The main objectives of this study are to determine the treatment preferences of children with narcolepsy type 1 between Extended-release sodium oxybate and Non-extended-release oxybates (Sodium oxybate or a medication with the combination of Calcium, magnesium, potassium, and sodium oxybates). The study will also assess the safety, convenience, and tolerability of Extended-release sodium oxybate. Additionally, the researchers aim to assess how well Extended-release sodium oxybate works in treating narcolepsy type 1.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
OTHER
Masking
NONE
Enrollment
36
Participants will take Extended-release sodium Oxybate (Lumryz) 4.5-9 g oral suspension in approximately ⅓ cup of water orally once a day before bedtime. Dosage of Lumryz will be the same as the equivalent total dose of the Non-Extended-release Oxybate (Xywav or Xyrem) that participants use before starting the study
Participants will take Non-Extended-release Oxybates (Xyrem oral solution or Xywav oral solution) 4.5-9 g orally per night (divided into two doses, once before bedtime and another dose 2-4 hours later) at the same dose of Xyrem or Xywav that participants use before starting the study will be continued to compare with Extended-release sodium Oxybate (Lumryz)
Stanford University
Redwood City, California, United States
RECRUITINGParticipant preference for selecting the extended vs non-extended release oxybates
Participants will be asked at the end of the study whether participants prefer extended-release sodium oxybate vs sodium oxybate vs calcium, magnesium, potassium and sodium oxybates
Time frame: Baseline, up to 8-12 weeks through study completion
Change in Epworth Sleepiness Scales for Children and Adolescents (ESS-CHAD) score
ESS-CHAD is a multi-item questionnaire which asks the participants to rate their chances of falling asleep during various normal daily activities. 4-point Likert scale will be used. The score ranges from 0-24. A higher score means higher chance of falling asleep.
Time frame: Baseline, up to 8-12 weeks through study completion
Number of cataplexy attacks
The study team will ask the participants to report the number of cataplexy attacks as measured by Sleep and Symptoms diary when participants switch to the other intervention.
Time frame: Baseline, up to 8-12 weeks through study completion
Change in Patient Global Impression of Change (PGIc) scale score
The PGIc is a 7-point scale. Participants will be asked to rate their narcolepsy impression since the end of the Stable-Dose Period. The scale is Very much better, Much better, A little better, No change, A little worse, Much worse, and Very much worse.
Time frame: Baseline, up to 8-12 weeks through study completion
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