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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

Study Type Interventional
Study Design Allocation: N/A;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Alternating Hemiplegia of Childhood
Intervention Drug: Sodium Oxybate
Enrollment 6
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Sodium Oxybate
Hide Arm/Group Description
  • 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.

Sodium Oxybate : dosage is by weight

Period Title: Overall Study
Started 6
Completed 6
Not Completed 0
Arm/Group Title Sodium Oxybate
Hide Arm/Group Description
  • 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.

Sodium Oxybate : dosage is by weight

Overall Number of Baseline Participants 6
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 6 participants
<=18 years
6
 100.0%
Between 18 and 65 years
0
   0.0%
>=65 years
0
   0.0%
Age, Continuous  
Mean (Full Range)
Unit of measure:  Years
Number Analyzed 6 participants
6.4
(1.1 to 11.2)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 6 participants
Female
4
  66.7%
Male
2
  33.3%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 6 participants
United States 5
Ireland 1
1.Primary Outcome
Title Time Duration of AHC Episodes
Hide Description [Not Specified]
Time Frame Week 7
Outcome Measure Data Not Reported
2.Primary Outcome
Title Observed Safety Data During 5-day Hospitalization for Drug Dose Identification
Hide Description [Not Specified]
Time Frame Week 7
Outcome Measure Data Not Reported
3.Primary Outcome
Title Number of Participants Who Reported Side Effects During Home Drug Maintenance Phase
Hide Description [Not Specified]
Time Frame 1 year
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Sodium Oxybate
Hide Arm/Group Description:
  • 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.

Sodium Oxybate : dosage is by weight

Overall Number of Participants Analyzed 6
Measure Type: Number
Unit of Measure: participants
4
4.Secondary Outcome
Title Mean Daily Percentage of Time That Functional Status is Affected Due to Episodes
Hide Description [Not Specified]
Time Frame Week 14
Outcome Measure Data Not Reported
5.Secondary Outcome
Title Quality of Life Questionnaires
Hide Description [Not Specified]
Time Frame Week 14
Outcome Measure Data Not Reported
6.Secondary Outcome
Title Mood and Behavior Questionnaires
Hide Description [Not Specified]
Time Frame Week 14
Outcome Measure Data Not Reported
7.Secondary Outcome
Title Functional Skills Questionnaires
Hide Description [Not Specified]
Time Frame Week 14
Outcome Measure Data Not Reported
8.Secondary Outcome
Title Neuropsychological Tests
Hide Description [Not Specified]
Time Frame Week 14
Outcome Measure Data Not Reported
Time Frame [Not Specified]
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Sodium Oxybate
Hide Arm/Group Description
  • 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.

Sodium Oxybate : dosage is by weight

All-Cause Mortality
Sodium Oxybate
Affected / at Risk (%)
Total   --/--    
Hide Serious Adverse Events
Sodium Oxybate
Affected / at Risk (%) # Events
Total   1/6 (16.67%)    
Gastrointestinal disorders   
Loss of consciousness/vomit event *  1/6 (16.67%)  2
Respiratory, thoracic and mediastinal disorders   
Respiratory Distress Event   1/6 (16.67%)  2
Indicates events were collected by systematic assessment
*
Indicates events were collected by non-systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Sodium Oxybate
Affected / at Risk (%) # Events
Total   6/6 (100.00%)    
Gastrointestinal disorders   
Diarrhea *  2/6 (33.33%)  2
Vomiting *  2/6 (33.33%)  2
General disorders   
Heat Rash *  2/6 (33.33%)  2
Fever *  1/6 (16.67%)  2
Infections and infestations   
Otitis Media   1/6 (16.67%)  1
Respiratory, thoracic and mediastinal disorders   
Desaturation   1/6 (16.67%)  10
Upper Respiratory Infection   3/6 (50.00%)  4
Bronchitis   1/6 (16.67%)  1
Indicates events were collected by systematic assessment
*
Indicates events were collected by non-systematic assessment
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Dr. Kathryn Swoboda
Organization: University of Utah
Phone: 801-585-9717
EMail: taran@genetics.utah.edu
Publications:
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.
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.
Layout table for additonal information
Responsible Party: Kathryn Swoboda, University of Utah
ClinicalTrials.gov Identifier: NCT00931164    
Other Study ID Numbers: 32164
103932
First Submitted: June 30, 2009
First Posted: July 2, 2009
Results First Submitted: January 8, 2014
Results First Posted: March 31, 2014
Last Update Posted: March 31, 2014