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• W1996779657 abstract "Objective: To investigate the safety, tolerability, dosage, and effect of sodium oxybate on ictal episodes in children with alternating hemiplegia of childhood (AHC). Background AHC is a sporadic neurodevelopmental syndrome of uncertain etiology, defined by paroxysmal episodes of complex abnormal movements. The clinical outcome is poor, with recurrent episodes, motor and cognitive disability, and unmanageable behavior. There is no effective pharmacologic treatment. Sodium oxybate (SO) is approved for narcolepsy to induce slow wave non-REM sleep. Hyperpolarization of neuronal cell membranes is a characteristic feature of both NREM slow wave sleep and SO-induced sleep. Theoretically, SO might be effective in aborting AHC episodes. Design/Methods: The study is an open-label, Phase I/II trial designed to obtain safety parameters and to explore potential benefit of SO. Inclusion criteria: confirmed AHC, age 0.5-25 years, >3 episodes/week, and completion of a daily AHC online event log for 6 weeks. Results: A safe and effective SO dose was identified for all 6 children who participated in a dose-escalation and safety-monitoring inpatient phase, range 40-80mg/kg/day divided qd-bid prn. Data on home SO use and its effects on episodes was collected via daily online event logs. The total episode duration, the primary outcome at 6 weeks, improved in 4 participants and worsened in 2. Adverse events included difficulty breathing, desaturations, worsening behaviors, and excessive sleepiness. The complexity of presentation and outcomes of these participants is instructive and will be reviewed. Conclusions: Impact of treatment with SO in children with AHC was difficult to interpret. Administration of SO on an as-needed basis to abort AHC episodes proved ineffective or poorly tolerated in most with prolonged use. However, 2 children remain on medication due to long-term benefits of decreased AHC episode duration and severity. Supported by: Alternating Hemiplegia of Childhood Foundation; Award Number UL1RR025763 and UL1RR025764 from the National Center for Research Resources. Disclosure: Dr. Lewelt has nothing to disclose. Dr. Sweney has nothing to disclose. Dr. Reyna has nothing to disclose. Dr. Silver has nothing to disclose. Dr. Katchan has nothing to disclose. Dr. Mamelak has nothing to disclose. Dr. Swoboda has received research support from BioMarin Pharmaceuticals and Orphamed," @default.
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• W1996779657 date "2012-04-22" @default.
• W1996779657 modified "2023-09-27" @default.
• W1996779657 title "Single-Center Phase I/II Trial of Sodium Oxybate in Patients with Alternating Hemiplegia of Childhood (IN10-1.007)" @default.
• W1996779657 doi "https://doi.org/10.1212/wnl.78.1_meetingabstracts.in10-1.007" @default.
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