Clinical Trial: Sodium Oxybate in Patients With Alternating Hemiplegia of Childhood (AHC-SO)

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: Single-center Phase I/II Trial of Sodium Oxybate in Patients With Alternating Hemiplegia of Childhood (AHC-SO Trial)

Brief Summary:

WHO: The investigators are recruiting children and young adults to participate in a research study who:

  1. Have been diagnosed with Alternating Hemiplegia of Childhood (AHC)
  2. Are between the ages of 6 months - 25 years old
  3. Have at least three 10-minute-long AHC episodes during a typical week
  4. Can commit to 12 weeks of completing of a daily log describing AHC episodes and to a multi-night hospital stay at the University of Utah Center for Clinical and Translational Science (CCTS)

WHY: The goal of this study is to evaluate whether the study drug can safely and effectively decrease AHC episodes and improve the quality of life of individuals with AHC.

WHAT and WHERE: This study involves at least 12 weeks of completing and submitting daily AHC Episode Logs and a five-day hospital stay at the University of Utah CCTS.

There are 4 phases of the study, and they include:

  1. Six weeks of daily log completion prior to starting study drug
  2. Five day stay at the University of Utah CCTS
  3. Six additional weeks of daily log completion while using study drug at home
  4. One day clinic visit to the University of Utah for follow up

COMPENSATION:

There is no fee to participate. All procedures performed specifically for this study will be covered by the study and will not be billed to you or your insurance company.

  • Time Duration of AHC Episodes [ Time Frame: Week 7 ]
  • Observed Safety Data During 5-day Hospitalization for Drug Dose Identification [ Time Frame: Week 7 ]
  • Number of Participants Who Reported Side Effects During Home Drug Maintenance Phase [ Time Frame: 1 year ]


    • Original Primary Outcome:

    • Time Duration of AHC Episodes [ Time Frame: Week 7 ]
    • Observed Safety Data During 5-day Hospitalization for Drug Dose Identification [ Time Frame: Week 7 ]
    • Reported side effects during 6-week home drug maintenance phase [ Time Frame: Week 7 ]


    Current Secondary Outcome:

    • Mean Daily Percentage of Time That Functional Status is Affected Due to Episodes [ Time Frame: Week 14 ]
    • Quality of Life Questionnaires [ Time Frame: Week 14 ]
    • Mood and Behavior Questionnaires [ Time Frame: Week 14 ]
    • Functional Skills Questionnaires [ Time Frame: Week 14 ]
    • Neuropsychological Tests [ Time Frame: Week 14 ]


    Original Secondary Outcome: Same as current

    Information By: University of Utah

    Dates:
    Date Received: June 30, 2009
    Date Started: August 2009
    Date Completion:
    Last Updated: March 3, 2014
    Last Verified: March 2014