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

NCT00931164 | Completed Phase 1 Results DMC

• 2 other identifiers: 32164103932
• Study Type: interventional
• Target: 6
• Locations: 1 country
• Sites: 1

Brief Summary

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

1. 1.Have been diagnosed with Alternating Hemiplegia of Childhood (AHC)
2. 2.Are between the ages of 6 months - 25 years old
3. 3.Have at least three 10-minute-long AHC episodes during a typical week
4. 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)
5. 5.Six weeks of daily log completion prior to starting study drug
6. 6.Five day stay at the University of Utah CCTS
7. 7.Six additional weeks of daily log completion while using study drug at home
8. 8.One day clinic visit to the University of Utah for follow up

Conditions

Alternating Hemiplegia of Childhood

Keywords

phase I/II study; effects of sodium oxybate; cohort of 6 children and young adults; AHC

Outcome Measures

Primary Outcomes (3)

• Time Duration of AHC Episodes

  Week 7

• Observed Safety Data During 5-day Hospitalization for Drug Dose Identification

  Week 7

• Number of Participants Who Reported Side Effects During Home Drug Maintenance Phase

  1 year

Secondary Outcomes (5)

• Mean Daily Percentage of Time That Functional Status is Affected Due to Episodes

  Week 14

• Quality of Life Questionnaires

  Week 14

• Mood and Behavior Questionnaires

  Week 14

• Functional Skills Questionnaires

  Week 14

• Neuropsychological Tests

  Week 14

Study Arms (1)

Sodium oxybate

EXPERIMENTAL

* 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

Interventions

Sodium Oxybate DRUG

dosage is by weight

Sodium oxybate

Eligibility Criteria

• Age: 6 Months - 25 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• University of Utah: lead
• Alternating Hemiplegia of Childhood Foundation: collaborator
• Jazz Pharmaceuticals: collaborator

Study Sites (1)

University of Utah Hospital

Salt Lake City, Utah, 84132, United States

Location

Related Publications (43)

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MeSH Terms

Conditions

Alternating hemiplegia of childhood

Interventions

Sodium Oxybate

Intervention Hierarchy (Ancestors)

Hydroxybutyrates; Butyrates; Acids, Acyclic; Carboxylic Acids; Organic Chemicals; Hydroxy Acids

Results Point of Contact

• Title: Dr. Kathryn Swoboda
• Organization: University of Utah

taran@genetics.utah.edu

801-585-9717

Study Officials

• Kathryn J. Swoboda, M.D.

  University of Utah/Primary Children's Medical Center

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Associate Professor, Neurology and Pediatrics Director, Pediatric Motor Disorders Research Program

Study Record Dates

• First Submitted: June 30, 2009
• First Posted: July 2, 2009
• Study Start: August 1, 2009
• Primary Completion: January 1, 2011
• Study Completion: September 1, 2011
• Last Updated: March 31, 2014
• Results First Posted: March 31, 2014

Record last verified: 2014-03

Locations

US (1)