Safety, Convenience, and Tolerability of a Nightly Single Oral Dose of Extended-release Sodium Oxybate in Children
Trial Snapshot
- Phase
- Phase 2
- Status
- Recruiting
- Sponsor
- Stanford University
- Enrollment
- 36
- Locations
- 1
- Primary Endpoint
- Participant preference for selecting the extended vs non-extended release oxybates
Study Overview
Brief Summary
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 Design
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Crossover
- Primary Purpose
- Other
- Masking
- None
Eligibility Criteria
- Ages
- 7 Years to 17 Years (Child)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •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
Exclusion Criteria
- •Participants who have any of the following conditions will not be included in the study
- •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
- •Currently pregnant
Arms & Interventions
Extended-release sodium oxybate
Participants in this arm will take extended-release sodium oxybate
Intervention: Extended-release sodium oxybate (Drug)
Extended-release sodium oxybate
Participants in this arm will take extended-release sodium oxybate
Intervention: Non-extended-release oxybates (Drug)
Non-extended-release oxybates
Participants in this arm will take non-extended-release sodium oxybate
Intervention: Extended-release sodium oxybate (Drug)
Non-extended-release oxybates
Participants in this arm will take non-extended-release sodium oxybate
Intervention: Non-extended-release oxybates (Drug)
Outcomes
Primary Outcomes
Participant preference for selecting the extended vs non-extended release oxybates
Time Frame: Baseline, up to 8-12 weeks through study completion
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
Secondary Outcomes
- Change in Epworth Sleepiness Scales for Children and Adolescents (ESS-CHAD) score(Baseline, up to 8-12 weeks through study completion)
- Number of cataplexy attacks(Baseline, up to 8-12 weeks through study completion)
- Change in Patient Global Impression of Change (PGIc) scale score(Baseline, up to 8-12 weeks through study completion)
Investigators
Oliver Sum-Ping
Clinical Associate Professor in Psych/Sleep Medicine
Stanford University