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

This study has been completed.
Sponsor:
Collaborators:
Alternating Hemiplegia of Childhood Foundation
Jazz Pharmaceuticals
Information provided by (Responsible Party):
Kathryn Swoboda, University of Utah
ClinicalTrials.gov Identifier:
NCT00931164
First received: June 30, 2009
Last updated: March 3, 2014
Last verified: March 2014
  Purpose

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.


Condition Intervention Phase
Alternating Hemiplegia of Childhood
Drug: Sodium Oxybate
Phase 1
Phase 2

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Single-center Phase I/II Trial of Sodium Oxybate in Patients With Alternating Hemiplegia of Childhood (AHC-SO Trial)

Resource links provided by NLM:


Further study details as provided by University of Utah:

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

Secondary Outcome Measures:
  • Mean Daily Percentage of Time That Functional Status is Affected Due to Episodes [ Time Frame: Week 14 ] [ Designated as safety issue: Yes ]
  • Quality of Life Questionnaires [ Time Frame: Week 14 ] [ Designated as safety issue: Yes ]
  • Mood and Behavior Questionnaires [ Time Frame: Week 14 ] [ Designated as safety issue: Yes ]
  • Functional Skills Questionnaires [ Time Frame: Week 14 ] [ Designated as safety issue: Yes ]
  • Neuropsychological Tests [ Time Frame: Week 14 ] [ Designated as safety issue: Yes ]

Enrollment: 6
Study Start Date: August 2009
Study Completion Date: September 2011
Primary Completion Date: January 2011 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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.
Drug: Sodium Oxybate
dosage is by weight

  Eligibility

Ages Eligible for Study:   6 Months to 25 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00931164

Locations
United States, Utah
University of Utah Hospital
Salt Lake City, Utah, United States, 84132
Sponsors and Collaborators
University of Utah
Alternating Hemiplegia of Childhood Foundation
Jazz Pharmaceuticals
Investigators
Principal Investigator: Kathryn J. Swoboda, M.D. University of Utah/Primary Children's Medical Center
  More Information

Publications:
Bourgeois M, Aicardi J, Goutieres F. Alternating hemiplegia of childhood. J Pediatr. 1993;122:673-679.
Bourgeois M, Aicardi J. The treatment of alternating hemiplegia of childhood with flunarizine: experience with 17 patients. Alternating Hemiplegia of Childhood, edited by F Andermann, J Aicardi, and F Vigevano. 1995;191-194.
Katchan B, Mamelak M, Hwang P. Alternating Hemiplegia of Childhood: Rationale for the use of Gammahydroxybutyrate, Reprints from the 10th International Child Neurology Congress, Montreal, June 2006.
Mikati M. Alternating hemiplegia of childhood. Pediatr Neurol. 1999;21:764.
Ricci S. Sleep studies of children with alternating hemiplegia of childhood. Alternating Hemiplegia of Childhood, edited by Andermann, Aicardi, and Vigevano. 1995;95-98.
Silver K, Andermann F. Alternating hemiplegia of childhood: treatment with flunarizine. Alternating Hemiplegia of Childhood, edited by F Andermann, J Aicardi, and F Vigevano. 1995;195-198.

Responsible Party: Kathryn Swoboda, Associate Professor, Neurology and Pediatrics Director, Pediatric Motor Disorders Research Program, University of Utah
ClinicalTrials.gov Identifier: NCT00931164     History of Changes
Other Study ID Numbers: 32164, 103932
Study First Received: June 30, 2009
Results First Received: January 8, 2014
Last Updated: March 3, 2014
Health Authority: United States: Food and Drug Administration

Keywords provided by University of Utah:
phase I/II study
effects of sodium oxybate
cohort of 6 children and young adults
AHC

Additional relevant MeSH terms:
Hemiplegia
Paralysis
Neurologic Manifestations
Nervous System Diseases
Signs and Symptoms
Sodium Oxybate
Adjuvants, Anesthesia
Central Nervous System Agents
Therapeutic Uses
Pharmacologic Actions
Anesthetics, Intravenous
Anesthetics, General
Anesthetics
Central Nervous System Depressants
Physiological Effects of Drugs

ClinicalTrials.gov processed this record on July 24, 2014