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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT00931164
Recruitment Status : Completed
First Posted : July 2, 2009
Results First Posted : March 31, 2014
Last Update Posted : March 31, 2014
Sponsor:
Collaborators:
Alternating Hemiplegia of Childhood Foundation
Jazz Pharmaceuticals
Information provided by (Responsible Party):
Kathryn Swoboda, University of Utah

Tracking Information
First Submitted Date  ICMJE June 30, 2009
First Posted Date  ICMJE July 2, 2009
Results First Submitted Date  ICMJE January 8, 2014
Results First Posted Date  ICMJE March 31, 2014
Last Update Posted Date March 31, 2014
Study Start Date  ICMJE August 2009
Actual Primary Completion Date January 2011   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: March 3, 2014)
  • 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 Measures  ICMJE
 (submitted: July 1, 2009)
  • 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 ]
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: July 1, 2009)
  • 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 Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Sodium Oxybate in Patients With Alternating Hemiplegia of Childhood (AHC-SO)
Official Title  ICMJE 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.

The study drug will be dispensed to you free of charge. Meals and lodging at the study site will be provided free of charge for the study participant and one care provider. Additionally, the research team will assist subjects with booking and obtaining free transportation, such as an airline tickets, to and from the study site.

Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Phase 2
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Alternating Hemiplegia of Childhood
Intervention  ICMJE Drug: Sodium Oxybate
dosage is by weight
Study Arms  ICMJE Experimental: Sodium oxybate
  • The study is an open-label, Phase I/II trial designed to obtain additional safety and pharmacokinetic parameters for use of sodium oxybate in children and adolescents afflicted with AHC.
  • Given the limited number of children carrying the diagnosis of AHC, typical controls will not be available for our study. In lieu of this, the subjective recording of ictal episodes in the 6 week period prior to drug initiation will serve as reference in determining drug efficacy.
Intervention: Drug: Sodium Oxybate
Publications *

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: July 1, 2009)
6
Original Estimated Enrollment  ICMJE Same as current
Actual Study Completion Date  ICMJE September 2011
Actual Primary Completion Date January 2011   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

Subjects must meet all of the following inclusion criteria.

  • AHC classic criteria:

    • Onset of symptomatology prior to 18 months of age
    • Repeated attacks of hemiplegia involving either side of the body
    • Paroxysmal disturbances, including tonic or dystonic spells, oculomotor abnormalities, and various autonomic phenomena, during hemiplegic bouts or in isolation
    • Episodes of bilateral hemiplegia or quadriplegia starting either as generalization of a hemiplegic episode or bilateral from the beginning
    • Immediate disappearance of symptoms upon sleeping, with possible resumption 10-20 minutes after waking
    • Evidence of developmental delay and neurologic abnormalities including choreoathetosis, dystonia, or ataxia

In addition:

  • Provision of a complete daily event log for 6 weeks prior to initiation of sodium oxybate therapy, including the: (1) frequency, (2) duration, (3) type, (4) severity of episodes, and (5) therapies, if any, used in an attempt to abort the episode
  • Ages 6 months to 25 years at the time of study enrollment
  • Minimum of 3 AHC episodes weekly, lasting at least 10 minutes each, on average prior to study enrollment
  • Brain neuroimaging studies excluding alternative etiology for symptoms
  • Documented absence of epileptiform features on EEG during typical ictal events
  • Ability of primary caregivers of study participants to submit online daily AHC Episode Logs indicating frequency and duration of ictal episodes over the entire study period
  • Written informed consent from parents/guardians and assent from children 7 years or older
  • Girls/women > 12 years of age will be required to be on birth control continuously if they are considered at risk to become pregnant. Those not on birth control will be required to have a screening pregnancy test at baseline, and to confirm their willingness to practice birth control or abstain from sexual activity for the duration of treatment with study medication.

Exclusion Criteria:

Subjects who meet any of the following criteria will be excluded from participation.

  • History of hepatic insufficiency, renal insufficiency, significant respiratory disease, cardiac arrhythmia, congenital heart defect, hypertension, or ischemic stroke
  • History of allergy/sensitivity to sodium oxybate
  • Use of sodium oxybate within 30 days of study enrollment
  • Serious illness requiring systemic treatment and/or hospitalization within two weeks prior to study enrollment
  • Change in neurologic medication regimen within 30 days of study enrollment.
  • Inability to stay at the Center for Clinical and Translational Science (CCTS) for 5 days due to behavioral issues
  • Unwillingness or inability to travel to study site during the necessary 1 week titration period to determine the most appropriate dose of GHB for subsequent administration
  • Noncompliance with AHC Episode Log or study visit requirements
  • Sleep apnea not adequately treated with C-PAP and oxygen saturation monitoring prior to drug therapy initiation
  • Succinic semialdehyde dehydrogenase deficiency
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 6 Months to 25 Years   (Child, Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT00931164
Other Study ID Numbers  ICMJE 32164
103932
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Kathryn Swoboda, University of Utah
Study Sponsor  ICMJE University of Utah
Collaborators  ICMJE
  • Alternating Hemiplegia of Childhood Foundation
  • Jazz Pharmaceuticals
Investigators  ICMJE
Principal Investigator: Kathryn J. Swoboda, M.D. University of Utah/Primary Children's Medical Center
PRS Account University of Utah
Verification Date March 2014

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP