Extended-release Sodium Oxybate in Children

Phase 2

Recruiting

• Conditions: Narcolepsy Type 1 (NT 1)
• Interventions: Drug: Extended-release sodium oxybate; Drug: Non-extended-release oxybates

• Registration Number: NCT06809803
• Lead Sponsor: Stanford University

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.

Detailed Description

Not available

Study Timeline

• Status Verified: 2025-01
• Study First Submitted: 2025-01-30
• Study First Submitted QC: 2025-01-30
• Study First Posted: 2025-02-05
• Last Update Submitted: 2025-06-20
• Last Update Posted: 2025-06-26
• Study Start: 2025-07-01
• Primary Completion: 2027-06
• Study Completion: 2027-07-01

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 36

Inclusion Criteria

1. Participants must be under the care of a doctor at the Stanford Sleep Clinic.
2. 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.
3. Parent(s), or guardian(s) have signed a consent form and the child must agree to participate.
4. 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

1. Uncontrolled mental health problems
2. Uncontrolled sleep problems that lead to sleepiness.
3. Currently having thought about ending one's life or sadness or loss of interest
4. Currently having a problem with illegal drug use
5. Currently pregnant

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: CROSSOVER

Arm && Interventions

Group	Intervention	Description
Extended-release sodium oxybate	Extended-release sodium oxybate	Participants in this arm will take extended-release sodium oxybate
Extended-release sodium oxybate	Non-extended-release oxybates	Participants in this arm will take extended-release sodium oxybate
Non-extended-release oxybates	Extended-release sodium oxybate	Participants in this arm will take non-extended-release sodium oxybate
Non-extended-release oxybates	Non-extended-release oxybates	Participants in this arm will take non-extended-release sodium oxybate

Primary Outcome Measures

Name	Time	Method
Participant preference for selecting the extended vs non-extended release oxybates	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 Outcome Measures

Name	Time	Method
Change in Epworth Sleepiness Scales for Children and Adolescents (ESS-CHAD) score	Baseline, up to 8-12 weeks through study completion	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.
Number of cataplexy attacks	Baseline, up to 8-12 weeks through study completion	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.
Change in Patient Global Impression of Change (PGIc) scale score	Baseline, up to 8-12 weeks through study completion	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.

Trial Locations

Locations (1)

Stanford University; 🇺🇸 Redwood City, California, United States