Extended-release Sodium Oxybate in Children
ConditionNarcolepsy Type 1 (NT 1)
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age7-17
SponsorStanford University
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Participants must be under the care of a doctor at the Stanford Sleep Clinic.
Participants must have a documented diagnosis of narcolepsy type 1 shown by sleepiness and either: a spinal fluid marker for narcolepsy (hypocretin-1) at a specific level, or a history of sudden loss of muscle control (cataplexy), or a particular genetic marker for narcolepsy, or a sleep study showing a specific sleep pattern for narcolepsy.
Parent(s), or guardian(s) have signed a consent form and the child must agree to participate.
Participants are on a stable dose of medications
Disqualifiers
Uncontrolled mental health problems
Uncontrolled sleep problems that lead to sleepiness.
Currently having thought about ending one's life or sadness or loss of interest
Currently having a problem with illegal drug use
Trial design
Design model
Crossover
Treatments tested in this trial
Extended-release sodium oxybate
DrugParticipants 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
Non-extended-release oxybates
DrugParticipants 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)
Treatment groups
36 Participants
are divided into 2 treatment groupsGroup A: Extended-release sodium oxybateExperimental treatment 2 interventions
Group B: Non-extended-release oxybatesActive comparator 2 interventions
Trial outcomes
Primary outcomes
1
Participant 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 completionSecondary outcomes
1
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 completion2
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 completion3
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 completionSponsors and contacts
Click on the lead sponsor to view all of their trials.
Stanford University
Lead sponsor
Avadel
Collaborator
Source ClinicalTrials.gov