Extended-release Sodium Oxybate in Children

NCT06809803 | Recruiting Phase 2 FDA Drug

• 1 other identifier: 75896
• Study Type: interventional
• Target: 36
• Locations: 1 country
• Sites: 1

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.

Conditions

Narcolepsy Type 1 (NT 1)

Keywords

Narcolepsy; Children; Sodium oxybate; treatment

Outcome Measures

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

  Baseline, up to 8-12 weeks through study completion

Secondary Outcomes (3)

• 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

Study Arms (2)

Extended-release sodium oxybate

EXPERIMENTAL

Participants in this arm will take extended-release sodium oxybate

Drug: Extended-release sodium oxybate; Drug: Non-extended-release oxybates

Non-extended-release oxybates

ACTIVE COMPARATOR

Participants in this arm will take non-extended-release sodium oxybate

Drug: Extended-release sodium oxybate; Drug: Non-extended-release oxybates

Interventions

Extended-release sodium oxybate DRUG

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

Also known as: Lumryz

Extended-release sodium oxybate; Non-extended-release oxybates

Non-extended-release oxybates DRUG

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)

Also known as: Xywav, Xyrem

Extended-release sodium oxybate; Non-extended-release oxybates

Eligibility Criteria

• Age: 7 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Stanford University: lead
• Avadel: collaborator

Study Sites (1)

Stanford University

Redwood City, California, 94063, United States

RECRUITING

MeSH Terms

Conditions

Narcolepsy

Interventions

Sodium Oxybate

Condition Hierarchy (Ancestors)

Disorders of Excessive Somnolence; Sleep Disorders, Intrinsic; Dyssomnias; Sleep Wake Disorders; Nervous System Diseases; Mental Disorders

Intervention Hierarchy (Ancestors)

Hydroxybutyrates; Butyrates; Acids, Acyclic; Carboxylic Acids; Organic Chemicals; Hydroxy Acids

Study Officials

• Oliver Sum-Ping, MD

  Stanford University

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Miran Cho

CONTACT

650-724-4149; mirancho@stanford.edu

Mila Trabanino

CONTACT

milaludi@stanford.edu

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: OTHER
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Clinical Associate Professor in Psych/Sleep Medicine

Study Record Dates

• First Submitted: January 30, 2025
• First Posted: February 5, 2025
• Study Start: October 27, 2025
• Primary Completion (Estimated): June 1, 2027
• Study Completion (Estimated): July 1, 2027
• Last Updated: November 6, 2025

Record last verified: 2025-11

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)