations": [
        "NYU Langone Medical Center, New York, NY, USA"
      ],
      "name": "Devinsky Orrin"
    },
    {
      "affiliations": [
        "Member, EpiCARE",
        "University of Leuven, Leuven, BE"
      ],
      "name": "Lagae Lieven"
    },
    {
      "affiliations": [
        "Meyer Children’s Hospital IRCCS",
        "University of Florence, Florence, IT"
      ],
      "name": "Guerrini Renzo"
    },
    {
      "affiliations": [
        "Paediatric Neurosciences Research Group, Royal Hospital for Children, Glasgow, UK"
      ],
      "name": "Zuberi Sameer M"
    },
    {
      "affiliations": [
        "UCB, Smyrna, GA, USA"
      ],
      "name": "Healy Patrick"
    },
    {
      "affiliations": [
        "UCB, Colombes, FR"
      ],
      "name": "Langlois Mélanie"
    },
    {
      "affiliations": [
        "UCB, Colombes, FR"
      ],
      "name": "Lothe Amélie"
    },
    {
      "affiliations": [
        "University of Colorado, Children’s Hospital Colorado, Aurora, CO, USA"
      ],
      "name": "Knupp Kelly G"
    }
  ],
  "title": "176 Fenfluramine Efficacy trajectories from randomized controlled trial to open-label extension in lennox-gastaut patients",
  "uid": "9e7a452f-5940-58e8-bbcb-811eda08f738"
}