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

• Register: ClinicalTrials.gov
• Last refreshed on: 19 October 2017
• Main ID: NCT00931164
• Date of registration: 30/06/2009
• Prospective Registration: Yes
• Primary sponsor: University of Utah
• Public title: Sodium Oxybate in Patients With Alternating Hemiplegia of Childhood (AHC-SO) AHC-SO
• Scientific title: Single-center Phase I/II Trial of Sodium Oxybate in Patients With Alternating Hemiplegia of Childhood (AHC-SO Trial)
• Date of first enrolment: August 2009
• Target sample size: 6
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT00931164
• Study type: Interventional
• Phase: Phase 1/Phase 2

Countries of recruitment

United States

Contacts

• Name: Kathryn J. Swoboda, M.D.
• Affiliation: University of Utah/Primary Children's Medical Center

Key inclusion & exclusion 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



Age minimum: 6 Months
Age maximum: 25 Years
Gender: All

Health Condition(s) or Problem(s) studied

Alternating Hemiplegia of Childhood

Intervention(s)

Drug: Sodium Oxybate

Primary Outcome(s)

Number of Participants Who Reported Side Effects During Home Drug Maintenance Phase [Time Frame: 1 year]

Time Duration of AHC Episodes [Time Frame: Week 7]

Observed Safety Data During 5-day Hospitalization for Drug Dose Identification [Time Frame: Week 7]

Secondary Outcome(s)

Mood and Behavior Questionnaires [Time Frame: Week 14]

Functional Skills Questionnaires [Time Frame: Week 14]

Quality of Life Questionnaires [Time Frame: Week 14]

Neuropsychological Tests [Time Frame: Week 14]

Mean Daily Percentage of Time That Functional Status is Affected Due to Episodes [Time Frame: Week 14]

Secondary ID(s)

32164

103932

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Alternating Hemiplegia of Childhood Foundation

Jazz Pharmaceuticals

Results

• Results available: Yes
• Date Posted: 31/03/2014
• URL: https://clinicaltrials.gov/ct2/show/results/NCT00931164