Trial Outcomes & Findings for Sodium Oxybate in Patients With Alternating Hemiplegia of Childhood (AHC-SO) (NCT NCT00931164)

Results Overview

Recruitment status

COMPLETED

Study phase

PHASE1/PHASE2

Target enrollment

6 participants

Primary outcome timeframe

Week 7

Results posted on

2014-03-31

Participant Flow

Participant milestones

Participant milestones
Measure	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. Sodium Oxybate : dosage is by weight
Overall Study STARTED	6
Overall Study COMPLETED	6
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

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

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Sodium Oxybate n=6 Participants * 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
Age, Categorical <=18 years	6 Participants n=93 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=93 Participants
Age, Categorical >=65 years	0 Participants n=93 Participants
Age, Continuous	6.4 years n=93 Participants
Sex: Female, Male Female	4 Participants n=93 Participants
Sex: Female, Male Male	2 Participants n=93 Participants
Region of Enrollment United States	5 participants n=93 Participants
Region of Enrollment Ireland	1 participants n=93 Participants

PRIMARY outcome

Timeframe: Week 7

Outcome measures

Outcome data not reported

PRIMARY outcome

Timeframe: Week 7

Outcome measures

Outcome data not reported

PRIMARY outcome

Timeframe: 1 year

Outcome measures

Outcome measures
Measure	Sodium Oxybate n=6 Participants * 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
Number of Participants Who Reported Side Effects During Home Drug Maintenance Phase	4 participants

SECONDARY outcome

Timeframe: Week 14

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Week 14

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Week 14

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Week 14

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Week 14

Outcome measures

Outcome data not reported

Adverse Events

Sodium Oxybate

Serious events: 1 serious events

Other events: 6 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Sodium Oxybate n=6 participants at risk * 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
Gastrointestinal disorders Loss of consciousness/vomit event	16.7% 1/6 • Number of events 2
Respiratory, thoracic and mediastinal disorders Respiratory Distress Event	16.7% 1/6 • Number of events 2

Other adverse events

Other adverse events
Measure	Sodium Oxybate n=6 participants at risk * 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
Respiratory, thoracic and mediastinal disorders Desaturation	16.7% 1/6 • Number of events 10
Gastrointestinal disorders Diarrhea	33.3% 2/6 • Number of events 2
Infections and infestations Otitis Media	16.7% 1/6 • Number of events 1
Gastrointestinal disorders Vomiting	33.3% 2/6 • Number of events 2
Respiratory, thoracic and mediastinal disorders Upper Respiratory Infection	50.0% 3/6 • Number of events 4
General disorders Heat Rash	33.3% 2/6 • Number of events 2
Respiratory, thoracic and mediastinal disorders Bronchitis	16.7% 1/6 • Number of events 1
General disorders Fever	16.7% 1/6 • Number of events 2

Additional Information

Dr. Kathryn Swoboda

University of Utah

Phone: 801-585-9717

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place